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114th Congress } { Rept. 114-190
1st Session } { Part 1
HOUSE OF REPRESENTATIVES
_______________________________________________________________________
21ST CENTURY CURES ACT
----------
R E P O R T
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
[TO ACCOMPANY H.R. 6]
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
July 7, 2015.--Committed to the Committee of the Whole House on the
State of the Union and ordered to be printed
114th Congress } { Rept. 114-190
HOUSE OF REPRESENTATIVES
1st Session } { Part 1
======================================================================
21ST CENTURY CURES ACT
_______
July 7, 2015.--Committed to the Committee of the Whole House on the
State of the Union and ordered to be printed
_______
Mr. Upton, from the Committee on Energy and Commerce, submitted the
following
R E P O R T
[To accompany H.R. 6]
[Including cost estimate of the Congressional Budget Office]
The Committee on Energy and Commerce, to whom was referred
the bill (H.R. 6) to accelerate the discovery, development, and
delivery of 21st century cures, and for other purposes, having
considered the same, report favorably thereon with an amendment
and recommend that the bill as amended do pass.
CONTENTS
Page
Purpose and Summary.............................................. 85
Background and Need for Legislation.............................. 85
Hearings......................................................... 85
Committee Consideration.......................................... 88
Committee Votes.................................................. 88
Committee Oversight Findings..................................... 90
Statement of General Performance Goals and Objectives............ 90
New Budget Authority, Entitlement Authority, and Tax Expenditures 90
Earmark, Limited Tax Benefits, and Limited Tariff Benefits....... 90
Committee Cost Estimate.......................................... 90
Congressional Budget Office Estimate............................. 90
Federal Mandates Statement....................................... 104
Duplication of Federal Programs.................................. 104
Disclosure of Directed Rule Makings.............................. 105
Advisory Committee Statement..................................... 105
Applicability to Legislative Branch.............................. 105
Section-by-Section Analysis of the Legislation................... 105
Changes in Existing Law Made by the Bill, as Reported............ 129
The amendment is as follows:
Strike all after the enacting clause and insert the
following:
SECTION 1. SHORT TITLE; TABLE OF CONTENTS.
(a) Short Title.--This Act may be cited as the ``21st Century Cures
Act''.
(b) Table of Contents.--The table of contents for this Act is as
follows:
Sec. 1. Short title; table of contents.
TITLE I--DISCOVERY
Subtitle A--National Institutes of Health Funding
Sec. 1001. National Institutes of Health reauthorization.
Sec. 1002. NIH Innovation Fund.
Subtitle B--National Institutes of Health Planning and Administration
Sec. 1021. NIH research strategic plan.
Sec. 1022. Increasing accountability at the National Institutes of
Health.
Sec. 1023. Reducing administrative burdens of researchers.
Sec. 1024. Exemption for the National Institutes of Health from the
Paperwork Reduction Act requirements.
Sec. 1025. NIH travel.
Sec. 1026. Other transactions authority.
Sec. 1027. NCATS phase IIB restriction.
Sec. 1028. High-risk, high-reward research.
Sec. 1029. Sense of Congress on increased inclusion of underrepresented
communities in clinical trials.
Subtitle C--Supporting Young Emerging Scientists
Sec. 1041. Improvement of loan repayment programs of the National
Institutes of Health.
Sec. 1042. Report.
Subtitle D--Capstone Grant Program
Sec. 1061. Capstone award.
Subtitle E--Promoting Pediatric Research Through the National
Institutes of Health
Sec. 1081. National pediatric research network.
Sec. 1082. Global pediatric clinical study network sense of Congress.
Sec. 1083. Appropriate age groupings in clinical research.
Subtitle F--Advancement of the National Institutes of Health Research
and Data Access
Sec. 1101. Sharing of data generated through NIH-funded research.
Sec. 1102. Standardization of data in Clinical Trial Registry Data Bank
on eligibility for clinical trials.
Subtitle G--Facilitating Collaborative Research
Sec. 1121. Clinical trial data system.
Sec. 1122. National neurological diseases surveillance system.
Sec. 1123. Data on natural history of diseases.
Sec. 1124. Accessing, sharing, and using health data for research
purposes.
Subtitle H--Council for 21st Century Cures
Sec. 1141. Council for 21st Century Cures.
TITLE II--DEVELOPMENT
Subtitle A--Patient-Focused Drug Development
Sec. 2001. Development and use of patient experience data to enhance
structured risk-benefit assessment framework.
Subtitle B--Qualification and Use of Drug Development Tools
Sec. 2021. Qualification of drug development tools.
Sec. 2022. Accelerated approval development plan.
Subtitle C--FDA Advancement of Precision Medicine
Sec. 2041. Precision medicine guidance and other programs of Food and
Drug Administration.
Subtitle D--Modern Trial Design and Evidence Development
Sec. 2061. Broader application of Bayesian statistics and adaptive
trial designs.
Sec. 2062. Utilizing evidence from clinical experience.
Sec. 2063. Streamlined data review program.
Subtitle E--Expediting Patient Access
Sec. 2081. Sense of Congress.
Sec. 2082. Expanded access policy.
Sec. 2083. Finalizing draft guidance on expanded access.
Subtitle F--Facilitating Responsible Manufacturer Communications
Sec. 2101. Facilitating dissemination of health care economic
information.
Sec. 2102. Facilitating responsible communication of scientific and
medical developments.
Subtitle G--Antibiotic Drug Development
Sec. 2121. Approval of certain drugs for use in a limited population of
patients.
Sec. 2122. Susceptibility test interpretive criteria for
microorganisms.
Sec. 2123. Encouraging the development and use of new antimicrobial
drugs.
Subtitle H--Vaccine Access, Certainty, and Innovation
Sec. 2141. Timely review of vaccines by the Advisory Committee on
Immunization Practices.
Sec. 2142. Review of processes and consistency of ACIP recommendations.
Sec. 2143. Meetings between CDC and vaccine developers.
Subtitle I--Orphan Product Extensions Now; Incentives for Certain
Products for Limited Populations
Sec. 2151. Extension of exclusivity periods for a drug approved for a
new indication for a rare disease or condition.
Sec. 2152. Reauthorization of rare pediatric disease priority review
voucher incentive program.
Subtitle J--Domestic Manufacturing and Export Efficiencies
Sec. 2161. Grants for studying the process of continuous drug
manufacturing.
Sec. 2162. Re-exportation among members of the European Economic Area.
Subtitle K--Enhancing Combination Products Review
Sec. 2181. Enhancing combination products review.
Subtitle L--Priority Review for Breakthrough Devices
Sec. 2201. Priority review for breakthrough devices.
Subtitle M--Medical Device Regulatory Process Improvements
Sec. 2221. Third-party quality system assessment.
Sec. 2222. Valid scientific evidence.
Sec. 2223. Training and oversight in least burdensome appropriate means
concept.
Sec. 2224. Recognition of standards.
Sec. 2225. Easing regulatory burden with respect to certain class I and
class II devices.
Sec. 2226. Advisory committee process.
Sec. 2227. Humanitarian device exemption application.
Sec. 2228. CLIA waiver study design guidance for in vitro diagnostics.
Subtitle N--Sensible Oversight for Technology Which Advances Regulatory
Efficiency
Sec. 2241. Health software.
Sec. 2242. Applicability and inapplicability of regulation.
Sec. 2243. Exclusion from definition of device.
Subtitle O--Streamlining Clinical Trials
Sec. 2261. Protection of human subjects in research; applicability of
rules.
Sec. 2262. Use of non-local institutional review boards for review of
investigational device exemptions and human device exemptions.
Sec. 2263. Alteration or waiver of informed consent for clinical
investigations.
Subtitle P--Improving Scientific Expertise and Outreach at FDA
Sec. 2281. Silvio O. Conte Senior Biomedical Research Service.
Sec. 2282. Enabling FDA scientific engagement.
Sec. 2283. Reagan-Udall Foundation for the Food and Drug
Administration.
Sec. 2284. Collection of certain voluntary information exempted from
Paperwork Reduction Act.
Sec. 2285. Hiring authority for scientific, technical, and professional
personnel.
Subtitle Q--Exempting From Sequestration Certain User Fees
Sec. 2301. Exempting from sequestration certain user fees of Food and
Drug Administration.
TITLE III--DELIVERY
Subtitle A--Interoperability
Sec. 3001. Ensuring interoperability of health information technology.
Subtitle B--Telehealth
Sec. 3021. Telehealth services under the Medicare program.
Subtitle C--Encouraging Continuing Medical Education for Physicians
Sec. 3041. Exempting from manufacturer transparency reporting certain
transfers used for educational purposes.
Subtitle D--Disposable Medical Technologies
Sec. 3061. Treatment of certain items and devices.
Subtitle E--Local Coverage Decision Reforms
Sec. 3081. Improvements in the Medicare local coverage determination
(LCD) process.
Subtitle F--Medicare Pharmaceutical and Technology Ombudsman
Sec. 3101. Medicare pharmaceutical and technology ombudsman.
Subtitle G--Medicare Site-of-Service Price Transparency
Sec. 3121. Medicare site-of-Service price transparency.
Subtitle H--Medicare Part D Patient Safety and Drug Abuse Prevention
Sec. 3141. Programs to prevent prescription drug abuse under Medicare
parts C and D.
TITLE IV--MEDICAID, MEDICARE, AND OTHER REFORMS
Subtitle A--Medicaid and Medicare Reforms
Sec. 4001. Limiting Federal Medicaid reimbursement to States for
durable medical equipment (DME) to Medicare payment rates.
Sec. 4002. Medicare payment incentive for the transition from
traditional x-ray imaging to digital radiography and other Medicare
imaging payment provision.
Sec. 4003. Implementation of Office of Inspector General recommendation
to delay certain Medicare prescription drug plan prepayments.
Subtitle B--Cures Innovation Fund
Sec. 4041. Cures Innovation Fund.
Subtitle C--Other Reforms
Sec. 4061. SPR drawdown.
Subtitle D--Miscellaneous
Sec. 4081. Lyme disease and other tick-borne diseases.
TITLE I--DISCOVERY
Subtitle A--National Institutes of Health Funding
SEC. 1001. NATIONAL INSTITUTES OF HEALTH REAUTHORIZATION.
Section 402A(a)(1) of the Public Health Service Act (42 U.S.C.
282a(a)(1)) is amended--
(1) in subparagraph (B), by striking at the end ``and'';
(2) in subparagraph (C), by striking at the end the period
and inserting a semicolon; and
(3) by adding at the end the following new subparagraphs:
``(D) $31,811,000,000 for fiscal year 2016;
``(E) $33,331,000,000 for fiscal year 2017; and
``(F) $34,851,000,000 for fiscal year 2018.''.
SEC. 1002. NIH INNOVATION FUND.
(a) Use of Innovation Fund.--Section 402(b) of the Public Health
Service Act (42 U.S.C. 282(b)) is amended--
(1) in paragraph (23), by striking at the end ``and'';
(2) in paragraph (24), by striking at the end the period and
inserting ``; and''; and
(3) by inserting after paragraph (24), the following new
paragraph:
``(25) shall, with respect to funds appropriated under
section 402A(e) to the NIH Innovation Fund, allocate such funds
to the national research institutes and national centers for
conducting and supporting innovation fund initiatives
identified under paragraph (3) of such section.''.
(b) Establishment of Innovation Fund.--Section 402A of the Public
Health Service Act (42 U.S.C. 282a)is amended--
(1) by redesignating subsection (e) as subsection (f); and
(2) by inserting after subsection (d) the following new
subsection:
``(e) NIH Innovation Fund.--
``(1) Establishment.--For the purpose of allocations under
section 402(b)(25), there is established a fund to be known as
the NIH Innovation Fund. The Director of NIH shall, with
respect to funds appropriated to the NIH Innovation Fund,
allocate such funds to support biomedical research through the
funding of basic, translational, and clinical research.
``(2) Amounts made available to fund.--
``(A) In general.--Subject to subparagraph (B), there
is authorized to be appropriated, and appropriated, to
the NIH Innovation Fund out of any funds in the
Treasury not otherwise appropriated, $2,000,000,000 for
each of fiscal years 2016 through 2020. The amounts
appropriated to the Fund by the preceding sentence
shall be in addition to any amounts otherwise made
available to the National Institutes of Health.
``(B) Availability subject to appropriations.--
Amounts in the Fund shall not be available except to
the extent and in such amounts as are provided in
advance in appropriation Acts.
``(C) Allocation of amounts.--Of the amounts made
available from the NIH Innovation Fund for allocations
under section 402(b)(25) for a fiscal year--
``(i) not less than $500,000,000 shall be for
the Accelerating Advancement Program under
paragraph (5);
``(ii) not less than 35 percent of such
amounts remaining after subtracting the
allocation for the Accelerating Advancement
Program shall be for early stage investigators
(as defined in paragraph (7));
``(iii) not less than 20 percent of such
amounts remaining after subtracting the
allocation for the Accelerating Advancement
Program shall be for high-risk, high-reward
research under section 409K; and
``(iv) not more than 10 percent of such
amounts (without subtracting the allocation for
the Accelerating Advancement Program) shall be
for intramural research.
``(D) Inapplicability of certain provisions.--Amounts
in the NIH Innovation Fund shall not be subject to--
``(i) any transfer authority of the Secretary
or the Director of NIH under section 241,
subsection (c), subsection (d), or any other
provision of law (other than section 402(b)(25)
and this subsection); or
``(ii) the Nonrecurring expenses fund under
section 223 of division G of the Consolidated
Appropriations Act, 2008 (42 U.S.C. 3514a).
``(3) Authorized uses.--Amounts in the NIH Innovation Fund
established under paragraph (1) may be used only to conduct or
support innovative biomedical research through the following:
``(A) Research in which--
``(i) a principal investigator has a specific
project or specific objectives; and
``(ii) funding is tied to pursuit of such
project or objectives.
``(B) Research in which--
``(i) a principal investigator has shown
promise in biomedical research; and
``(ii) funding is not tied to a specific
project or specific objectives.
``(C) Research to be carried out by an early stage
investigator (as defined in paragraph (7)).
``(D) Research to be carried out by a small business
concern (as defined in section 3 of the Small Business
Act).
``(E) The Accelerating Advancement Program under
paragraph (5).
``(F) Development and implementation of the strategic
plan under paragraph (6).
``(4) Coordination.--In funding programs and activities
through the NIH Innovation Fund, the Secretary, acting through
the Director of NIH, shall--
``(A) ensure coordination among the national research
institutes, the national centers, and other
departments, agencies, and offices of the Federal
Government; and
``(B) minimize unnecessary duplication.
``(5) Accelerating advancement program.--The Director of NIH
shall establish a program, to be known as the Accelerating
Advancement Program, under which--
``(A) the Director of NIH partners with national
research institutes and national centers to accomplish
important biomedical research objectives; and
``(B) for every $1 made available by the Director of
NIH to a national research institute or national center
for a research project, the institute or center makes
$1 available for such project from funds that are not
derived from the NIH Innovation Fund.
``(6) Strategic plan.--
``(A) In general.--The Director of NIH shall ensure
that scientifically based strategic planning is
implemented in support of research priorities,
including through development, use, and updating of a
research strategic plan that--
``(i) is designed to increase the efficient
and effective focus of biomedical research in a
manner that leverages the best scientific
opportunities through a deliberative planning
process;
``(ii) identifies areas, to be known as
strategic focus areas, in which the resources
of the NIH Innovation Fund can contribute to
the goals of expanding knowledge to address,
and find more effective treatments for, unmet
medical needs in the United States, including
the areas of--
``(I) biomarkers;
``(II) precision medicine;
``(III) infectious diseases,
including pathogens listed as a
qualifying pathogen under section
505E(f) of the Federal Food, Drug, and
Cosmetic Act or listed or designated as
a tropical disease under section 524 of
such Act; and
``(IV) antibiotics;
``(iii) includes objectives for each such
strategic focus area; and
``(iv) ensures that basic research remains a
priority.
``(B) Updates and reviews.--The Director shall review
and, as appropriate, update the research strategic plan
under subparagraph (A) not less than every 18 months.
``(7) Definition.--In this subsection, the term `early stage
investigator' means an investigator who--
``(A) will be the principal investigator or the
program director of the proposed research;
``(B) has never been awarded, or has been awarded
only once, a substantial, competing grant by the
National Institutes of Health for independent research;
and
``(C) is within 10 years of having completed--
``(i) the investigator's terminal degree; or
``(ii) a medical residency (or the
equivalent).''.
(c) Supplement, Not Supplant; Prohibition Against Transfer.--Funds
appropriated pursuant to section 402A(e) of the Public Health Service
Act, as inserted by subsection (b)--
(1) shall be used to supplement, not supplant, the funds
otherwise allocated by the National Institutes of Health for
biomedical research; and
(2) notwithstanding any transfer authority in any
appropriation Act, shall not be used for any purpose other than
allocating funds for conducting and supporting innovation fund
initiatives as described in section 402(b)(25) of the Public
Health Service Act, as added by subsection (a).
Subtitle B--National Institutes of Health Planning and Administration
SEC. 1021. NIH RESEARCH STRATEGIC PLAN.
Section 402 of the Public Health Service Act (42 U.S.C. 282) is
amended--
(1) in subsection (b), by amending paragraph (5) to read as
follows:
``(5) shall ensure that scientifically based strategic
planning is implemented in support of research priorities as
determined by the agencies of the National Institutes of
Health, including through development, use, and updating of the
research strategic plan under subsection (m);''; and
(2) by adding at the end the following:
``(m) Research Strategic Plan.--
``(1) Five-year plans for biomedical research strategy.--
``(A) In general.--For each successive five-year
period beginning with the period of fiscal years 2016
through 2020, the Director of NIH, in consultation with
the entities described in subparagraph (B), shall
develop and maintain a biomedical research strategic
plan that--
``(i) is designed to increase the efficient
and effective focus of biomedical research in a
manner that leverages the best scientific
opportunities through a deliberative planning
process;
``(ii) identifies areas, to be known as
strategic focus areas, in which the resources
of the National Institutes of Health can best
contribute to the goal of expanding knowledge
on human health in the United States through
biomedical research; and
``(iii) includes objectives for each such
strategic focus area.
``(B) Entities described.--The entities described in
this subparagraph are the directors of the national
research institutes and national centers, researchers,
patient advocacy groups, and industry leaders.
``(2) Use of plan.--The Director of NIH and the directors of
the national research institutes and national centers shall use
the strategic plan--
``(A) to identify research opportunities; and
``(B) to develop individual strategic plans for the
research activities of each of the national research
institutes and national centers that--
``(i) have a common template; and
``(ii) identify strategic focus areas in
which the resources of the national research
institutes and national centers can best
contribute to the goal of expanding knowledge
on human health in the United States through
biomedical research.
``(3) Contents of plans.--
``(A) Strategic focus areas.--The strategic focus
areas identified pursuant to paragraph (1)(A)(ii)
shall--
``(i) be identified in a manner that--
``(I) considers the return on
investment to the United States public
through the investments of the National
Institutes of Health in biomedical
research; and
``(II) contributes to expanding
knowledge to improve the United States
public's health through biomedical
research; and
``(ii) include overarching and trans-National
Institutes of Health strategic focus areas, to
be known as Mission Priority Focus Areas, which
best serve the goals of preventing or
eliminating the burden of a disease or
condition and scientifically merit enhanced and
focused research over the next 5 years.
``(B) Rare and pediatric diseases and conditions.--In
developing and maintaining a strategic plan under this
subsection, the Director of NIH shall ensure that rare
and pediatric diseases and conditions remain a
priority.
``(C) Workforce.--In developing and maintaining a
strategic plan under this subsection, the Director of
NIH shall ensure that maintaining the biomedical
workforce of the future, including the participation by
scientists from groups traditionally underrepresented
in the scientific workforce, remains a priority.
``(4) Initial plan.--Not later than 270 days after the date
of enactment of this subsection, the Director of NIH and the
directors of the national research institutes and national
centers shall--
``(A) complete the initial strategic plan required by
paragraphs (1) and (2); and
``(B) make such initial strategic plan publicly
available on the website of the National Institutes of
Health.
``(5) Review; updates.--
``(A) Progress reviews.--Not less than annually, the
Director of NIH, in consultation with the directors of
the national research institutes and national centers,
shall conduct progress reviews for each strategic focus
area identified under paragraph (1)(A)(ii).
``(B) Updates.--Not later than the end of the 5-year
period covered by the initial strategic plan under this
subsection, and every 5 years thereafter, the Director
of NIH, in consultation with the directors of the
national research institutes and national centers,
stakeholders in the scientific field, advocates, and
the public at large, shall--
``(i) conduct a review of the plan, including
each strategic focus area identified under
paragraph (2)(B); and
``(ii) update such plan in accordance with
this section.''.
SEC. 1022. INCREASING ACCOUNTABILITY AT THE NATIONAL INSTITUTES OF
HEALTH.
(a) Appointment and Terms of Directors of National Research
Institutes and National Centers.--Subsection (a) of section 405 of the
Public Health Service Act (42 U.S.C. 284) is amended to read as
follows: ``(a) Appointment; Terms.--
``(1) Appointment.--The Director of the National Cancer
Institute shall be appointed by the President and the directors
of the other national research institutes, as well as the
directors of the national centers, shall be appointed by the
Director of NIH. The directors of the national research
institutes, as well as national centers, shall report directly
to the Director of NIH.
``(2) Terms.--
``(A) In general.--The term of office of a director
of a national research institute or national center
shall be 5 years.
``(B) Removal.--The director of a national research
institute or national center may be removed from office
by the Director of NIH prior to the expiration of such
director's 5-year term.
``(C) Reappointment.--At the end of the term of a
director of a national research institute or national
center, the director may be reappointed. There is no
limit on the number of terms a director may serve.
``(D) Vacancies.--If the office of a director of a
national research institute or national center becomes
vacant before the end of such director's term, the
director appointed to fill the vacancy shall be
appointed for a 5-year term starting on the date of
such appointment.
``(E) Transitional provision.--Each director of a
national research institute or national center serving
on the date of enactment of the 21st Century Cures Act
is deemed to be appointed for a 5-year term under this
subsection starting on such date of enactment.''.
(b) Compensation to Consultants or Individual Scientists.--Section
202 of the Departments of Labor, Health and Human Services, and
Education, and Related Agencies Appropriations Act, 1993 (Public Law
102-394; 42 U.S.C. 238f note) is amended by striking ``portable
structures;'' and all that follows and inserting ``portable
structures.''.
(c) Review of Certain Awards by Directors.--Section 405(b) of the
Public Health Service Act (42 U.S.C. 284(b)) is amended by adding at
the end the following:
``(3) Before an award is made by a national research institute or by
a national center for a grant for a research program or project
(commonly referred to as an `R-series grant'), other than an award
constituting a noncompeting renewal of such grant, or a noncompeting
administrative supplement to such grant, the director of such national
research institute or national center--
``(A) shall review and approve the award; and
``(B) shall take into consideration--
``(i) the mission of the national research institute
or national center and the scientific priorities
identified in the strategic plan under section 402(m);
and
``(ii) whether other agencies are funding programs or
projects to accomplish the same goal.''.
(d) IOM Study on Duplication in Federal Biomedical Research.--The
Secretary of Health and Human Services shall enter into an arrangement
with the Institute of Medicine of the National Academies (or, if the
Institute declines, another appropriate entity) under which the
Institute (or other appropriate entity) not later than 2 years after
the date of enactment of this Act will--
(1) complete a study on the extent to which biomedical
research conducted or supported by Federal agencies is
duplicative; and
(2) submit a report to the Congress on the results of such
study, including recommendations on how to prevent such
duplication.
SEC. 1023. REDUCING ADMINISTRATIVE BURDENS OF RESEARCHERS.
(a) Plan Preparation and Implementation of Measures To Reduce
Administrative Burdens.--The Director of the National Institutes of
Health shall prepare a plan, including time frames, and implement
measures to reduce the administrative burdens of researchers funded by
the National Institutes of Health, taking into account the
recommendations, evaluations, and plans researched by the following
entities:
(1) The Scientific Management Review Board.
(2) The National Academy of Sciences.
(3) The 2007 and 2012 Faculty Burden Survey conducted by The
Federal Demonstration Partnership.
(4) Relevant recommendations from the Research Business
Models Working Group.
(b) Report.--Not later than two years after the date of enactment of
this Act, the Director of the National Institutes of Health shall
submit to Congress a report on the extent to which the Director has
implemented measures pursuant to subsection (a).
SEC. 1024. EXEMPTION FOR THE NATIONAL INSTITUTES OF HEALTH FROM THE
PAPERWORK REDUCTION ACT REQUIREMENTS.
Section 3518(c)(1) of title 44, United States Code, is amended--
(1) in subparagraph (C), by striking ``; or'' and inserting a
semicolon;
(2) in subparagraph (D), by striking the period at the end
and inserting ``; or''; and
(3) by inserting at the end the following new subparagraph:
``(E) during the conduct of research by the National
Institutes of Health.''.
SEC. 1025. NIH TRAVEL.
It is the sense of Congress that participation in or sponsorship of
scientific conferences and meetings is essential to the mission of the
National Institutes of Health.
SEC. 1026. OTHER TRANSACTIONS AUTHORITY.
Section 480 of the Public Health Service Act (42 U.S.C. 287a) is
amended--
(1) in subsection (b), by striking ``the appropriation of
funds as described in subsection (g)'' and inserting ``the
availability of funds as described in subsection (f)'';
(2) in subsection (e)(3), by amending subparagraph (C) to
read as follows:
``(C) Other transactions authority.--The Director of
the Center shall have other transactions authority in
entering into transactions to fund projects in
accordance with the terms and conditions of this
section.'';
(3) by striking subsection (f); and
(4) by redesignating subsection (g) as subsection (f).
SEC. 1027. NCATS PHASE IIB RESTRICTION.
Section 479 of the Public Health Service Act (42 U.S.C. 287) is
amended--
(1) prior to making the amendments under paragraph (2), by
striking ``IIB'' each place it appears and inserting ``III'';
and
(2) by striking ``IIA'' each place it appears and inserting
``IIB''.
SEC. 1028. HIGH-RISK, HIGH-REWARD RESEARCH.
Part B of title IV of the Public Health Service Act (42 U.S.C. 284 et
seq.) is amended by adding at the end the following:
``SEC. 409K. HIGH-RISK, HIGH-REWARD RESEARCH PROGRAM.
``The director of each national research institute shall, as
appropriate--
``(1) establish programs to conduct or support research
projects that pursue innovative approaches to major
contemporary challenges in biomedical research that involve
inherent high risk, but have the potential to lead to
breakthroughs; and
``(2) set aside a specific percentage of funding, to be
determined by the Director of NIH for each national research
institute, for such projects.''.
SEC. 1029. SENSE OF CONGRESS ON INCREASED INCLUSION OF UNDERREPRESENTED
COMMUNITIES IN CLINICAL TRIALS.
It is the sense of Congress that the National Institute on Minority
Health and Health Disparities (NIMHD) should include within its
strategic plan ways to increase representation of underrepresented
communities in clinical trials.
Subtitle C--Supporting Young Emerging Scientists
SEC. 1041. IMPROVEMENT OF LOAN REPAYMENT PROGRAMS OF THE NATIONAL
INSTITUTES OF HEALTH.
(a) In General.--Part G of title IV of the Public Health Service (42
U.S.C. 288 et seq.) is amended--
(1) by redesignating the second section 487F (42 U.S.C. 288-
6; relating to pediatric research loan repayment program) as
section 487G; and
(2) by inserting after section 487G, as so redesignated, the
following:
``SEC. 487H. LOAN REPAYMENT PROGRAM.
``(a) In General.--The Secretary shall establish a program, based on
workforce and scientific needs, of entering into contracts with
qualified health professionals under which such health professionals
agree to engage in research in consideration of the Federal Government
agreeing to pay, for each year of engaging in such research, not more
than $50,000 of the principal and interest of the educational loans of
such health professionals.
``(b) Adjustment for Inflation.--Beginning with respect to fiscal
year 2017, the Secretary may increase the maximum amount specified in
subsection (a) by an amount that is determined by the Secretary, on an
annual basis, to reflect inflation.
``(c) Limitation.--The Secretary may not enter into a contract with a
health professional pursuant to subsection (a) unless such professional
has a substantial amount of educational loans relative to income.
``(d) Applicability of Certain Provisions Regarding Obligated
Service.--Except to the extent inconsistent with this section, the
provisions of sections 338B, 338C, and 338E shall apply to the program
established under this section to the same extent and in the same
manner as such provisions apply to the National Health Service Corps
Loan Repayment Program established under section 338B.
``(e) Availability of Appropriations.--Amounts appropriated for a
fiscal year for contracts under subsection (a) are authorized to remain
available until the expiration of the second fiscal year beginning
after the fiscal year for which the amounts were appropriated.''.
(b) Update of Other Loan Repayment Programs.--
(1) Section 464z-5(a) of the Public Health Service Act (42
U.S.C.285t-2(a)) is amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
subsection in the same manner as it applies to the
maximum amount specified in subsection (a) of such
section.''.
(2) Section 487A(a) of such Act (42 U.S.C. 288-1(a)) is
amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
subsection in the same manner as it applies to the
maximum amount specified in subsection (a) of such
section.''.
(3) Section 487B(a) of such Act (42 U.S.C. 288-2(a)) is
amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
subsection in the same manner as it applies to the
maximum amount specified in such subsection (a) of such
section.''.
(4) Section 487C(a)(1) of such Act (42 U.S.C. 288-3(a)(1)) is
amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
paragraph in the same manner as it applies to the
maximum amount specified in such subsection (a) of such
section.''.
(5) Section 487E(a)(1) of such Act (42 U.S.C. 288-5(a)(1)) is
amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
paragraph in the same manner as it applies to the
maximum amount specified in such subsection (a) of such
section.''.
(6) Section 487F(a) of such Act (42 U.S.C. 288-5a(a)), as
added by section 205 of Public Law 106-505, is amended--
(A) by striking ``$35,000'' and inserting
``$50,000''; and
(B) by adding at the end the following new sentence:
``Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this
subsection in the same manner as it applies to the
maximum amount specified in such subsection (a) of such
section.''.
(7) Section 487G of such Act (42 U.S.C. 288-6, as
redesignated by section 1041(a)(1)), is further amended--
(A) in subsection (a)(1), by striking ``$35,000'' and
inserting ``$50,000''; and
(B) in subsection (b), by adding at the end the
following new sentence: ``Subsection (b) of section
487H shall apply with respect to the maximum amount
specified in subsection (a)(1) in the same manner as it
applies to the maximum amount specified in such
subsection (a) of such section.''.
SEC. 1042. REPORT.
Not later than 18 months after the date of the enactment of this Act,
the Director of the National Institutes of Health shall submit to
Congress a report on efforts of the National Institutes of Health to
attract, retain, and develop emerging scientists.
Subtitle D--Capstone Grant Program
SEC. 1061. CAPSTONE AWARD.
Part G of title IV of the Public Health Service Act (42 U.S.C. 288 et
seq.) is amended by adding at the end the following:
``SEC. 490. CAPSTONE AWARD.
``(a) In General.--The Secretary may make awards (each of which,
hereafter in this section, referred to as a `Capstone Award') to
support outstanding scientists who have been funded by the National
Institutes of Health.
``(b) Purpose.--Capstone Awards shall be made to facilitate the
successful transition or conclusion of research programs, or for other
purposes, as determined by the Director of NIH, in consultation with
the directors of the national research institutes and national centers.
``(c) Duration and Amount.--The duration and amount of each Capstone
Award shall be determined by the Director of NIH in consultation with
the directors of the national research institutes and national centers.
``(d) Limitation.--Individuals who have received a Capstone Award
shall not be eligible to have principle investigator status on
subsequent awards from the National Institutes of Health.''.
Subtitle E--Promoting Pediatric Research Through the National
Institutes of Health
SEC. 1081. NATIONAL PEDIATRIC RESEARCH NETWORK.
Section 409D(d) of the Public Health Service Act (42 U.S.C. 284h(d))
is amended--
(1) in paragraph (1)--
(A) by striking ``in consultation with the Director
of the Eunice Kennedy Shriver National Institute of
Child Health and Human Development and in collaboration
with other appropriate national research institutes and
national centers that carry out activities involving
pediatric research'' and inserting ``in collaboration
with the national research institutes and national
centers that carry out activities involving pediatric
research'';
(B) by striking subparagraph (B);
(C) by striking ``may be comprised of, as
appropriate'' and all that follows through ``the
pediatric research consortia'' and inserting ``may be
comprised of, as appropriate, the pediatric research
consortia''; and
(D) by striking ``; or'' at the end and inserting a
period; and
(2) in paragraph (1), paragraph (2)(A), the first sentence of
paragraph (2)(E), and paragraph (4), by striking ``may'' each
place it appears and inserting ``shall''.
SEC. 1082. GLOBAL PEDIATRIC CLINICAL STUDY NETWORK SENSE OF CONGRESS.
It is the sense of Congress that--
(1) the National Institutes of Health should encourage a
global pediatric clinical study network through the allocation
of grants, contracts, or cooperative agreements to supplement
the salaries of new and early investigators who participate in
the global pediatric clinical study network;
(2) National Institutes of Health grants, contracts, or
cooperative agreements should be awarded, solely for the
purpose of supplementing the salaries of new and early
investigators, to entities that participate in the global
pediatric clinical study network;
(3) the Food and Drug Administration should engage the
European Medicines Agency and other foreign regulatory entities
during the formation of the global pediatric clinical study
network to encourage their participation; and
(4) once a global pediatric clinical study network is
established and becomes operational, the Food and Drug
Administration should continue to engage the European Medicines
Agency and other foreign regulatory entities to encourage and
facilitate their participation in the network with the goal of
enhancing the global reach of the network.
SEC. 1083. APPROPRIATE AGE GROUPINGS IN CLINICAL RESEARCH.
(a) Input From Experts.--Not later than 180 days after the date of
enactment of this Act, the Director of the National Institutes of
Health shall convene a workshop of experts on pediatrics and experts on
geriatrics to provide input on--
(1) appropriate age groupings to be included in research
studies involving human subjects; and
(2) acceptable scientific justifications for excluding
participants from a range of age groups from human subjects
research studies.
(b) Guidelines.--Not later than 180 days after the conclusion of the
workshop under subsection (a), the Director of the National Institutes
of Health shall publish guidelines--
(1) addressing the consideration of age as an inclusion
variable in research involving human subjects; and
(2) identifying criteria for justifications for any age-
related exclusions in such research.
(c) Public Availability of Findings and Conclusions.--The Director of
the National Institutes of Health shall--
(1) make the findings and conclusions resulting from the
workshop under subsection (a) available to the public on the
website of the National Institutes of Health; and
(2) not less than biennially, disclose to the public on such
website the number of children included in research that is
conducted or supported by the National Institutes of Health,
disaggregated by developmentally appropriate age group, race,
and gender.
Subtitle F--Advancement of the National Institutes of Health Research
and Data Access
SEC. 1101. SHARING OF DATA GENERATED THROUGH NIH-FUNDED RESEARCH.
Section 402 of the Public Health Service Act (42 U.S.C. 282) (as
amended by section 1021(2)) is further amended by adding at the end the
following:
``(n) Sharing of Data Generated Through NIH-funded Research.--
``(1) Authority.--Subject to paragraph (2), the Director of
NIH may require recipients of the award of an NIH grant or
other financial support, provided that the research is fully
funded through such grant or other support, to share scientific
data generated from research conducted through such support for
research purposes.
``(2) Limitation.--The Director of NIH shall not require the
sharing of data that is inconsistent with applicable law and
policy protecting--
``(A) privacy and confidentiality;
``(B) proprietary interests;
``(C) business confidential information;
``(D) intellectual property rights; and
``(E) other relevant rights.''.
SEC. 1102. STANDARDIZATION OF DATA IN CLINICAL TRIAL REGISTRY DATA BANK
ON ELIGIBILITY FOR CLINICAL TRIALS.
(a) Standardization.--
(1) In general.--Section 402(j) of the Public Health Service
Act (42 U.S.C. 282(j)) is amended--
(A) by redesignating paragraph (7) as paragraph (8);
and
(B) by inserting after paragraph (6) the following:
``(7) Standardization.--The Director of NIH shall--
``(A) ensure that the registry and results data bank
is easily used by the public;
``(B) ensure that entries in the registry and results
data bank are easily compared;
``(C) ensure that information required to be
submitted to the registry and results data bank,
including recruitment information under paragraph
(2)(A)(ii)(II), is submitted by persons and posted by
the Director of NIH in a standardized format and
includes at least--
``(i) the disease or indication being
studied;
``(ii) inclusion criteria such as age,
gender, diagnosis or diagnoses, laboratory
values, or imaging results; and
``(iii) exclusion criteria such as specific
diagnosis or diagnoses, laboratory values, or
prohibited medications; and
``(D) to the extent possible, in carrying out this
paragraph, make use of standard health care
terminologies, such as the International Classification
of Diseases or the Current Procedural Terminology, that
facilitate electronic matching to data in electronic
health records or other relevant health information
technologies.''.
(2) Conforming amendment.--Clause (iv) of section
402(j)(2)(B) of the Public Health Service Act (42 U.S.C.
282(j)(2)(B)) is hereby stricken.
(b) Consultation.--Not later than 90 days after the date of enactment
of this Act, the Secretary of Health and Human Services shall consult
with stakeholders (including patients, researchers, physicians,
industry representatives, health information technology providers, the
Food and Drug Administration, and standard setting organizations such
as CDISC that have experience working with Federal agencies to
standardize health data submissions) to receive advice on enhancements
to the clinical trial registry data bank under section 402(j) of the
Public Health Service Act (42 U.S.C. 282(j)) (including enhancements to
usability, functionality, and search capability) that are necessary to
implement paragraph (7) of section 402(j) of such Act, as added by
subsection (a).
(c) Applicability.--Not later than 18 months after the date of
enactment of this Act, the Secretary of Health and Human Services shall
begin implementation of paragraph (7) of section 402(j) of the Public
Health Service Act, as added by subsection (a).
Subtitle G--Facilitating Collaborative Research
SEC. 1121. CLINICAL TRIAL DATA SYSTEM.
(a) Establishment.--The Secretary, acting through the Commissioner of
Food and Drugs and the Director of the National Institutes of Health,
shall enter into a cooperative agreement, contract, or grant for a
period of 7 years, to be known as the Clinical Trial Data System
Agreement, with one or more eligible entities to implement a pilot
program with respect to all clinical trial data obtained from qualified
clinical trials for purposes of registered users conducting further
research on such data.
(b) Application.--Eligible entities seeking to enter into a
cooperative agreement, contract, or grant with the Secretary under this
section shall submit to the Secretary an application in such time and
manner, and containing such information, as the Secretary may require
in accordance with this section. The Secretary shall not enter into a
cooperative agreement, contract, or grant under this section with an
eligible entity unless such entity submits an application including the
following:
(1) A certification that the eligible entity is not currently
and does not plan to be involved in sponsoring, operating, or
participating in a clinical trial nor collaborating with
another entity for the purposes of sponsoring, operating, or
participating in a clinical trial.
(2) Information demonstrating that the eligible entity can
compile clinical trial data in standardized formats using
terminologies and standards that have been developed by
recognized standards developing organizations with input from
diverse stakeholder groups, and information demonstrating that
the eligible entity can de-identify clinical trial data
consistent with the requirements of section 164.514 of title
45, Code of Federal Regulations (or successor regulations).
(3) A description of the system the eligible entity will use
to store and maintain such data, and information demonstrating
that this system will comply with applicable standards and
requirements for ensuring the security of the clinical trial
data.
(4) A certification that the eligible entity will allow only
registered users to access and use de-identified clinical trial
data, gathered from qualified clinical trials, and that the
eligible entity will allow each registered user to access and
use such data only after such registered user agrees in writing
to the terms described in (e)(4)(B), and such other carefully
controlled contractual terms as may be defined by the
Secretary.
(5) Evidence demonstrating the ability of the eligible entity
to ensure that registered users disseminate the results of the
research conducted in accordance with this section to
interested parties to serve as a guide to future medical
product development or scientific research.
(6) The plan of the eligible entity for securing funding for
the activities it would conduct under the clinical trial data
system agreement from governmental sources and private
foundations, entities, and individuals.
(7) Evidence demonstrating a proven track record of--
(A) being a neutral third party in working with
medical product manufacturers, academic institutions,
and the Food and Drug Administration; and
(B) having the ability to protect confidential data.
(8) An agreement that the eligible entity will work with the
Comptroller General of the United States for purposes of the
study and report under subsection (d).
(c) Extension, Expansion, Termination.--The Secretary, acting through
the Commissioner of Food and Drugs and the Director of the National
Institutes of Health, upon the expiration of the 7-year period referred
to in subsection (a), may extend (including permanently), expand, or
terminate the pilot program established under such subsection, in whole
or in part.
(d) Study and Report.--
(1) In general.--The Comptroller General of the United States
shall conduct a study and issue a report to the Congress and
the Secretary with respect to the pilot program established
under subsection (a), not later than 6 years after the date on
which the pilot program is established under subsection (a).
(2) Study.--The study under paragraph (1) shall--
(A) review the effectiveness of the pilot program
established under subsection (a); and
(B) be designed to formulate recommendations on
improvements to the program.
(3) Report.--The report under paragraph (1) shall contain at
least the following information:
(A) The new discoveries, research inquiries, or
clinical trials that have resulted from accessing
clinical trial data under the pilot program established
under subsection (a).
(B) The number of times scientists have accessed such
data, disaggregated by research area and clinical trial
phase.
(C) An analysis of whether the program has helped to
reduce adverse events in clinical trials.
(D) An analysis of whether scientists have raised any
concerns about the burden of having to share data with
the system established under the program and a
description, if any, of such burden.
(E) An analysis of privacy and data integrity
practices used in the program.
(e) Definitions.--In this section:
(1) The term ``eligible entity'' means an entity that has
experienced personnel with clinical and other technical
expertise in the biomedical sciences and biomedical ethics and
that is--
(A) an institution of higher education (as such term
is defined in section 1001 of the Higher Education Act
of 1965 (20 U.S.C. 1001)) or a consortium of such
institutions; or
(B) an organization described in section 501(c)(3) of
title 26 of the Internal Revenue Code of 1986 and
exempt from tax under section 501(a) of such title.
(2) The term ``medical product'' means a drug (as defined in
section 201(g) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 331(g))), a device (as defined in section 201(h) of such
Act (21 U.S.C. 331(h)), a biological product (as defined in
section 351 of the Public Health Service Act (42 U.S.C. 262)),
or any combination thereof.
(3) The term ``qualified clinical trial'' means a clinical
trial sponsored solely by an agency of the Department of Health
and Human Services with respect to a medical product--
(A) that--
(i) was approved or cleared under section
505, 510(k), or 515, or has an exemption for
investigational use in effect under section 505
or 520(m), of the Federal Food, Drug, and
Cosmetic Act (42 U.S.C. 301 et seq.); or
(ii) was licensed under section 351 of the
Public Health Service Act (42 U.S.C. 262) or
has an exemption for investigational use in
effect under such section 351; or
(B) that is an investigational product for which the
original development was discontinued and with respect
to which--
(i) no additional work to support approval,
licensure, or clearance of such medical product
is being or is planned to be undertaken by the
sponsor of the original development program,
its successors, assigns, or collaborators; and
(ii) the sponsor of the original
investigational development program has
provided its consent to the Secretary for
inclusion of data regarding such product in the
system established under this section.
(4) The term ``registered user'' means a scientific or
medical researcher who has--
(A) a legitimate biomedical research purpose for
accessing information from the clinical trials data
system and has appropriate qualifications to conduct
such research; and
(B) agreed in writing not to transfer to any other
person that is not a registered user de-identified
clinical trial data from qualified clinical trials
accessed through an eligible entity, use such data for
reasons not specified in the research proposal, or seek
to re-identify qualified clinical trial participants.
(5) The term ``Secretary'' means the Secretary of Health and
Human Services.
SEC. 1122. NATIONAL NEUROLOGICAL DISEASES SURVEILLANCE SYSTEM.
Part P of title III of the Public Health Service Act (42 U.S.C. 280g
et seq.) is amended by adding at the end the following:
``SEC. 399V-6 SURVEILLANCE OF NEUROLOGICAL DISEASES.
``(a) In General.--The Secretary, acting through the Director of the
Centers for Disease Control and Prevention and in coordination with
other agencies as determined appropriate by the Secretary, shall--
``(1) enhance and expand infrastructure and activities to
track the epidemiology of neurological diseases, including
multiple sclerosis and Parkinson's disease; and
``(2) incorporate information obtained through such
activities into a statistically sound, scientifically credible,
integrated surveillance system, to be known as the National
Neurological Diseases Surveillance System.
``(b) Research.--The Secretary shall ensure that the National
Neurological Diseases Surveillance System is designed in a manner that
facilitates further research on neurological diseases.
``(c) Content.--In carrying out subsection (a), the Secretary--
``(1) shall provide for the collection and storage of
information on the incidence and prevalence of neurological
diseases in the United States;
``(2) to the extent practicable, shall provide for the
collection and storage of other available information on
neurological diseases, such as information concerning--
``(A) demographics and other information associated
or possibly associated with neurological diseases, such
as age, race, ethnicity, sex, geographic location, and
family history;
``(B) risk factors associated or possibly associated
with neurological diseases, including genetic and
environmental risk factors; and
``(C) diagnosis and progression markers;
``(3) may provide for the collection and storage of
information relevant to analysis on neurological diseases, such
as information concerning--
``(A) the epidemiology of the diseases;
``(B) the natural history of the diseases;
``(C) the prevention of the diseases;
``(D) the detection, management, and treatment
approaches for the diseases; and
``(E) the development of outcomes measures; and
``(4) may address issues identified during the consultation
process under subsection (d).
``(d) Consultation.--In carrying out this section, the Secretary
shall consult with individuals with appropriate expertise, including--
``(1) epidemiologists with experience in disease surveillance
or registries;
``(2) representatives of national voluntary health
associations that--
``(A) focus on neurological diseases, including
multiple sclerosis and Parkinson's disease; and
``(B) have demonstrated experience in research, care,
or patient services;
``(3) health information technology experts or other
information management specialists;
``(4) clinicians with expertise in neurological diseases; and
``(5) research scientists with experience conducting
translational research or utilizing surveillance systems for
scientific research purposes.
``(e) Grants.--The Secretary may award grants to, or enter into
contracts or cooperative agreements with, public or private nonprofit
entities to carry out activities under this section.
``(f) Coordination With Other Federal, State, and Local Agencies.--
Subject to subsection (h), the Secretary shall make information and
analysis in the National Neurological Diseases Surveillance System
available, as appropriate--
``(1) to Federal departments and agencies, such as the
National Institutes of Health, the Food and Drug
Administration, the Centers for Medicare & Medicaid Services,
the Agency for Healthcare Research and Quality, the Department
of Veterans Affairs, and the Department of Defense; and
``(2) to State and local agencies.
``(g) Public Access.--Subject to subsection (h), the Secretary shall
make information and analysis in the National Neurological Diseases
Surveillance System available, as appropriate, to the public, including
researchers.
``(h) Privacy.--The Secretary shall ensure that privacy and security
protections applicable to the National Neurological Diseases
Surveillance System are at least as stringent as the privacy and
security protections under HIPAA privacy and security law (as defined
in section 3009(a)(2)).
``(i) Report.--Not later than 4 years after the date of the enactment
of this section, the Secretary shall submit a report to the Congress
concerning the implementation of this section. Such report shall
include information on--
``(1) the development and maintenance of the National
Neurological Diseases Surveillance System;
``(2) the type of information collected and stored in the
System;
``(3) the use and availability of such information, including
guidelines for such use; and
``(4) the use and coordination of databases that collect or
maintain information on neurological diseases.
``(j) Definition.--In this section, the term `national voluntary
health association' means a national nonprofit organization with
chapters, other affiliated organizations, or networks in States
throughout the United States.
``(k) Authorization of Appropriations.--To carry out this section,
there is authorized to be appropriated $5,000,000 for each of fiscal
years 2016 through 2020.''.
SEC. 1123. DATA ON NATURAL HISTORY OF DISEASES.
(a) Sense of Congress.--It is the sense of the Congress that studies
on the natural history of diseases can help to facilitate and expedite
the development of medical products for such diseases.
(b) Authority.--Part A of title II of the Public Health Service Act
(42 U.S.C. 202 et seq.) is amended by adding at the end the following:
``SEC. 229A. DATA ON NATURAL HISTORY OF DISEASES.
``(a) In General.--The Secretary may, for the purposes described in
subsection (b)--
``(1) participate in public-private partnerships engaged in
one or more activities specified in subsection (c); and
``(2) award grants to patient advocacy groups or other
organizations determined appropriate by the Secretary.
``(b) Purposes Described.--The purposes described in this subsection
are to establish or facilitate the collection, maintenance, analysis,
and interpretation of data regarding the natural history of diseases,
with a particular focus on rare diseases.
``(c) Activities of Public-Private Partnerships.--The activities of
public-private partnerships in which the Secretary may participate for
purposes of this section include--
``(1) cooperating with other entities that sponsor or
maintain disease registries, including disease registries and
disease registry platforms for rare diseases;
``(2) developing or enhancing a secure information technology
system that--
``(A) has the capacity to support data needs across a
wide range of disease studies;
``(B) is easily modified as knowledge is gained
during such studies; and
``(C) is capable of handling increasing amounts of
data as more studies are carried out; and
``(3) providing advice to clinical researchers, patient
advocacy groups, and other entities with respect to--
``(A) the design and conduct of disease studies;
``(B) the modification of any such ongoing studies;
and
``(C) addressing associated patient privacy issues.
``(d) Availability of Data on Natural History of Diseases.--Data
relating to the natural history of diseases obtained, aggregated, or
otherwise maintained by a public-private partnership in which the
Secretary participates under subsection (a) shall be made available,
consistent with otherwise applicable Federal and State privacy laws, to
the public (including patient advocacy groups, researchers, and drug
developers) to help to facilitate and expedite medical product
development programs.
``(e) Confidentiality.--Notwithstanding subsection (d), nothing in
this section authorizes the disclosure of any information that is a
trade secret or commercial or financial information that is privileged
or confidential and subject to section 552(b)(4) of title 5, United
States Code, or section 1905 of title 18, United States Code.
``(f) Authorization of Appropriations.--There is authorized to be
appropriated to carry out this section $5,000,000 for each of fiscal
years 2016 through 2020.''.
SEC. 1124. ACCESSING, SHARING, AND USING HEALTH DATA FOR RESEARCH
PURPOSES.
(a) In General.--(1) The HITECH Act (title XIII of division A of
Public Law 111-5) is amended by adding at the end of subtitle D of such
Act (42 U.S.C. 17921 et seq.) the following:
``PART 4--ACCESSING, SHARING, AND USING HEALTH DATA FOR RESEARCH
PURPOSES
``SEC. 13441. REFERENCES.
``In this part:
``(1) The rule.--References to `the Rule' refer to part 160
or part 164, as appropriate, of title 45, Code of Federal
Regulations (or any successor regulation).
``(2) Part 164.--References to a specified section of `part
164', refer to such specified section of part 164 of title 45,
Code of Federal Regulations (or any successor section).
``SEC. 13442. DEFINING HEALTH DATA RESEARCH AS PART OF HEALTH CARE
OPERATIONS.
``(a) In General.--Subject to subsection (b), the Secretary shall
revise or clarify the Rule to allow the use and disclosure of protected
health information by a covered entity for research purposes, including
studies whose purpose is to obtain generalizable knowledge, to be
treated as the use and disclosure of such information for health care
operations described in subparagraph (1) of the definition of health
care operations in section 164.501 of part 164.
``(b) Modifications to Rules for Disclosures for Health Care
Operations.--In applying section 164.506 of part 164 to the disclosure
of protected health information described in subsection (a)--
``(1) the Secretary shall revise or clarify the Rule so that
the disclosure may be made by the covered entity to only--
``(A) another covered entity for health care
operations (as defined in section 164.501 of part 164);
``(B) a business associate that has entered into a
contract under section 164.504(e) of part 164 with a
disclosing covered entity to perform health care
operations; or
``(C) a business associate that has entered into a
contract under section 164.504(e) of part 164 for the
purpose of data aggregation (as defined in section
164.501 of part 164); and
``(2) the Secretary shall further revise or clarify the Rule
so that the limitation specified by section 164.506(c)(4) of
part 164 does not apply to disclosures that are described by
subsection (a).
``(c) Rule of Construction.--This section shall not be construed as
prohibiting or restricting a use or disclosure of protected health
information for research purposes that is otherwise permitted under
part 164.
``SEC. 13443. TREATING DISCLOSURES OF PROTECTED HEALTH INFORMATION FOR
RESEARCH SIMILARLY TO DISCLOSURES OF SUCH
INFORMATION FOR PUBLIC HEALTH PURPOSES.
``(a) Remuneration.--The Secretary shall revise or clarify the Rule
so that disclosures of protected health information for research
purposes are not subject to the limitation on remuneration described in
section 164.502(a)(5)(ii)(B)(2)(ii) of part 164.
``(b) Permitted Uses and Disclosures.--The Secretary shall revise or
clarify the Rule so that research activities, including comparative
research activities, related to the quality, safety, or effectiveness
of a product or activity that is regulated by the Food and Drug
Administration are included as public health activities for purposes of
which a covered entity may disclose protected health information to a
person described in section 164.512(b)(1)(iii) of part 164.
``SEC. 13444. PERMITTING REMOTE ACCESS TO PROTECTED HEALTH INFORMATION
BY RESEARCHERS.
``The Secretary shall revise or clarify the Rule so that subparagraph
(B) of section 164.512(i)(1)(ii) of part 164 (prohibiting the removal
of protected health information by a researcher) shall not prohibit
remote access to health information by a researcher so long as--
``(1) appropriate security and privacy safeguards are
maintained by the covered entity and the researcher; and
``(2) the protected health information is not copied or
otherwise retained by the researcher.
``SEC. 13445. ALLOWING ONE-TIME AUTHORIZATION OF USE AND DISCLOSURE OF
PROTECTED HEALTH INFORMATION FOR RESEARCH PURPOSES.
``(a) In General.--The Secretary shall revise or clarify the Rule to
specify that an authorization for the use or disclosure of protected
health information, with respect to an individual, for future research
purposes shall be deemed to contain a sufficient description of the
purpose of the use or disclosure if the authorization--
``(1) sufficiently describes the purposes such that it would
be reasonable for the individual to expect that the protected
health information could be used or disclosed for such future
research;
``(2) either--
``(A) states that the authorization will expire on a
particular date or on the occurrence of a particular
event; or
``(B) states that the authorization will remain valid
unless and until it is revoked by the individual; and
``(3) provides instruction to the individual on how to revoke
such authorization at any time.
``(b) Revocation of Authorization.--The Secretary shall revise or
clarify the Rule to specify that, if an individual revokes an
authorization for future research purposes such as is described by
subsection (a), the covered entity may not make any further uses or
disclosures based on that authorization, except, as provided in
paragraph (b)(5) of section 164.508 of part 164, to the extent that the
covered entity has taken action in reliance on the authorization.''.
(2) The table of sections in section 13001(b) of such Act is amended
by adding at the end of the items relating to subtitle D the following
new items:
``Part 4--Accessing, Sharing, and Using Health Data for Research
Purposes
``Sec. 13441. References.
``Sec. 13442. Defining health data research as part of health care
operations.
``Sec. 13443. Treating disclosures of protected health information for
research similarly to disclosures of such information for public health
purposes.
``Sec. 13444. Permitting remote access to protected health information
by researchers.
``Sec. 13445. Allowing one-time authorization of use and disclosure of
protected health information for research purposes.''.
(b) Revision of Regulations.--Not later than 12 months after the date
of the enactment of this Act, the Secretary of Health and Human
Services shall revise and clarify the provisions of title 45, Code of
Federal Regulations, for consistency with part 4 of subtitle D of the
HITECH Act, as added by subsection (a).
Subtitle H--Council for 21st Century Cures
SEC. 1141. COUNCIL FOR 21ST CENTURY CURES.
Title II of the Public Health Service Act (42 U.S.C. 202 et seq.) is
amended by adding at the end the following:
``PART E--COUNCIL FOR 21ST CENTURY CURES
``SEC. 281. ESTABLISHMENT.
``A nonprofit corporation to be known as the Council for 21st Century
Cures (referred to in this part as the `Council') shall be established
in accordance with this section. The Council shall be a public-private
partnership headed by an Executive Director (referred to in this part
as the `Executive Director'), appointed by the members of the Board of
Directors. The Council shall not be an agency or instrumentality of the
United States Government.
``SEC. 281A. PURPOSE.
``The purpose of the Council is to accelerate the discovery,
development, and delivery in the United States of innovative cures,
treatments, and preventive measures for patients.
``SEC. 281B. DUTIES.
``For the purpose described in section 281A, the Council shall--
``(1) foster collaboration and coordination among the
entities that comprise the Council, including academia,
government agencies, industry, health care payors and
providers, patient advocates, and others engaged in the cycle
of discovery, development, and delivery of life-saving and
health-enhancing innovative interventions;
``(2) undertake communication and dissemination activities;
``(3) publish information on the activities funded under
section 281D;
``(4) establish a strategic agenda for accelerating the
discovery, development, and delivery in the United States of
innovative cures, treatments, and preventive measures for
patients;
``(5) identify gaps and opportunities within and across the
discovery, development, and delivery cycle;
``(6) develop and propose recommendations based on the gaps
and opportunities so identified;
``(7) facilitate the interoperability of the components of
the discovery, development, and delivery cycle;
``(8) propose recommendations that will facilitate
precompetitive collaboration;
``(9) identify opportunities to work with, but not duplicate
the efforts of, nonprofit organizations and other public-
private partnerships; and
``(10) identify opportunities for collaboration with
organizations operating outside of the United States, such as
the Innovative Medicines Initiative of the European Union.
``SEC. 281C. ORGANIZATION; ADMINISTRATION.
``(a) Board of Directors.--
``(1) Establishment.--
``(A) In general.--The Council shall have a Board of
Directors (in this part referred to as the `Board of
Directors'), which shall be composed of the ex officio
members under subparagraph (B) and the appointed
members under subparagraph (C). All members of the
Board shall be voting members.
``(B) Ex officio members.--The ex officio members of
the Board shall be the following individuals or their
designees:
``(i) The Director of the National Institutes
of Health.
``(ii) The Commissioner of Food and Drugs.
``(iii) The Administrator of the Centers for
Medicare & Medicaid Services.
``(iv) The heads of five other Federal
agencies deemed by the Secretary to be engaged
in biomedical research and development.
``(C) Appointed members.--The appointed members of
the Board shall consist of 17 individuals, of whom--
``(i) 8 shall be appointed by the Comptroller
General of the United States from a list of
nominations submitted by leading trade
associations--
``(I) 4 of whom shall be
representatives of the
biopharmaceutical industry;
``(II) 2 of whom shall be
representatives of the medical device
industry; and
``(III) 2 of whom shall be
representatives of the information and
digital technology industry; and
``(ii) 9 shall be appointed by the
Comptroller General of the United States, after
soliciting nominations--
``(I) 2 of whom shall be
representatives of academic
researchers;
``(II) 3 of whom shall be
representatives of patients;
``(III) 2 of whom shall be
representatives of health care
providers; and
``(IV) 2 of whom shall be
representatives of health care plans
and insurers.
``(D) Chair.--The Chair of the Board shall be
selected by the members of the Board by majority vote
from among the members of the Board.
``(2) Terms and vacancies.--
``(A) In general.--The term of office of each member
of the Board appointed under paragraph (1)(C) shall be
5 years.
``(B) Vacancy.--Any vacancy in the membership of the
Board--
``(i) shall not affect the power of the
remaining members to execute the duties of the
Board; and
``(ii) shall be filled by appointment by the
appointed members described in paragraph (1)(C)
by majority vote.
``(C) Partial term.--If a member of the Board does
not serve the full term applicable under subparagraph
(A), the individual appointed under subparagraph (B) to
fill the resulting vacancy shall be appointed for the
remainder of the term of the predecessor of the
individual.
``(3) Responsibilities.--Not later than 90 days after the
date on which the Council is incorporated and its Board of
Directors is fully constituted, the Board of Directors shall
establish bylaws and policies for the Council that--
``(A) are published in the Federal Register and
available for public comment;
``(B) establish policies for the selection and, as
applicable, appointment of--
``(i) the officers, employees, agents, and
contractors of the Council; and
``(ii) the members of any committees of the
Council;
``(C) establish policies, including ethical
standards, for the conduct of programs and other
activities under section 281D; and
``(D) establish specific duties of the Executive
Director.
``(4) Meetings.--
``(A) In general.--The Board of Directors shall--
``(i) meet on a quarterly basis; and
``(ii) submit to Congress, and make publicly
available, the minutes of such meetings.
``(B) Agenda.--The Board of Directors shall, not
later than 3 months after the incorporation of the
Council--
``(i) issue an agenda (in this part referred
to as the `agenda') outlining how the Council
will achieve the purpose described in section
281A; and
``(ii) annually thereafter, in consultation
with the Executive Director, review and update
such agenda.
``(b) Appointment and Incorporation.--Not later than 6 months after
the date of enactment of the 21st Century Cures Act--
``(1) the Comptroller General of the United States shall
appoint the appointed members of the Board of Directors under
subsection (a)(1)(C); and
``(2) the ex officio members of the Board of Directors under
subsection (a)(1)(B) shall serve as incorporators and shall
take whatever actions are necessary to incorporate the Council.
``(c) Nonprofit Status.--In carrying out this part, the Board of
Directors shall establish such policies and bylaws, and the Executive
Director shall carry out such activities, as may be necessary to ensure
that the Council maintains status as an organization that--
``(1) is described in subsection (c)(3) of section 501 of the
Internal Revenue Code of 1986; and
``(2) is, under subsection (a) of such section, exempt from
taxation.
``(d) Executive Director.--The Executive Director shall--
``(1) be the chief executive officer of the Council; and
``(2) subject to the oversight of the Board of Directors, be
responsible for the day-to-day management of the Council.
``SEC. 281D. OPERATIONAL ACTIVITIES AND ASSISTANCE.
``(a) In General.--The Council shall establish a sufficient
operational infrastructure to fulfill the duties specified in section
281B.
``(b) Private Sector Matching Funds.--The Council may accept
financial or in-kind support from participating entities or private
foundations or organizations when such support is deemed appropriate.
``SEC. 281E. TERMINATION; REPORT.
``(a) In General.--The Council shall terminate on September 30, 2023.
``(b) Report.--Not later than one year after the date on which the
Council is established and each year thereafter, the Executive Director
shall submit to the appropriate congressional committees a report on
the performance of the Council. In preparing such report, the Council
shall consult with a nongovernmental consultant with appropriate
expertise.
``SEC. 281F. FUNDING.
``For the each of fiscal years 2016 through 2023, there is authorized
to be appropriated $10,000,000 to the Council for purposes of carrying
out the duties of the Council under this part.''.
TITLE II--DEVELOPMENT
Subtitle A--Patient-Focused Drug Development
SEC. 2001. DEVELOPMENT AND USE OF PATIENT EXPERIENCE DATA TO ENHANCE
STRUCTURED RISK-BENEFIT ASSESSMENT FRAMEWORK.
(a) In General.--Section 505 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355) is amended--
(1) in subsection (d), by striking ``The Secretary shall
implement'' and all that follows through ``premarket approval
of a drug.''; and
(2) by adding at the end the following new subsections:
``(x) Structured Risk-Benefit Assessment Framework.--
``(1) In general.--The Secretary shall implement a structured
risk-benefit assessment framework in the new drug approval
process--
``(A) to facilitate the balanced consideration of
benefits and risks; and
``(B) to develop and implement a consistent and
systematic approach to the discussion of, regulatory
decisionmaking with respect to, and the communication
of, the benefits and risks of new drugs.
``(2) Rule of construction.--Nothing in paragraph (1) shall
alter the criteria for evaluating an application for premarket
approval of a drug.
``(y) Development and Use of Patient Experience Data To Enhance
Structured Risk-Benefit Assessment Framework.--
``(1) In general.--Not later than two years after the date of
the enactment of this subsection, the Secretary shall establish
and implement processes under which--
``(A) an entity seeking to develop patient experience
data may submit to the Secretary--
``(i) initial research concepts for feedback
from the Secretary; and
``(ii) with respect to patient experience
data collected by the entity, draft guidance
documents, completed data, and summaries and
analyses of such data;
``(B) the Secretary may request such an entity to
submit such documents, data, and summaries and
analyses; and
``(C) patient experience data may be developed and
used to enhance the structured risk-benefit assessment
framework under subsection (x).
``(2) Patient experience data.--In this subsection, the term
`patient experience data' means data collected by patients,
parents, caregivers, patient advocacy organizations, disease
research foundations, medical researchers, research sponsors,
or other parties determined appropriate by the Secretary that
is intended to facilitate or enhance the Secretary's risk-
benefit assessments, including information about the impact of
a disease or a therapy on patients' lives.''.
(b) Guidance.--
(1) In general.--The Secretary of Health and Human Services
shall publish guidance on the implementation of subsection (y)
of section 505 of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355), as added by subsection (a). Such guidance shall
include--
(A) with respect to draft guidance documents, data,
or summaries and analyses submitted to the Secretary
under paragraph (1)(A) of such subsection, guidance--
(i) specifying the timelines for the review
of such documents, data, or summaries and
analyses by the Secretary; and
(ii) on how the Secretary will use such
documents, data, or summaries and analyses to
update any guidance documents published under
this subsection or publish new guidance;
(B) with respect to the collection and analysis of
patient experience data (as defined in paragraph (2) of
such subsection (y)), guidance on--
(i) methodological considerations for the
collection of patient experience data, which
may include structured approaches to gathering
information on--
(I) the experience of a patient
living with a particular disease;
(II) the burden of living with or
managing the disease;
(III) the impact of the disease on
daily life and long-term functioning;
and
(IV) the effect of current
therapeutic options on different
aspects of the disease; and
(ii) the establishment and maintenance of
registries designed to increase understanding
of the natural history of a disease;
(C) methodological approaches that may be used to
assess patients' beliefs with respect to the benefits
and risks in the management of the patient's disease;
and
(D) methodologies, standards, and potential
experimental designs for patient-reported outcomes.
(2) Timing.--Not later than 3 years after the date of the
enactment of this Act, the Secretary of Health and Human
Services shall issue draft guidance on the implementation of
subsection (y) of section 505 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355), as added by subsection (a). The
Secretary shall issue final guidance on the implementation of
such subsection not later than one year after the date on which
the comment period for the draft guidance closes.
(3) Workshops.--
(A) In general.--Not later than 6 months after the
date of the enactment of this Act and once every 6
months during the following 12-month period, the
Secretary of Health and Human Services shall convene a
workshop to obtain input regarding methodologies for
developing the guidance under paragraph (1), including
the collection of patient experience data.
(B) Attendees.--A workshop convened under this
paragraph shall include--
(i) patients;
(ii) representatives from patient advocacy
organizations, biopharmaceutical companies, and
disease research foundations;
(iii) representatives of the reviewing
divisions of the Food and Drug Administration;
and
(iv) methodological experts with significant
expertise in patient experience data.
(4) Public meeting.--Not later than 90 days after the date on
which the draft guidance is published under this subsection,
the Secretary of Health and Human Services shall convene a
public meeting to solicit input on the guidance.
Subtitle B--Qualification and Use of Drug Development Tools
SEC. 2021. QUALIFICATION OF DRUG DEVELOPMENT TOOLS.
(a) Findings.--Congress finds the following:
(1) Development of new drugs has become increasingly
challenging and resource intensive.
(2) Development of drug development tools can benefit the
availability of new medical therapies by helping to translate
scientific discoveries into clinical applications.
(3) Biomedical research consortia (as defined in section
507(f) of the Federal Food, Drug, and Cosmetic Act, as added by
subsection (c)) can play a valuable role in helping to develop
and qualify drug development tools.
(b) Sense of Congress.--It is the sense of Congress that--
(1) Congress should promote and facilitate a collaborative
effort among the biomedical research consortia described in
subsection (a)(3)--
(A) to develop, through a transparent public process,
data standards and scientific approaches to data
collection accepted by the medical and clinical
research community for purposes of qualifying drug
development tools;
(B) to coordinate efforts toward developing and
qualifying drug development tools in key therapeutic
areas; and
(C) to encourage the development of accessible
databases for collecting relevant drug development tool
data for such purposes; and
(2) an entity seeking to qualify a drug development tool
should be encouraged, in addition to consultation with the
Secretary, to consult with biomedical research consortia and
other individuals and entities with expert knowledge and
insights that may assist the requestor and benefit the process
for such qualification.
(c) Qualification of Drug Development Tools.--Chapter V of the
Federal Food, Drug, and Cosmetic Act is amended by inserting after
section 506F the following new section:
``SEC. 507. QUALIFICATION OF DRUG DEVELOPMENT TOOLS.
``(a) Process for Qualification.--
``(1) In general.--The Secretary shall establish a process
for the qualification of drug development tools for a proposed
context of use under which--
``(A)(i) a requestor initiates such process by
submitting a letter of intent to the Secretary; and
``(ii) the Secretary shall accept or decline to
accept such letter of intent;
``(B)(i) if the Secretary accepts the letter of
intent, a requestor shall submit a qualification plan
to the Secretary; and
``(ii) the Secretary shall accept or decline to
accept the qualification plan; and
``(C)(i) if the Secretary accepts the qualification
plan, the requestor submits to the Secretary a full
qualification package;
``(ii) the Secretary shall determine whether to
accept such qualification package for review; and
``(iii) if the Secretary accepts such qualification
package for review, the Secretary shall conduct such
review in accordance with this section.
``(2) Acceptance and review of submissions.--
``(A) In general.--The succeeding provisions of this
paragraph shall apply with respect to the treatment of
a letter of intent, a qualification plan, or a full
qualification package submitted under paragraph (1)
(referred to in this paragraph as `qualification
submissions').
``(B) Acceptance factors; nonacceptance.--The
Secretary shall determine whether to accept a
qualification submission based on factors which may
include the scientific merit of the submission and the
available resources of the Food and Drug Administration
to review the qualification submission. A determination
not to accept a submission under paragraph (1) shall
not be construed as a final determination by the
Secretary under this section regarding the
qualification of a drug development tool for its
proposed context of use.
``(C) Prioritization of qualification review.--The
Secretary may prioritize the review of a full
qualification package submitted under paragraph (1)
with respect to a drug development tool, based on
factors determined appropriate by the Secretary,
including--
``(i) as applicable, the severity, rarity, or
prevalence of the disease or condition targeted
by the drug development tool and the
availability or lack of alternative treatments
for such disease or condition; and
``(ii) the identification, by the Secretary
or by biomedical research consortia and other
expert stakeholders, of such a drug development
tool and its proposed context of use as a
public health priority.
``(D) Engagement of external experts.--The Secretary
may, for purposes of the review of qualification
submissions, through the use of cooperative agreements,
grants, or other appropriate mechanisms, consult with
biomedical research consortia and may consider the
recommendations of such consortia with respect to the
review of any qualification plan submitted under
paragraph (1) or the review of any full qualification
package under paragraph (3).
``(3) Review of full qualification package.--The Secretary
shall--
``(A) conduct a comprehensive review of a full
qualification package accepted under paragraph (1)(C);
and
``(B) determine whether the drug development tool at
issue is qualified for its proposed context of use.
``(4) Qualification.--The Secretary shall determine whether a
drug development tool is qualified for a proposed context of
use based on the scientific merit of a full qualification
package reviewed under paragraph (3).
``(b) Effect of Qualification.--
``(1) In general.--A drug development tool determined to be
qualified under subsection (a)(4) for a proposed context of use
specified by the requestor may be used by any person in such
context of use for the purposes described in paragraph (2).
``(2) Use of a drug development tool.--Subject to paragraph
(3), a drug development tool qualified under this section may
be used for--
``(A) supporting or obtaining approval or licensure
(as applicable) of a drug or biological product
(including in accordance with section 506(c)) under
section 505 of this Act or section 351 of the Public
Health Service Act; or
``(B) supporting the investigational use of a drug or
biological product under section 505(i) of this Act or
section 351(a)(3) of the Public Health Service Act.
``(3) Rescission or modification.--
``(A) In general.--The Secretary may rescind or
modify a determination under this section to qualify a
drug development tool if the Secretary determines that
the drug development tool is not appropriate for the
proposed context of use specified by the requestor.
Such a determination may be based on new information
that calls into question the basis for such
qualification.
``(B) Meeting for review.--If the Secretary rescinds
or modifies under subparagraph (A) a determination to
qualify a drug development tool, the requestor involved
shall be granted a request for a meeting with the
Secretary to discuss the basis of the Secretary's
decision to rescind or modify the determination before
the effective date of the rescission or modification.
``(c) Transparency.--
``(1) In general.--Subject to paragraph (3), the Secretary
shall make publicly available, and update on at least a
biannual basis, on the Internet website of the Food and Drug
Administration the following:
``(A) Information with respect to each qualification
submission under the qualification process under
subsection (a), including--
``(i) the stage of the review process
applicable to the submission;
``(ii) the date of the most recent change in
stage status;
``(iii) whether the external scientific
experts were utilized in the development of a
qualification plan or the review of a full
qualification package; and
``(iv) submissions from requestors under the
qualification process under subsection (a),
including any data and evidence contained in
such submissions, and any updates to such
submissions.
``(B) The Secretary's formal written determinations
in response to such qualification submissions.
``(C) Any rescissions or modifications under
subsection (b)(3) of a determination to qualify a drug
development tool.
``(D) Summary reviews that document conclusions and
recommendations for determinations to qualify drug
development tools under subsection (a).
``(E) A comprehensive list of--
``(i) all drug development tools qualified
under subsection (a); and
``(ii) all surrogate endpoints which were the
basis of approval or licensure (as applicable)
of a drug or biological product (including in
accordance with section 506(c)) under section
505 of this Act or section 351 of the Public
Health Service Act.
``(2) Relation to trade secrets act.--Information made
publicly available by the Secretary under paragraph (1) shall
be considered a disclosure authorized by law for purposes of
section 1905 of title 18, United States Code.
``(3) Applicability.--Nothing in this section shall be
construed as authorizing the Secretary to disclose any
information contained in an application submitted under section
505 of this Act or section 351 of the Public Health Service Act
that is confidential commercial or trade secret information
subject to section 552(b)(4) of title 5, United States Code, or
section 1905 of title 18, United States Code.
``(d) Rule of Construction.--Nothing in this section shall be
construed--
``(1) to alter the standards of evidence under subsection (c)
or (d) of section 505, including the substantial evidence
standard in such subsection (d), or under section 351 of the
Public Health Service Act (as applicable); or
``(2) to limit the authority of the Secretary to approve or
license products under this Act or the Public Health Service
Act, as applicable (as in effect before the date of the
enactment of the 21st Century Cures Act).
``(e) Authorization of Appropriations.--There are authorized to be
appropriated to carry out this section, $10,000,000 for each of fiscal
years 2016 through 2020.
``(f) Definitions.--In this section:
``(1) Biomarker.--(A) The term `biomarker' means a
characteristic (such as a physiologic, pathologic, or anatomic
characteristic or measurement) that is objectively measured and
evaluated as an indicator of normal biologic processes,
pathologic processes, or biological responses to a therapeutic
intervention; and
``(B) such term includes a surrogate endpoint.
``(2) Biomedical research consortia.--The term `biomedical
research consortia' means collaborative groups that may take
the form of public-private partnerships and may include
government agencies, institutions of higher education (as
defined in section 101(a) of the Higher Education Act of 1965,
patient advocacy groups, industry representatives, clinical and
scientific experts, and other relevant entities and
individuals.
``(3) Clinical outcome assessment.--(A) The term `clinical
outcome assessment' means a measurement of a patient's
symptoms, overall mental state, or the effects of a disease or
condition on how the patient functions; and
``(B) such term includes a patient-reported outcome.
``(4) Context of use.--The term `context of use' means, with
respect to a drug development tool, a statement that describes
the circumstances under which the drug development tool is to
be used in drug development and regulatory review.
``(5) Drug development tool.--The term `drug development
tool' includes--
``(A) a biomarker;
``(B) a clinical outcome assessment; and
``(C) any other method, material, or measure that the
Secretary determines aids drug development and
regulatory review for purposes of this section.
``(6) Patient-reported outcome.--The term `patient-reported
outcome' means a measurement based on a report from a patient
regarding the status of the patient's health condition without
amendment or interpretation of the patient's report by a
clinician or any other person.
``(7) Qualification.--The terms `qualification' and
`qualified' mean a determination by the Secretary that a drug
development tool and its proposed context of use can be relied
upon to have a specific interpretation and application in drug
development and regulatory review under this Act.
``(8) Requestor.--The term `requestor' means an entity or
entities, including a drug sponsor or a biomedical research
consortia, seeking to qualify a drug development tool for a
proposed context of use under this section.
``(9) Surrogate endpoint.--The term `surrogate endpoint'
means a marker, such as a laboratory measurement, radiographic
image, physical sign, or other measure, that is not itself a
direct measurement of clinical benefit, and--
``(A) is known to predict clinical benefit and could
be used to support traditional approval of a drug or
biological product; or
``(B) is reasonably likely to predict clinical
benefit and could be used to support the accelerated
approval of a drug or biological product in accordance
with section 506(c).''.
(d) Guidance.--
(1) In general.--The Secretary of Health and Human Services
shall, in consultation with biomedical research consortia (as
defined in subsection (f) of section 507 the Federal Food,
Drug, and Cosmetic Act (as added by subsection (c))) and other
interested parties through a collaborative public process,
issue guidance to implement such section 507 that--
(A) provides a conceptual framework describing
appropriate standards and scientific approaches to
support the development of biomarkers delineated under
the taxonomy established under paragraph (3);
(B) makes recommendations for demonstrating that a
surrogate endpoint is reasonably likely to predict
clinical benefit for the purpose of supporting the
accelerated approval of a drug under section 506(c) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
356(c));
(C) with respect to the qualification process under
such section 507--
(i) describes the requirements that entities
seeking to qualify a drug development tool
under such section shall observe when engaging
in such process;
(ii) outlines reasonable timeframes for the
Secretary's review of letters, qualification
plans, or full qualification packages submitted
under such process; and
(iii) establishes a process by which such
entities or the Secretary may consult with
biomedical research consortia and other
individuals and entities with expert knowledge
and insights that may assist the Secretary in
the review of qualification plans and full
qualification submissions under such section;
and
(D) includes such other information as the Secretary
determines appropriate.
(2) Timing.--Not later than 24 months after the date of the
enactment of this Act, the Secretary of Health and Human
Services shall issue draft guidance under paragraph (1) on the
implementation of section 507 of the Federal Food, Drug, and
Cosmetic Act (as added by subsection (c)). The Secretary shall
issue final guidance on the implementation of such section not
later than 6 months after the date on which the comment period
for the draft guidance closes.
(3) Taxonomy.--
(A) In general.--For purposes of informing guidance
under this subsection, the Secretary of Health and
Human Services shall, in consultation with biomedical
research consortia and other interested parties through
a collaborative public process, establish a taxonomy
for the classification of biomarkers (and related
scientific concepts) for use in drug development.
(B) Public availability.--Not later than 12 months
after the date of the enactment of this Act, the
Secretary of Health and Human Services shall make such
taxonomy publicly available in draft form for public
comment. The Secretary shall finalize the taxonomy not
later than 12 months after the close of the public
comment period.
(e) Meeting and Report.--
(1) Meeting.--Not later than 12 months after the date of the
enactment of this Act, the Secretary of Health and Human
Services shall convene a public meeting to describe and solicit
public input regarding the qualification process under section
507 of the Federal Food, Drug, and Cosmetic Act, as added by
subsection (c).
(2) Report.--Not later than 5 years after the date of the
enactment of this Act, the Secretary shall make publicly
available on the Internet website of the Food and Drug
Administration a report. Such report shall include, with
respect to the qualification process under section 507 of the
Federal Food, Drug, and Cosmetic Act, as added by subsection
(c), information on--
(A) the number of requests submitted, as a letter of
intent, for qualification of a drug development tool
(as defined in subsection (f) of such section);
(B) the number of such requests accepted and
determined to be eligible for submission of a
qualification plan or full qualification package (as
such terms are defined in such subsection),
respectively;
(C) the number of such requests for which external
scientific experts were utilized in the development of
a qualification plan or review of a full qualification
package; and
(D) the number of qualification plans and full
qualification packages, respectively, submitted to the
Secretary; and
(3) the drug development tools qualified through such
qualification process, specified by type of tool, such as a
biomarker or clinical outcome assessment (as such terms are
defined in subsection (f) of such section 507).
SEC. 2022. ACCELERATED APPROVAL DEVELOPMENT PLAN.
(a) In General.--Section 506 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 356) is amended by adding the following subsection:
``(g) Accelerated Approval Development Plan.--
``(1) In general.--In the case of a drug that the Secretary
determines may be eligible for accelerated approval in
accordance with subsection (c), the sponsor of such drug may
request, at any time after the submission of an application for
the investigation of the drug under section 505(i) of this Act
or section 351(a)(3) of the Public Health Service Act, that the
Secretary agree to an accelerated approval development plan
described in paragraph (2).
``(2) Plan described.--A plan described in this paragraph,
with respect to a drug described in paragraph (1), is an
accelerated approval development plan, which shall include
agreement on--
``(A) the surrogate endpoint to be assessed under
such plan;
``(B) the design of the study that will utilize the
surrogate endpoint; and
``(C) the magnitude of the effect of the drug on the
surrogate endpoint that is the subject of the agreement
that would be sufficient to form the primary basis of a
claim that the drug is effective.
``(3) Modification; termination.--The Secretary may require
the sponsor of a drug that is the subject of an accelerated
approval development plan to modify or terminate the plan if
additional data or information indicates that--
``(A) the plan as originally agreed upon is no longer
sufficient to demonstrate the safety and effectiveness
of the drug involved; or
``(B) the drug is no longer eligible for accelerated
approval under subsection (c).
``(4) Sponsor consultation.--If the Secretary requires the
modification or termination of an accelerated approval
development plan under paragraph (3), the sponsor shall be
granted a request for a meeting to discuss the basis of the
Secretary's decision before the effective date of the
modification or termination.
``(5) Definition.--In this section, the term `accelerated
approval development plan' means a development plan agreed upon
by the Secretary and the sponsor submitting the plan that
contains study parameters for the use of a surrogate endpoint
that--
``(A) is reasonably likely to predict clinical
benefit; and
``(B) is intended to be the basis of the accelerated
approval of a drug in accordance with subsection
(c).''.
(b) Technical Amendments.--Section 506 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 356) is amended--
(1) by striking ``(f) Awareness Efforts'' and inserting ``(e)
Awareness Efforts''; and
(2) by striking ``(e) Construction'' and inserting ``(f)
Construction''.
Subtitle C--FDA Advancement of Precision Medicine
SEC. 2041. PRECISION MEDICINE GUIDANCE AND OTHER PROGRAMS OF FOOD AND
DRUG ADMINISTRATION.
Chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351
et seq.) is amended by adding at the end the following:
``Subchapter J--Precision Medicine
``SEC. 591. GENERAL AGENCY GUIDANCE ON PRECISION MEDICINE.
``(a) In General.--The Secretary shall issue and periodically update
guidance to assist sponsors in the development of a precision drug or
biological product. Such guidance shall--
``(1) define the term `precision drug or biological product';
and
``(2) address the topics described in subsection (b).
``(b) Certain Issues.--The topics to be addressed by guidance under
subsection (a) are--
``(1) the evidence needed to support the use of biomarkers
(as defined in section 507(e)) that identify subsets of
patients as likely responders to therapies in order to
streamline the conduct of clinical trials;
``(2) recommendations for the design of studies to
demonstrate the validity of a biomarker as a predictor of drug
or biological product response;
``(3) the manner and extent to which a benefit-risk
assessment may be affected when clinical trials are limited to
patient population subsets that are identified using
biomarkers;
``(4) the development of companion diagnostics in the context
of a drug development program; and
``(5) considerations for developing biomarkers that inform
prescribing decisions for a drug or biological product, and
when information regarding a biomarker may be included in the
approved prescription labeling for a precision drug or
biological product.
``(c) Date Certain for Initial Guidance.--The Secretary shall issue
guidance under subsection (a) not later than 18 months after the date
of the enactment of the 21st Century Cures Act.
``SEC. 592. PRECISION MEDICINE REGARDING ORPHAN-DRUG AND EXPEDITED-
APPROVAL PROGRAMS.
``(a) In General.--In the case of a precision drug or biological
product that is the subject of an application submitted under section
505(b)(1), or section 351(a) of the Public Health Service Act, for the
treatment of a serious or life-threatening disease or condition and has
been designated under section 526 as a drug for a rare disease or
condition, the Secretary may--
``(1) consistent with applicable standards for approval, rely
upon data or information previously submitted by the sponsor of
the precision drug or biological product, or another sponsor,
provided that the sponsor of the precision drug or biological
product has obtained a contractual right of reference to such
other sponsor's data and information, in an application
approved under section 505(c) or licensed under section 351(a)
of the Public Health Service Act, as applicable--
``(A) for a different drug or biological product; or
``(B) for a different indication for such precision
drug or biological product,
in order to expedite clinical development for a precision drug
or biological product that is using the same or similar
approach as that used to support approval of the prior approved
application or license, as appropriate; and
``(2) as appropriate, consider the application for approval
of such precision drug or biological product to be eligible for
expedited review and approval programs described in section
506, including accelerated approval in accordance with
subsection (c) of such section.
``(b) Rule of Construction.--Nothing in this section shall be
construed to--
``(1) limit the authority of the Secretary to approve
products pursuant to this Act and the Public Health Service Act
as authorized prior to the date of enactment of this section;
or
``(2) confer any new rights, beyond those authorized under
this Act prior to enactment of this section, with respect to a
sponsor's ability to reference information contained in another
application submitted under section 505(b)(1) of this Act or
section 351(a) of the Public Health Service Act.''.
Subtitle D--Modern Trial Design and Evidence Development
SEC. 2061. BROADER APPLICATION OF BAYESIAN STATISTICS AND ADAPTIVE
TRIAL DESIGNS.
(a) Proposals for Use of Innovative Statistical Methods in Clinical
Protocols for Drugs and Biological Products.--For purposes of assisting
sponsors in incorporating adaptive trial design and Bayesian methods
into proposed clinical protocols and applications for new drugs under
section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355)
and biological products under section 351 of the Public Health Service
Act (42 U.S.C. 262), the Secretary shall conduct a public meeting and
issue guidance in accordance with subsection (b).
(b) Guidance Addressing Use of Adaptive Trial Designs and Bayesian
Methods.--
(1) In general.--The Secretary of Health and Human Services,
acting through the Commissioner of Food and Drugs (in this
subsection referred to as the ``Secretary''), shall--
(A) update and finalize the draft guidance addressing
the use of adaptive trial design for drugs and
biological products; and
(B) issue draft guidance on the use of Bayesian
methods in the development and regulatory review and
approval or licensure of drugs and biological products.
(2) Contents.--The guidances under paragraph (1) shall
address--
(A) the use of adaptive trial designs and Bayesian
methods in clinical trials, including clinical trials
proposed or submitted to help to satisfy the
substantial evidence standard under section 505(d) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355(d));
(B) how sponsors may obtain feedback from the
Secretary on technical issues related to modeling and
simulations prior to--
(i) completion of such modeling or
simulations; or
(ii) the submission of resulting information
to the Secretary;
(C) the types of quantitative and qualitative
information that should be submitted for review; and
(D) recommended analysis methodologies.
(3) Public meeting.--Prior to updating or developing the
guidances required by paragraph (1), the Secretary shall
consult with stakeholders, including representatives of
regulated industry, academia, patient advocacy organizations,
and disease research foundations, through a public meeting to
be held not later than 1 year after the date of enactment of
this Act.
(4) Schedule.--The Secretary shall publish--
(A) the final guidance required by paragraph (1)(A)
not later than 18 months after the date of the public
meeting required by paragraph (3); and
(B) the guidance required by paragraph (1)(B) not
later than 48 months after the date of the public
meeting required by paragraph (3).
SEC. 2062. UTILIZING EVIDENCE FROM CLINICAL EXPERIENCE.
Chapter V of the Federal Food, Drug, and Cosmetic Act is amended by
inserting after section 505E of such Act (21 U.S.C. 355f) the
following:
``SEC. 505F. UTILIZING EVIDENCE FROM CLINICAL EXPERIENCE.
``(a) In General.--The Secretary shall establish a program to
evaluate the potential use of evidence from clinical experience--
``(1) to help to support the approval of a new indication for
a drug approved under section 505(b); and
``(2) to help to support or satisfy postapproval study
requirements.
``(b) Evidence From Clinical Experience Defined.--In this section,
the term `evidence from clinical experience' means data regarding the
usage, or the potential benefits or risks, of a drug derived from
sources other than randomized clinical trials, including from
observational studies, registries, and therapeutic use.
``(c) Program Framework.--
``(1) In general.--Not later than 18 months after the date of
enactment of this section, the Secretary shall establish a
draft framework for implementation of the program under this
section.
``(2) Contents of framework.--The framework shall include
information describing--
``(A) the current sources of data developed through
clinical experience, including ongoing safety
surveillance, registry, claims, and patient-centered
outcomes research activities;
``(B) the gaps in current data collection activities;
``(C) the current standards and methodologies for
collection and analysis of data generated through
clinical experience; and
``(D) the priority areas, remaining challenges, and
potential pilot opportunities that the program
established under this section will address.
``(3) Consultation.--
``(A) In general.--In developing the program
framework under this subsection, the Secretary shall
consult with regulated industry, academia, medical
professional organizations, representatives of patient
advocacy organizations, disease research foundations,
and other interested parties.
``(B) Process.--The consultation under subparagraph
(A) may be carried out through approaches such as--
``(i) a public-private partnership with the
entities described in such subparagraph in
which the Secretary may participate; or
``(ii) a contract, grant, or other
arrangement, as determined appropriate by the
Secretary with such a partnership or an
independent research organization.
``(d) Program Implementation.--The Secretary shall, not later than 24
months after the date of enactment of this section and in accordance
with the framework established under subsection (c), implement the
program to evaluate the potential use of evidence from clinical
experience.
``(e) Guidance for Industry.--The Secretary shall--
``(1) utilize the program established under subsection (a),
its activities, and any subsequent pilots or written reports,
to inform a guidance for industry on--
``(A) the circumstances under which sponsors of drugs
and the Secretary may rely on evidence from clinical
experience for the purposes described in subsection
(a)(1) or (a)(2); and
``(B) the appropriate standards and methodologies for
collection and analysis of evidence from clinical
experience submitted for such purposes;
``(2) not later than 36 months after the date of enactment of
this section, issue draft guidance for industry as described in
paragraph (1); and
``(3) not later than 48 months after the date of enactment of
this section, after providing an opportunity for public comment
on the draft guidance, issue final guidance.
``(f) Rule of Construction.--
``(1) Subject to paragraph (2), nothing in this section
prohibits the Secretary from using evidence from clinical
experience for purposes not specified in this section, provided
the Secretary determines that sufficient basis exists for any
such nonspecified use.
``(2) This section shall not be construed to alter--
``(A) the standards of evidence under--
``(i) subsection (c) or (d) of section 505,
including the substantial evidence standard in
such subsection (d); or
``(ii) section 351(a) of the Public Health
Service Act; or
``(B) the Secretary's authority to require
postapproval studies or clinical trials, or the
standards of evidence under which studies or trials are
evaluated.
``SEC. 505G. COLLECTING EVIDENCE FROM CLINICAL EXPERIENCE THROUGH
TARGETED EXTENSIONS OF THE SENTINEL SYSTEM.
``(a) In General.--The Secretary shall, in parallel to implementing
the program established under section 505F and in order to build
capacity for utilizing the evidence from clinical experience described
in that section, identify and execute pilot demonstrations to extend
existing use of the Sentinel System surveillance infrastructure
authorized under section 505(k).
``(b) Pilot Demonstrations.--
``(1) In general.--The Secretary--
``(A) shall design and implement pilot demonstrations
to utilize data captured through the Sentinel System
surveillance infrastructure authorized under section
505(k) for purposes of, as appropriate--
``(i) generating evidence from clinical
experience to improve characterization or
assessment of risks or benefits of a drug
approved under section 505(c);
``(ii) protecting the public health; or
``(iii) advancing patient-centered care; and
``(B) may make strategic linkages with sources of
complementary public health data and infrastructure the
Secretary determines appropriate and necessary.
``(2) Consultation.--In developing the pilot demonstrations
under this subsection, the Secretary shall--
``(A) consult with regulated industry, academia,
medical professional organizations, representatives of
patient advocacy organizations, disease research
foundations, and other interested parties through a
public process; and
``(B) develop a framework to promote appropriate
transparency and dialogue about research conducted
under these pilot demonstrations, including by--
``(i) providing adequate notice to a sponsor
of a drug approved under section 505 or section
351 of the Public Health Service Act of the
Secretary's intent to conduct analyses of such
sponsor's drug or drugs under these pilot
demonstrations;
``(ii) providing adequate notice of the
findings related to analyses described in
clause (i) and an opportunity for the sponsor
of such drug or drugs to comment on such
findings; and
``(iii) ensuring the protection from public
disclosure of any information that is a trade
secret or confidential information subject to
section 552(b)(4) of title 5, United States
Code, or section 1905 of title 18, United
States Code.
``(3) Public health exemption.--The Secretary may--
``(A) deem such pilot demonstrations public health
activities, permitting the use and disclosure of
protected health information as described in section
164.512(b)(1)(iii) of title 45, Code of Federal
Regulations (or any successor regulation) and exempted
as a public health activity as described in section
46.101(b)(5) of title 46, Code of Federal Regulations
(or any successor regulation); and
``(B) deem safety surveillance performed at the
request of the Food and Drug Administration or under
such jurisdiction by a sponsor with responsibility for
a drug approved under this section or section 351 of
the Public Health Services Act using the Sentinel
System surveillance infrastructure authorized under
section 505(k), including use of analytic tools and
querying capabilities developed to implement the active
postmarket surveillance system described in this
section, public health activities as described in
section 164.512(b)(1)(iii) of title 45, Code of Federal
Regulations (or any successor regulation) and exempted
as a public health activity as described in section
46.101(b)(5) of title 46, Code of Federal Regulations
(or any successor regulation).
``(c) Authorization of Appropriations.--There are authorized to be
appropriated to carry out this section $3,000,000 for each of fiscal
years 2016 through 2020.''.
SEC. 2063. STREAMLINED DATA REVIEW PROGRAM.
(a) In General.--Chapter V of the Federal Food, Drug, and Cosmetic
Act, as amended by section 2062, is further amended by inserting after
section 505G of such Act the following:
``SEC. 505H. STREAMLINED DATA REVIEW PROGRAM.
``(a) In General.--The Secretary shall establish a streamlined data
review program under which a holder of an approved application
submitted under section 505(b)(1) or under section 351(a) of the Public
Health Service Act may, to support the approval or licensure (as
applicable) of the use of the drug that is the subject of such approved
application for a new qualified indication, submit qualified data
summaries.
``(b) Eligibility.--In carrying out the streamlined data review
program under subsection (a), the Secretary may authorize the holder of
the approved application to include one or more qualified data
summaries described in subsection (a) in a supplemental application
if--
``(1) the drug has been approved under section 505(c) of this
Act or licensed under section 351(a) of the Public Health
Service Act for one or more indications, and such approval or
licensure remains in effect;
``(2) the supplemental application is for approval or
licensure (as applicable) under such section 505(c) or 351(a)
of the use of the drug for a new qualified indication under
such section 505(c) or 351(a);
``(3) there is an existing database acceptable to the
Secretary regarding the safety of the drug developed for one or
more indications of the drug approved under such section 505(c)
or licensed under such section 351(a);
``(4) the supplemental application incorporates or
supplements the data submitted in the application for approval
or licensure referred to in paragraph (1); and
``(5) the full data sets used to develop the qualified data
summaries are submitted, unless the Secretary determines that
the full data sets are not required.
``(c) Public Availability of Information on Program.--The Secretary
shall post on the public website of the Food and Drug Administration
and update annually--
``(1) the number of applications reviewed under the
streamlined data review program;
``(2) the average time for completion of review under the
streamlined data review program versus other review of
applications for new indications; and
``(3) the number of applications reviewed under the
streamlined data review program for which the Food and Drug
Administration made use of full data sets in addition to the
qualified data summary.
``(d) Definitions.--In this section:
``(1) The term `qualified indication' means--
``(A) an indication for the treatment of cancer, as
determined appropriate by the Secretary; or
``(B) such other types of indications as the
Secretary determines to be subject to the streamlined
data review program under this section.
``(2) The term `qualified data summary' means a summary of
clinical data intended to demonstrate safety and effectiveness
with respect to a qualified indication for use of a drug.''.
(b) Sense of Congress.--It is the sense of Congress that the
streamlined data review program under section 505H of the Federal Food,
Drug, and Cosmetic Act, as added by subsection (a), should enable the
Food and Drug Administration to make approval decisions for certain
supplemental applications based on qualified data summaries (as defined
in such section 505H).
(c) Guidance; Regulations.--The Commissioner of Food and Drugs--
(1) shall--
(A) issue final guidance for implementation of the
streamlined data review program established under
section 505H of the Federal Food, Drug, and Cosmetic
Act, as added by subsection (a), not later than 24
months after the date of enactment of this Act; and
(B) include in such guidance the process for
expanding the types of indications to be subject to the
streamlined data review program, as authorized by
section 505H(c)(1)(B) of such Act; and
(2) in addition to issuing guidance under paragraph (1), may
issue such regulations as may be necessary for implementation
of the program.
Subtitle E--Expediting Patient Access
SEC. 2081. SENSE OF CONGRESS.
It is the sense of Congress that the Food and Drug Administration
should continue to expedite the approval of drugs designated as
breakthrough therapies pursuant to section 506(a) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 356(a)) by approving drugs so
designated as early as possible in the clinical development process,
regardless of the phase of development, provided that the Secretary of
Health and Human Services determines that an application for such a
drug meets the standards of evidence of safety and effectiveness under
section 505 of such Act (21 U.S.C. 355), including the substantial
evidence standard under subsection (d) of such section or under section
351(a) of the Public Health Service Act (42 U.S.C. 262(a)).
SEC. 2082. EXPANDED ACCESS POLICY.
Chapter V of the Federal Food, Drug, and Cosmetic Act is amended by
inserting after section 561 (21 U.S.C. 360bbb) the following:
``SEC. 561A. EXPANDED ACCESS POLICY REQUIRED FOR INVESTIGATIONAL DRUGS.
``(a) In General.--The manufacturer or distributor of one or more
investigational drugs for the diagnosis, monitoring, or treatment of
one or more serious diseases or conditions shall make publicly
available the policy of the manufacturer or distributor on evaluating
and responding to requests submitted under section 561(b) for provision
of such a drug. A manufacturer or distributor may satisfy the
requirement of the preceding sentence by posting such policy as
generally applicable to all of such manufacturer's or distributor's
investigational drugs.
``(b) Content of Policy.--A policy described in subsection (a) shall
include making publicly available--
``(1) contact information for the manufacturer or distributor
to facilitate communication about requests described in
subsection (a);
``(2) procedures for making such requests;
``(3) the general criteria the manufacturer or distributor
will consider or use to approve such requests; and
``(4) the length of time the manufacturer or distributor
anticipates will be necessary to acknowledge receipt of such
requests.
``(c) No Guarantee of Access.--The posting of policies by
manufacturers and distributors under subsection (a) shall not serve as
a guarantee of access to any specific investigational drug by any
individual patient.
``(d) Revised Policy.--A manufacturer or distributor that has made a
policy publicly available as required by this section may revise the
policy at any time.
``(e) Application.--This section shall apply to a manufacturer or
distributor with respect to an investigational drug beginning on the
later of--
``(1) the date that is 60 days after the date of enactment of
the 21st Century Cures Act; or
``(2) the first initiation of a phase 2 or phase 3 study (as
such terms are defined in section 312.21(b) and (c) of title
21, Code of Federal Regulations (or any successor regulations))
with respect to such investigational new drug.''.
SEC. 2083. FINALIZING DRAFT GUIDANCE ON EXPANDED ACCESS.
(a) In General.--Not later than 12 months after the date of enactment
of this Act, the Secretary of Health and Human Services shall finalize
the draft guidance entitled ``Expanded Access to Investigational Drugs
for Treatment Use--Qs & As'' and dated May 2013.
(b) Contents.--The final guidance referred to in subsection (a) shall
clearly define how the Secretary of Health and Human Services
interprets and uses adverse drug event data reported by investigators
in the case of data reported from use under a request submitted under
section 561(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360bbb(b)).
Subtitle F--Facilitating Responsible Manufacturer Communications
SEC. 2101. FACILITATING DISSEMINATION OF HEALTH CARE ECONOMIC
INFORMATION.
Section 502(a) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
352(a)) is amended--
(1) by striking ``(a) If its'' and inserting ``(a)(1) If
its'';
(2) by striking ``a formulary committee, or other similar
entity, in the course of the committee or the entity carrying
out its responsibilities for the selection of drugs for managed
care or other similar organizations'' and inserting ``a payor,
formulary committee, or other similar entity with knowledge and
expertise in the area of health care economic analysis,
carrying out its responsibilities for the selection of drugs
for coverage or reimbursement'';
(3) by striking ``directly relates'' and inserting
``relates'';
(4) by striking ``and is based on competent and reliable
scientific evidence. The requirements set forth in section
505(a) or in section 351(a) of the Public Health Service Act
shall not apply to health care economic information provided to
such a committee or entity in accordance with this paragraph''
and inserting ``, is based on competent and reliable scientific
evidence, and includes, where applicable, a conspicuous and
prominent statement describing any material differences between
the health care economic information and the labeling approved
for the drug under section 505 or under section 351 of the
Public Health Service Act. The requirements set forth in
section 505(a) or in subsections (a) and (k) of section 351 of
the Public Health Service Act shall not apply to health care
economic information provided to such a payor, committee, or
entity in accordance with this paragraph''; and
(5) by striking ``In this paragraph, the term'' and all that
follows and inserting the following:
``(2)(A) For purposes of this paragraph, the term `health care
economic information' means any analysis (including the clinical data,
inputs, clinical or other assumptions, methods, results, and other
components underlying or comprising the analysis) that identifies,
measures, or describes the economic consequences, which may be based on
the separate or aggregated clinical consequences of the represented
health outcomes, of the use of a drug. Such analysis may be comparative
to the use of another drug, to another health care intervention, or to
no intervention.
``(B) Such term does not include any analysis that relates only to an
indication that is not approved under section 505 or under section 351
of the Public Health Service Act for such drug.''.
SEC. 2102. FACILITATING RESPONSIBLE COMMUNICATION OF SCIENTIFIC AND
MEDICAL DEVELOPMENTS.
(a) Guidance.--Not later than 18 months after the date of enactment
of this Act, the Secretary of Health and Human Services shall issue
draft guidance on facilitating the responsible dissemination of
truthful and nonmisleading scientific and medical information not
included in the approved labeling of drugs and devices.
(b) Definition.--In this section, the terms ``drug'' and ``device''
have the meaning given to such terms in section 201 of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 321).
Subtitle G--Antibiotic Drug Development
SEC. 2121. APPROVAL OF CERTAIN DRUGS FOR USE IN A LIMITED POPULATION OF
PATIENTS.
(a) Purpose.--The purpose of this section is to help to expedite the
development and availability of treatments for serious or life-
threatening bacterial or fungal infections in patients with unmet
needs, while maintaining safety and effectiveness standards for such
treatments, taking into account the severity of the infection and the
availability or lack of alternative treatments.
(b) Approval of Certain Antibacterial and Antifungal Drugs.--Section
505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355), as
amended by section 2001, is further amended by adding at the end the
following new subsection:
``(z) Approval of Certain Antibacterial and Antifungal Drugs for Use
in a Limited Population of Patients.--
``(1) Process.--At the request of the sponsor of an
antibacterial or antifungal drug that is intended to treat a
serious or life-threatening infection, the Secretary--
``(A) may execute a written agreement with the
sponsor on the process for developing data to support
an application for approval of such drug, for use in a
limited population of patients in accordance with this
subsection;
``(B) shall proceed in accordance with this
subsection only if a written agreement is reached under
subparagraph (A);
``(C) shall provide the sponsor with an opportunity
to request meetings under paragraph (2);
``(D) if a written agreement is reached under
subparagraph (A), may approve the drug under this
subsection for such use--
``(i) in a limited population of patients for
which there is an unmet medical need;
``(ii) based on a streamlined development
program; and
``(iii) only if the standards for approval
under subsections (c) and (d) of this section
or licensure under section 351 of the Public
Health Service Act, as applicable, are met; and
``(E) in approving a drug in accordance with this
subsection, subject to subparagraph (D)(iii), may rely
upon--
``(i) traditional endpoints, alternate
endpoints, or a combination of traditional and
alternate endpoints, and, as appropriate, data
sets of a limited size; and
``(ii)(I) additional data, including
preclinical, pharmacologic, or pathophysiologic
evidence;
``(II) nonclinical susceptibility and
pharmacokinetic data;
``(III) data from phase 2 clinical trials;
and
``(IV) such other confirmatory evidence as
the Secretary determines appropriate to approve
the drug.
``(2) Formal meetings.--
``(A) In general.--To help to expedite and facilitate
the development and review of a drug for which a
sponsor intends to request approval in accordance with
this subsection, the Secretary may, at the request of
the sponsor, conduct meetings that provide early
consultation, timely advice, and sufficient
opportunities to develop an agreement described in
paragraph (1)(A) and help the sponsor design and
conduct a drug development program as efficiently as
possible, including the following types of meetings:
``(i) An early consultation meeting.
``(ii) An assessment meeting.
``(iii) A postapproval meeting.
``(B) No altering of goals.--Nothing in this
paragraph shall be construed to alter agreed upon goals
and procedures identified in the letters described in
section 101(b) of the Prescription Drug User Fee
Amendments of 2012.
``(C) Breakthrough therapies.--In the case of a drug
designated as a breakthrough therapy under section
506(a), the sponsor of such drug may elect to utilize
meetings provided under such section with respect to
such drug in lieu of meetings described in subparagraph
(A).
``(3) Labeling requirement.--The labeling of an antibacterial
or antifungal drug approved in accordance with this subsection
shall contain the statement `Limited Population' in a prominent
manner and adjacent to, and not more prominent than, the brand
name of the product. The prescribing information for such
antibacterial or antifungal drug required by section 201.57 of
title 21, Code of Federal Regulations (or any successor
regulation) shall also include the following statement: `This
drug is indicated for use in a limited and specific population
of patients.'.
``(4) Promotional materials.--The provisions of section
506(c)(2)(B) shall apply with respect to approval in accordance
with this subsection to the same extent and in the same manner
as such provisions apply with respect to accelerated approval
in accordance with section 506(c)(1).
``(5) Termination of requirements or conditions.--If a drug
is approved in accordance with this subsection for an
indication in a limited population of patients and is
subsequently approved or licensed under this section or section
351 of the Public Health Service Act, other than in accordance
with this subsection, for--
``(A) the same indication and the same conditions of
use, the Secretary shall remove any labeling
requirements or postmarketing conditions that were made
applicable to the drug under this subsection; or
``(B) a different indication or condition of use, the
Secretary shall not apply the labeling requirements and
postmarketing conditions that were made applicable to
the drug under this subsection to the subsequent
approval of the drug for such different indication or
condition of use.
``(6) Relation to other provisions.--Nothing in this
subsection shall be construed to prohibit the approval of a
drug for use in a limited population of patients in accordance
with this subsection, in combination with--
``(A) an agreement on the design and size of a
clinical trial pursuant to subparagraphs (B) and (C) of
subsection (b)(5);
``(B) designation and treatment of the drug as a
breakthrough therapy under section 506(a);
``(C) designation and treatment of the drug as a fast
track product under section 506(b); or
``(D) accelerated approval of the drug in accordance
with section 506(c).
``(7) Rule of construction.--Nothing in this subsection shall
be construed--
``(A) to alter the standards of evidence under
subsection (c) or (d) (including the substantial
evidence standard in subsection (d));
``(B) to waive or otherwise preclude the application
of requirements under subsection (o);
``(C) to otherwise, in any way, limit the authority
of the Secretary to approve products pursuant to this
Act and the Public Health Service Act as authorized
prior to the date of enactment of this subsection; or
``(D) to restrict in any manner, the prescribing of
antibiotics or other products by health care providers,
or to otherwise limit or restrict the practice of
health care.
``(8) Effective immediately.--The Secretary shall have the
authorities vested in the Secretary by this subsection
beginning on the date of enactment of this subsection,
irrespective of when and whether the Secretary promulgates
final regulations or guidance.
``(9) Definitions.--In this subsection:
``(A) Early consultation meeting.--The term `early
consultation meeting' means a pre-investigational new
drug meeting or an end-of-phase-1 meeting that--
``(i) is conducted to review and reach a
written agreement--
``(I) on the scope of the streamlined
development plan for a drug for which a
sponsor intends to request approval in
accordance with this subsection; and
``(II) which, as appropriate, may
include agreement on the design and
size of necessary preclinical and
clinical studies early in the
development process, including clinical
trials whose data are intended to form
the primary basis for an effectiveness
claim; and
``(ii) provides an opportunity to discuss
expectations of the Secretary regarding studies
or other information that the Secretary deems
appropriate for purposes of applying paragraph
(5), relating to the termination of labeling
requirements or postmarketing conditions.
``(B) Assessment meeting.--The term `assessment
meeting' means an end-of-phase 2 meeting, pre-new drug
application meeting, or pre-biologics license
application meeting conducted to resolve questions and
issues raised during the course of clinical
investigations, and details addressed in the written
agreement regarding postapproval commitments or
expansion of approved uses.
``(C) Postapproval meeting.--The term `postapproval
meeting' means a meeting following initial approval or
licensure of the drug for use in a limited population,
to discuss any issues identified by the Secretary or
the sponsor regarding postapproval commitments or
expansion of approved uses.''.
(c) Guidance.--Not later than 18 months after the date of enactment
of this Act, the Secretary of Health and Human Services, acting through
the Commissioner of Food and Drugs, shall issue draft guidance
describing criteria, process, and other general considerations for
demonstrating the safety and effectiveness of antibacterial and
antifungal drugs to be approved for use in a limited population in
accordance with section 505(z) of the Federal Food, Drug, and Cosmetic
Act, as added by subsection (b).
(d) Conforming Amendments.--
(1) Licensure of certain biological products.--Section 351(j)
of the Public Health Service Act (42 U.S.C. 262(j)) is
amended--
(A) by striking ``(j)'' and inserting ``(j)(1)'';
(B) by inserting ``505(z),'' after ``505(p),''; and
(C) by adding at the end the following new paragraph:
``(2) In applying section 505(z) of the Federal Food, Drug, and
Cosmetic Act to the licensure of biological products under this
section--
``(A) references to an antibacterial or antifungal drug that
is intended to treat a serious or life-threatening infection
shall be construed to refer to a biological product intended to
treat a serious or life-threatening bacterial or fungal
infection; and
``(B) references to approval of a drug under section 505(c)
of such Act shall be construed to refer to a licensure of a
biological product under subsection (a) of this section.''.
(2) Misbranding.--Section 502 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 352) is amended by adding at the end
the following new subsection:
``(dd) If it is a drug approved in accordance with section 505(z) and
its labeling does not meet the requirements under paragraph (3) of such
subsection, subject to paragraph (5) of such subsection.''.
(e) Evaluation.--
(1) Assessment.--Not later than 48 months after the date of
enactment of this Act, the Secretary of Health and Human
Services shall publish for public comment an assessment of the
program established under section 505(z) of the Federal Food,
Drug, and Cosmetic Act, as added by subsection (b). Such
assessment shall determine if the limited-use pathway
established under such section 505(z) has improved or is likely
to improve patient access to novel antibacterial or antifungal
treatments and assess how the pathway could be expanded to
cover products for serious or life-threatening diseases or
conditions beyond bacterial and fungal infections.
(2) Meeting.--Not later than 90 days after the date of the
publication of such assessment, the Secretary, acting through
the Commissioner of Food and Drugs, shall hold a public meeting
to discuss the findings of the assessment, during which public
stakeholders may present their views on the success of the
program established under section 505(z) of the Federal Food,
Drug, and Cosmetic Act, as added by subsection (b), and the
appropriateness of expanding such program.
(f) Expansion of Program.--If the Secretary of Health and Human
Services determines, based on the assessment under subsection (e)(1),
evaluation of the assessment, and any other relevant information, that
the public health would benefit from expansion of the limited-use
pathway established under section 505(z) of the Federal Food, Drug, and
Cosmetic Act (as added by subsection (b)) beyond the drugs approved in
accordance with such section, the Secretary may expand such limited-use
pathway in accordance with such a determination. The approval of any
drugs under any such expansion shall be subject to the considerations
and requirements described in such section 505(z) for purposes of
expansion to other serious or life-threatening diseases or conditions.
(g) Monitoring.--The Public Health Service Act is amended by
inserting after section 317T (42 U.S.C. 247b-22) the following:
``SEC. 317U. MONITORING ANTIBACTERIAL AND ANTIFUNGAL DRUG USE AND
RESISTANCE.
``(a) Monitoring.--The Secretary shall use an appropriate monitoring
system to monitor--
``(1) the use of antibacterial and antifungal drugs,
including those receiving approval or licensure for a limited
population pursuant to section 505(z) of the Federal Food,
Drug, and Cosmetic Act; and
``(2) changes in bacterial and fungal resistance to drugs.
``(b) Public Availability of Data.--The Secretary shall make
summaries of the data derived from monitoring under this section
publicly available for the purposes of--
``(1) improving the monitoring of important trends in
antibacterial and antifungal resistance; and
``(2) ensuring appropriate stewardship of antibacterial and
antifungal drugs, including those receiving approval or
licensure for a limited population pursuant to section 505(z)
of the Federal Food, Drug, and Cosmetic Act.''.
SEC. 2122. SUSCEPTIBILITY TEST INTERPRETIVE CRITERIA FOR
MICROORGANISMS.
(a) In General.--Section 511 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360a) is amended to read as follows:
``SEC. 511. IDENTIFYING AND UPDATING SUSCEPTIBILITY TEST INTERPRETIVE
CRITERIA FOR MICROORGANISMS.
``(a) Purpose; Identification of Criteria.--
``(1) Purpose.--The purpose of this section is to provide the
Secretary with an expedited, flexible method for--
``(A) clearance or premarket approval of
antimicrobial susceptibility testing devices utilizing
updated, recognized susceptibility test interpretive
criteria to characterize the in vitro susceptibility of
particular bacteria, fungi, or other microorganisms to
antimicrobial drugs; and
``(B) providing public notice of the availability of
recognized interpretive criteria to meet premarket
submission requirements or other requirements under
this Act for antimicrobial susceptibility testing
devices.
``(2) In general.--The Secretary shall identify appropriate
susceptibility test interpretive criteria with respect to
antimicrobial drugs--
``(A) if such criteria are available on the date of
approval of the drug under section 505 of this Act or
licensure of the drug under section 351 of the Public
Health Service Act (as applicable), upon such approval
or licensure; or
``(B) if such criteria are unavailable on such date,
on the date on which such criteria are available for
such drug.
``(3) Bases for initial identification.--The Secretary shall
identify appropriate susceptibility test interpretive criteria
under paragraph (2), based on the Secretary's review of, to the
extent available and relevant--
``(A) preclinical and clinical data, including
pharmacokinetic, pharmacodynamic, and epidemiological
data;
``(B) Bayesian and pharmacometric statistical
methodologies; and
``(C) such other evidence and information as the
Secretary considers appropriate.
``(b) Susceptibility Test Interpretive Criteria Website.--
``(1) In general.--Not later than 1 year after the date of
the enactment of the 21st Century Cures Act, the Secretary
shall establish, and maintain thereafter, on the website of the
Food and Drug Administration, a dedicated website that contains
a list of any appropriate new or updated susceptibility test
interpretive criteria standards in accordance with paragraph
(2) (referred to in this section as the `Interpretive Criteria
Website').
``(2) Listing of susceptibility test interpretive criteria
standards.--
``(A) In general.--The list described in paragraph
(1) shall consist of any new or updated susceptibility
test interpretive criteria standards that are--
``(i) established by a nationally or
internationally recognized standard development
organization that--
``(I) establishes and maintains
procedures to address potential
conflicts of interest and ensure
transparent decisionmaking;
``(II) holds open meetings to ensure
that there is an opportunity for public
input by interested parties, and
establishes and maintains processes to
ensure that such input is considered in
decisionmaking; and
``(III) permits its standards to be
made publicly available, through the
National Library of Medicine or another
similar source acceptable to the
Secretary; and
``(ii) recognized in whole, or in part, by
the Secretary under subsection (c).
``(B) Other list.--The Interpretive Criteria Website
shall, in addition to the list described in
subparagraph (A), include a list of interpretive
criteria, if any, that the Secretary has determined to
be appropriate with respect to legally marketed
antimicrobial drugs, where--
``(i) the Secretary does not recognize, in
whole or in part, an interpretive criteria
standard described under subparagraph (A)
otherwise applicable to such a drug;
``(ii) the Secretary withdraws under
subsection (c)(1)(B) recognition of a standard,
in whole or in part, otherwise applicable to
such a drug;
``(iii) the Secretary approves an application
under section 505 of this Act or section 351 of
the Public Health Service Act, as applicable,
with respect to marketing of such a drug for
which there are no relevant interpretive
criteria included in a standard recognized by
the Secretary under subsection (c); or
``(iv) because the characteristics of such a
drug differ from other drugs with the same
active ingredient, the interpretive criteria
with respect to such drug--
``(I) differ from otherwise
applicable interpretive criteria
included in a standard listed under
subparagraph (A) or interpretive
criteria otherwise listed under this
subparagraph; and
``(II) are determined by the
Secretary to be appropriate for the
drug.
``(C) Required statements of limitations of
information.--The Interpretive Criteria Website shall
include the following:
``(i) A statement that--
``(I) the website provides
information about the susceptibility of
bacteria, fungi, or other
microorganisms to a certain drug (or
drugs); and
``(II) the safety and efficacy of the
drug in treating clinical infections
due to such bacteria, fungi, or other
microorganisms may not have been
established in adequate and well-
controlled clinical trials and the
clinical significance of such
susceptibility information in such
trials is unknown.
``(ii) A statement that directs health care
practitioners to consult the approved product
labeling for specific drugs to determine the
uses for which the Food and Drug Administration
has approved the product.
``(iii) Any other statement that the
Secretary determines appropriate to adequately
convey the limitations of the data supporting
susceptibility test interpretive criteria
standard listed on the website.
``(3) Notice.--Not later than the date on which the
Interpretive Criteria Website is established, the Secretary
shall publish a notice of that establishment in the Federal
Register.
``(4) Inapplicability of misbranding provision.--The
inclusion in the approved labeling of an antimicrobial drug of
a reference or hyperlink to the Interpretive Criteria Website,
in and of itself, shall not cause the drug to be misbranded in
violation of section 502, or the regulations promulgated
thereunder.
``(5) Trade secrets and confidential information.--Nothing in
this section shall be construed as authorizing the Secretary to
disclose any information that is a trade secret or confidential
information subject to section 552(b)(4) of title 5, United
States Code.
``(c) Recognition of Susceptibility Test Interpretive Criteria From
Standard Development Organizations.--
``(1) In general.--Beginning on the date of the establishment
of the Interpretive Criteria Website, and at least every 6
months thereafter, the Secretary shall--
``(A) evaluate any appropriate new or updated
susceptibility test interpretive criteria standards
established by a nationally or internationally
recognized standard development organization described
in subsection (b)(2)(A)(i); and
``(B) publish on the public website of the Food and
Drug Administration a notice--
``(i) withdrawing recognition of any
different susceptibility test interpretive
criteria standard, in whole or in part;
``(ii) recognizing the new or updated
standards;
``(iii) recognizing one or more parts of the
new or updated interpretive criteria specified
in such a standard and declining to recognize
the remainder of such standard; and
``(iv) making any necessary updates to the
lists under subsection (b)(2).
``(2) Bases for updating interpretive criteria standards.--In
evaluating new or updated susceptibility test interpretive
criteria standards under paragraph (1)(A), the Secretary may
consider--
``(A) the Secretary's determination that such a
standard is not applicable to a particular drug because
the characteristics of the drug differ from other drugs
with the same active ingredient;
``(B) information provided by interested third
parties, including public comment on the annual
compilation of notices published under paragraph (3);
``(C) any bases used to identify susceptibility test
interpretive criteria under subsection (a)(2); and
``(D) such other information or factors as the
Secretary determines appropriate.
``(3) Annual compilation of notices.--Each year, the
Secretary shall compile the notices published under paragraph
(1)(B) and publish such compilation in the Federal Register and
provide for public comment. If the Secretary receives comments,
the Secretary will review such comments and, if the Secretary
determines appropriate, update pursuant to this subsection
susceptibility test interpretive criteria standards--
``(A) recognized by the Secretary under this
subsection; or
``(B) otherwise listed on the Interpretive Criteria
Website under subsection (b)(2).
``(4) Relation to section 514(c).--Any susceptibility test
interpretive standard recognized under this subsection or any
criteria otherwise listed under subsection (b)(2)(B) shall be
deemed to be recognized as a standard by the Secretary under
section 514(c)(1).
``(5) Voluntary use of interpretive criteria.--Nothing in
this section prohibits a person from seeking approval or
clearance of a drug or device, or changes to the drug or the
device, on the basis of susceptibility test interpretive
criteria standards which differ from those recognized pursuant
to paragraph (1).
``(d) Antimicrobial Drug Labeling.--
``(1) Drugs marketed prior to establishment of interpretive
criteria website.--With respect to an antimicrobial drug
lawfully introduced or delivered for introduction into
interstate commerce for commercial distribution before the
establishment of the Interpretive Criteria Website, a holder of
an approved application under section 505 of this Act or
section 351 of the Public Health Service Act, as applicable,
for each such drug--
``(A) not later than 1 year after establishment of
the Interpretive Criteria Website, shall submit to the
Secretary a supplemental application for purposes of
changing the drug's labeling to substitute a reference
or hyperlink to such Website for any susceptibility
test interpretive criteria and related information; and
``(B) may begin distribution of the drug involved
upon receipt by the Secretary of the supplemental
application for such change.
``(2) Drugs marketed subsequent to establishment of
interpretive criteria website.--With respect to antimicrobial
drugs lawfully introduced or delivered for introduction into
interstate commerce for commercial distribution on or after the
date of the establishment of the Interpretive Criteria Website,
the labeling for such a drug shall include, in lieu of
susceptibility test interpretive criteria and related
information, a reference to such Website.
``(e) Special Condition for Marketing of Antimicrobial Susceptibility
Testing Devices.--
``(1) In general.--Notwithstanding sections 501, 502, 510,
513, and 515, if the conditions specified in paragraph (2) are
met (in addition to other applicable provisions under this
chapter) with respect to an antimicrobial susceptibility
testing device described in subsection (f)(1), the Secretary
may authorize the marketing of such device for a use described
in such subsection.
``(2) Conditions applicable to antimicrobial susceptibility
testing devices.--The conditions specified in this paragraph
are the following:
``(A) The device is used to make a determination of
susceptibility using susceptibility test interpretive
criteria that are--
``(i) included in a standard recognized by
the Secretary under subsection (c); or
``(ii) otherwise listed on the Interpretive
Criteria Website under subsection (b)(2).
``(B) The labeling of such device prominently and
conspicuously--
``(i) includes a statement that--
``(I) the device provides information
about the susceptibility of bacteria
and fungi to certain drugs; and
``(II) the safety and efficacy of
such drugs in treating clinical
infections due to such bacteria or
fungi may not have been established in
adequate and well-controlled clinical
trials and the clinical significance of
such susceptibility information in
those instances is unknown;
``(ii) includes a statement directing health
care practitioners to consult the approved
labeling for drugs tested using such a device,
to determine the uses for which the Food and
Drug Administration has approved such drugs;
and
``(iii) includes any other statement the
Secretary determines appropriate to adequately
convey the limitations of the data supporting
the interpretive criteria described in
subparagraph (A).
``(f) Definitions.--In this section:
``(1) The term `antimicrobial susceptibility testing device'
means a device that utilizes susceptibility test interpretive
criteria to determine and report the in vitro susceptibility of
certain microorganisms to a drug (or drugs).
``(2) The term `qualified infectious disease product' means a
qualified infectious disease product designated under section
505E(d).
``(3) The term `susceptibility test interpretive criteria'
means--
``(A) one or more specific numerical values which
characterize the susceptibility of bacteria or other
microorganisms to the drug tested; and
``(B) related categorizations of such susceptibility,
including categorization of the drug as susceptible,
intermediate, resistant, or such other term as the
Secretary determines appropriate.
``(4)(A) The term `antimicrobial drug' means, subject to
subparagraph (B), a systemic antibacterial or antifungal drug
that--
``(i) is intended for human use in the treatment of a
disease or condition caused by a bacterium or fungus;
``(ii) may include a qualified infectious disease
product designated under section 505E(d); and
``(iii) is subject to section 503(b)(1).
``(B) If provided by the Secretary through regulations, such
term may include--
``(i) drugs other than systemic antibacterial and
antifungal drugs; and
``(ii) biological products (as such term is defined
in section 351 of the Public Health Service Act) to the
extent such products exhibit antimicrobial activity.
``(g) Rule of Construction.--Nothing in this section shall be
construed--
``(1) to alter the standards of evidence--
``(A) under subsection (c) or (d) of section 505,
including the substantial evidence standard in section
505(d), or under section 351 of the Public Health
Service Act (as applicable); or
``(B) with respect to marketing authorization for
devices, under section 510, 513, or 515;
``(2) to apply with respect to any drug, device, or
biological product, in any context other than--
``(A) an antimicrobial drug; or
``(B) an antimicrobial susceptibility testing device
that uses susceptibility test interpretive criteria to
characterize and report the in vitro susceptibility of
certain bacteria, fungi, or other microorganisms to
antimicrobial drugs in accordance with this section; or
``(3) unless specifically stated, to have any effect on
authorities provided under other sections of this Act,
including any regulations issued under such sections.''.
(b) Conforming Amendments.--
(1) Repeal of related authority.--Section 1111 of the Food
and Drug Administration Amendments Act of 2007 (42 U.S.C. 247d-
5a; relating to identification of clinically susceptible
concentrations of antimicrobials) is repealed.
(2) Clerical amendment.--The table of contents in section 2
of the Food and Drug Administration Amendments Act of 2007 is
amended by striking the item relating to section 1111.
(3) Misbranding.--Section 502 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 352), as amended by section 2121, is
further amended by adding at the end the following:
``(ee) If it is an antimicrobial drug and its labeling fails to
conform with the requirements under section 511(d).''.
(4) Recognition of interpretive criteria as device
standard.--Section 514(c)(1)(A) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360d(c)(1)(A)) is amended by inserting
after ``the Secretary shall, by publication in the Federal
Register'' the following: ``(or, with respect to susceptibility
test interpretive criteria or standards recognized or otherwise
listed under section 511, by posting on the Interpretive
Criteria Website in accordance with such section)''.
(c) Report to Congress.--Not later than two years after the date of
enactment of this Act, the Secretary of Health and Human Services shall
submit to the Committee on Energy and Commerce of the House of
Representatives and the Committee on Health, Education, Labor and
Pensions of the Senate a report on the progress made in implementing
section 511 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360a), as amended by this section.
(d) Requests for Updates to Interpretive Criteria Website.--Chapter
35 of title 44, United States Code, shall not apply to the collection
of information from interested parties regarding the updating of lists
under paragraph (2) of subsection (b) section 511 of the Federal Food,
Drug, and Cosmetic Act (as amended by subsection (a)) and posted on the
Interpretive Criteria Website established under paragraph (1) of such
subsection (b).
(e) No Effect on Health Care Practice.--Nothing in this subtitle
(including the amendments made by this subtitle) shall be construed to
restrict, in any manner, the prescribing or administering of
antibiotics or other products by health care practitioners, or to limit
the practice of health care.
SEC. 2123. ENCOURAGING THE DEVELOPMENT AND USE OF NEW ANTIMICROBIAL
DRUGS.
(a) Additional Payment for New Antimicrobial Drugs Under Medicare.--
(1) In general.--Section 1886(d)(5) of the Social Security
Act (42 U.S.C. 1395ww(d)(5)) is amended by adding at the end
the following new subparagraph:
``(M)(i) As part of the annual rulemaking under this subsection for
payment for subsection (d) hospitals for each fiscal year beginning
with fiscal year 2018, the Secretary shall--
``(I) include publication of a list of the new antimicrobial
drugs for such fiscal year; and
``(II) with respect to discharges by eligible hospitals that
involve a drug so published, provide for an additional payment
to be made under this subsection in accordance with the
provisions of this subparagraph.
``(ii) Additional payments may not be made for a drug under this
subparagraph--
``(I) other than during the 5-fiscal-year period beginning
with the fiscal year for which the drug is first included in
the publication described in clause (i)(I); and
``(II) with respect to which payment has ever been made
pursuant to subparagraph (K).
``(iii) For purposes of this subparagraph, the term `new
antimicrobial drug' means a product that is approved for use, or a
product for which an indication is first approved for use, by the Food
and Drug Administration on or after December 1, 2014, and that the Food
and Drug Administration determines--
``(I) either--
``(aa) is intended to treat an infection caused by,
or likely to be caused by, a qualifying pathogen (as
defined under section 505E(f) of the Federal Food,
Drug, and Cosmetic Act); or
``(bb) meets the definition of a qualified infectious
disease product under section 505E(g) of the Federal
Food, Drug, and Cosmetic Act; and
``(II) is intended to treat an infection--
``(aa) for which there is an unmet medical need; and
``(bb) which is associated with high rates of
mortality or significant patient morbidity, as
determined in consultation with the Director of the
Centers for Disease Control and Prevention and the
infectious disease professional community.
Such determination may be revoked only upon a finding that the request
for such determination contained an untrue statement of material fact.
``(iv) For purposes of this subparagraph, the term `eligible
hospital' means a subsection (d) hospital that participates in the
National Healthcare Safety Network of the Centers for Disease Control
and Prevention (or, to the extent a similar surveillance system
reporting program that includes reporting about antimicrobial drugs is
determined by the Secretary to be available to such hospitals, such
similar surveillance system as the Secretary may specify).
``(v)(I) Subject to the succeeding provisions of this clause, the
additional payment under this subparagraph, with respect to a drug,
shall be in the amount provided for such drug under section 1847A.
``(II) The Secretary shall, as part of the rulemaking referred to in
clause (i) for each fiscal year, estimate--
``(aa) the total amount of the additional payments that will
be made under this subsection pursuant to this subparagraph for
discharges in such fiscal year without regard to the
application of subclause (III); and
``(bb) the total program payments to be made under this
subsection for all discharges in such fiscal year.
``(III) If the estimated total amount described in subclause (II)(aa)
for a fiscal year exceeds the applicable percentage of the estimated
total program payments described in subclause (II)(bb) for such fiscal
year, the Secretary shall reduce in a pro rata manner the amount of
each additional payment under this subsection pursuant to this
subparagraph for such fiscal year in order to ensure that the total
amount of the additional payments under this subsection pursuant to
this subparagraph for such fiscal year do not exceed the applicable
percentage of the estimated total program payments described in
subclause (II)(bb) for such fiscal year.
``(IV) For purposes of subclause (III), the term `applicable
percentage' means 0.03 percent.''.
(2) Conforming amendments.--
(A) No duplicative ntap payments.--Section
1886(d)(5)(K)(vi) of the Social Security Act (42 U.S.C.
1395ww(d)(5)(K)(vi)) is amended by inserting ``if
additional payment has never been made under this
subsection pursuant to subparagraph (M) with respect to
the service or technology'' after ``if the service or
technology''.
(B) Access to price information.--Section
1927(b)(3)(A)(iii) of the Social Security Act (42
U.S.C. 1396r-8(b)(3)(A)(iii)) is amended--
(i) in subclause (II), by inserting ``or
under section 1886(d) pursuant to paragraph
(5)(M) of such section,'' after ``1847A,''; and
(ii) in the matter following subclause (III),
by inserting ``or section 1886(d)(5)(M)'' after
``1881(b)(13)(A)(ii)''.
(b) Study and Report on Removing Barriers to Development of New
Antimicrobial Drugs.--
(1) Study.--The Comptroller General of the United States
shall, in consultation with the Director of the National
Institutes of Health, the Commissioner of Food and Drugs, and
the Director of the Centers for Disease Control and Prevention,
conduct a study to--
(A) identify and examine the barriers that prevent
the development of new antimicrobial drugs, as defined
in section 1886(d)(5)(M)(iii) of the Social Security
Act (42 U.S.C. 1395ww(d)(5)(M)(iii)), as added by
subsection (a)(1); and
(B) develop recommendations for actions to be taken
in order to overcome any barriers identified under
subparagraph (A).
(2) Report.--Not later than 1 year after the date of the
enactment of this Act, the Comptroller General shall submit to
Congress a report on the study conducted under paragraph (1).
Subtitle H--Vaccine Access, Certainty, and Innovation
SEC. 2141. TIMELY REVIEW OF VACCINES BY THE ADVISORY COMMITTEE ON
IMMUNIZATION PRACTICES.
Section 2102(a) of the Public Health Service Act (42 U.S.C. 300aa-
2(a)) is amended by adding at the end the following:
``(10) Advisory committee on immunization practices.--
``(A) Standard periods of time for making
recommendations.--Upon the licensure of any vaccine or
any new indication for a vaccine, the Director of the
Program shall direct the Advisory Committee on
Immunization Practices, at its next regularly scheduled
meeting, to consider the use of the vaccine.
``(B) Expedited review pursuant to request by sponsor
or manufacturer.--If the Advisory Committee does not
make recommendations with respect to the use of a
vaccine at the Advisory Committee's first regularly
scheduled meeting after the licensure of the vaccine or
any new indication for the vaccine, the Advisory
Committee, at the request of the sponsor of the
vaccine, shall make such recommendations on an
expedited basis.
``(C) Expedited review for breakthrough therapies and
for use during public health emergencies.--If a vaccine
is designated as a breakthrough therapy under section
506 of the Federal Food, Drug, and Cosmetic Act and is
licensed under section 351 of this Act, the Advisory
Committee shall make recommendations with respect to
the use of the vaccine on an expedited basis.
``(D) Definition.--In this paragraph, the terms
`Advisory Committee on Immunization Practices' and
`Advisory Committee' mean the advisory committee on
immunization practices established by the Secretary
pursuant to section 222, acting through the Director of
the Centers for Disease Control and Prevention.''.
SEC. 2142. REVIEW OF PROCESSES AND CONSISTENCY OF ACIP RECOMMENDATIONS.
(a) Review.--The Director of the Centers for Disease Control and
Prevention shall conduct a review of the process used by the Advisory
Committee on Immunization Practices to evaluate consistency in
formulating and issuing recommendations pertaining to vaccines.
(b) Considerations.--The review under subsection (a) shall include
assessment of--
(1) the criteria used to evaluate new and existing vaccines;
(2) the Grading of Recommendations, Assessment, Development,
and Evaluation (GRADE) approach to the review and analysis of
scientific and economic data, including the scientific basis
for such approach; and
(3) the extent to which the processes used by the working
groups of the Advisory Committee on Immunization Practices are
consistent among groups.
(c) Stakeholders.--In carrying out the review under subsection (a),
the Director of the Centers for Disease Control and Prevention shall
solicit input from vaccine stakeholders.
(d) Report.--Not later than 18 months after the date of enactment of
this Act, the Director of the Centers for Disease Control and
Prevention shall submit to the appropriate committees of the Congress
and make publicly available a report on the results of the review under
subsection (a), including recommendations on improving the consistency
of the process described in such subsection.
(e) Definition.--In this section, the term ``Advisory Committee on
Immunization Practices'' means the advisory committee on immunization
practices established by the Secretary of Health and Human Services
pursuant to section 222 of the Public Health Service Act (42 U.S.C.
217a), acting through the Director of the Centers for Disease Control
and Prevention.
SEC. 2143. MEETINGS BETWEEN CDC AND VACCINE DEVELOPERS.
Section 310 of the Public Health Service Act (42 U.S.C. 242o) is
amended by adding at the end the following:
``(c)(1) In this subsection, the term `vaccine developer' means a
nongovernmental entity engaged in--
``(A)(i) the development of a vaccine with the intent to
pursue licensing of the vaccine by the Food and Drug
Administration; or
``(ii) the production of a vaccine licensed by the Food and
Drug Administration; and
``(B) vaccine research.
``(2)(A) Upon the submission of a written request for a meeting by a
vaccine developer, that includes a justification for the meeting, the
Secretary, acting through the Director of the Centers for Disease
Control and Prevention, shall convene a meeting of representatives of
the vaccine developer and experts from the Centers for Disease Control
and Prevention in immunization programs, epidemiology, and other
relevant areas at which the Director (or the Director's designee), for
the purpose of informing the vaccine developer's understanding of
public health needs and priorities, shall provide the perspectives of
the Centers for Disease Control and Prevention and other relevant
Federal agencies regarding--
``(i) public health needs, epidemiology, and implementation
considerations with regard to a vaccine developer's potential
vaccine profile; and
``(ii) potential implications of such perspectives for the
vaccine developer's vaccine research and development planning.
``(B) In addition to the representatives specified in subparagraph
(A), the Secretary may, with the agreement of the vaccine developer
requesting a meeting under such subparagraph, include in such meeting
representatives of--
``(i) the Food and Drug Administration; and
``(ii) the National Vaccine Program.
``(C) The Secretary shall convene a meeting requested under
subparagraph (A) not later than 120 days after receipt of the request
for the meeting.
``(3)(A) Upon the submission of a written request by a vaccine
developer, the Secretary, acting through the Director of the Centers
for Disease Control and Prevention, shall provide to the vaccine
developer any age-based or other demographically assessed disease
epidemiological analyses or data that--
``(i) are specified in the request;
``(ii) have been published;
``(iii) have been performed by or are in the possession of
the Centers;
``(iv) are not a trade secret or commercial or financial
information that is privileged or confidential and subject to
section 552(b)(4) of title 5, United States Code, or section
1905 of title 18, United States Code; and
``(v) do not contain individually identifiable information.
``(B) The Secretary shall provide analyses requested by a vaccine
manufacturer under subparagraph (A) not later than 120 calendar days
after receipt of the request for the analyses.
``(4) The Secretary shall promptly notify a vaccine developer if--
``(A) the Secretary becomes aware of any change to
information that was--
``(i) shared by the Secretary with the vaccine
developer during a meeting under paragraph (2); or
``(ii) provided by the Secretary to the vaccine
developer in one or more analyses under paragraph (3);
and
``(B) the change to such information may have implications
for the vaccine developer's vaccine research and
development.''.
Subtitle I--Orphan Product Extensions Now; Incentives for Certain
Products for Limited Populations
SEC. 2151. EXTENSION OF EXCLUSIVITY PERIODS FOR A DRUG APPROVED FOR A
NEW INDICATION FOR A RARE DISEASE OR CONDITION.
(a) In General.--Chapter V of the Federal Food, Drug, and Cosmetic
Act, as amended by sections 2062 and 2063, is further amended by
inserting after section 505H of such Act the following:
``SEC. 505I. EXTENSION OF EXCLUSIVITY PERIODS FOR A DRUG APPROVED FOR A
NEW INDICATION FOR A RARE DISEASE OR CONDITION.
``(a) Designation.--
``(1) In general.--The Secretary shall designate a drug as a
drug approved for a new indication to prevent, diagnose, or
treat a rare disease or condition for purposes of granting the
extensions under subsection (b) if--
``(A) prior to approval of an application or
supplemental application for the new indication, the
drug was approved or licensed for marketing under
section 505(c) of this Act or section 351(a) of the
Public Health Service Act, but was not so approved or
licensed for the new indication;
``(B)(i) the sponsor of the approved or licensed drug
files an application or a supplemental application for
approval of the new indication for use of the drug to
prevent, diagnose, or treat the rare disease or
condition; and
``(ii) the Secretary approves the application or
supplemental application; and
``(C) the application or supplemental application for
the new indication contains the consent of the
applicant to notice being given by the Secretary under
paragraph (4) respecting the designation of the drug.
``(2) Revocation of designation.--
``(A) In general.--Except as provided in subparagraph
(B), a designation under paragraph (1) shall not be
revoked for any reason.
``(B) Exception.--The Secretary may revoke a
designation of a drug under paragraph (1) if the
Secretary finds that the application or supplemental
application resulting in such designation contained an
untrue statement of material fact.
``(3) Notification prior to discontinuance of production for
solely commercial reasons.--A designation of a drug under
paragraph (1) shall be subject to the condition that the
sponsor of the drug will notify the Secretary of any
discontinuance of the production of the drug for solely
commercial reasons at least one year before such
discontinuance.
``(4) Notice to public.--Notice respecting the designation of
a drug under paragraph (1) shall be made available to the
public.
``(b) Extension.--If the Secretary designates a drug as a drug
approved for a new indication for a rare disease or condition, as
described in subsection (a)(1)--
``(1)(A) the 4-, 5-, and 7\1/2\-year periods described in
subsections (c)(3)(E)(ii) and (j)(5)(F)(ii) of section 505, the
3-year periods described in clauses (iii) and (iv) of
subsection (c)(3)(E) and clauses (iii) and (iv) of subsection
(j)(5)(F) of section 505, and the 7-year period described in
section 527, as applicable, shall be extended by 6 months; or
``(B) the 4- and 12-year periods described in subparagraphs
(A) and (B) of section 351(k)(7) of the Public Health Service
Act and the 7-year period described in section 527, as
applicable, shall be extended by 6 months; and
``(2)(A) if the drug is the subject of a listed patent for
which a certification has been submitted under subsection
(b)(2)(A)(ii) or (j)(2)(A)(vii)(II) of section 505 or a listed
patent for which a certification has been submitted under
subsections (b)(2)(A)(iii) or (j)(2)(A)(vii)(III) of section
505, the period during which an application may not be approved
under section 505(c)(3) or section 505(j)(5)(B) shall be
extended by a period of 6 months after the date the patent
expires (including any patent extensions); or
``(B) if the drug is the subject of a listed patent for which
a certification has been submitted under subsection
(b)(2)(A)(iv) or (j)(2)(A)(vii)(IV) of section 505, and in the
patent infringement litigation resulting from the certification
the court determines that the patent is valid and would be
infringed, the period during which an application may not be
approved under section 505(c)(3) or section 505(j)(5)(B) shall
be extended by a period of 6 months after the date the patent
expires (including any patent extensions).
``(c) Relation to Pediatric and Qualified Infectious Disease Product
Exclusivity.--Any extension under subsection (b) of a period shall be
in addition to any extension of the periods under sections 505A and
505E of this Act and section 351(m) of the Public Health Service Act,
as applicable, with respect to the drug.
``(d) Limitations.--The extension described in subsection (b) shall
not apply if the drug designated under subsection (a)(1) has previously
received an extension by operation of subsection (b).
``(e) Definition.--In this section, the term `rare disease or
condition' has the meaning given to such term in section 526(a)(2).''.
(b) Application.--Section 505G of the Federal Food, Drug, and
Cosmetic Act, as added by subsection (a), applies only with respect to
a drug for which an application or supplemental application described
in subsection (a)(1)(B)(i) of such section 505G is first approved under
section 505(c) of such Act (21 U.S.C. 355(c)) or section 351(a) of the
Public Health Service Act (42 U.S.C. 262(a)) on or after the date of
the enactment of this Act.
(c) Conforming Amendments.--
(1) Relation to pediatric exclusivity for drugs.--Section
505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) is amended--
(A) in subsection (b), by adding at the end the
following:
``(3) Relation to exclusivity for a drug approved for a new
indication for a rare disease or condition.--Notwithstanding
the references in paragraph (1) to the lengths of the
exclusivity periods after application of pediatric exclusivity,
the 6-month extensions described in paragraph (1) shall be in
addition to any extensions under section 505G.''; and
(B) in subsection (c), by adding at the end the
following:
``(3) Relation to exclusivity for a drug approved for a new
indication for a rare disease or condition.--Notwithstanding
the references in paragraph (1) to the lengths of the
exclusivity periods after application of pediatric exclusivity,
the 6-month extensions described in paragraph (1) shall be in
addition to any extensions under section 505G.''.
(2) Relation to exclusivity for new qualified infectious
disease products that are drugs.--Subsection (b) of section
505E of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355f) is amended--
(A) by amending the subsection heading to read as
follows: ``Relation to Pediatric Exclusivity and
Exclusivity for a Drug Approved for a New Indication
for a Rare Disease or Condition.--''; and
(B) by striking ``any extension of the period under
section 505A'' and inserting ``any extension of the
periods under sections 505A and 505G, as applicable,''.
(3) Relation to pediatric exclusivity for biological
products.--Section 351(m) of the Public Health Service Act (42
U.S.C. 262(m)) is amended by adding at the end the following:
``(5) Relation to exclusivity for a biological product
approved for a new indication for a rare disease or
condition.--Notwithstanding the references in paragraphs
(2)(A), (2)(B), (3)(A), and (3)(B) to the lengths of the
exclusivity periods after application of pediatric exclusivity,
the 6-month extensions described in such paragraphs shall be in
addition to any extensions under section 505G.''.
SEC. 2152. REAUTHORIZATION OF RARE PEDIATRIC DISEASE PRIORITY REVIEW
VOUCHER INCENTIVE PROGRAM.
(a) In General.--Section 529 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360ff) is amended--
(1) in subsection (a)--
(A) in paragraph (3), by amending subparagraph (A) to
read as follows:
``(A) The disease is a serious or life-threatening
disease in which the serious or life-threatening
manifestations primarily affect individuals aged from
birth to 18 years, including age groups often called
neonates, infants, children, and adolescents.''; and
(B) in paragraph (4)--
(i) in subparagraph (E), by striking ``and''
at the end;
(ii) in subparagraph (F), by striking the
period at the end and inserting ``; and''; and
(iii) by adding at the end the following:
``(G) is for a drug or biological product for which a
priority review voucher has not been issued under
section 524 (relating to tropical disease products).'';
and
(2) in subsection (b), by striking paragraph (5) and
inserting the following:
``(5) Termination of authority.--The Secretary may not award
any priority review vouchers under paragraph (1) after December
31, 2018.''.
(b) GAO Study and Report.--
(1) Study.--The Comptroller General of the United States
shall conduct a study on the effectiveness of awarding priority
review vouchers under section 529 of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 360ff) in providing incentives for
the development of drugs that treat or prevent rare pediatric
diseases (as defined in subsection (a)(3) of such section) that
would not otherwise have been developed. In conducting such
study, the Comptroller General shall examine the following:
(A) The indications for which each drug for which a
priority review voucher was awarded under such section
529 was approved under section 505 of such Act (21
U.S.C. 355) or section 351 of the Public Health Service
Act (42 U.S.C. 262).
(B) Whether the priority review voucher impacted a
sponsor's decision to invest in developing a drug to
treat or prevent a rare pediatric disease.
(C) An analysis of the drugs that utilized such
priority review vouchers, which shall include--
(i) the indications for which such drugs were
approved under section 505 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355) or
section 351 of the Public Health Service Act
(42 U.S.C. 262);
(ii) whether unmet medical needs were
addressed through the approval of such drugs,
including, for each such drug--
(I) if an alternative therapy was
previously available to treat the
indication; and
(II) the benefit or advantage the
drug provided over another available
therapy;
(iii) the number of patients potentially
treated by such drugs;
(iv) the value of the priority review voucher
if transferred; and
(v) the length of time between the date on
which a priority review voucher was awarded and
the date on which it was used.
(D) With respect to the priority review voucher
program under section 529 of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 360ff)--
(i) the resources used by, and burden placed
on, the Food and Drug Administration in
implementing such program, including the effect
of such program on the Food and Drug
Administration's review of drugs for which a
priority review voucher was not awarded or
used;
(ii) the impact of the program on the public
health as a result of the expedited review of
applications for drugs that treat or prevent
non-serious indications that are generally used
by the broader public; and
(iii) alternative approaches to improving
such program so that the program is
appropriately targeted toward providing
incentives for the development of clinically
important drugs that--
(I) prevent or treat rare pediatric
diseases; and
(II) would likely not otherwise have
been developed to prevent or treat such
diseases.
(2) Report.--Not later than December 31, 2017, the
Comptroller General of the United States shall submit to the
Committee on Energy and Commerce of the House of
Representatives and the Committee on Health, Education, Labor
and Pensions of the Senate a report containing the results of
the study of conducted under paragraph (1).
Subtitle J--Domestic Manufacturing and Export Efficiencies
SEC. 2161. GRANTS FOR STUDYING THE PROCESS OF CONTINUOUS DRUG
MANUFACTURING.
(a) In General.--The Commissioner of Food and Drugs may award grants
to institutions of higher education and nonprofit organizations for the
purpose of studying and recommending improvements to the process of
continuous manufacturing of drugs and biological products and similar
innovative monitoring and control techniques.
(b) Definitions.--In this section:
(1) The term ``drug'' has the meaning given to such term in
section 201 of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 321).
(2) The term ``biological product'' has the meaning given to
such term in section 351(i) of the Public Health Service Act
(42 U.S.C. 262(i)).
(3) The term ``institution of higher education'' has the
meaning given to such term in section 101 of the Higher
Education Act of 1965 (20 U.S.C. 1001).
(c) Authorization of Appropriations.--There is authorized to be
appropriated to carry out this section $5,000,000 for each of fiscal
years 2016 through 2020.
SEC. 2162. RE-EXPORTATION AMONG MEMBERS OF THE EUROPEAN ECONOMIC AREA.
Section 1003 of the Controlled Substances Import and Export Act (21
U.S.C. 953) is amended--
(1) in subsection (f)--
(A) in paragraph (5)--
(i) by striking ``(5)'' and inserting
``(5)(A)'';
(ii) by inserting ``, except that the
controlled substance may be exported from the
second country to another country that is a
member of the European Economic Area'' before
the period at the end; and
(iii) by adding at the end the following:
``(B) Subsequent to any re-exportation described in
subparagraph (A), a controlled substance may continue to be
exported from any country that is a member of the European
Economic Area to any other such country, provided that--
``(i) the conditions applicable with respect to the
first country under paragraphs (1), (2), (3), (4), (6),
and (7) are met by each subsequent country from which
the controlled substance is exported pursuant to this
paragraph; and
``(ii) the conditions applicable with respect to the
second country under such paragraphs are met by each
subsequent country to which the controlled substance is
exported pursuant to this paragraph.''; and
(B) in paragraph (6)--
(i) by striking ``(6)'' and inserting
``(6)(A)''; and
(ii) by adding at the end the following:
``(B) In the case of re-exportation among members of the
European Economic Area, within 30 days after each re-
exportation, the person who exported the controlled substance
from the United States delivers to the Attorney General--
``(i) documentation certifying that such re-
exportation has occurred; and
``(ii) information concerning the consignee, country,
and product.''; and
(2) by adding at the end the following:
``(g) Limitation.--The Attorney General shall not promulgate nor
enforce any regulation, subregulatory guidance, or enforcement policy
which impedes re-exportation among European Economic Area countries (as
provided in subsection (f)(5)), including by promulgating or enforcing
any requirement that--
``(1) re-exportation from the first country to the second
country or re-exportation from the second country to another
country (as such terms are used in subsection (f)) occur within
a specified period of time; or
``(2) information concerning the consignee, country, and
product be provided prior to exportation of the controlled
substance from the United States or prior to each re-
exportation among members of the European Economic Area.''.
Subtitle K--Enhancing Combination Products Review
SEC. 2181. ENHANCING COMBINATION PRODUCTS REVIEW.
Section 503(g)(4)(C) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 353(g)(4)(C)) is amended by adding at the end the following new
clause:
``(iii) Not later than 18 months after the date of the enactment of
the 21st Century Cures Act, the Secretary shall issue final guidance
that describes the responsibilities of each agency center regarding its
review of combination products. The Secretary shall, after soliciting
public comment, review and update the guidance periodically.''.
Subtitle L--Priority Review for Breakthrough Devices
SEC. 2201. PRIORITY REVIEW FOR BREAKTHROUGH DEVICES.
(a) In General.--Chapter V of the Federal Food, Drug, and Cosmetic
Act is amended--
(1) in section 515(d)--
(A) by striking paragraph (5); and
(B) by redesignating paragraph (6) as paragraph (5);
and
(2) by inserting after section 515A (21 U.S.C. 360e-1) the
following:
``SEC. 515B. PRIORITY REVIEW FOR BREAKTHROUGH DEVICES.
``(a) In General.--In order to provide for more effective treatment
or diagnosis of life-threatening or irreversibly debilitating human
diseases or conditions, the Secretary shall establish a program to
provide priority review for devices--
``(1) representing breakthrough technologies;
``(2) for which no approved alternatives exist;
``(3) offering significant advantages over existing approved
or cleared alternatives, including the potential to, compared
to existing approved or cleared alternatives, reduce or
eliminate the need for hospitalization, improve patient quality
of life, facilitate patients' ability to manage their own care
(such as through self-directed personal assistance), or
establish long-term clinical efficiencies; or
``(4) the availability of which is in the best interest of
patients.
``(b) Request for Designation.--A sponsor of a device may request
that the Secretary designate the device for priority review under this
section. Any such request for designation may be made at any time prior
to the submission of an application under section 515(c), a petition
for classification under section 513(f)(2), or a notification under
section 510(k).
``(c) Designation Process.--
``(1) In general.--Not later than 60 calendar days after the
receipt of a request under subsection (b), the Secretary shall
determine whether the device that is the subject of the request
meets the criteria described in subsection (a). If the
Secretary determines that the device meets the criteria, the
Secretary shall designate the device for priority review.
``(2) Review.--Review of a request under subsection (b) shall
be undertaken by a team that is composed of experienced staff
and managers of the Food and Drug Administration and is chaired
by a senior manager.
``(3) Designation determination.--A determination approving
or denying a request under subsection (b) shall be considered a
significant decision under section 517A and the Secretary shall
provide a written, substantive summary of the basis for the
determination in accordance with section 517A(a).
``(4) Reconsideration.--
``(A) Request for reconsideration.--Any person whose
request under subsection (b) is denied may, within 30
days of the denial, request reconsideration of the
denial in accordance with section 517A(b)--
``(i) based upon the submission of documents
by such person; or
``(ii) based upon such documents and a
meeting or teleconference.
``(B) Response.--Reconsideration of a designation
determination under this paragraph shall be conducted
in accordance with section 517A(b).
``(5) Withdrawal.--If the Secretary approves a priority
review designation for a device under this section, the
Secretary may not withdraw the designation based on the fact
that the criteria specified in subsection (a) are no longer met
because of the subsequent clearance or approval of another
device that was designated under--
``(A) this section; or
``(B) section 515(d)(5) (as in effect immediately
prior to the enactment of the 21st Century Cures Act).
``(d) Priority Review.--
``(1) Actions.--For purposes of expediting the development
and review of devices designated under subsection (c), the
Secretary shall--
``(A) assign a team of staff, including a team leader
with appropriate subject matter expertise and
experience, for each device for which a request is
submitted under subsection (b);
``(B) provide for oversight of the team by senior
agency personnel to facilitate the efficient
development of the device and the efficient review of
any submission described in subsection (b) for the
device;
``(C) adopt an efficient process for timely dispute
resolution;
``(D) provide for interactive communication with the
sponsor of the device during the review process;
``(E) expedite the Secretary's review of
manufacturing and quality systems compliance, as
applicable;
``(F) disclose to the sponsor in advance the topics
of any consultation concerning the sponsor's device
that the Secretary intends to undertake with external
experts or an advisory committee and provide the
sponsor an opportunity to recommend such external
experts;
``(G) for applications submitted under section
515(c), provide for advisory committee input, as the
Secretary determines appropriate (including in response
to the request of the sponsor); and
``(H) assign staff to be available within a
reasonable time to address questions posed by
institutional review committees concerning the
conditions and clinical testing requirements applicable
to the investigational use of the device pursuant to an
exemption under section 520(g).
``(2) Additional actions.--In addition to the actions
described in paragraph (1), for purposes of expediting the
development and review of devices designated under subsection
(c), the Secretary, in collaboration with the device sponsor,
may, as appropriate--
``(A) coordinate with the sponsor regarding early
agreement on a data development plan;
``(B) take steps to ensure that the design of
clinical trials is as efficient as practicable, such as
through adoption of shorter or smaller clinical trials,
application of surrogate endpoints, and use of adaptive
trial designs and Bayesian statistics, to the extent
scientifically appropriate;
``(C) facilitate, to the extent scientifically
appropriate, expedited and efficient development and
review of the device through utilization of timely
postmarket data collection, with regard to applications
for approval under section 515(c); and
``(D) agree to clinical protocols that the Secretary
will consider binding on the Secretary and the sponsor,
subject to--
``(i) changes agreed to by the sponsor and
the Secretary;
``(ii) changes that the Secretary determines
are required to prevent an unreasonable risk to
the public health; or
``(iii) the identification of a substantial
scientific issue determined by the Secretary to
be essential to the safety or effectiveness of
the device involved.
``(e) Priority Review Guidance.--
``(1) Content.--The Secretary shall issue guidance on the
implementation of this section. Such guidance shall include the
following:
``(A) The process for a person to seek a priority
review designation.
``(B) A template for requests under subsection (b).
``(C) The criteria the Secretary will use in
evaluating a request for priority review.
``(D) The standards the Secretary will use in
assigning a team of staff, including team leaders, to
review devices designated for priority review,
including any training required for such personnel on
effective and efficient review.
``(2) Process.--Prior to finalizing the guidance under
paragraph (1), the Secretary shall propose such guidance for
public comment.
``(f) Construction.--
``(1) Purpose.--This section is intended to encourage the
Secretary and provide the Secretary sufficient authorities to
apply efficient and flexible approaches to expedite the
development of, and prioritize the agency's review of, devices
that represent breakthrough technologies.
``(2) Construction.--Nothing in this section shall be
construed to alter the criteria and standards for evaluating an
application pursuant to section 515(c), a report and request
for classification under section 513(f)(2), or a report under
section 510(k), including the recognition of valid scientific
evidence as described in section 513(a)(3)(B), and
consideration of the least burdensome means of evaluating
device effectiveness or demonstrating substantial equivalence
between devices with differing technological characteristics,
as applicable. Nothing in this section alters the authority of
the Secretary to act on an application pursuant to section
515(d) before completion of an establishment inspection, as the
Secretary deems appropriate.''.
(b) Conforming Amendment Related to Designation Determinations.--
Section 517A(a)(1) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360g-1(a)(1)) is amended by inserting ``a request for
designation under section 515B,'' after ``an application under section
515,''.
Subtitle M--Medical Device Regulatory Process Improvements
SEC. 2221. THIRD-PARTY QUALITY SYSTEM ASSESSMENT.
(a) Establishment of Third-Party Quality System Assessment Program.--
Chapter V of the Federal Food, Drug, and Cosmetic Act is amended by
inserting after section 524A (21 U.S.C. 360n-1) the following new
section:
``SEC. 524B. THIRD-PARTY QUALITY SYSTEM ASSESSMENT.
``(a) Accreditation and Assessment.--
``(1) In general; certification of device quality system.--
The Secretary shall, in accordance with this section, establish
a third-party quality system assessment program--
``(A) to accredit persons to assess whether a
requestor's quality system, including its design
controls, can reasonably assure the safety and
effectiveness of in-scope devices subject to device-
related changes;
``(B) under which accredited persons shall (as
applicable) certify that a requestor's quality system
meets the criteria included in the guidance issued
under paragraph (5) with respect to the in-scope
devices at issue; and
``(C) under which the Secretary shall rely on such
certifications for purposes of determining the safety
and effectiveness (or as applicable, substantial
equivalence) of in-scope devices subject to the device-
related changes involved, in lieu of compliance with
the following submission requirements:
``(i) A premarket notification.
``(ii) A thirty-day notice.
``(iii) A Special PMA supplement.
``(2) Definitions.--For purposes of this section-
``(A) the term `device-related changes' means changes
made by a requestor with respect to in-scope devices,
which are--
``(i) changes to a device found to be
substantially equivalent under sections 513(i)
and 510(k) to a predicate device, that--
``(I) would otherwise be subject to a
premarket notification; and
``(II) do not alter--
``(aa) the intended use of
the changed device; or
``(bb) the fundamental
scientific technology of such
device;
``(ii) manufacturing changes subject to a 30-
day notice;
``(iii) changes that qualify for a Special
PMA Supplement; and
``(iv) such other changes relating to the
devices or the device manufacturing process as
the Secretary determines appropriate;
``(B) the term `in-scope device' means a device
within the scope of devices agreed to by the requestor
and the accredited person for purposes of a request for
certification under this section;
``(C) the term `premarket notification' means a
premarket notification under section 510(k);
``(D) the term `quality system' means the methods
used in, and the facilities and controls used for, the
design, manufacture, packaging, labeling, storage,
installation, and servicing of devices, as described in
section 520(f);
``(E) the term `requestor' means a device
manufacturer that is seeking certification under this
section of a quality system used by such manufacturer;
``(F) the term `Special PMA' means a Special PMA
supplement under section 814.39(d) of title 21, Code of
Federal Regulations (or any successor regulations); and
``(G) the term `thirty-day notice' means a notice
described in section 515(d)(6).
``(3) Accreditation process; accreditation renewal.--Except
as inconsistent with this section, the process and
qualifications for accreditation of persons and renewal of such
accreditation under section 704(g) shall apply with respect to
accreditation of persons and renewal of such accreditation
under this section.
``(4) Use of accredited parties to conduct assessments.--
``(A) Initiation of assessment services.--
``(i) Date assessments authorized.--Beginning
after the date on which the final guidance is
issued under paragraph (5), an accredited
person may conduct an assessment under this
section.
``(ii) Initiation of assessments.--Use of one
or more accredited persons to assess a
requestor's quality system under this section
with respect to in-scope devices shall be at
the initiation of the person who registers and
lists the devices at issue under section 510.
``(B) Compensation.--Compensation for such accredited
persons shall--
``(i) be determined by agreement between the
accredited person and the person who engages
the services of the accredited person; and
``(ii) be paid by the person who engages such
services.
``(C) Accredited person selection.--Each person who
chooses to use an accredited person to assess a
requestor's quality system, as described in this
section, shall select the accredited person from a list
of such persons published by the Secretary in
accordance with section 704(g)(4).
``(5) Guidance; criteria for certification.--
``(A) In general.--The criteria for certification of
a quality system under this section shall be as
specified by the Secretary in guidance issued under
this paragraph.
``(B) Contents; certification criteria.--The guidance
under this paragraph shall include specification of--
``(i) evaluative criteria to be used by an
accredited person to assess and, as applicable,
certify a requestor's quality system under this
section with respect to in-scope devices; and
``(ii) criteria for accredited persons to
apply for a waiver of, and exemptions from, the
certification criteria under clause (i).
``(C) Timeframe for issuing guidance.--The Secretary
shall issue under this paragraph--
``(i) draft guidance not later than 12 months
after the enactment of the 21st Century Cures
Act; and
``(ii) final guidance not later than 12
months after issuance of the draft guidance
under clause (i).
``(b) Use of Third-Party Assessment.--
``(1) Assessment summary; certification.--
``(A) Submission of assessment to secretary.--An
accredited person who assesses a requestor's quality
system under subsection (a) shall submit to the
Secretary a summary of the assessment--
``(i) within 30 days of the assessment; and
``(ii) which shall include (as applicable)--
``(I) the accredited person's
certification that the requestor has
satisfied the criteria specified in the
guidance issued under subsection (a)(5)
for quality system certification with
respect to the in-scope devices at
issue; and
``(II) any waivers or exemptions from
such criteria applied by the accredited
person.
``(B) Treatment of assessments.--Subject to action by
the Secretary under subparagraph (C), with respect to
assessments which include a certification under this
section--
``(i) the Secretary's review of the
assessment summary shall be deemed complete on
the day that is 30 days after the date on which
the Secretary receives the summary under
subparagraph (A); and
``(ii) the assessment summary and
certification of the quality system of a
requestor shall be deemed accepted by the
Secretary on such 30th day.
``(C) Actions by secretary.--
``(i) In general.--Within 30 days of
receiving an assessment summary and
certification under subparagraph (A), the
Secretary may, by written notice to the
accredited person submitting such assessment
certification, deem any such certification to
be provisional beyond such 30-day period,
suspended pending further review by the
Secretary, or otherwise qualified or cancelled,
based on the Secretary's determination that (as
applicable)--
``(I) additional information is
needed to support such certification;
``(II) such assessment or
certification is unwarranted; or
``(III) such action with regard to
the certification is otherwise
justified according to such factors and
criteria as the Secretary finds
appropriate.
``(ii) Acceptance of certification.--If
following action by the Secretary under clause
(i) with respect to a certification, the
Secretary determines that such certification is
acceptable, the Secretary shall issue written
notice to the applicable accredited person
indicating such acceptance.
``(2) Notifications to secretary by certified requestors or
accredited persons for program evaluation purposes.--
``(A) Annual summary report for device-related
changes otherwise subject to premarket notification.--A
requestor whose quality system is certified under this
section that effectuates device-related changes with
respect to in-scope devices, without prior submission
of a premarket notification, shall ensure that an
annual summary report is submitted to the Secretary by
the accredited person which--
``(i) describes the changes made to the in-
scope device; and
``(ii) indicates the effective dates of such
changes.
``(B) Periodic notification for manufacturing changes
otherwise subject to thirty-day notice.--A requestor
whose quality system is certified under this section
that effectuates device-related changes with respect to
in-scope devices, without prior submission of a thirty-
day notice, shall provide notification to the Secretary
of such changes in the requestor's next periodic report
under section 814.84(b) of title 21, Code of Federal
Regulations (or any successor regulation). Such
notification shall--
``(i) describe the changes made; and
``(ii) indicate the effective dates of such
changes.
``(C) Periodic notification for device-related
changes otherwise subject to special pma supplement.--A
requestor whose quality system is certified under this
section that effectuates device-related changes with
respect to in-scope devices, without prior submission
of a Special PMA Supplement, shall provide notification
to the Secretary of such changes in the requestor's
next periodic report under section 814.84(b) of title
21, Code of Federal Regulations (or any successor
regulation). Such notification shall--
``(i) describe the changes made, including a
full explanation of the basis for the changes;
and
``(ii) indicate the effective dates of such
changes.
``(D) Use of notifications for program evaluation
purposes.--Information submitted to the Secretary under
subparagraphs (A) through (C) shall be used by the
Secretary for purposes of the program evaluation under
subsection (d).
``(c) Duration and Effect of Certification.--A certification under
this section--
``(1) shall remain in effect for a period of 2 years from the
date such certification is accepted by the Secretary, subject
to paragraph (6);
``(2) may be renewed through the process described in
subsection (a)(3);
``(3) shall continue to apply with respect to device-related
changes made during such 2-year period, provided the
certification remains in effect, irrespective of whether such
certification is renewed after such 2-year period;
``(4) shall have no effect on the need to comply with
applicable submission requirements specified in subsection
(a)(1)(C) with respect to any change pertaining to in-scope
devices which is not a device-related change under subsection
(a)(2);
``(5) shall have no effect on the authority of the Secretary
to conduct an inspection or otherwise determine whether the
requestor has complied with the applicable requirements of this
Act; and
``(6) may be revoked by the Secretary upon a determination
that the requestor's quality system no longer meets the
certification criteria specified in the guidance issued under
subsection (a)(5) with respect to the in-scope devices at
issue.
``(d) Notice of Revocation.--The Secretary shall provide written
notification to the requestor of a revocation pursuant to subsection
(c)(6) not later than 10 business days after the determination
described in such subsection. Upon receipt of the written notification,
the requestor shall satisfy the applicable submission requirements
specified in subsection (a)(1)(C) for any device-related changes
effectuated after the date of such determination. After such
revocation, such requestor is eligible to seek re-certification under
this section of its quality system.
``(e) Program Evaluation; Sunset.--
``(1) Program evaluation and report.--
``(A) Evaluation.--The Secretary shall complete an
evaluation of the third-party quality system assessment
program under this section no later than January 31,
2021, based on--
``(i) analysis of information from a
representative group of device manufacturers
obtained from notifications provided by
certified requestors or accredited persons
under subsection (b)(2); and
``(ii) such other available information and
data as the Secretary determines appropriate.
``(B) Report.--No later than 1 year after completing
the evaluation under subparagraph (A), the Secretary
shall issue a report of the evaluation's findings on
the website of the Food and Drug Administration, which
shall include the Secretary's recommendations with
respect to continuation and as applicable expansion of
the program under this section to encompass--
``(i) device submissions beyond those
identified in subsection (a)(1)(C); and
``(ii) device changes beyond those described
in subsection (a)(2)(A).
``(2) Sunset.--This section shall cease to be effective
October 1, 2022.
``(f) Rule of Construction.--Nothing in this section shall be
construed to limit the authority of the Secretary to request and review
the complete assessment of a certified requestor under this section on
a for-cause basis.''.
(b) Conforming Amendments.--
(1) Requirements for premarket approval supplements.--Section
515(d)(5)(A)(i) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360e(d)(5)(A)(i)), as redesignated by section 2201, is
further amended by inserting ``, subject to section 524B''
after ``that affects safety or effectiveness''.
(2) Requirements for thirty-day notice.--Section
515(d)(5)(A)(ii) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360e(d)(5)(A)(ii)), as redesignated by section 2201,
is further amended by inserting ``, subject to section 524B''
after ``the date on which the Secretary receives the notice''.
(3) Requirements for premarket notification; technical
correction to reference to section 510(k).--Section 510(l) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360(l)) is
amended by striking ``of this subsection under subsection (m)''
and inserting ``of subsection (k) under subsection (m) or
section 524B''.
(4) Misbranded devices.--Section 502(t) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 352(t)) is amended by
inserting ``or 524B'' after ``section 519''.
SEC. 2222. VALID SCIENTIFIC EVIDENCE.
Section 513(a)(3)(B) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360c(a)(3)(B)) is amended--
(1) by redesignating clauses (i) and (ii) as subclauses (I)
and (II), respectively;
(2) by striking ``(B) If the Secretary'' and inserting
``(B)(i) If the Secretary''; and
(3) by adding at the end the following:
``(ii) For purposes of clause (i), valid scientific evidence may
include--
``(I) evidence described in well-documented case histories,
including registry data, that are collected and monitored under
an acceptable protocol;
``(II) studies published in peer-reviewed journals; and
``(III) data collected in countries other than the United
States so long as such data otherwise meet the criteria
specified in this subparagraph.
``(iii) In the case of a study published in a peer-reviewed journal
that is offered as valid scientific evidence for purposes of clause
(i), the Secretary may request data underlying the study if--
``(I) the Secretary, in making such request, complies with
the requirement of subparagraph (D)(ii) to consider the least
burdensome appropriate means of evaluating device effectiveness
or subsection (i)(1)(D) to consider the least burdensome means
of determining substantial equivalence, as applicable;
``(II) the Secretary furnishes a written rationale for so
requesting the underlying data together with such request; and
``(III) if the requested underlying data for such a study are
unavailable, the Secretary shall consider such study to be part
of the totality of the evidence with respect to the device, as
the Secretary determines appropriate.''.
SEC. 2223. TRAINING AND OVERSIGHT IN LEAST BURDENSOME APPROPRIATE MEANS
CONCEPT.
(a) In General.--Section 513 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360c) is amended by adding at the end the following:
``(j) Training and Oversight in Least Burdensome Appropriate Means
Concept.--
``(1) Training.--Each employee of the Food and Drug
Administration who is involved in the review of premarket
submissions under section 515 or section 510(k), including
supervisors, shall receive training regarding the meaning and
implementation of the least burdensome appropriate means
concept in the context of the use of that term in subsections
(a)(3)(D) and (i)(1)(D) of this section and in section
515(c)(5).
``(2) Guidance documents.--
``(A) Draft updated guidance.--Not later than 12
months after the date of enactment of the 21st Century
Cures Act, the Secretary shall issue a draft guidance
document updating the October 4, 2002, guidance
document entitled `The Least Burdensome Provision of
the FDA Modernization Act of 1997: Concept and
Principles; Final Guidance for FDA and Industry'.
``(B) Meeting of stakeholders.--In developing such
draft guidance document, the Secretary shall convene a
meeting of stakeholders to ensure a full record to
support the publication of such document.
``(3) Ombudsman audit.--Not later than 18 months after the
date of issuance of final version of the draft guidance under
paragraph (2), the ombudsman for the organizational unit of the
Food and Drug Administration responsible for the premarket
review of devices shall--
``(A) conduct, or have conducted, an audit of the
training described in paragraph (1); and
``(B) include in such audit interviews with a
representative sample of persons from industry
regarding their experience in the device premarket
review process.''.
(b) Additional Information Regarding Premarket Applications.--
Subsection (c) of section 515 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360e) is amended by adding at the end the following:
``(5)(A) Whenever the Secretary requests additional information from
an applicant regarding an application under paragraph (1), the
Secretary shall consider the least burdensome appropriate means
necessary to demonstrate device safety and effectiveness, and request
information accordingly.
``(B) For purposes of subparagraph (A), the term `necessary' means
the minimum required information that would support a determination by
the Secretary that an application provides a reasonable assurance of
the safety and effectiveness of the device.
``(C) Nothing in this paragraph alters the standards for premarket
approval of a device.''.
SEC. 2224. RECOGNITION OF STANDARDS.
Section 514(c) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360d(c)) is amended--
(1) in paragraph (1), by inserting after subparagraph (B) the
following new subparagraphs:
``(C)(i) Any person may submit a request for recognition under
subparagraph (A) of all or part of an appropriate standard established
by a nationally or internationally recognized standard organization.
``(ii) Not later than 60 days after the Secretary receives such a
request, the Secretary shall--
``(I) make a determination to recognize all, part, or none of
the standard that is the subject of the request; and
``(II) issue to the person who submitted such request a
response in writing that states the Secretary's rationale for
that determination, including the scientific, technical,
regulatory, or other basis for such determination.
``(iii) The Secretary shall make a response issued under clause
(ii)(II) publicly available, in such manner as the Secretary determines
appropriate.
``(iv) The Secretary shall take such actions as may be necessary to
implement all or part of a standard recognized under clause (i)(I), in
accordance with subparagraph (A).
``(D) The Secretary shall make publicly available, in such manner as
the Secretary determines appropriate, the rationale for recognition
under subparagraph (A) of part of a standard, including the scientific,
technical, regulatory, or other basis for such recognition.''; and
(2) by adding at the end the following new paragraphs:
``(4) Training on use of standards.--The Secretary shall
provide to all employees of the Food and Drug Administration
who review premarket submissions for devices periodic training
on the concept and use of recognized standards for purposes of
meeting a premarket submission requirement or other applicable
requirement under this Act, including standards relevant to an
employee's area of device review.
``(5) Guidance.--
``(A) Draft guidance.--The Secretary shall publish
guidance identifying the principles for recognizing
standards under this section. In publishing such
guidance, the Secretary shall consider--
``(i) the experience with, and reliance on, a
standard by other Federal regulatory
authorities and the device industry; and
``(ii) whether recognition of a standard will
promote harmonization among regulatory
authorities in the regulation of devices.
``(B) Timing.--The Secretary shall publish--
``(i) draft guidance under subparagraph (A)
not later than 12 months after the date of the
enactment of the 21st Century Cures Act; and
``(ii) final guidance not later than 12
months after the close of the public comment
period for the draft guidance under clause
(i).''.
SEC. 2225. EASING REGULATORY BURDEN WITH RESPECT TO CERTAIN CLASS I AND
CLASS II DEVICES.
(a) Class I Devices.--Section 510(l) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360(l)) is amended--
(1) by striking ``A report under subsection (k)'' and
inserting ``(1) A report under subsection (k)''; and
(2) by adding at the end the following new paragraph:
``(2) Not later than 120 days after the date of the enactment of the
21st Century Cures Act, the Secretary shall identify, through
publication in the Federal Register, any type of class I device that
the Secretary determines no longer requires a report under subsection
(k) to provide reasonable assurance of safety and effectiveness. Upon
such publication--
``(A) each type of class I device so identified shall be
exempt from the requirement for a report under subsection (k);
and
``(B) the classification regulation applicable to each such
type of device shall be deemed amended to incorporate such
exemption.''.
(b) Class II Devices.--Section 510(m) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360(m)) is amended--
(1) by striking paragraph (1) and inserting the following new
paragraph: ``(1) The Secretary shall--
``(A) not later than 60 days after the date of the enactment
of the 21st Century Cures Act--
``(i) publish in the Federal Register a notice that
contains a list of each type of class II device that
the Secretary determines no longer requires a report
under subsection (k) to provide reasonable assurance of
safety and effectiveness; and
``(ii) provide for a period of not less than 60 days
for public comment beginning on the date of the
publication of such notice; and
``(B) not later than 180 days after the date of the enactment
of 21st Century Cures Act, publish in the Federal Register a
list representing the Secretary's final determination with
respect to the devices included in the list published under
subparagraph (A).'';
(2) in paragraph (2)--
(A) by striking ``1 day after the date of the
publication of a list under this subsection,'' and
inserting ``1 day after the date of publication of the
final list under paragraph (1)(B),''; and
(B) by striking ``30-day period'' and inserting ``60-
day period''; and
(3) by adding at the end the following new paragraph:
``(3) Upon the publication of the final list under paragraph (1)(B)--
``(A) each type of class II device so listed shall be exempt
from the requirement for a report under subsection (k); and
``(B) the classification regulation applicable to each such
type of device shall be deemed amended to incorporate such
exemption.''.
SEC. 2226. ADVISORY COMMITTEE PROCESS.
(a) Classification Panels.--Paragraph (5) of section 513(b) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360c(b)) is amended--
(1) by striking ``(5)'' and inserting ``(5)(A)''; and
(2) by adding at the end the following:
``(B) When a device is specifically the subject of review by a
classification panel, the Secretary shall--
``(i) ensure that adequate expertise is represented on the
classification panel to assess--
``(I) the disease or condition which the device is
intended to cure, treat, mitigate, prevent, or
diagnose; and
``(II) the technology of the device; and
``(ii) as part of the process to ensure adequate expertise
under clause (i), give due consideration to the recommendations
of the person whose premarket submission is subject to panel
review on the expertise needed among the voting members of the
panel.
``(C) For review by a classification panel of a premarket submission
for a device, the Secretary shall--
``(i) provide an opportunity for the person whose premarket
submission is subject to panel review to provide
recommendations on the expertise needed among the voting
members of the panel; and
``(ii) give due consideration to such recommendations and
ensure that adequate expertise is represented on advisory
panels to assess--
``(I) the disease or condition for which the device
is intended to cure, treat, mitigate, prevent, or
diagnose; and
``(II) the technology of the device.
``(D) For purposes of subparagraph (B)(ii), the term `adequate
expertise' means, with respect to the membership of the classification
panel reviewing a premarket submission, that such membership includes--
``(i) two or more voting members, with a specialty or other
expertise clinically relevant to the device under review; and
``(ii) at least one voting member who is knowledgeable about
the technology of the device.''.
(b) Panel Review Process.--Section 513(b)(6) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360c(b)(6)) is amended--
(1) in subparagraph (A)(iii), by inserting before the period
at the end ``, including by designating a representative who
will be provided a time during the panel meeting to address the
panel individually (or accompanied by experts selected by such
representative) for the purpose of correcting misstatements of
fact or providing clarifying information, subject to the
discretion of the panel chairperson''; and
(2) by striking subparagraph (B) and inserting the following
new subparagraph:
``(B)(i) Any meeting of a classification panel for a device that is
specifically the subject of review shall--
``(I) provide adequate time for initial presentations by the
person whose device is specifically the subject of a
classification panel review and by the Secretary; and
``(II) encourage free and open participation by all
interested persons.
``(ii) Following the initial presentations described in clause (i),
the panel may--
``(I) pose questions to a designated representative described
in subparagraph (A)(iii); and
``(II) consider the responses to such questions in the
panel's review of the device that is specifically the subject
of review by the panel.''.
SEC. 2227. HUMANITARIAN DEVICE EXEMPTION APPLICATION.
(a) In General.--Section 520(m) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360j) is amended--
(1) in paragraph (1) by striking ``fewer than 4,000'' and
inserting ``not more than 8,000'';
(2) in paragraph (2)(A) by striking ``fewer than 4,000'' and
inserting ``not more than 8,000''; and
(3) in paragraph (6)(A)(ii), by striking ``4,000'' and
inserting ``8,000''
(b) Guidance Document on Probable Benefit.--Not later than 18 months
after the date of enactment of this Act, the Secretary of Health and
Human Services, acting through the Commissioner of Food and Drugs,
shall publish a draft guidance document that defines the criteria for
establishing ``probable benefit'' as that term is used in section
520(m)(2)(C) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360j(m)(2)(C)).
SEC. 2228. CLIA WAIVER STUDY DESIGN GUIDANCE FOR IN VITRO DIAGNOSTICS.
(a) Draft Revised Guidance.--Not later than 12 months after the date
of the enactment of this Act, the Secretary of Health and Human
Services shall publish a draft guidance that--
(1) revises ``Section V. Demonstrating Insignificant Risk of
an Erroneous Result--`Accuracy''' of the guidance entitled
``Recommendations for Clinical Laboratory Improvement
Amendments of 1988 (CLIA) Waiver Applications for Manufacturers
of In Vitro Diagnostic Devices'' and dated January 30, 2008;
and
(2) includes guidance on the appropriate use of comparable
performance between a waived user and a moderately complex
laboratory user to demonstrate accuracy.
(b) Final Revised Guidance.--The Secretary of Health and Human
Services shall finalize the draft guidance published under subsection
(a) not later than 12 months after the comment period for such draft
guidance closes.
Subtitle N--Sensible Oversight for Technology Which Advances Regulatory
Efficiency
SEC. 2241. HEALTH SOFTWARE.
Section 201 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
321) is amended by adding at the end the following:
``(ss)(1) The term `health software' means software that does not,
through use of an in vitro diagnostic device or signal acquisition
system, acquire, process, or analyze an image or physiological signal,
is not an accessory, is not an integral part of a device necessary to
support the use of the device, is not used in the manufacture and
transfusion of blood and blood components to assist in the prevention
of disease in humans, and--
``(A) is intended for use for administrative or operational
support or the processing and maintenance of financial records;
``(B) is intended for use in clinical, laboratory, or
administrative workflow and related recordkeeping;
``(C)(i) is intended for use solely in the transfer,
aggregation, conversion (in accordance with a present
specification), storage, management, retrieval, or transmission
of data or information;
``(ii) utilizes a connectivity software platform, electronic
or electrical hardware, or a physical communications
infrastructure; and
``(iii) is not intended for use--
``(I) in active patient monitoring; or
``(II) in controlling or altering the functions or
parameters of a device that is connected to such
software;
``(D) is intended for use to organize and present information
for health or wellness education or for use in maintaining a
healthy lifestyle, including medication adherence and health
management tools;
``(E) is intended for use to analyze information to provide
general health information that does not include patient-
specific recommended options to consider in the prevention,
diagnosis, treatment, cure, or mitigation of a particular
disease or condition; or
``(F) is intended for use to analyze information to provide
patient-specific recommended options to consider in the
prevention, diagnosis, treatment, cure, or mitigation of a
particular disease or condition.
``(2) The term `accessory' means a product that--
``(A) is intended for use with one or more parent devices;
``(B) is intended to support, supplement, or augment the
performance of one or more parent devices; and
``(C) shall be classified by the Secretary--
``(i) according to its intended use; and
``(ii) independently of any classification of any
parent device with which it is used.''.
SEC. 2242. APPLICABILITY AND INAPPLICABILITY OF REGULATION.
Subchapter A of chapter V of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 351 et seq.), as amended by section 2221(a), is further
amended by adding at the end the following:
``SEC. 524C. HEALTH SOFTWARE.
``(a) Inapplicability of Regulation to Health Software.--Except as
provided in subsection (b), health software shall not be subject to
regulation under this Act.
``(b) Exception.--
``(1) In general.--Subsection (a) shall not apply with
respect to a software product--
``(A) of a type described in subparagraph (F) of
section 201(ss)(1); and
``(B) that the Secretary determines poses a
significant risk to patient safety.
``(2) Considerations.--In making a determination under
subparagraph (B) of paragraph (1) with respect to a product to
which such paragraph applies, the Secretary shall consider the
following:
``(A) The likelihood and severity of patient harm if
the product were to not perform as intended.
``(B) The extent to which the product is intended to
support the clinical judgment of a medical
professional.
``(C) Whether there is a reasonable opportunity for a
medical professional to review the basis of the
information or treatment recommendation provided by the
product.
``(D) The intended user and user environment, such as
whether a medical professional will use a software
product of a type described in subparagraph (F) of
section 201(ss)(1).
``(c) Delegation.--The Secretary shall delegate primary jurisdiction
for regulating a software product determined under subsection (b) to be
subject to regulation under this Act to the center at the Food and Drug
Administration charged with regulating devices.
``(d) Regulation of Software.--
``(1) In general.--The Secretary shall review existing
regulations and guidance regarding the regulation of software
under this Act. The Secretary may implement a new framework for
the regulation of software and shall, as appropriate, modify
such regulations and guidance or issue new regulations or
guidance.
``(2) Issuance by order.--Notwithstanding subchapter II of
chapter 5 of title 5, United States Code, the Secretary may
modify or issue regulations for the regulation of software
under this Act by administrative order published in the Federal
Register following the publication of a proposed order.
``(3) Areas under review.--The review of existing regulations
and guidance under paragraph (1) may include review of the
following areas:
``(A) Classification of software.
``(B) Standards for development of software.
``(C) Standards for validation and verification of
software.
``(D) Review of software.
``(E) Modifications to software.
``(F) Manufacturing of software.
``(G) Quality systems for software.
``(H) Labeling requirements for software.
``(I) Postmarketing requirements for reporting of
adverse events.
``(4) Process for issuing proposed regulations,
administrative order, and guidance.--Not later than 18 months
after the date of enactment of this section, the Secretary
shall consult with external stakeholders (including patients,
industry, health care providers, academia, and government) to
gather input before issuing regulations, an administrative
order, and guidance under this subsection.
``(e) Rule of Construction.--Nothing in this section shall be
construed as providing the Secretary with the authority to regulate
under this Act any health software product of the type described in
subparagraph (F) of section 201(ss)(1) unless and until the Secretary
has made a determination described in subsection (b)(1)(B) with respect
to such product.''.
SEC. 2243. EXCLUSION FROM DEFINITION OF DEVICE.
Section 201(h) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
321) is amended--
(1) in subparagraph (2), by striking ``or'' after ``or other
animals,'';
(2) in subparagraph (3), by striking ``and'' and inserting
``or''; and
(3) by inserting after subparagraph (3) the following:
``(4) not health software (other than software determined to
be a risk to patient safety under section 524B(b)), and''.
Subtitle O--Streamlining Clinical Trials
SEC. 2261. PROTECTION OF HUMAN SUBJECTS IN RESEARCH; APPLICABILITY OF
RULES.
(a) In General.--In order to simplify and facilitate compliance by
researchers with applicable regulations for the protection of human
subjects in research, the Secretary of Health and Human Services shall,
to the extent possible and consistent with other statutory provisions,
harmonize differences between the HHS Human Subject Regulations and the
FDA Human Subject Regulations in accordance with subsection (b).
(b) Avoiding Regulatory Duplication and Unnecessary Delays.--
(1) In general.--The Secretary shall--
(A) make such modifications to the provisions of the
HHS Human Subject Regulations, the FDA Human Subject
Regulations, and the vulnerable-populations rules as
may be necessary--
(i) to reduce regulatory duplication and
unnecessary delays;
(ii) to modernize such provisions in the
context of multisite and cooperative research
projects; and
(iii) to incorporate local considerations,
community values, and mechanisms to protect
vulnerable populations; and
(B) ensure that human subject research that is
subject to the HHS Human Subject Regulations or to the
FDA Human Subject Regulations may--
(i) use joint or shared review;
(ii) rely upon the review of--
(I) an independent institutional
review board; or
(II) an institutional review board of
an entity other than the sponsor of the
research; or
(iii) use similar arrangements to avoid
duplication of effort.
(2) Regulations and guidance.--Not later than 36 months after
the date of enactment of this Act, the Secretary, acting
through the relevant agencies and offices of the Department of
Health and Human Services, including the Office for Human
Research Protections and relevant agencies and offices of the
Food and Drug Administration, shall issue such regulations and
guidance and take such other actions as may be necessary to
implement this section and help to facilitate the broader use
of single, central, or lead institutional review boards. Such
regulations and guidance shall clarify the requirements and
policies relating to the following:
(A) Arrangements to avoid duplication described in
paragraph (1)(A)(i), including--
(i) delineating the roles of institutional
review boards in multisite or cooperative,
multisite studies where one or more local
institutional review boards are relied upon, or
similar arrangements are used;
(ii) the risks and benefits to human
subjects;
(iii) standardizing the informed consent and
other processes and legal documents; and
(iv) incorporating community values through
the use of local institutional review boards
while continuing to use central or lead
institutional review boards.
(B) Concerns about regulatory and legal liability
contributing to decisions by the sponsors of research
to rely on local institutional review boards for
multisite research.
(3) Consultation.--In issuing regulations or guidance under
paragraph (2), the Secretary shall consult with stakeholders
(including researchers, academic organizations, hospitals,
institutional research boards, pharmaceutical, biotechnology
and medical device developers, clinical research organizations,
patient groups, and others).
(c) Timing.--The Secretary shall complete the harmonization described
in subsection (a) not later than 36 months after the date of enactment
of this Act.
(d) Progress Report.--Not later than 24 months after the date of
enactment of this Act, the Secretary shall submit to Congress a report
on the progress made toward completing such harmonization.
(e) Draft NIH Policy.--Not later than 12 months after the date of
enactment of this Act, the Secretary, acting through the Director of
the National Institutes of Health, shall finalize the draft policy
entitled ``Draft NIH Policy on Use of a Single Institutional Review
Board for Multi-Site Research''.
(f) Definitions.--
(1) Human subject regulations.--In this section:
(A) FDA human subject regulations.--The term ``FDA
Human Subject Regulations'' means the provisions of
parts 50, 56, 312, and 812 of title 21, Code of Federal
Regulations (or any successor regulations).
(B) HHS human subject regulations.--The term ``HHS
Human Subject Regulations'' means the provisions of
subpart A of part 46 of title 45, Code of Federal
Regulations (or any successor regulations).
(C) Vulnerable-populations rules.--The term
``vulnerable-populations rules''--
(i) subject to clause (ii), means the
provisions of subparts B through D of such part
46 (or any successor regulations); or
(ii) as applicable to research that is
subject to the FDA Human Subject Regulations,
means the provisions applicable to vulnerable
populations under part 56 of such title 21 (or
any successor regulations) and subpart D of
part 50 of such title 21 (or any successor
regulations).
(2) Other definitions.--In this section:
(A) Institutional review board.--The term
``institutional review board'' has the meaning that
applies to the term ``institutional review board''
under the HHS Human Subject Regulations.
(B) Lead institutional review board.--The term ``lead
institutional review board'' means an institutional
review board that otherwise meets the requirements of
the HHS Human Subject Regulations and enters into a
written agreement with an institution, another
institutional review board, a sponsor, or a principal
investigator to approve and oversee human subject
research that is conducted at multiple locations.
References to an institutional review board include an
institutional review board that serves a single
institution as well as a lead institutional review
board.
SEC. 2262. USE OF NON-LOCAL INSTITUTIONAL REVIEW BOARDS FOR REVIEW OF
INVESTIGATIONAL DEVICE EXEMPTIONS AND HUMAN DEVICE
EXEMPTIONS.
(a) In General.--Section 520 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360(j)) is amended--
(1) in subsection (g)(3)--
(A) by striking ``local'' each place it appears; and
(B) in subparagraph (A)(i), by striking ``which has
been''; and
(2) in subsection (m)(4)--
(A) by striking ``local'' each place it appears; and
(B) by striking subparagraph (A) and inserting the
following new subparagraph:
``(A) in facilities in which clinical testing of devices is
supervised by an institutional review committee established in
accordance with the regulations of the Secretary, and''.
(b) Regulations.--Not later than 12 months after the date of the
enactment of this Act, the Secretary of Health and Human Services shall
revise or issue such regulations or guidance as may be necessary to
carry out the amendments made by subsection (a).
SEC. 2263. ALTERATION OR WAIVER OF INFORMED CONSENT FOR CLINICAL
INVESTIGATIONS.
(a) Devices.--Section 520(g)(3) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360j(g)(3)) is amended--
(1) in subparagraph (D), by striking ``except where subject
to such conditions as the Secretary may prescribe, the
investigator'' and inserting the following: ``except where,
subject to such conditions as the Secretary may prescribe--
``(i) the proposed clinical testing poses no more
than minimal risk to the human subject and includes
appropriate safeguards to protect the rights, safety,
and welfare of the human subject; or
``(ii) the investigator''; and
(2) in the matter following subparagraph (D), by striking
``subparagraph (D)'' and inserting ``subparagraph (D)(ii)''.
(b) Drugs.--Section 505(i)(4) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355(i)(4)) is amended by striking ``except where it is
not feasible or it is contrary to the best interests of such human
beings'' and inserting ``except where it is not feasible, it is
contrary to the best interests of such human beings, or the proposed
clinical testing poses no more than minimal risk to such human beings
and includes appropriate safeguards as prescribed to protect the
rights, safety, and welfare of such human beings''.
Subtitle P--Improving Scientific Expertise and Outreach at FDA
SEC. 2281. SILVIO O. CONTE SENIOR BIOMEDICAL RESEARCH SERVICE.
(a) Hiring and Retention Authority.--Section 228 of the Public Health
Service Act (42 U.S.C. 237) is amended--
(1) in the section heading, by inserting ``and biomedical
product assessment'' after ``research'';
(2) in subsection (a)(1), by striking ``Silvio O. Conte
Senior Biomedical Research Service, not to exceed 500 members''
and inserting ``Silvio O. Conte Senior Biomedical Research and
Biomedical Product Assessment Service (in this section referred
to as the `Service'), the purpose of which is to recruit and
retain competitive and qualified scientific and technical
experts outstanding in the field of biomedical research,
clinical research evaluation, and biomedical product
assessment'';
(3) by amending subsection (a)(2) to read as follows:
``(2) The authority established in paragraph (1) may not be construed
to require the Secretary to reduce the number of employees serving
under any other employment system in order to offset the number of
members serving in the Service.'';
(4) in subsection (b)--
(A) in the matter preceding paragraph (1), by
striking ``or clinical research evaluation'' and
inserting ``, clinical research evaluation or
biomedical product assessment''; and
(B) in paragraph (1), by inserting ``or a master's
level degree in engineering, bioinformatics, or a
related or emerging field,'' after the comma;
(5) in subsection (d)(2), by striking ``and shall not exceed
the rate payable for level I of the Executive Schedule unless
approved by the President under section 5377(d)(2) of title 5,
United States Code'' and inserting ``and shall not exceed the
rate payable for the President'';
(6) by striking subsection (e); and
(7) by redesignating subsections (f) and (g) as subsections
(e) and (f), respectively.
(b) Report.--Not later than 3 years after the date of the enactment
of this Act, the Secretary of Health and Human Services shall submit,
and publish on the website of the Department of Health and Human
Services a report on the implementation of the amendments made by
subsection (a), including whether the amendments have improved the
ability of the Food and Drug Administration to hire and retain
qualified experts to fulfill obligations specified under user fee
agreements.
SEC. 2282. ENABLING FDA SCIENTIFIC ENGAGEMENT.
It is the sense of Congress that the participation in, or sponsorship
of, scientific conferences and meetings is essential to the mission of
the Food and Drug Administration.
SEC. 2283. REAGAN-UDALL FOUNDATION FOR THE FOOD AND DRUG
ADMINISTRATION.
(a) Board of Directors.--
(1) Composition and size.--Section 770(d)(1)(C) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 379dd(d)(1)(C))
is amended--
(A) by redesignating clause (ii) as clause (iii);
(B) by inserting after clause (i) the following:
``(ii) Additional members.--The Board,
through amendments to the bylaws of the
Foundation, may provide that the number of
voting members of the Board shall be a number
(to be specified in such amendment) greater
than 14. Any Board positions that are
established by any such amendment shall be
appointed (by majority vote) by the individuals
who, as of the date of such amendment, are
voting members of the Board and persons so
appointed may represent any of the categories
specified in subclauses (I) through (V) of
clause (i), so long as no more than 30 percent
of the total voting members of the Board
(including members whose positions are
established by such amendment) are
representatives of the general pharmaceutical,
device, food, cosmetic, and biotechnology
industries.''; and
(C) in clause (iii)(I), as redesignated by
subparagraph (A), by striking ``The ex officio members
shall ensure'' and inserting ``The ex officio members,
acting pursuant to clause (i), and the Board, acting
pursuant to clause (ii), shall ensure''.
(2) Federal employees allowed to serve on board.--Clause
(iii)(II) of section 770(d)(1)(C) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 379dd(d)(1)(C)), as redesignated by
paragraph (1)(A), is amended by adding at the end the
following: ``For purposes of this section, the term `employee
of the Federal Government' does not include a `special
Government employee', as that term is defined in section 202(a)
of title 18, United States Code.''.
(3) Staggered terms.--Subparagraph (A) of section 770(d)(3)
of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
379dd(d)(3)) is amended to read as follows:
``(A) Term.--The term of office of each member of the
Board appointed under paragraph (1)(C)(i), and the term
of office of any member of the Board whose position is
established pursuant to paragraph (1)(C)(ii), shall be
4 years, except that--
``(i) the terms of offices for the members of
the Board initially appointed under paragraph
(1)(C)(i) shall expire on a staggered basis as
determined by the ex officio members; and
``(ii) the terms of office for the persons
initially appointed to positions established
pursuant to paragraph (1)(C)(ii) may be made to
expire on a staggered basis, as determined by
the individuals who, as of the date of the
amendment establishing such positions, are
members of the Board.''.
(b) Executive Director Compensation.--Section 770(g)(2) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 379dd(g)(2)) is amended
by striking ``but shall not be greater than the compensation of the
Commissioner''.
(c) Separation of Funds.--Section 770(m) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 379dd(m)) is amended by striking ``are held
in separate accounts from funds received from entities under subsection
(i)'' and inserting ``are managed as individual programmatic funds
under subsection (i), according to best accounting practices''.
SEC. 2284. COLLECTION OF CERTAIN VOLUNTARY INFORMATION EXEMPTED FROM
PAPERWORK REDUCTION ACT.
Chapter VII of the Federal Food, Drug, and Cosmetic Act is amended
by inserting after section 708 of such Act (21 U.S.C. 379) the
following:
``SEC. 708A. COLLECTION OF CERTAIN VOLUNTARY INFORMATION EXEMPTED FROM
PAPERWORK REDUCTION ACT.
``Chapter 35 of title 44, United States Code, shall not apply to the
collection from patients, industry, academia, and other stakeholders,
of voluntary information such as through voluntary surveys or
questionnaires, initiated by the Secretary.''.
SEC. 2285. HIRING AUTHORITY FOR SCIENTIFIC, TECHNICAL, AND PROFESSIONAL
PERSONNEL.
(a) In General.--The Federal Food, Drug, and Cosmetic Act is amended
by inserting after section 714 (21 U.S.C. 379d-3) the following:
``SEC. 714A. ADDITIONAL HIRING AUTHORITY.
``(a) In General.--The Secretary may, without regard to the
provisions of title 5, United States Code, governing appointments in
the competitive service, appoint qualified candidates to scientific,
technical, or professional positions within the following centers of
the Food and Drug Administration:
``(1) The Center for Drug Evaluation and Research.
``(2) The Center for Biologics Evaluation and Research.
``(3) The Center for Devices and Radiological Health.
Such positions shall be within the competitive service.
``(b) Compensation.--
``(1) In general.--Notwithstanding any other provision of
law, including any requirement with respect to General Schedule
pay rates under subchapter III of chapter 53 of title 5, United
States Code, and consistent with the requirements of paragraph
(2), the Secretary may determine and fix--
``(A) the annual rate of pay of any individual
appointed under subsection (a); and
``(B) for purposes of retaining qualified employees,
the annual rate of pay for any highly qualified
scientific, technical, or professional personnel
appointed to a position at any of the centers listed
under subsection (a) before the date of enactment of
this section.
``(2) Limitation.--The annual rate of pay established
pursuant to paragraph (1) may not exceed the annual rate of pay
of the President.
``(c) Sunset.--The authority to appoint employees under this section
shall terminate on September 30, 2022.
``(d) Report.--
``(1) In general.--Not later than September 30, 2021, the
Secretary shall submit a report to Congress that examines the
extent to which the authority to appoint and retain personnel
under this section enhanced the Food and Drug Administration's
ability to meet the agency's critical need for highly qualified
individuals for scientific, technical, or professional
positions.
``(2) Recommendations.--The report under paragraph (1) shall
include the recommendations of the Secretary on--
``(A) whether the authority to appoint personnel
under this section should be reauthorized; and
``(B) other personnel authorities that would help the
Food and Drug Administration to better recruit and
retain highly qualified individuals for scientific,
technical, or professional positions in the agency's
medical product centers.''.
(b) Rule of Construction.--The authority provided by section 714A of
the Federal Food, Drug, and Cosmetic Act (as added by subsection (a))
shall not be construed to affect the authority provided under section
714 of such Act.
Subtitle Q--Exempting From Sequestration Certain User Fees
SEC. 2301. EXEMPTING FROM SEQUESTRATION CERTAIN USER FEES OF FOOD AND
DRUG ADMINISTRATION.
The Balanced Budget and Emergency Deficit Control Act of 1985 is
amended--
(1) in section 255(g)(1)(A) (2 U.S.C. 905(g)(1)(A)), by
inserting after the item relating to ``Financial Agent
Services'' the following new item:
``Food and Drug Administration, Salaries and
Expenses, but only the portion of appropriations under
such account corresponding to fees collected under
sections 736, 738, 740, 741, 744B, and 744H of the
Federal Food, Drug, and Cosmetic Act (75-9911-0-1-
554).''; and
(2) in section 256(h) (2 U.S.C. 906(h)), by adding at the end
the following new paragraph:
``(5) Notwithstanding any other provision of law, this
subsection shall not apply with respect to the portion of
administrative expenses incurred by the Food and Drug
Administration that are funded through fees collected under
sections 736, 738, 740, 741, 744B, and 744H of the Federal
Food, Drug, and Cosmetic Act.''.
TITLE III--DELIVERY
Subtitle A--Interoperability
SEC. 3001. ENSURING INTEROPERABILITY OF HEALTH INFORMATION TECHNOLOGY.
(a) Interoperability Standards.--
(1) In general.--Subtitle A of title XXX of the Public Health
Service Act (42 U.S.C. 300jj-11 et seq.) is amended by adding
at the end the following new section:
``SEC. 3010. ENSURING INTEROPERABILITY OF HEALTH INFORMATION
TECHNOLOGY.
``(a) Interoperability.--In order for health information technology
to be considered interoperable, such technology must satisfy the
following criteria:
``(1) Secure transfer.--The technology allows the secure
transfer of the entirety of a patient's data from any and all
health information technology for authorized use under
applicable law.
``(2) Complete access to health data.--The technology allows
access to the entirety of a patient's available data for
authorized use under applicable law without special effort, as
defined by recommendations for interoperability standards
adopted under section 3004, by the requestor of such data
unless such data is not disclosable under applicable law.
``(3) No information blocking.--The technology is not
configured, set up, or implemented to engage in information
blocking, as defined in section 3010A(f).
``(b) Categories for Interoperability Standards.--The categories
described in this subsection, with respect to standards for determining
if health information technology is interoperable, consistent with the
criteria described in subsection (a), include the following categories
of standards:
``(1) Standards with respect to vocabulary and terminology.
``(2) Standards with respect to content and structure.
``(3) Standards with respect to transport of information.
``(4) Security standards.
``(5) Service standards.''.
(2) Guidance.--Not later than January 1, 2017, the Secretary
of Health and Human Services, through the National Coordinator
of the Office of the National Coordinator for Health
Information Technology, shall issue guidance with respect to
the implementation of section 3010 of the Public Health Service
Act, as added by paragraph (1), including with respect to
defining and providing examples of authorized use of health
information technology, as described in such section.
(b) Improvements to Recommendation Process.--
(1) HIT policy committee to incorporate policies for updates
to interoperability standards.--Section 3002 of the Public
Health Service Act (42 U.S.C. 300jj-12) is amended--
(A) in subsection (a)--
(i) by striking ``National Coordinator'' and
inserting ``Secretary, in consultation with the
National Coordinator,''; and
(ii) by adding at the end the following new
sentence: ``The HIT Policy Committee is
authorized only to provide policy and priority
recommendations to the Secretary and not
authorized to otherwise affect the development
or modification of any standard, implementation
specification, or certification criterion under
this title.''; and
(B) in subsection (b)(2)--
(i) in subparagraph (A), in the first
sentence--
(I) by striking ``The HIT Policy
Committee'' and inserting ``Subject to
subparagraph (D), the HIT Policy
Committee''; and
(II) by inserting ``(including the
areas in which modifications and
additions to interoperability standards
under section 3010 are needed for the
electronic exchange and use of health
information for purposes of adoption of
such modifications and additions under
section 3004)'' after ``section 3004''.
(ii) by adding at the end the following new
subparagraph:
``(D) Special rule related to interoperability.--Any
recommendation made by the HIT Policy Committee on or
after the date of the enactment of this subparagraph
with respect to interoperability of health information
technology shall be consistent with the criteria
described in subsection (a) of section 3010.''.
(2) Sunset of hit standards committee.--Section 3003 of the
Public Health Service Act (42 U.S.C. 300jj-13) is amended by
adding at the end the following new subsection:
``(f) Termination.--The HIT Standards Committee shall terminate on
the date that is 90 days after the date of the enactment of this
subsection.''.
(3) Standards development organizations.--Title XXX of the
Public Health Service Act is amended by inserting after section
3003 the following new section:
``SEC. 3003A. RECOMMENDATIONS FOR STANDARDS THROUGH CONTRACTS WITH
STANDARDS DEVELOPMENT ORGANIZATIONS.
``(a) Contracts.--
``(1) In general.--For purposes of activities conducted under
this title, the Secretary shall enter into contracts with
health care standards development organizations accredited by
the American National Standards Institute to carry out the
duties described in subsection (b), as applicable.
``(2) Timing for first contract.--As soon as practicable
after the date of the enactment of this section, the Secretary
shall enter into the first contract under paragraph (1).
``(3) Period of contract.--Each contract under paragraph (1)
shall be for a period determined necessary by the Secretary, in
consultation with the National Coordinator, to carry out the
applicable duties described in subsection (b).
``(4) Appropriate organizations.--The Secretary shall ensure
the most appropriate organizations described in paragraph (1)
are selected for each contract under such paragraph.
``(5) Allowance for variations.--Standards developed pursuant
to a contract under this subsection, and the methods to test
such standards, shall allow for variations on such standards as
long as such variations are consistent with the standards so
developed under this section.
``(b) Duties.--
``(1) Initial contract.--Under the initial contract under
subsection (a)(1), the standards development organizations--
``(A) shall provide to the Secretary, in consultation
with the National Coordinator, for adoption under
section 3004, recommendations, in accordance with
section 3010, for interoperability standards, and
methods to test such standards, consistent with the
criteria described in subsection (a) of such section
and with respect to the categories described in
subsection (b)(1) of such section; and
``(B) may provide to the Secretary recommendations
described in paragraph (2).
``(2) Subsequent contracts.--Under each subsequent contract,
the organizations shall provide to the Secretary, in
consultation with the National Coordinator, for adoption under
section 3004 recommendations for any standards (including
interoperability standards and methods to test such standards),
implementation specifications, and certification criteria (and
modifications, including additions, to such standards,
specifications, and criteria), which are in accordance with the
policies and priorities developed by the Secretary, in
consultation with the National Coordinator.
``(3) Multiple methods to test interoperability standards.--
For the purposes of developing methods to test interoperability
standards for adoption under section 3004, the Secretary shall
ensure that contracts under this section allow for multiple
methods to test such standards to account for variations in the
adoption of such standards that do not conflict with section
3010(a).
``(c) Modifications and Subsequent Contracts.--
``(1) In general.--The Secretary, in consultation with the
National Coordinator, shall periodically conduct hearings to
evaluate and review the standards, implementation
specifications, and certification criteria adopted under
section 3004 for purposes of determining if modifications,
including any additions, are needed with respect to such
standards, specifications, and criteria.
``(2) Contract trigger.--Based on the needs for standards,
implementation specifications, and certification criteria (and
modifications, including additions, to such standards,
specifications, and criteria) under this title, as determined
by the Secretary, in consultation with the National
Coordinator, the Secretary shall, as needed, enter into
contracts under subsection (a) in addition to the initial
contract.
``(d) Authorization of Appropriations.--There is authorized to be
appropriated $10,000,000 for contracts under subsection (a), to remain
available until expended.''.
(4) Modifications to role of onchit.--Section 3001(c)(1)(A)
of the Public Health Service Act (42 U.S.C. 300jj-11(c)(1)(A))
is amended by inserting ``for recommendations made before the
date of the enactment of the 21st Century Cures Act,'' before
``review and determine''.
(c) Adoption.--Section 3004 of the Public Health Service Act (42
U.S.C. 300jj-14) is amended--
(1) in subsection (a)--
(A) in paragraph (1), by inserting after ``section
3001(c)'' the following: ``(or, subject to subsection
(c), in the case of a standard, specification, or
criterion recommended on or after the date of the
enactment of the 21st Century Cures Act, after the date
of submission of the recommendation to the Secretary
under section 3003A)''; and
(B) in paragraph (2)(B), by striking ``and the HIT
Standards Committee'';
(2) in subsection (b), by adding at the end the following new
paragraph:
``(4) Limitation.--The Secretary may not adopt any standards,
implementation specifications, or certification criteria under
this subsection or subsection (a) that are inconsistent with or
duplicative of an interoperability standard adopted under this
section, in accordance with subsections (c) and (d). In the
case of a standard, specification, or criterion that has been
adopted under this section and is inconsistent or duplicative
of such an interoperability standard that is subsequently
adopted under this section, such interoperability standard
shall supercede such other standard, specification, or
criterion and such other standard, specification, or criterion
shall no longer be considered adopted under this section
beginning on the date that such interoperability standard
becomes effective.''; and
(3) by adding at the end the following new subsections:
``(c) Adoption of Initial Interoperability Standards.--
Notwithstanding the previous subsections of this section, the following
shall apply in the case of the initial set of interoperability
standards recommended under section 3003A:
``(1) Review of standards.--Not later than 90 days after the
date of receipt of recommendations for such interoperability
standards, the Secretary, in consultation with the National
Coordinator and representatives of other relevant Federal
agencies, shall jointly review such standards and shall
determine whether or not to propose adoption of such standards.
``(2) Determination to adopt.--If the Secretary determines--
``(A) to propose adoption of such standards, the
Secretary shall, by regulation under section 553 of
title 5, United States Code, determine whether or not
to adopt such standards; or
``(B) not to propose adoption of such standards, the
Secretary shall notify the applicable standards
development organizations with a contract under section
3003A in writing of such determination and the reasons
for not proposing the adoption of the recommendation
for such standards.
``(3) Publication.--The Secretary shall provide for
publication in the Federal Register of all determinations made
by the Secretary under paragraph (1).
``(4) Application.--Any standard adopted under this
subsection shall be effective 12 months after the date of
publication of the determination to adopt such standard.
``(d) Rules for Adoption.--In the case of a standard (including
interoperability standard), implementation specification, or
certification criteria adopted under this section on or after the date
of the enactment of the 21st Century Cures Act, the following shall
apply:
``(1) In general.--Except as provided in paragraph (2), any
such standard (including interoperability standard),
implementation specification, or certification criterion shall
be a standard, specification, or criterion that has been
recommended by the standards development organizations with
which the Secretary has entered into a contract under section
3003A.
``(2) Special rule if no standard, specification, or
criterion recommended.--If no standard is recommended under
paragraph (1)--
``(A) in the case of interoperability standards,
relating to a category described in section 3010(b)--
``(i) paragraph (1) shall not apply; and
``(ii) paragraph (4) shall apply; or
``(B) in the case of any other standard,
implementation specification, or certification
criteria, relating to a policy or priority to carry out
this title, as determined by the Secretary, in
consultation with the National Coordinator--
``(i) paragraph (1) shall not apply; and
``(ii) paragraph (4) shall apply.
``(3) Effective date.--Any standard, implementation
specification, or certification criterion adopted under this
section shall be effective 12 months after the date of
publication of the final rule to adopt such standard,
implementation specification, or certification criterion.
``(4) Assistance to the secretary.--In complying with the
requirements of this subsection, the Secretary shall rely on
the recommendations of the National Committee on Vital and
Health Statistics established under section 306(k), and shall
consult with appropriate Federal and State agencies and private
organizations. The Secretary shall publish in the Federal
Register any recommendation of the National Committee on Vital
and Health Statistics regarding the adoption of a standard,
implementation specification, or certification criterion under
this section. Any standard, implementation specification, or
certification criterion adopted pursuant to this paragraph
shall be promulgated in accordance with the rulemaking
procedures of subchapter III of chapter 5 of title 5, United
States Code.''.
(d) Reports and Notifications.--Section 3010 of the Public Health
Service Act, as added by subsection (a), is amended by adding at the
end the following new subsection:
``(c) Dissemination of Information.--
``(1) Initial summary report.--Not later than July 1, 2017,
the Secretary, after consultation with relevant stakeholders,
shall submit to Congress and provide for publication in the
Federal Register and the posting on the Internet website of the
Office of the National Coordinator for Health Information
Technology of a report on the following:
``(A) The initial set of interoperability standards
adopted under section 3004(c).
``(B) The strategies for achieving widespread
interoperability.
``(C) An overview of the extent to which electronic
health records and health information technology
offered as of such date satisfy such initial set.
``(D) Any barriers that are preventing widespread
interoperability.
``(E) The plan and milestones, including specific
steps, to achieve widespread interoperability.
``(2) Followup determination and report on widespread
interoperability.--Not later than December 31, 2019, the
Secretary shall provide for publication in the Federal Register
and the posting on the Internet website of the Office of the
National Coordinator for Health Information Technology of the
following:
``(A) A determination by the Secretary whether the
goal of widespread interoperability has been achieved.
``(B) A list identifying the vendors of, or other
entities offering, qualified electronic health records,
which categorizes such entities, with respect to such
records, as in compliance or not in compliance with the
certification criteria described in section
3001(c)(5)(B)(ii) and with the requirements under
clause (i) of section 3001(c)(5)(C) (including with the
terms of the attestation and other requirements under
such clause).
``(C) Actions that may be taken by entities
identified under subparagraph (B) as not being in
compliance with such criteria and requirements in order
for such entities to become in compliance with such
criteria and requirements.
``(D) Penalties described in section 3010A(d) to
which entities, with respect to such qualified
electronic health records, beginning January 1, 2019,
are subject if such technology and entities are not in
compliance with the certification criteria described in
section 3001(c)(5)(B)(ii) and with the requirements
under clause (i) of section 3001(c)(5)(C),
respectively.
``(3) Ongoing publication of recommendations.--The Secretary
shall provide for publication in the Federal Register and the
posting on the Internet website of the Office of the National
Coordinator for Health Information Technology of all
recommendations made under this section.''.
(e) Certification and Other Enforcement Provisions.--
(1) Certification of qualified electronic health records.--
(A) In general.--Section 3007(b) of the Public Health
Service Act (42 U.S.C. 300jj-17(b)) is amended by
striking ``under section 3001(c)(3) to be in compliance
with'' and all that follows through the period at the
end and inserting ``under section 3001(c)(3)--
``(1) for certifications made before January 1, 2018, to be
in compliance with applicable standards adopted under
subsections (a) and (b) of section 3004; and
``(2) for certifications made on or after January 1, 2018, to
be in compliance with applicable standards adopted under
subsections (a) and (b) of section 3004 and to be interoperable
in accordance with section 3010, including by being in
compliance with interoperability standards adopted under
section 3004.''.
(B) Requirements of secretary.--Section 3001(c)(5) of
the Public Health Service Act (42 U.S.C. 300jj-
11(c)(5)) is amended--
(i) by amending subparagraph (B) of such
section to read as follows:
``(B) Certification criteria described.--In this
title, the term `certification criteria' means, with
respect to qualified electronic health records--
``(i) for certifications made before January
1, 2018, criteria to establish that the records
meet standards and implementation
specifications adopted under subsections (a)
and (b) of section 3004 for qualified
electronic health records; and
``(ii) for certifications made on or after
January 1, 2018, criteria described in clause
(i) and criteria to establish that the records
are interoperable, in accordance with section
3010, including by being in compliance with
interoperability standards adopted under
section 3004.''; and
(ii) by adding at the end the following new
subparagraph:
``(C) Enforcement; decertifications.--
``(i) Requirements.--Under any program kept
or recognized under subparagraph (A), the
Secretary shall ensure that any vendor of or
other entity offering qualified electronic
health records seeking a certification of such
records under such program on or after January
1, 2018, shall, as a condition of certification
(and maintenance of certification) of such a
record under such program--
``(I) provide to the Secretary an
attestation--
``(aa) that the entity,
unless for a legitimate purpose
specified by the Secretary, has
not taken any action, including
through any financial,
administrative, or
technological barrier, which
the entity knows or should know
(as defined in section
1128A(i)(7) of the Social
Security Act), is to limit or
restrict the exchange of
information or to prevent or
disincentivize widespread
interoperability between any
providers using such records or
other health information
technology in connection with
such record;
``(bb) on the pricing
information described in clause
(v) for purposes of the portal
created under paragraph (9);
that such information will be
available on a public Web site
of such entity and in marketing
materials, communications
statements, and other
assertions of such entity
related to such record; and
that the entity will
voluntarily provide such
information to customers prior
to providing any qualified
electronic health records or
related product or service
(including subsequent updates,
add-ons, or additional products
or services to be provided
during the course of an on-
going contract), prospective
customers (such as persons who
request or receive a quotation,
estimate, or other similar
marketing or promotional
material), and other persons
who request such information;
``(cc) that the software with
respect to such records have
published application
programming interfaces for
medical records data, search
and indexing, semantic
harmonization and vocabulary
translation, and user interface
applications;
``(dd) that the entity has
successfully tested the use of
the record in the type of
setting in which it would be
marketed;
``(ee) the entity has in
place implementation guidelines
for such record that support
interoperability, consistent
with section 3010; and
``(ff) that the entity has in
place data sharing programs or
capabilities based on common
data elements through
application programming
interfaces without the
requirement for vendor-specific
interfaces;
``(II) publish application
programming interfaces and associated
documentation, with respect to such
records, for medical records data,
search and indexing, semantic
harmonization and vocabulary
translation, and user interface
applications; and
``(III) demonstrate to the
satisfaction of the Secretary that data
from such records are able to be
exchanged through the use of
application programming interfaces and
used in a manner that allows for
exchange and everyday use, as
authorized under applicable law, of
such records.
``(ii) Decertification.--Under any program
kept or recognized under subparagraph (A), the
Secretary shall ensure that beginning January
1, 2019, any qualified electronic health
records that do not satisfy the certification
criteria described in section 3001(c)(5)(B)(ii)
or with respect to which the vendor or other
entity described in clause (i) does not satisfy
the requirements under such clause (or is
determined to be in violation of the terms of
the attestation or other requirements under
such clause) shall no longer be considered as
certified under such program.
``(iii) Annual publication.--For 2019 and
each subsequent year, the Secretary shall post
on the public Internet website of the
Department of Health and Human Services a list
of any vendors of or other entities offering
qualified electronic health records with
respect to which certification has been
withdrawn under clause (ii) during such year.
``(iv) Periodic review.--The Secretary shall
periodically review and confirm that vendors of
and other entities offering qualified
electronic health records have publicly
published application programming interfaces
and associated documentation as required by
clause (i)(II) for purposes of certification
and maintaining certification under any program
kept or recognized under subparagraph (A).
``(v) Pricing information.--For purposes of
clause (i)(I)(bb), the pricing information
described in this clause, with respect to a
vendor of or other entity offering a qualified
electronic health record, is the following:
``(I) Additional types of costs or
fees (whether fixed, recurring,
transaction based, or otherwise)
imposed by the entity (or any third-
party from whom the entity purchases,
licenses, or obtains any technology,
products, or services in connection
with the qualified electronic health
record) to purchase, license,
implement, maintain, upgrade, use, or
otherwise enable and support the use of
capabilities to which such record is to
be certified under this section; or in
connection with any data generated in
the course of using any capability to
which the record is to be so certified.
``(II) Limitations, whether by
contract or otherwise, on the use of
any capability to which the record is
to be certified under this section for
any purpose within the scope of the
record's certification; or in
connection with any data generated in
the course of using any capability to
which the record is to be certified
under this section.
``(III) Limitations, including
technical or practical limitations of
technology or its capabilities, that
could prevent or impair the successful
implementation, configuration,
customization, maintenance, support, or
use of any capabilities to which the
record is to be certified under this
section; or that could prevent or limit
the use, exchange, or portability of
any data generated in the course of
using any capability to which the
record is to be so certified.''.
(2) Additional enforcement provisions under the public health
service act.--Subtitle A of title XXX of the Public Health
Service Act (42 U.S.C. 300jj-11 et seq.), as amended by
subsections (a)(1) and (d), is further amended by adding at the
end the following new section:
``SEC. 3010A. ENFORCEMENT MECHANISMS.
``(a) Inspector General Authority.--The Inspector General of the
Department of Health and Human Services shall have the authority to
investigate claims of--
``(1) vendors of, or other entities offering, qualified
electronic health records--
``(A) being in violation of an attestation made under
section 3001(c)(5)(C)(i)(I), with respect to the use of
such records by a health care provider under a
specified meaningful use incentive program; and
``(B) having engaged in information blocking (as
defined in subsection (f)), unless for a legitimate
purpose specified by the Secretary, with respect to the
use of such records by a health care provider under
such a program;
``(2) health care providers, with respect to the use of such
records under a specified meaningful use incentive program,
having, unless for a legitimate purpose specified by the
Secretary, engaged in information blocking (as so defined);
``(3) health information system providers described in
subsection (b) having engaged in information blocking (as so
defined), unless for a legitimate purpose specified by the
Secretary, with respect to the use of such records under a
specified meaningful use incentive program; and
``(4) vendors of, or other entities offering, health
information technology (other than technology described in
paragraph (1)), health care providers, with respect to the use
of such technology, and health information system providers,
with respect to such technology, unless for a legitimate
purpose specified by the Secretary, having engaged in
information blocking (as so defined).
``(b) Health Information System Providers.--The Inspector General of
the Department of Health and Human Services shall, in coordination with
the Federal Trade Commission, ensure that health information system
providers (such as operators of health information exchanges and other
systems that facilitate the exchange of information) investigate claims
of information blocking, with respect to the use of such records under
a specified meaningful use incentive program.
``(c) Information Sharing Provisions.--
``(1) In general.--The National Coordinator may serve as a
technical consultant to the Inspector General of the Department
of Health and Human Services and the Federal Trade Commission
for purposes of carrying out this section. As such technical
consultant, the National Coordinator may, notwithstanding any
other provision of law, share information related to claims or
investigations under subsection (a) or (b) with the Federal
Trade Commission for purposes of such investigations.
``(2) Protection from disclosure of information.--Any
information shared by the National Coordinator under paragraph
(1) shall not be subject to the provisions of section 552 of
title 5, United States Code (commonly referred to as the
Freedom of Information Act). Any information acquired pursuant
to paragraph (1) shall be held in confidence and shall not be
disclosed to any person except as may be necessary to carry out
the purposes of subsection (a).
``(3) Non-application of paperwork reduction act.--Chapter 35
of title 44, United States Code (commonly referred to as the
Paperwork Reduction Act of 1995) shall not apply to the
National Coordinator or to the Office of the National
Coordinator for Health Information Technology with respect to
the collection of complaints relating to claims described in
subsection (a).
``(d) Penalty.--Any person or entity determined to have committed an
act described in paragraph (1), (2), or (3) of subsection (a), in
connection with a specified meaningful use incentive program, shall be
subject to a civil monetary penalty of not more than $10,000 for each
such act. The provisions of section 1128A (other than subsections (a)
and (b)) shall apply to a civil money penalty applied under this
subsection in the same manner as they apply to a civil money penalty or
proceeding under section 1128A(a).
``(e) Specified Meaningful Use Incentive Program.--For purposes of
this section, the term `specified meaningful use incentive program'
includes the following:
``(1) The incentive payments under subsection (o) of section
1848 of the Social Security Act (42 U.S.C. 1395w-4) and
adjustments under subsection (a)(7) of such section.
``(2) The incentive payments under subsection (n) of section
1848 of such Act (42 U.S.C. 1395ww) and adjustments under
subsection (b)(3)(B) of such section.
``(3) The incentive payments and adjustments made under
subsections (l) and (m) of section 1853 of such Act (42 U.S.C.
1395w-23).
``(4) The incentive payment under paragraph (3) of section
1814(l) of such Act (42 U.S.C. 1395f(l)) and adjustment under
paragraph (4) of such section.
``(5) The shared savings program under section 1899 of such
Act (42 U.S.C. 1395jjj).
``(6) The payments to Medicaid providers described in section
1903(t) of such Act (42 U.S.C. 1396b(t)).
``(f) Information Blocking.--
``(1) In general.--For purposes of this section and section
3010, the term `information blocking' means, with respect to
the use of qualified electronic health records or other health
information technology under a specified meaningful use
incentive program, business, technical, and organizational
practices, including practices described in paragraph (2),
that--
``(A) prevent or materially discourage the exchange
of electronic health information;
``(B) the actor knows or should know (as defined in
section 1128A(i)(7) of the Social Security Act) are
likely to interfere with the exchange or use of
electronic health information; and
``(C) do not serve to protect patient safety,
maintain the privacy and security of individuals'
health information or promote competition and consumer
welfare.
``(2) Practices described.--For purposes of paragraph (1),
the practices described in this paragraph are the following:
``(A) Contract terms, policies, or other business or
organizational practices that restrict individuals'
access to their electronic health information or
restrict the exchange or use of that information for
treatment and other permitted purposes.
``(B) Charging prices or fees (such as for data
exchange, portability, and interfaces) that make
exchanging and using electronic health information cost
prohibitive.
``(C) Developing or implementing health information
technology in nonstandard ways that are likely to
substantially increase the costs, complexity, or burden
of sharing electronic health information, especially in
cases in which relevant interoperability standards or
methods to measure interoperability have been adopted
by the Secretary.
``(D) Developing or implementing health information
technology in ways that are likely to lock in users or
electronic health information, such as not allowing for
the full export of data; lead to fraud, waste, or
abuse; or impede innovations and advancements in health
information exchange and health information technology-
enabled care delivery.
``(g) Treatment of Vendors With Respect to Patient Safety
Organizations.--In applying part C of title IX--
``(1) vendors shall be treated as a provider (as defined in
section 921) for purposes of reporting requirements under such
part, to the extent that such reports are related to
attestation requirements under section 3001(c)(5)(C)(i)(I);
``(2) claims of information blocking described in subsection
(a) shall be treated as a patient safety activity under such
part for purposes of reporting requirements under such part;
and
``(3) health care providers that are not members of patient
safety organizations shall be treated in the same manner as
health care providers that are such members for purposes of
such reporting requirements with respect to claims of
information blocking described in subsection (a).''.
(3) ONCHIT.--
(A) Portal.--Section 3001(c) of the Public Health
Service Act (42 U.S.C. 300jj-11(c)) is amended by
adding at the end the following new paragraph:
``(9) Portal.--Not later than January 1, 2019, the National
Coordinator shall create a portal to make the information
described in paragraph (5)(C)(I)(i)(bb) available to the public
in a manner that allows for comparison of price information
among health information technology products and that aids in
making informed decisions for purchasing such a product.''.
(B) Information blocking.--Not later than 12 months
after the date of the enactment of this Act, the
National Coordinator of the Office of the National
Coordinator of Health Information Technology shall,
through rulemaking, implement the provisions of this
section, and amendments made by this section, relating
to information blocking.
(C) HIPAA.--Not later than January 1, 2017, the
National Coordinator shall publish guidance to clarify
the relationship of the HIPAA privacy and security law,
as defined in section 3009(a)(2) of the Public Health
Service Act (42 U.S.C. 300jj-19(a)(2)) as such
provisions relate to information blocking (as defined
in section 3010A(f) of such Act, as added by paragraph
(2)), including examples of how such provisions may
result in information blocking.
(4) Demonstration required for meaningful ehr use incentives
under medicare.--
(A) Incentives for professionals.--
(i) In general.--Section 1848(o)(2)(C) of the
Social Security Act (42 U.S.C. 1395w-
4(o)(2)(C)) is amended by adding at the end the
following new clause:
``(iii) Interoperability.--With respect to
EHR reporting periods for payment years
beginning with 2018, the means described in
clause (i) specified by the Secretary shall
include a demonstration, through means such as
an attestation, that the professional has not
taken any action described in subsection (a)(2)
of section 3010A of the Public Health Service
Act, with respect to the use of any certified
EHR technology.''.
(ii) Hardship exemption in case of
decertified ehr.--Subparagraph (B) of section
1848(a)(7) of the Social Security Act (42
U.S.C. 1395w-4(a)(7)) is amended to read as
follows:
``(B) Significant hardship exception.--
``(i) In general.--The Secretary may, on a
case-by-case basis, exempt an eligible
professional from the application of the
payment adjustment under subparagraph (A) if
the Secretary determines, subject to annual
renewal, that compliance with the requirement
for being a meaningful EHR user would result in
a significant hardship, such as in the case of
an eligible professional who practices in a
rural area without sufficient Internet access.
``(ii) Decertification.--
``(I) In general.--The Secretary may,
on a case-by-case basis, exempt an
eligible professional from the
application of the payment adjustment
under subparagraph (A) if the Secretary
determines that such professional was
determined to not be a meaningful EHR
user because the qualified electronic
health record used by such professional
was decertified under section
3001(c)(5)(C) of the Public Health
Service Act. An exemption under the
previous sentence may be applied to an
eligible professional only, subject to
subclause (II), during the first
payment year with respect to the first
EHR reporting period to which such
decertification applies.
``(II) Duration.--
``(aa) In general.--In no
case shall an exemption by
reason of this clause be for a
period of less than 12 months.
``(bb) Extension.--An
exemption under this clause may
be extended for a period of an
additional 12 months subject to
the limitation described in
clause (ii).
``(iii) Limitation.--Subject to clause
(ii)(II)(aa), in no case may an eligible
professional be granted an exemption under this
subparagraph for more than 5 years.''.
(B) Incentives for hospitals.--
(i) In general.--Section 1886(o)(1) of the
Social Security Act (42 U.S.C. 1395ww(o)(1)) is
amended--
(I) in subparagraph (A), by inserting
before the period at the end the
following: ``and, for performance
periods for fiscal year 2018 or a
subsequent fiscal year, that provide a
demonstration described in subparagraph
(D) to the Secretary''; and
(II) by adding at the end the
following new subparagraph:
``(D) Demonstration described.--The demonstration
described in this subparagraph is a demonstration,
through means such as an attestation, that the hospital
has not taken any action described in subsection (a)(2)
of section 3010A of the Public Health Service Act, with
respect to the use of any certified EHR technology.''.
(ii) Hardship exemption in case of
decertified ehr.--Subclause (II) of section
1886(b)(3)(B)(ix) of the Social Security Act
(42 U.S.C. 1395ww(b)(3)(B)(ix)) is amended to
read as follows:
``(II)(aa) The Secretary may, on a case-by-case basis, exempt a
subsection (d) hospital from the application of subclause (I) with
respect to a fiscal year if the Secretary determines, subject to annual
renewal, that requiring such hospital to be a meaningful EHR user
during such fiscal year would result in a significant hardship, such as
in the case of a hospital in a rural area without sufficient Internet
access.
``(bb) The Secretary may, on a case-by-case basis, exempt a
subsection (d) hospital from the application of subclause (I) with
respect to a fiscal year if the Secretary determines, subject to annual
renewal, that such hospital was determined to not be a meaningful EHR
user because the qualified electronic health record used by such
hospital was decertified under section 3001(c)(5)(C) of the Public
Health Service Act. An exemption under the previous sentence may be
applied to a subsection (d) hospital only, subject to items (cc) and
(dd), during the first payment year with respect to the first EHR
reporting period to which such decertification applies.
``(cc) In no case shall an exemption by reason of item (bb) be for a
period of less than 12 months.
``(dd) An exemption under item (bb) may be extended for a period of
an additional 12 months subject to the limitation described in item
(ee).
``(ee) Subject to item (cc), in no case may a hospital be granted an
exemption under this subclause for more than 5 years.''.
(C) Demonstration required for meaningful ehr use
incentives under medicaid.--Section 1903(t)(2) of the
Social Security Act (42 U.S.C. 1396b(t)(2)) is amended
by adding at the end the following: ``An eligible
professional shall not qualify as a Medicaid provider
under this subsection, with respect to a year beginning
with 2018, unless such provider demonstrates to the
Secretary, through means such as an attestation, that
the provider has not taken any action described in
subsection (a)(2) of section 3010A of the Public Health
Service Act with respect to which the provider knows or
should know (as defined in section 1128A(i)(7) of the
Social Security Act) about, with respect to the use of
any certified EHR technology.''.
(f) Definitions.--
(1) Certified ehr technology.--Paragraph (1) of section 3000
of the Public Health Service Act (42 U.S.C. 300jj) is amended
to read as follows:
``(1) Certified ehr technology.--The term `certified EHR
technology' means a qualified electronic health record that is
certified pursuant to section 3001(c)(5) as meeting the
certification criteria defined in subparagraph (B) of such
section that are applicable to the type of record involved (as
determined by the Secretary, such as an ambulatory electronic
health record for office-based physicians or an inpatient
hospital electronic health record for hospitals) including,
beginning January 1, 2018, with respect to which the vendor or
other entity offering such technology is in compliance with the
requirements under section 3001(c)(5)(C)(i).''.
(2) Widespread interoperability.--Section 3000 of the Public
Health Service Act (42 U.S.C. 300jj) is amended by adding at
the end the following new paragraph:
``(15) Widespread interoperability.--The term `widespread
interoperability' means that, on a nationwide basis--
``(A) health information technology is interoperable,
in accordance with section 3010; and
``(B) such technology is employed by meaningful EHR
users under the specified meaningful use incentive
programs (as defined in section 3010A(e)) and by other
clinicians and health care providers.''.
(g) Conforming Amendments.--
(1) Voluntary use of standards.--Section 3006 of the Public
Health Service Act (42 U.S.C. 300jj-16) is amended--
(A) in subsection (a)(1), by inserting ``, including
an interoperability standard adopted under such
section'' after ``section 3004''.
(B) in subsection (b), by inserting ``, including the
interoperability standards adopted under such section''
after ``section 3004''.
(2) HIPAA privacy and security law definition correction.--
Section 3009(a)(2)(A) of the Public Health Service Act (42
U.S.C. 300jj-19(a)(2)(A)) is amended by striking ``title IV''
and inserting ``title XIII''.
(3) Coordination of federal activities.--Section 13111 of the
HITECH Act is amended--
(A) in subsection (a), by inserting before the period
at the end the following: ``(and, beginning on January
1, 2018, that are also interoperable under section 3010
of such Act, including by being in compliance with
interoperability standards adopted under section 3004
of such Act)''; and
(B) in subsection (b), by inserting ``(and, beginning
on January 1, 2018, including an interoperability
standard adopted under section 3004 of such Act)''
before ``the President''.
(4) Application to private entities.--Section 13112 of the
HITECH Act is amended by inserting before the period at the end
the following: ``(and, beginning on January 1, 2018, that are
also interoperable under section 3010 of such Act, including by
being in compliance with interoperability standards adopted
under section 3004 of such Act)''.
(5) Coordination with recommendations for achieving
widespread ehr interoperability.--Section 106 of the Medicare
Access and CHIP Reauthorization Act of 2015 (Public Law 114-10)
is amended by striking subsection (b).''.
(h) Patient Empowerment.--It is the sense of Congress that--
(1) patients have the right to the entirety of the health
information of such patients, including such information
contained in an electronic health record of such patients;
(2) such right extends to both structured and unstructured
data; and
(3) to further facilitate patient ownership over health
information of such patient--
(A) health care providers should not have the ability
to deny a patient's request for access to the entirety
of such health information of such patient; and
(B) health care providers do not need the consent of
their patients to share personal health information of
such patients with other covered entities, in
compliance with the HIPAA privacy regulations
promulgated pursuant to section 264(c) of the Health
Insurance Portability and Accountability Act of 1996
for the purposes of supporting patient care, except in
situations where consent is specifically required under
such regulations, such as in cases related to the
psychiatric records of the patient.
Subtitle B--Telehealth
SEC. 3021. TELEHEALTH SERVICES UNDER THE MEDICARE PROGRAM.
(a) Provision of Information by Centers for Medicare & Medicaid
Services.--Not later than 1 year after the date of the enactment of
this Act, the Administrator of the Centers for Medicare & Medicaid
Services shall provide to the committees of jurisdiction of the House
of Representatives and the Senate information on the following:
(1) The populations of Medicare beneficiaries, such as those
who are dually eligible for the Medicare program under title
XVIII of the Social Security Act (42 U.S.C. 1395 et seq.) and
the Medicaid program under title XIX of such Act (42 U.S.C.
1396 et seq.) and those with chronic conditions, whose care may
be improved most in terms of quality and efficiency by the
expansion, in a manner that meets or exceeds the existing in-
person standard of care under the Medicare program under title
XVIII of such Act, of telehealth services under section
1834(m)(4) of such Act (42 U.S.C. 1395m(m)(4)).
(2) Activities by the Center for Medicare and Medicaid
Innovation which examine the use of telehealth services in
models, projects, or initiatives funded through section 1115A
of the Social Security Act (42 U.S.C. 1315a).
(3) The types of high volume procedures codes or diagnoses
under such title XVIII which might be suitable to the
furnishing of services via telehealth.
(4) Barriers that might prevent the expansion of telehealth
services under section 1834(m)(4) of the Social Security Act
(42 U.S.C. 1395m(m)(4)) beyond such services that are in effect
as of the date of the enactment of this Act.
(b) Provision of Information by MedPAC.--Not later than 1 year after
the date of the enactment of this Act, the Medicare Payment Advisory
Commission established under section 1805 of the Social Security Act
(42 U.S.C. 1395b-6) shall, using data from the Medicare Advantage
program under part C of title XVIII of such Act (42 U.S.C. 1395w-21 et
seq.), provide information to the committees of jurisdiction of the
House of Representatives and the Senate that identifies--
(1) services--
(A) for which payment could not be made, as of the
date of the enactment of this Act, under the fee-for-
service program under parts A and B of such title by
reason of any limitation imposed under section 1834(m)
of such Act (42 U.S.C. 1395m(m)); and
(B) that are services that are recommended by the
Commission to be included as telehealth services for
which payment may be made under the fee-for-service
program under parts A and B of such title; and
(2) barriers to furnishing telehealth services for which
payment may be made under such title XVIII and solutions to
address such barriers.
(c) Sense of Congress.--It is the sense of Congress that--
(1) States should collaborate, through the use of State
health board compacts or other mechanisms, to create common
licensure requirements services in order to facilitate
multistate practices and allow for health care providers to
provide such services across State lines;
(2) health care providers should be appropriately licensed in
the physical location where the patient is receiving services;
(3) eligible originating sites should be expanded beyond
those originating sites described in section 1834(m)(4)(C) of
the Social Security Act (42 U.S.C. 1395m(m)(4)(C)); and
(4) any expansion of telehealth services under the Medicare
program should--
(A) recognize that telemedicine is the delivery of
safe, effective, quality health care services, by a
health care provider, using technology as the mode of
care delivery;
(B) meet or exceed the conditions of coverage and
payment with respect to the Medicare program under
title XVIII unless specifically address in subsequent
statute, of such Act if the service were furnished in
person, including standards of care; and
(C) involve clinically appropriate means to furnish
such services.
Subtitle C--Encouraging Continuing Medical Education for Physicians
SEC. 3041. EXEMPTING FROM MANUFACTURER TRANSPARENCY REPORTING CERTAIN
TRANSFERS USED FOR EDUCATIONAL PURPOSES.
(a) In General.--Section 1128G(e)(10)(B) of the Social Security Act
(42 U.S.C. 1320a-7h(e)(10)(B)) is amended--
(1) in clause (iii), by inserting ``, including peer-reviewed
journals, journal reprints, journal supplements, medical
conference reports, and medical textbooks'' after ``patient
use''; and
(2) by adding at the end the following new clause:
``(xiii) In the case of a covered recipient
who is a physician, an indirect payment or
transfer of value to the covered recipient--
``(I) for speaking at, or preparing
educational materials for, an
educational event for physicians or
other health care professionals that
does not commercially promote a covered
drug, device, biological, or medical
supply; or
``(II) that serves the sole purpose
of providing the covered recipient with
medical education, such as by providing
the covered recipient with the tuition
required to attend an educational event
or with materials provided to
physicians at an educational event.''.
(b) Effective Date.--The amendments made by this section shall apply
with respect to transfers of value made on or after the date of the
enactment of this Act.
Subtitle D--Disposable Medical Technologies
SEC. 3061. TREATMENT OF CERTAIN ITEMS AND DEVICES.
(a) In General.--Section 1834 of the Social Security Act (42 U.S.C.
1395m) is amended by adding at the end the following new subsection:
``(r) Payment for Certain Disposable Devices.--
``(1) In general.--The Secretary shall make separate payment
in the amount established under paragraph (3) to a home health
agency for a device described in paragraph (2) when furnished
to an individual who receives home health services for which
payment is made under section 1895(b).
``(2) Device described.--For purposes of paragraph (1), a
device described in this paragraph is a disposable device for
which, as of January 1, 2015, there is--
``(A) a Level I Healthcare Common Procedure Coding
System (HCPCS) code for which the description for a
professional service includes the furnishing of such
device; and
``(B) a separate Level I HCPCS code for a
professional service that uses durable medical
equipment instead of such device.
``(3) Payment amount.--The Secretary shall establish the
separate payment amount for such a device such that such amount
does not exceed the payment that would be made for the HCPCS
code described in paragraph (2)(A) under section 1833(t)
(relating to payment for covered OPD services).''.
(b) Conforming Amendment.--Section 1861(m)(5) of the Social Security
Act (42 U.S.C. 1395x(m)(5)) is amended by inserting ``and devices
described in section 1834(r)(2)'' after ``durable medical equipment''.
(c) Effective Date.--The amendments made by this section shall apply
to devices furnished on or after January 1, 2017.
Subtitle E--Local Coverage Decision Reforms
SEC. 3081. IMPROVEMENTS IN THE MEDICARE LOCAL COVERAGE DETERMINATION
(LCD) PROCESS.
(a) In General.--Section 1862(l)(5) of the Social Security Act (42
U.S.C. 1395y(l)(5)) is amended by adding at the end the following new
subparagraph:
``(D) Local coverage determinations.--The Secretary
shall require each medicare administrative contractor
that develops a local coverage determination to make
available on the website of such contractor and in the
coverage database on the Medicare website, at least 45
days before the effective date of such determination,
the following information:
``(i) Such determination in its entirety.
``(ii) Where and when the proposed
determination was first made public.
``(iii) Hyperlinks to the proposed
determination and a response to comments
submitted to the contractor with respect to
such proposed determination.
``(iv) A summary of evidence that was
considered by the contractor during the
development of such determination and a list of
the sources of such evidence.
``(v) An explanation of the rationale that
supports such determination.''.
(b) Effective Date.--The amendment made by subsection (a) shall apply
with respect to local coverage determinations that are proposed or
revised on or after the date that is 180 days after the date of the
enactment of this Act.
Subtitle F--Medicare Pharmaceutical and Technology Ombudsman
SEC. 3101. MEDICARE PHARMACEUTICAL AND TECHNOLOGY OMBUDSMAN.
Section 1808(c) of the Social Security Act (42 U.S.C. 1395b-9(c)) is
amended by adding at the end the following new paragraph:
``(4) Pharmaceutical and technology ombudsman.--Not later
than 12 months after the date of the enactment of this
paragraph, the Secretary shall provide for a pharmaceutical and
technology ombudsman within the Centers for Medicare & Medicaid
Services who shall receive and respond to complaints,
grievances, and requests that--
``(A) are from entities that manufacture
pharmaceutical, biotechnology, medical device, or
diagnostic products that are covered or for which
coverage is being sought under this title; and
``(B) are with respect to coverage, coding, or
payment under this title for such products.''.
Subtitle G--Medicare Site-of-Service Price Transparency
SEC. 3121. MEDICARE SITE-OF-SERVICE PRICE TRANSPARENCY.
Section 1834 of the Social Security Act (42 U.S.C. 1395m), as amended
by section 3061, is further amended by adding at the end the following
new subsection:
``(s) Site-of-Service Price Transparency.--
``(1) In general.--In order to facilitate price transparency
with respect to items and services for which payment may be
made either to a hospital outpatient department or to an
ambulatory surgical center under this title, the Secretary
shall, for 2017 and each year thereafter, make available to the
public via a searchable website, with respect to an appropriate
number of such items and services--
``(A) the estimated payment amount for the item or
service under the outpatient department fee schedule
under subsection (t) of section 1833 and the ambulatory
surgical center payment system under subsection (i) of
such section; and
``(B) the estimated amount of beneficiary liability
applicable to the item or service.
``(2) Calculation of estimated beneficiary liability.--For
purposes of paragraph (1)(B), the estimated amount of
beneficiary liability, with respect to an item or service, is
the amount for such item or service for which an individual who
does not have coverage under a medicare supplemental policy
certified under section 1882 or any other supplemental
insurance coverage is responsible.
``(3) Implementation.--In carrying out this subsection, the
Secretary--
``(A) shall include in the notice described in
section 1804(a) a notification of the availability of
the estimated amounts made available under paragraph
(1); and
``(B) may utilize mechanisms in existence on the date
of the enactment of this subsection, such as the
portion of the website of the Centers for Medicare &
Medicaid Services on which information comparing
physician performance is posted (commonly referred to
as the Physician Compare website), to make available
such estimated amounts under such paragraph.
``(4) Funding.--For purposes of implementing this subsection,
the Secretary shall provide for the transfer, from the
Supplemental Medical Insurance Trust Fund under section 1841 to
the Centers for Medicare & Medicaid Services Program Management
Account, of $6,000,000 for fiscal year 2015, to remain
available until expended.''.
Subtitle H--Medicare Part D Patient Safety and Drug Abuse Prevention
SEC. 3141. PROGRAMS TO PREVENT PRESCRIPTION DRUG ABUSE UNDER MEDICARE
PARTS C AND D.
(a) Drug Management Program for At-Risk Beneficiaries.--
(1) In general.--Section 1860D-4(c) of the Social Security
Act (42 U.S.C. 1395w-10(c)) is amended by adding at the end the
following:
``(5) Drug management program for at-risk beneficiaries.--
``(A) Authority to establish.--A PDP sponsor may
establish a drug management program for at-risk
beneficiaries under which, subject to subparagraph (B),
the PDP sponsor may, in the case of an at-risk
beneficiary for prescription drug abuse who is an
enrollee in a prescription drug plan of such PDP
sponsor, limit such beneficiary's access to coverage
for frequently abused drugs under such plan to
frequently abused drugs that are prescribed for such
beneficiary by one or more prescribers selected under
subparagraph (D), and dispensed for such beneficiary by
one or more pharmacies selected under such
subparagraph.
``(B) Requirement for notices.--
``(i) In general.--A PDP sponsor may not
limit the access of an at-risk beneficiary for
prescription drug abuse to coverage for
frequently abused drugs under a prescription
drug plan until such sponsor--
``(I) provides to the beneficiary an
initial notice described in clause (ii)
and a second notice described in clause
(iii); and
``(II) verifies with the providers of
the beneficiary that the beneficiary is
an at-risk beneficiary for prescription
drug abuse.
``(ii) Initial notice.--An initial notice
described in this clause is a notice that
provides to the beneficiary--
``(I) notice that the PDP sponsor has
identified the beneficiary as
potentially being an at-risk
beneficiary for prescription drug
abuse;
``(II) information describing all
State and Federal public health
resources that are designed to address
prescription drug abuse to which the
beneficiary has access, including
mental health services and other
counseling services;
``(III) notice of, and information
about, the right of the beneficiary to
appeal such identification under
subsection (h) and the option of an
automatic escalation to external
review;
``(IV) a request for the beneficiary
to submit to the PDP sponsor
preferences for which prescribers and
pharmacies the beneficiary would prefer
the PDP sponsor to select under
subparagraph (D) in the case that the
beneficiary is identified as an at-risk
beneficiary for prescription drug abuse
as described in clause (iii)(I);
``(V) an explanation of the meaning
and consequences of the identification
of the beneficiary as potentially being
an at-risk beneficiary for prescription
drug abuse, including an explanation of
the drug management program established
by the PDP sponsor pursuant to
subparagraph (A);
``(VI) clear instructions that
explain how the beneficiary can contact
the PDP sponsor in order to submit to
the PDP sponsor the preferences
described in subclause (IV) and any
other communications relating to the
drug management program for at-risk
beneficiaries established by the PDP
sponsor; and
``(VII) contact information for other
organizations that can provide the
beneficiary with assistance regarding
such drug management program (similar
to the information provided by the
Secretary in other standardized notices
provided to part D eligible individuals
enrolled in prescription drug plans
under this part).
``(iii) Second notice.--A second notice
described in this clause is a notice that
provides to the beneficiary notice--
``(I) that the PDP sponsor has
identified the beneficiary as an at-
risk beneficiary for prescription drug
abuse;
``(II) that such beneficiary is
subject to the requirements of the drug
management program for at-risk
beneficiaries established by such PDP
sponsor for such plan;
``(III) of the prescriber (or
prescribers) and pharmacy (or
pharmacies) selected for such
individual under subparagraph (D);
``(IV) of, and information about, the
beneficiary's right to appeal such
identification under subsection (h) and
the option of an automatic escalation
to external review;
``(V) that the beneficiary can, in
the case that the beneficiary has not
previously submitted to the PDP sponsor
preferences for which prescribers and
pharmacies the beneficiary would prefer
the PDP sponsor select under
subparagraph (D), submit such
preferences to the PDP sponsor; and
``(VI) that includes clear
instructions that explain how the
beneficiary can contact the PDP
sponsor.
``(iv) Timing of notices.--
``(I) In general.--Subject to
subclause (II), a second notice
described in clause (iii) shall be
provided to the beneficiary on a date
that is not less than 60 days after an
initial notice described in clause (ii)
is provided to the beneficiary.
``(II) Exception.--In the case that
the PDP sponsor, in conjunction with
the Secretary, determines that concerns
identified through rulemaking by the
Secretary regarding the health or
safety of the beneficiary or regarding
significant drug diversion activities
require the PDP sponsor to provide a
second notice described in clause (iii)
to the beneficiary on a date that is
earlier than the date described in
subclause (I), the PDP sponsor may
provide such second notice on such
earlier date.
``(C) At-risk beneficiary for prescription drug
abuse.--
``(i) In general.--For purposes of this
paragraph, the term `at-risk beneficiary for
prescription drug abuse' means a part D
eligible individual who is not an exempted
individual described in clause (ii) and--
``(I) who is identified through the
use of clinical guidelines developed by
the Secretary in consultation with PDP
sponsors and other stakeholders
described in section 3141(f)(2)(A) of
the 21st Century Cures Act; or
``(II) with respect to whom the PDP
sponsor of a prescription drug plan,
upon enrolling such individual in such
plan, received notice from the
Secretary that such individual was
identified under this paragraph to be
an at-risk beneficiary for prescription
drug abuse under the prescription drug
plan in which such individual was most
recently previously enrolled and such
identification has not been terminated
under subparagraph (F).
``(ii) Exempted individual described.--An
exempted individual described in this clause is
an individual who--
``(I) receives hospice care under
this title;
``(II) is a resident of a long-term
care facility, of an intermediate care
facility for the mentally retarded, or
of another facility for which
frequently abused drugs are dispensed
for residents through a contract with a
single pharmacy; or
``(III) the Secretary elects to treat
as an exempted individual for purposes
of clause (i).
``(D) Selection of prescribers and pharmacies.--
``(i) In general.--With respect to each at-
risk beneficiary for prescription drug abuse
enrolled in a prescription drug plan offered by
such sponsor, a PDP sponsor shall, based on the
preferences submitted to the PDP sponsor by the
beneficiary pursuant to clauses (ii)(IV) and
(iii)(V) of subparagraph (B), select--
``(I) one or more individuals who are
authorized to prescribe frequently
abused drugs (referred to in this
paragraph as `prescribers') who may
write prescriptions for such drugs for
such beneficiary; and
``(II) one or more pharmacies that
may dispense such drugs to such
beneficiary.
``(ii) Reasonable access.--In making the
selections under this subparagraph--
``(I) a PDP sponsor shall ensure that
the beneficiary continues to have
reasonable access to frequently abused
drugs (as defined in subparagraph (G)),
taking into account geographic
location, beneficiary preference,
impact on costsharing, and reasonable
travel time; and
``(II) a PDP sponsor shall ensure
such access (including access to
prescribers and pharmacies with respect
to frequently abused drugs) in the case
of individuals with multiple residences
and in the case of natural disasters
and similar emergency situations.
``(iii) Beneficiary preferences.--
``(I) In general.--If an at-risk
beneficiary for prescription drug abuse
submits preferences for which in-
network prescribers and pharmacies the
beneficiary would prefer the PDP
sponsor select in response to a notice
under subparagraph (B), the PDP sponsor
shall--
``(aa) review such
preferences;
``(bb) select or change the
selection of prescribers and
pharmacies for the beneficiary
based on such preferences; and
``(cc) inform the beneficiary
of such selection or change of
selection.
``(II) Exception.--In the case that
the PDP sponsor determines that a
change to the selection of prescriber
or pharmacy under item (bb) by the PDP
sponsor is contributing or would
contribute to prescription drug abuse
or drug diversion by the beneficiary,
the PDP sponsor may change the
selection of prescriber or pharmacy for
the beneficiary without regard to the
preferences of the beneficiary
described in subclause (I).
``(iv) Confirmation.--Before selecting a
prescriber (or prescribers) or pharmacy (or
pharmacies) under this subparagraph, a PDP
sponsor must request and receive confirmation
from such a prescriber or pharmacy
acknowledging and accepting that the
beneficiary involved is in the drug management
program for at-risk beneficiaries.
``(E) Terminations and appeals.--The identification
of an individual as an at-risk beneficiary for
prescription drug abuse under this paragraph, a
coverage determination made under a drug management
program for at-risk beneficiaries, and the selection of
prescriber or pharmacy under subparagraph (D) with
respect to such individual shall be subject to
reconsideration and appeal under subsection (h) and the
option of an automatic escalation to external review to
the extent provided by the Secretary.
``(F) Termination of identification.--
``(i) In general.--The Secretary shall
develop standards for the termination of
identification of an individual as an at-risk
beneficiary for prescription drug abuse under
this paragraph. Under such standards such
identification shall terminate as of the
earlier of--
``(I) the date the individual
demonstrates that the individual is no
longer likely, in the absence of the
restrictions under this paragraph, to
be an at-risk beneficiary for
prescription drug abuse described in
subparagraph (C)(i); and
``(II) the end of such maximum period
of identification as the Secretary may
specify.
``(ii) Rule of construction.--Nothing in
clause (i) shall be construed as preventing a
plan from identifying an individual as an at-
risk beneficiary for prescription drug abuse
under subparagraph (C)(i) after such
termination on the basis of additional
information on drug use occurring after the
date of notice of such termination.
``(G) Frequently abused drug.--For purposes of this
subsection, the term `frequently abused drug' means a
drug that is a controlled substance that the Secretary
determines to be frequently abused or diverted.
``(H) Data disclosure.--In the case of an at-risk
beneficiary for prescription drug abuse whose access to
coverage for frequently abused drugs under a
prescription drug plan has been limited by a PDP
sponsor under this paragraph, such PDP sponsor shall
disclose data, including any necessary individually
identifiable health information, in a form and manner
specified by the Secretary, about the decision to
impose such limitations and the limitations imposed by
the sponsor under this part.
``(I) Education.--The Secretary shall provide
education to enrollees in prescription drug plans of
PDP sponsors and providers regarding the drug
management program for at-risk beneficiaries described
in this paragraph, including education--
``(i) provided by medicare administrative
contractors through the improper payment
outreach and education program described in
section 1874A(h); and
``(ii) through current education efforts
(such as State health insurance assistance
programs described in subsection (a)(1)(A) of
section 119 of the Medicare Improvements for
Patients and Providers Act of 2008 (42 U.S.C.
1395b-3 note)) and materials directed toward
such enrollees.
``(J) Application under ma-pd plans.--Pursuant to
section 1860D--21(c)(1), the provisions of this
paragraph apply under part D to MA organizations
offering MA-PD plans to MA eligible individuals in the
same manner as such provisions apply under this part to
a PDP sponsor offering a prescription drug plan to a
part D eligible individual.''.
(2) Information for consumers.--Section 1860D-4(a)(1)(B) of
the Social Security Act (42 U.S.C. 1395w-104(a)(1)(B)) is
amended by adding at the end the following:
``(v) The drug management program for at-risk
beneficiaries under subsection (c)(5).''.
(b) Utilization Management Programs.--Section 1860D-4(c) of the
Social Security Act (42 U.S.C. 1395w-104(c)), as amended by subsection
(a)(1), is further amended--
(1) in paragraph (1), by inserting after subparagraph (D) the
following new subparagraph:
``(E) A utilization management tool to prevent drug
abuse (as described in paragraph (6)(A)).''; and
(2) by adding at the end the following new paragraph:
``(6) Utilization management tool to prevent drug abuse.--
``(A) In general.--A tool described in this paragraph
is any of the following:
``(i) A utilization tool designed to prevent
the abuse of frequently abused drugs by
individuals and to prevent the diversion of
such drugs at pharmacies.
``(ii) Retrospective utilization review to
identify--
``(I) individuals that receive
frequently abused drugs at a frequency
or in amounts that are not clinically
appropriate; and
``(II) providers of services or
suppliers that may facilitate the abuse
or diversion of frequently abused drugs
by beneficiaries.
``(iii) Consultation with the contractor
described in subparagraph (B) to verify if an
individual enrolling in a prescription drug
plan offered by a PDP sponsor has been
previously identified by another PDP sponsor as
an individual described in clause (ii)(I).
``(B) Reporting.--A PDP sponsor offering a
prescription drug plan (and an MA organization offering
an MA-PD plan) in a State shall submit to the Secretary
and the Medicare drug integrity contractor with which
the Secretary has entered into a contract under section
1893 with respect to such State a report, on a monthly
basis, containing information on--
``(i) any provider of services or supplier
described in subparagraph (A)(ii)(II) that is
identified by such plan sponsor (or
organization) during the 30-day period before
such report is submitted; and
``(ii) the name and prescription records of
individuals described in paragraph (5)(C).''.
(c) Expanding Activities of Medicare Drug Integrity Contractors
(MEDICs).--
(1) In general.--Section 1893 of the Social Security Act (42
U.S.C. 1395ddd) is amended by adding at the end the following
new subsection:
``(j) Expanding Activities of Medicare Drug Integrity Contractors
(MEDICs).--
``(1) Access to information.--Under contracts entered into
under this section with Medicare drug integrity contractors
(including any successor entity to a Medicare drug integrity
contractor), the Secretary shall authorize such contractors to
directly accept prescription and necessary medical records from
entities such as pharmacies, prescription drug plans, MA-PD
plans, and physicians with respect to an individual in order
for such contractors to provide information relevant to the
determination of whether such individual is an at-risk
beneficiary for prescription drug abuse, as defined in section
1860D-4(c)(5)(C).
``(2) Requirement for acknowledgment of referrals.--If a PDP
sponsor or MA organization refers information to a contractor
described in paragraph (1) in order for such contractor to
assist in the determination described in such paragraph, the
contractor shall--
``(A) acknowledge to the sponsor or organization
receipt of the referral; and
``(B) in the case that any PDP sponsor or MA
organization contacts the contractor requesting to know
the determination by the contractor of whether or not
an individual has been determined to be an individual
described such paragraph, shall inform such sponsor or
organization of such determination on a date that is
not later than 15 days after the date on which the
sponsor or organization contacts the contractor.
``(3) Making data available to other entities.--
``(A) In general.--For purposes of carrying out this
subsection, subject to subparagraph (B), the Secretary
shall authorize MEDICs to respond to requests for
information from PDP sponsors and MA organizations,
State prescription drug monitoring programs, and other
entities delegated by such sponsors or organizations
using available programs and systems in the effort to
prevent fraud, waste, and abuse.
``(B) HIPAA compliant information only.--Information
may only be disclosed by a MEDIC under subparagraph (A)
if the disclosure of such information is permitted
under the Federal regulations (concerning the privacy
of individually identifiable health information)
promulgated under section 264(c) of the Health
Insurance Portability and Accountability Act of 1996
(42 U.S.C. 1320d-2 note).''.
(2) OIG study and report on effectiveness of medics.--
(A) Study.--The Inspector General of the Department
of Health and Human Services shall conduct a study on
the effectiveness of Medicare drug integrity
contractors with which the Secretary of Health and
Human Services has entered into a contract under
section 1893 of the Social Security Act (42 U.S.C.
1395ddd) in identifying, combating, and preventing
fraud under the Medicare program, including under the
authority provided under section 1893(j) of the Social
Security Act, added by paragraph (1).
(B) Report.--Not later than 1 year after the date of
the enactment of this Act, the Inspector General shall
submit to Congress a report on the study conducted
under subparagraph (A). Such report shall include such
recommendations for improvements in the effectiveness
of such contractors as the Inspector General determines
appropriate.
(d) Treatment of Certain Complaints for Purposes of Quality or
Performance Assessment.--Section 1860D-42 of the Social Security Act
(42 U.S.C. 1395w-152) is amended by adding at the end the following new
subsection:
``(d) Treatment of Certain Complaints for Purposes of Quality or
Performance Assessment.--In conducting a quality or performance
assessment of a PDP sponsor, the Secretary shall develop or utilize
existing screening methods for reviewing and considering complaints
that are received from enrollees in a prescription drug plan offered by
such PDP sponsor and that are complaints regarding the lack of access
by the individual to prescription drugs due to a drug management
program for at-risk beneficiaries.''.
(e) Sense of Congress Regarding Use of Technology Tools To Combat
Fraud.--It is the sense of Congress that MA organizations and PDP
sponsors should consider using e-prescribing and other health
information technology tools to support combating fraud under MA-PD
plans and prescription drug plans under parts C and D of the Medicare
program.
(f) Effective Date.--
(1) In general.--The amendments made by this section shall
apply to prescription drug plans (and MA-PD plans) for plan
years beginning more than 1 year after the date of the
enactment of this Act.
(2) Stakeholder meetings prior to effective date.--
(A) In general.--Not later than January 1, 2016, the
Secretary of Health and Human Services shall convene
stakeholders, including individuals entitled to
benefits under part A of title XVIII of the Social
Security Act or enrolled under part B of such title of
such Act, advocacy groups representing such
individuals, physicians, pharmacists, and other
clinicians, retail pharmacies, plan sponsors, entities
delegated by plan sponsors, and biopharmaceutical
manufacturers for input regarding the topics described
in subparagraph (B).
(B) Topics described.--The topics described in this
subparagraph are the topics of--
(i) the impact on cost-sharing and ensuring
accessibility to prescription drugs for
enrollees in prescription drug plans of PDP
sponsors, and enrollees in MA-PD plans, who are
at-risk beneficiaries for prescription drug
abuse (as defined in subparagraph (C) of
paragraph (5) of section 1860D-4(c) of the
Social Security Act (42 U.S.C. 1395w-104(c)));
(ii) the use of an expedited appeals process
under which such an enrollee may appeal an
identification of such enrollee as an at-risk
beneficiary for prescription drug abuse under
such paragraph (similar to the processes
established under the Medicare Advantage
program under part C of title XVIII of the
Social Security Act that allow an automatic
escalation to external review of claims
submitted under such part);
(iii) the types of enrollees that should be
treated as exempted individuals, as described
in subparagraph (C)(ii) of such paragraph;
(iv) the manner in which terms and
definitions in such paragraph should be
applied, such as the use of clinical
appropriateness in determining whether an
enrollee is an at-risk beneficiary for
prescription drug abuse as defined in
subparagraph (C) of such paragraph;
(v) the information to be included in the
notices described in subparagraph (B) of such
paragraph and the standardization of such
notices; and
(vi) with respect to a PDP sponsor (or
Medicare Advantage organization) that
establishes a drug management program for at-
risk beneficiaries under such paragraph, the
responsibilities of such PDP sponsor (or
organization) with respect to the
implementation of such program.
(g) Rulemaking.--The Secretary of Health and Human Services shall
promulgate regulations based on the input gathered pursuant to
subsection (f)(2)(A).
TITLE IV--MEDICAID, MEDICARE, AND OTHER REFORMS
Subtitle A--Medicaid and Medicare Reforms
SEC. 4001. LIMITING FEDERAL MEDICAID REIMBURSEMENT TO STATES FOR
DURABLE MEDICAL EQUIPMENT (DME) TO MEDICARE PAYMENT
RATES.
(a) Medicaid Reimbursement.--
(1) In general.--Section 1903(i) of the Social Security Act
(42 U.S.C. 1396b(i)) is amended--
(A) in paragraph (25), by striking ``or'' at the end;
(B) in paragraph (26), by striking the period at the
end and inserting ``; or''; and
(C) by inserting after paragraph (26) the following
new paragraph:
``(27) with respect to any amounts expended by the State on
the basis of a fee schedule for items described in section
1861(n), as determined in the aggregate with respect to each
class of such items as defined by the Secretary, in excess of
the aggregate amount, if any, that would be paid for such items
within such class on a fee-for-service basis under the program
under part B of title XVIII, including, as applicable, under a
competitive acquisition program under section 1847 in an area
of the State.''.
(2) Effective date.--The amendments made by this subsection
shall be effective with respect to payments for items furnished
on or after January 1, 2020.
(b) Medicare Ombudsman.--Section 1808(c) of the Social Security Act
(42 U.S.C. 1395b(c)), as amended by section 3101, is further amended by
adding at the end the following new paragraph:
``(5) Monitoring dme reimbursement under medicaid.--The
ombudsmen under each of paragraphs (1) and (4) shall evaluate
the impact of the competitive acquisition program under section
1847, including as applied under section 1903(i)(27), on
beneficiary health status and health outcomes.''.
SEC. 4002. MEDICARE PAYMENT INCENTIVE FOR THE TRANSITION FROM
TRADITIONAL X-RAY IMAGING TO DIGITAL RADIOGRAPHY
AND OTHER MEDICARE IMAGING PAYMENT PROVISION.
(a) Physician Fee Schedule.--
(1) Payment incentive for transition.--
(A) In general.--Section 1848(b) of the Social
Security Act (42 U.S.C. 1395w-4(b)) is amended by
adding at the end the following new paragraph:
``(9) Special rule to incentivize transition from traditional
x-ray imaging to digital radiography.--
``(A) Limitation on payment for film x-ray imaging
services.--In the case of imaging services that are X
rays taken using film and that are furnished during
2017 or a subsequent year, the payment amount for the
technical component (including the technical component
portion of a global fee) of such services that would
otherwise be determined under this section (without
application of this paragraph and before application of
any other adjustment under this section) for such year
shall be reduced by 20 percent.
``(B) Phased-in limitation on payment for computed
radiography imaging services.--In the case of imaging
services that are X rays taken using computed
radiography technology--
``(i) in the case of such services furnished
during 2018, 2019, 2020, 2021, or 2022 the
payment amount for the technical component
(including the technical component portion of a
global fee) of such services that would
otherwise be determined under this section
(without application of this paragraph and
before application of any other adjustment
under this section) for such year shall be
reduced by 7 percent; and
``(ii) in the case of such services furnished
during 2023 or a subsequent year, the payment
amount for the technical component (including
the technical component portion of a global
fee) of such services that would otherwise be
determined under this section (without
application of this paragraph and before
application of any other adjustment under this
section) for such year shall be reduced by 10
percent.
``(C) Computed radiography technology defined.--For
purposes of this paragraph, the term `computed
radiography technology' means cassette-based imaging
which utilizes an imaging plate to create the image
involved.
``(D) Implementation.--In order to implement this
paragraph, the Secretary shall adopt appropriate
mechanisms which may include use of modifiers.''.
(B) Exemption from budget neutrality.--Section
1848(c)(2)(B)(v) of the Social Security Act (42 U.S.C.
1395w-4(c)(2)(B)(v)) is amended by adding at the end
the following new subclause:
``(X) Reduced expenditures
attributable to incentives to
transition to digital radiography.--
Effective for fee schedules established
beginning with 2017, reduced
expenditures attributable to
subparagraph (A) of subsection (b)(9)
and effective for fee schedules
established beginning with 2018,
reduced expenditures attributable to
subparagraph (B) of such subsection.''.
(2) Elimination of application of multiple procedure payment
reduction.--Section 1848(b)(4) of the Social Security Act (42
U.S.C. 1395w-4(b)(4)) is amended by adding at the end the
following new subparagraph:
``(E) Elimination of application of multiple
procedure payment reduction.--
``(i) In general.--Not later than January 1,
2016, the Secretary shall not apply a multiple
procedure payment reduction policy to the
professional component of imaging services
furnished in any subsequent year that is prior
to a year in which the Secretary conducts and
publishes, as part of the Medicare Physician
Fee Schedule Proposed Rule for a year, the
empirical analysis described in clause (ii).
``(ii) Empirical analysis described.--The
empirical analysis described in this clause is
an analysis of the Resource-Based Relative
Value Scale (commonly known as the `RBRVS')
Data Manager information that is used to
determine what, if any, efficiencies exist
within the professional component of imaging
services when two or more studies are performed
on the same patient on the same day. Such
empirical analysis shall include--
``(I) work sheets and other
information detailing which physician
work activities performed given the
typical vignettes were assigned
reduction percentages of 0, 25, 50, 75
and 100 percent;
``(II) a discussion of the clinical
aspects that informed the assignment of
the reduction percentages described in
subclause (I);
``(III) an explanation of how the
percentage reductions for pre-, intra-,
and post-service work were determined
and calculated; and
``(IV) a demonstration that the
Centers for Medicare & Medicaid
Services has consulted with practicing
radiologists to gain knowledge of how
radiologists interpret studies of
multiple body parts on the same
individual on the same day.''.
(b) Payment Incentive for Transition Under Hospital Outpatient
Prospective Payment System.--Section 1833(t)(16) of the Social Security
Act (42 U.S.C. 1395(t)(16)) is amended by adding at the end the
following new subparagraph:
``(F) Payment incentive for the transition from
traditional x-ray imaging to digital radiography.--
Notwithstanding the previous provisions of this
subsection:
``(i) Limitation on payment for film x-ray
imaging services.--In the case of imaging
services that are X rays taken using film and
that are furnished during 2017 or a subsequent
year, the payment amount for the technical
component (including the technical component
portion of a global fee) of such services that
would otherwise be determined under this
section (without application of this paragraph
and before application of any other adjustment
under this subsection) for such year shall be
reduced by 20 percent.
``(ii) Phased-in limitation on payment for
computed radiography imaging services.--In the
case of imaging services that are X rays taken
using computed radiography technology (as
defined in section 1848(b)(9)(C))--
``(I) in the case of such services
furnished during 2018, 2019, 2020,
2021, or 2022 the payment amount for
the technical component (including the
technical component portion of a global
fee) of such services that would
otherwise be determined under this
section (without application of this
paragraph and before application of any
other adjustment under this subsection)
for such year shall be reduced by 7
percent; and
``(II) in the case of such services
furnished during 2023 or a subsequent
year, the payment amount for the
technical component (including the
technical component portion of a global
fee) of such services that would
otherwise be determined under this
section (without application of this
paragraph and before application of any
other adjustment under this subsection)
for such year shall be reduced by 10
percent.
``(iii) Application without regard to budget
neutrality.--The reductions made under this
paragraph--
``(I) shall not be considered an
adjustment under paragraph (2)(E); and
``(II) shall not be implemented in a
budget neutral manner.''.
SEC. 4003. IMPLEMENTATION OF OFFICE OF INSPECTOR GENERAL RECOMMENDATION
TO DELAY CERTAIN MEDICARE PRESCRIPTION DRUG PLAN
PREPAYMENTS.
Section 1860D-15(d) of the Social Security Act (42 U.S.C. 1395w-
115(d)) is amended by adding at the end the following:
``(5) Timing of payments.--With respect to monthly
reinsurance payment amounts under this section to a PDP sponsor
for months in a year (beginning with 2020), such payment
amounts for a month shall be made on the first business day
occurring on or after the following date for that month:
``(A) For the month of January, January 2nd.
``(B) For the month of February, February 5th.
``(C) For the month of March, March 10th.
``(D) For the month of April, April 15th.
``(E) For the month of May, May 20th.
``(F) For the month of June, June 25th.
``(G) For the month of July and each succeeding month
(other than December) in a year, the first day of the
next month.
``(H) For the month of December, December 24th.''.
Subtitle B--Cures Innovation Fund
SEC. 4041. CURES INNOVATION FUND.
(a) Establishment.--There is hereby established in the Treasury of
the United States a fund to be known as the Cures Innovation Fund (in
this section referred to as the ``Fund'').
(b) Appropriations.--There is hereby appropriated to the Fund, out of
any funds in the Treasury not otherwise appropriated, $110,000,000 for
each of fiscal years 2016 through 2020.
(c) Expenditures.--Amounts in the Fund shall be available, as
provided by appropriation Acts, for making expenditures for carrying
out the following:
(1) Section 229A of the Public Health Service Act, as added
by section 1123 (relating to data on natural history of
diseases).
(2) Part E of title II of the Public Health Service Act, as
added by section 1141 (relating to Council for 21st Century
Cures).
(3) Section 2001 and the amendments made by such section
(relating to development and use of patient experience data to
enhance structured risk-benefit assessment framework).
(4) Section 2021 and the amendments made by such section
(relating to qualification of drug development tools).
(5) Section 2062 and the amendments made by such section
(relating to utilizing evidence from clinical experience).
(6) Section 2161 (relating to grants for studying the process
of continuous drug manufacturing).
(d) Supplement, Not Supplant; Prohibition Against Transfer.--Funds
appropriated by subsection (b)--
(1) shall be used to supplement, not supplant, amounts
otherwise made available to the National Institutes of Health
and the Food and Drug Administration; and
(2) notwithstanding any transfer authority in any
appropriation Act, shall not be used for any purpose other than
the expenditures listed in subsection (c).
Subtitle C--Other Reforms
SEC. 4061. SPR DRAWDOWN.
(a) Drawdown and Sale.--Notwithstanding section 161 of the Energy
Policy and Conservation Act (42 U.S.C. 6241), the Secretary of Energy
shall draw down and sell 8,000,000 barrels of crude oil from the
Strategic Petroleum Reserve during each of the fiscal years 2018
through 2025, except as provided in subsection (b). Amounts received
for a sale under this subsection shall be deposited in the general fund
of the Treasury during the fiscal year in which the sale occurs.
(b) Emergency Protection.--The Secretary shall not draw down and sell
crude oil under this section in amounts that would result in a
Strategic Petroleum Reserve that contains an inventory of petroleum
products representing less than 90 days of emergency reserves, based on
the average daily level of net imports of crude oil and petroleum
products in the previous calendar year.
(c) Proceeds.--Proceeds from a sale under this section shall be
deposited into the general fund of the Treasury of the United States.
Subtitle D--Miscellaneous
SEC. 4081. LYME DISEASE AND OTHER TICK-BORNE DISEASES.
(a) In General.--Title III of the Public Health Service Act (42
U.S.C. 241 et seq.) is amended by adding at the end the following new
part:
``PART W--LYME DISEASE AND OTHER TICK-BORNE DISEASES
``SEC. 399OO. RESEARCH.
``(a) In General.--The Secretary shall conduct or support
epidemiological, basic, translational, and clinical research regarding
Lyme disease and other tick-borne diseases.
``(b) Biennial Reports.--The Secretary shall ensure that each
biennial report under section 403 includes information on actions
undertaken by the National Institutes of Health to carry out subsection
(a) with respect to Lyme disease and other tick-borne diseases,
including an assessment of the progress made in improving the outcomes
of Lyme disease and such other tick-borne diseases.
``SEC. 399OO-1. WORKING GROUP.
``(a) Establishment.--The Secretary shall establish a permanent
working group, to be known as the Interagency Lyme and Tick-Borne
Disease Working Group (in this section and section 399OO-2 referred to
as the `Working Group'), to review all efforts within the Department of
Health and Human Services concerning Lyme disease and other tick-borne
diseases to ensure interagency coordination, minimize overlap, and
examine research priorities.
``(b) Responsibilities.--The Working Group shall--
``(1) not later than 24 months after the date of enactment of
this part, and every 24 months thereafter, develop or update a
summary of--
``(A) ongoing Lyme disease and other tick-borne
disease research related to causes, prevention,
treatment, surveillance, diagnosis, diagnostics,
duration of illness, intervention, and access to
services and supports for individuals with Lyme disease
or other tick-borne diseases;
``(B) advances made pursuant to such research;
``(C) the engagement of the Department of Health and
Human Services with persons that participate at the
public meetings required by paragraph (5); and
``(D) the comments received by the Working Group at
such public meetings and the Secretary's response to
such comments;
``(2) ensure that a broad spectrum of scientific viewpoints
is represented in each such summary;
``(3) monitor Federal activities with respect to Lyme disease
and other tick-borne diseases;
``(4) make recommendations to the Secretary regarding any
appropriate changes to such activities; and
``(5) ensure public input by holding annual public meetings
that address scientific advances, research questions,
surveillance activities, and emerging strains in species of
pathogenic organisms.
``(c) Membership.--
``(1) In general.--The Working Group shall be composed of a
total of 14 members as follows:
``(A) Federal members.--Seven Federal members,
consisting of one or more representatives of each of--
``(i) the Office of the Assistant Secretary
for Health;
``(ii) the Food and Drug Administration;
``(iii) the Centers for Disease Control and
Prevention;
``(iv) the National Institutes of Health; and
``(v) such other agencies and offices of the
Department of Health and Human Services as the
Secretary determines appropriate.
``(B) Non-federal public members.--Seven non-Federal
public members, consisting of representatives of the
following categories:
``(i) Physicians and other medical providers
with experience in diagnosing and treating Lyme
disease and other tick-borne diseases.
``(ii) Scientists or researchers with
expertise.
``(iii) Patients and their family members.
``(iv) Nonprofit organizations that advocate
for patients with respect to Lyme disease and
other tick-borne diseases.
``(v) Other individuals whose expertise is
determined by the Secretary to be beneficial to
the functioning of the Working Group.
``(2) Appointment.--The members of the Working Group shall be
appointed by the Secretary, except that of the non-Federal
public members under paragraph (1)(B)--
``(A) one shall be appointed by the Speaker of the
House of Representatives; and
``(B) one shall be appointed by the majority leader
of the Senate.
``(3) Diversity of scientific perspectives.--In making
appointments under paragraph (2), the Secretary, the Speaker of
the House of Representatives, and the majority leader of the
Senate shall ensure that the non-Federal public members of the
Working Group represent a diversity of scientific perspectives.
``(4) Terms.--The non-Federal public members of the Working
Group shall each be appointed to serve a 4-year term and may be
reappointed at the end of such term.
``(d) Meetings.--The Working Group shall meet as often as necessary,
as determined by the Secretary, but not less than twice each year.
``(e) Applicability of FACA.--The Working Group shall be treated as
an advisory committee subject to the Federal Advisory Committee Act.
``(f) Reporting.--Not later than 24 months after the date of
enactment of this part, and every 24 months thereafter, the Working
Group--
``(1) shall submit a report on its activities, including an
up-to-date summary under subsection (b)(1) and any
recommendations under subsection (b)(4), to the Secretary, the
Committee on Energy and Commerce of the House of
Representatives, and the Committee on Health, Education, Labor
and Pensions of the Senate;
``(2) shall make each such report publicly available on the
website of the Department of Health and Human Services; and
``(3) shall allow any member of the Working Group to include
in any such report minority views.
``SEC. 399OO-2. STRATEGIC PLAN.
``Not later than 3 years after the date of enactment of this section,
and every 5 years thereafter, the Secretary shall submit to the
Congress a strategic plan, informed by the most recent summary under
section 399OO-1(b)(1), for the conduct and support of Lyme disease and
tick-borne disease research, including--
``(1) proposed budgetary requirements;
``(2) a plan for improving outcomes of Lyme disease and other
tick-borne diseases, including progress related to chronic or
persistent symptoms and chronic or persistent infection and co-
infections;
``(3) a plan for improving diagnosis, treatment, and
prevention;
``(4) appropriate benchmarks to measure progress on achieving
the improvements described in paragraphs (2) and (3); and
``(5) a plan to disseminate each summary under section 399OO-
1(b)(1) and other relevant information developed by the Working
Group to the public, including health care providers, public
health departments, and other relevant medical groups.''.
(b) No Additional Authorization of Appropriations.--No additional
funds are authorized to be appropriated for the purpose of carrying out
this section and the amendment made by this section, and this section
and such amendment shall be carried out using amounts otherwise
available for such purpose.
Purpose and Summary
For decades, our nation's commitment to the discovery,
development, and delivery of new treatments and cures has made
the United States the biomedical innovation capital of the
world, bringing life-saving and life-improving drugs and
devices to patients. It is clear that the discovery,
development, and delivery process is a cycle. Information
captured and analyzed at the ``end'' of the process--the
delivery phase--can be used for new discovery and development
of better treatments. The purpose of this legislation is to
foster this cycle to help bring more cures and treatments to
patients and strengthen the innovation ecosystem in the United
States.
Background and Need for Legislation
For over a year, the Energy and Commerce Committee has
engaged in a public conversation with patients, innovators,
providers, regulators, and researchers about how to move
advances in science and medicine into new therapies. The fight
to treat and cure disease is an urgent, nonpartisan, national
priority. With 10,000 known diseases, 7,000 of which are rare,
and treatments for only 500 of them, it is clear that there is
much work to do. Disease management costs billions of dollars,
and its personal costs are much higher, causing pain and
heartbreak during the battle and with each loss of life. The
United States has been at the forefront of medical innovation,
developing many promising treatments and cures. Nonetheless,
improvement in the battle with disease and furthering the
nation's leadership position in health care innovation is
possible.
The yearlong 21st Century Cures listening session explored
the complete cycle of cures--from the discovery of clues in
basic science, to the development of new treatments, to the
delivery of those cures, and back again to further discovery.
As explained in the section by section below, this
legislation contains a number of policies to accelerate the
cycle and help find cures and treatments for the thousands of
diseases, particularly rare and serious disease.
Hearings
The Subcommittee on Health held a hearing entitled ``The
President's Council of Advisors on Science and Technology
(PCAST) Report on Drug Innovation'' on May 20, 2014. The
Subcommittee received testimony from:
Gary Neil, M.D., Global Head of Research and
Development, Medgenics;
Sara Radcliffe, Executive Vice President of
Health Section, Biotechnology Industry Organization;
Frank J. Sasinowski, Director, Hyman, Phelps
& McNamara, PC;
Jeff Allen, Executive Director, Friends of
Cancer Research; and
Sean Tunis, M.D., Founder and Chief
Executive Officer, Center for Medical Technology
Policy.
The Subcommittee on Health held a hearing entitled
``Examining the Role of Incentives in Advancing Treatments and
Cures for Patients'' on June 11, 2014. The Subcommittee
received testimony from:
Kenneth Davis, President and CEO, Mt. Sinai
Health System;
Marc Boutin, Executive Vice President and
Chief Operating Officer, National Health Council;
Alexis Borisy, Partner, Third Rock Ventures;
Mike Carusi, General Partner, Advanced
Technology Ventures;
Fred Ledley, Professor, Natural & Applied
Sciences and Management Director, Center for
Integration of Science and Industry, Bentley
University;
C. Scott Hemphill, Professor of Law,
Columbia Law School; and
Steven Miller, Senior Vice President and
Chief Medical Officer, Express Scripts Holding Company.
The Subcommittee on Health held a hearing entitled
``Modernizing Clinical Trials'' on July 9, 2014. The
Subcommittee received testimony from:
Roy S. Herbst, M.D., Ph.D., Chief of Medical
Oncology, Yale Cancer Center;
Sundeep Khosla, M.D., Director, Center for
Clinical and Translational Science, Mayo Clinic;
Jay P. Siegel, M.D., Chief Biotechnology
Office and Head, Scientific Strategy and Policy,
Johnson & Johnson;
William V. Murray, President and CEO,
Medical Device Innovation Consortium;
Robert J. Meyer, M.D., Director, Virginia
Center for Translational and Regulatory Sciences,
University of Virginia School of Medicine;
Paula Brown Stafford, M.P.H., President,
Clinical Development, Quintiles; and
Aaron S. Kesselheim, M.D., J.D., M.P.H,
Assistant Professor of Medicine at Harvard Medical
School.
The Subcommittee on Health held a hearing entitled
``Incorporating the Patient Perspective'' on July 11, 2014. The
Subcommittee received testimony from:
Janet Woodcock, M.D, Director, Center for
Drug Evaluation and Research, Food and Drug
Administration;
Richard F. Pops, Chairman and CEO, Alkermes;
Robert J. Beall, Ph.D., President and CEO,
Cystic Fibrosis Foundation;
Pat Furlong, Founding President and CEO,
Parent Project Muscular Dystrophy;
Leonard Lichtenfeld, M.D., Deputy Chief
Medical Officer, American Cancer Society; and
Marshall Summar, M.D., Director, Scientific
Advisory Committee, National Organization for Rare
Disorders.
The Subcommittee on Health held a hearing entitled
``Technology for 21st Century Cures'' on July 17, 2014. The
Subcommittee received testimony from:
Robert Jarrin, Senior Director, Government
Affairs, Qualcomm Incorporated;
Paul Misener, Vice President, Global Public
Policy, Amazon;
Jonathan Niloff, Chief Medical Officer and
Vice President, McKesson Connected Care and Analytics,
McKesson Corporation;
Dan Riskin, Founder, Health Fidelity; and
Dave Vockell, Chief Executive Officer,
LyfeChannel.
The Subcommittee on Health held a hearing entitled
``Examining Barriers to Ongoing Evidence Development and
Communication'' on July 22, 2014. The Subcommittee received
testimony from:
Mary Grealy, President, Healthcare
Leadership Council;
Michael A. Mussallem, Chairman and CEO,
Edwards Lifesciences;
Gregory Schimizzi, M.D., Cofounder, Carolina
Arthritis Associates, on behalf of the Alliance for
Specialty Medicine;
Josh Rising, M.D., M.P.H., Director, Medical
Devices, The Pew Charitable Trusts; and
Louis Jacques, Senior Vice President and
Chief Clinical Officer, ADVI.
The Subcommittee on Health held a hearing entitled
``Examining the Regulation of Laboratory Developed Tests'' on
September 9, 2014. The Subcommittee received testimony from:
Jeffrey Shuren, M.D., J.D., Director, Center
for Devices and Radiological Health, Food and Drug
Administration;
Christopher Newton-Cheh, M.D., Assistant
Professor of Medicine, Harvard Medical School,
Massachusetts General Hospital;
Andrew Fish, Executive Director, AdvaMed
Diagnostics;
Alan Mertz, President, American Clinical
Laboratory Association;
Charles Sawyers, M.D., Immediate-Past
President, American Association for Cancer Research;
and
Kathleen Wilsey, Ph.D., Co-Founder,
Coalition for 21st Century Medicine.
The Subcommittee on Health held a hearing entitled
``Examining Ways to Combat Antibiotic Resistance and Foster New
Drug Development'' on September 19, 2014. The Subcommittee
received testimony from:
Janet Woodcock, M.D., Director, Center for
Drug Evaluation and Research, U.S. Food and Drug
Administration;
Kenneth J. Hillan, Chief Executive Officer,
Achaogen, Inc.;
Barbara E. Murray, President, Infectious
Diseases Society of America;
Kevin Outterson, Professor of Law, Boston
University School of Law;
Adrian Thomas, Vice President, Global Market
Access and Public Health, Janssen Global Services, LLC;
Allan Coukell, Director, Medical Programs,
Pew Health Group, The Pew Charitable Trusts; and
John H. Powers, Assistant Clinical Professor
of Medicine, George Washington University School of
Medicine.
The Subcommittee on Health held a hearing to review H.R. 6,
21st Century Cures Act, on April 30, 2015. The Subcommittee
received testimony from:
Kathy Hudson, Deputy Director for Science,
Outreach, and Policy, National Institutes of Health;
Janet Woodcock, M.D., Director of the Center
for Drug Evaluation and Research, Center for Drug
Evaluation and Research, U.S. Food and Drug
Administration; and
Jeff Shuren, M.D., J.D., Director of the
Center for Devices and Radiological Health, Center for
Devices and Radiological Health, U.S. Food and Drug
Administration.
Committee Consideration
On May 14, 2015, the Subcommittee on Health met in open
markup session and forwarded H.R. 6 to the full Committee, as
amended, by a voice vote. On May 19, 2015, the full Committee
on Energy and Commerce met in open markup session and ordered
H.R. 6 reported to the House, as amended, by a recorded vote of
51 yeas and 0 nays.
Committee Votes
Clause 3(b) of rule XIII of the Rules of the House of
Representatives requires the Committee to list the recorded
votes on the motion to report legislation and amendments
thereto. A motion by Mr. Upton to order H.R. 6 reported to the
House, with amendment, was agreed to by a recorded vote of 51
ayes and 0 nays. The following reflects the recorded votes
taken during the Committee consideration:
Committee Oversight Findings
Pursuant to clause 3(c)(1) of rule XIII of the Rules of the
House of Representatives, the Committee the Committee held
hearings and made findings that are reflected in this report.
Statement of General Performance Goals and Objectives
The goal of this legislation is to help accelerate the
discovery, development, and delivery of promising new
treatments and cures for patients and strengthen the innovation
ecosystem in the United States.
New Budget Authority, Entitlement Authority, and Tax Expenditures
In compliance with clause 3(c)(2) of rule XIII of the Rules
of the House of Representatives, the Committee finds that H.R.
6, would result in no new or increased budget authority,
entitlement authority, or tax expenditures or revenues.
Earmark, Limited Tax Benefits, and Limited Tariff Benefits
In compliance with clause 9(e), 9(f), and 9(g) of rule XXI
of the Rules of the House of Representatives, the Committee
finds that H.R. 6 contains no earmarks, limited tax benefits,
or limited tariff benefits.
Committee Cost Estimate
The Committee adopts as its own the cost estimate prepared
by the Director of the Congressional Budget Office pursuant to
section 402 of the Congressional Budget Act of 1974.
Congressional Budget Office Estimate
Pursuant to clause 3(c)(3) of rule XIII of the Rules of the
House of Representatives, the following is the cost estimate
provided by the Congressional Budget Office pursuant to section
402 of the Congressional Budget Act of 1974:
U.S. Congress,
Congressional Budget Office,
Washington, DC, June 23, 2015.
Hon. Fred Upton,
Chairman, Committee on Energy and Commerce,
House of Representatives, Washington, DC.
Dear Mr. Chairman: The Congressional Budget Office has
prepared the enclosed cost estimate for H.R. 6, the 21st
Century Cures Act.
If you wish further details on this estimate, we will be
pleased to provide them. The CBO staff contacts are Tom Bradley
and Chad Chirico.
Sincerely,
Keith Hall, Director.
Enclosure.
H.R. 6--21st Century Cures Act
Summary: H.R. 6 would authorize appropriations for the
National Institutes of Health (NIH), the Food and Drug
Administration (FDA), and other agencies within the Department
of Health and Human Services (HHS) for programs aimed at
promoting the discovery and development of drugs and other
technologies that prevent, diagnose, and treat disease or to
support activities authorized by the legislation. The bill also
would make related changes to those agencies' programs.
In addition, H.R. 6 contains provisions that would:
Grant additional periods of exclusivity for
certain brand-name drugs approved for a new indication
that treats a rare disease or condition;
Require Medicare to make an additional
payment to hospitals when Medicare beneficiaries use
certain antimicrobial drugs during the course of their
hospital stay;
Direct the sale of 8 million barrels of oil
from the Strategic Petroleum Reserve (SPR) in each of
the fiscal years 2018 through 2025;
Delay monthly reinsurance payments to stand-
alone prescription drug plans in Medicare Part D by
shifting payments between certain fiscal years; and
Limit federal Medicaid reimbursement to
states for durable medical equipment (DME).
CBO estimates that implementing the legislation would cost
$106.4 billion over the 2016-2020 period, assuming the
appropriation of the authorized and necessary amounts.
CBO estimates that enacting H.R. 6 would reduce direct
spending, on net, by $11.9 billion over the 2016-2025 period.
(Of that amount, CBO estimates that off-budget costs for the
U.S. Postal Service would total $6 million over the 2016-2025
period.) Pay-as-you-go procedures apply because enacting the
legislation would affect direct spending. Enacting H.R. 6 would
not affect revenues.
H.R. 6 contains no intergovernmental mandates as defined in
the Unfunded Mandates Reform Act (UMRA). However, because the
bill would delay entry into the market of some generic drugs
and limit Medicaid payments to states for DME, the bill could
increase state Medicaid costs by $2.6 billion over the 2016-
2025 period, CBO estimates. States have flexibility in that
program to adjust their financial and programmatic
responsibilities, so those costs would not result from an
intergovernmental mandate.
The bill would impose private-sector mandates, as defined
in UMRA, on drug manufacturers. CBO estimates that the
aggregate cost of the mandates would fall below the annual
threshold established in UMRA ($154 million in 2015, adjusted
annually for inflation) in each of the first five years that
the mandates are in effect.
Estimated cost to the Federal Government: The estimated
budgetary effects of H.R. 6 are shown in Table 1. The effects
of the legislation fall primarily within budget functions 270
(energy), 550 (health) and 570 (Medicare).
Basis of estimate: For this estimate, CBO assumes that H.R.
6 will be enacted near the start of fiscal year 2016 and that
authorized amounts will be appropriated each year. Outlay
estimates are based on historical spending patterns for
affected programs.
TABLE 1. BUDGETARY EFFECTS OF H.R. 6
----------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
-------------------------------------------------------------------------
2016 2017 2018 2019 2020 2016-2020 2016-2025
----------------------------------------------------------------------------------------------------------------
CHANGES IN SPENDING SUBJECT TO APPROPRIATION
Department of Health and Human
Services
National Institutes of Health
Authorization Level........... 33,811 35,331 36,851 2,000 2,000 109,993 n.a.
Estimated Outlays............. 8,576 27,473 33,679 26,687 8,625 105,040 n.a.
Food and Drug Administrationa
Estimated Authorization Level. 194 223 240 256 257 1,171 n.a.
Estimated Outlays............. 59 127 178 234 274 872 n.a.
Centers for Disease Control and
Prevention
Estimated Authorization Level. 17 6 6 6 6 41 n.a.
Estimated Outlays............. 6 10 7 6 6 35 n.a.
Other HHS Programsb
Estimated Authorization Level. 112 104 106 109 111 543 n.a.
Estimated Outlays............. 35 82 97 104 109 427 n.a.
Other Departments and Agencies
Estimated Authorization Level. 1 * 4 6 9 21 n.a.
Estimated Outlays............. 1 * 4 6 9 21 n.a.
Subtotal
Estimated Authorization Level. 34,135 35,665 37,207 2,378 2,384 111,768 n.a.
Estimated Outlays............. 8,677 27,692 33,964 27,037 9,024 106,395 n.a.
CHANGES IN DIRECT SPENDINGc
Estimated Budget Authority............ 1 -12 -571 -532 -4,005 -5,119 -11,888
Estimated Outlays..................... 1 -12 -571 -532 -4,005 -5,119 -11,888
----------------------------------------------------------------------------------------------------------------
Notes: * = less than $500,000; n.a. = not applicable; HHS = Health and Human Services.
Numbers may not add up to totals because of rounding.
aAmounts include authorizations of appropriations of $110 million for each of fiscal years 2016 through 2020
from the Cures Innovation Fund established in title IV of the bill. Estimated outlays from the Cures
Innovation Fund are also reported here, assuming appropriation action consistent with the bill.
bH.R. 6 would provide authorizations of appropriations of $10 million for each of fiscal years 2016 through 2023
for the Council for 21st Century Cures.
cIn addition, CBO estimates that enacting H.R. 6 would increase off-budget costs for the U.S. Postal Service by
$6 million over the 2016-2025 period.
Spending subject to appropriation: H.R. 6 would authorize
funding and modify programs within HHS that support medical
research, oversee the development and marketing approval for
drugs, and monitor the use of drugs in the United States. The
legislation also would change the regulatory framework
surrounding medical devices and oversight of technology by FDA.
As shown in Table 2, CBO estimates that implementing H.R. 6
would cost $106.4 billion over the 2016-2020 period, assuming
appropriation of the authorized and estimated amounts. Of that
amount, $105.5 billion would be spent from amounts specifically
authorized by H.R. 6. CBO estimated other authorizations based
on information from NIH, FDA, Centers for Disease Control and
Prevention (CDC), and other government agencies.
Assuming appropriation action consistent with the bill, CBO
estimates that over the 2016-2020 period:
Provisions implemented by NIH would cost
$105.0 billion;
Provisions administered by FDA would cost
$872 million;
Provisions administered by CDC would cost
$35 million;
Provisions affecting discretionary spending
by other HHS programs would cost $427 million; and
Provisions affecting discretionary spending
by other Departments and agencies would cost $21
million.
TABLE 2. ESTIMATED AUTHORIZATIONS OF APPROPRIATIONS IN H.R. 6
----------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
-----------------------------------------------------------
2016-
2016 2017 2018 2019 2020 2020
----------------------------------------------------------------------------------------------------------------
CHANGES IN SPENDING SUBJECT TO APPROPRIATIONa
Title 1--Discovery
NIH Reauthorization
Authorization Level......................... 31,811 33,331 34,851 0 0 99,993
Estimated Outlays........................... 8,190 25,750 31,781 24,735 6,645 97,100
NIH Innovation Fund
Authorization Level......................... 2,000 2,000 2,000 2,000 2,000 10,000
Estimated Outlays........................... 386 1,723 1,897 1,952 1,980 7,939
Other Provisionsb
Estimated Authorization Level............... 21 22 22 21 21 107
Estimated Outlays........................... 7 17 19 20 21 84
Subtotal, Title I
Estimated Authorization Level........... 33,832 35,353 36,873 2,021 2,021 110,100
Estimated Outlays....................... 8,583 27,490 33,698 26,707 8,646 105,124
Title ll--Development
Development and Approval of Prescription Drugs
and Biologics
Estimated Authorization Level............... 51 55 66 69 73 315
Estimated Outlays........................... 22 45 62 69 73 270
Development and Regulation of Medical Devices
and Technology
Estimated Authorizations Level.............. 44 58 65 82 82 331
Estimated Outlays........................... 31 51 61 75 80 299
Subtotal, Title II
Estimated Authorization Level........... 95 113 131 151 155 646
Estimated Outlays....................... 52 96 123 145 153 569
Title III--Delivery
Health Information Technology and Other
Provisions
Estimated Authorization Level............... 12 2 2 2 2 19
Estimated Outlays........................... 6 4 3 3 2 18
Title IV--Medicaid, Medicare, and Other Reforms
Cures Innovation Fundc
Authorization Level......................... 110 110 110 110 110 550
Estimated Outlays........................... 11 36 58 94 129 327
SPR Drawdown
Estimated Authorization Level............... 0 0 2 2 2 6
Estimated Outlays........................... 0 0 2 2 2 6
Lyme Disease and Other Tick-borne Diseases
Estimated Authorization Level............... 86 87 90 92 94 448
Estimated Outlays........................... 25 67 81 87 92 351
Subtotal, Title IV
Estimated Authorization Level........... 193 197 202 204 206 1,004
Estimated Outlays....................... 36 102 141 182 223 684
Total
Estimated Authorization Level........... 34,135 35,665 37,207 2,378 2,384 111,768
Estimated Outlays....................... 8,677 27,692 33,964 27,037 9,024 106,395
----------------------------------------------------------------------------------------------------------------
Notes: NIH = National Institutes of Health; SPR = Strategic Petroleum Reserve.
Numbers may not add up to totals because of rounding.
aEstimated outlays for specified authorizations of appropriations in H.R. 6 are shown for the title in which the
authorization of appropriation is identified in the bill, assuming appropriation action is consistent with the
bill.
bIncludes authorizations of appropriations of $10 million for each of fiscal years 2016 through 2023 for the
Council for 21st Century Cures.
cReflects estimated outlays from the Cures Innovation Fund, including any amounts disbursed by the Fund for
activities that also are associated with separate authorizations identified in titles I and II of the bill.
Title I--Discovery. Title I would reauthorize NIH, make
several programmatic changes to the agency's research and loan
repayment programs, and authorize other initiatives aimed at
promoting medical research. CBO estimates that implementing
title I would cost $105.1 billion over the 2016-2020 period,
assuming the availability of appropriated funds.
NIH Reauthorization. Section 1001 would authorize the
appropriation of almost $100 billion over the next three years
for NIH. The authority for research programs at NIH that are
subject to future appropriations expired at the end of fiscal
year 2009. Since then, however, the Congress has appropriated
an average of about $30 billion annually to continue operating
those programs across all areas of research at NIH. CBO
estimates that reauthorizing NIH would cost $97.1 billion over
the 2016-2020 period.
NIH Innovation Fund. Section 1002 would direct the
Secretary of HHS to establish an ``NIH Innovation Fund'' in the
U.S. Treasury to support biomedical research. The bill would
authorize the appropriation of $2 billion from that fund for
each of fiscal years 2016 through 2020. CBO estimates that
spending from the NIH Innovation Fund would total $7.9 billion
over the 2016-2020 period.
Other Provisions. Other provisions of title I would aim to
promote medical research and accelerate the availability of new
therapies. CBO estimates that implementing those provisions of
title I would cost $84 million over the 2016-2020 period. That
amount includes:
$45 million for the Council for 21st Century
Cures, a public-private partnership intended to help
accelerate the discovery, development, and delivery of
treatments for patients in the United States. (The bill
would authorize the appropriation of $10 million for
each of fiscal years 2016 through 2023 for such
activities.);\1\
---------------------------------------------------------------------------
\1\The legislation also would authorize the appropriation of
additional funds for such purposes from the Cures Innovation Fund
established in title IV of the bill. See discussion of spending by the
Cures Innovation Fund later in the cost estimate.
---------------------------------------------------------------------------
$21 million for CDC to develop a
surveillance system for neurological diseases. (The
bill would authorize the appropriation of $5 million
for each of fiscal years 2016 through 2020 for such
activities.)
$12 million for the Secretary of HHS to
participate in public-private partnerships and award
grants to facilitate the collection, analysis, and
availability of data on diseases. (The bill would
authorize the appropriation of $5 million for each of
fiscal years 2016 through 2020 for such
activities.);\1\ and
$6 million for FDA to establish a pilot
program to create a research sharing system (in
coordination with NIH) that would give third parties
direct access to data generated from clinical trials
funded exclusively by the federal government and to
assist NIH with standardizing certain information in
the registry data bank involving eligibility for
clinical trials.
Title II--Development. Title II of H.R. 6 would modify
FDA's approach to regulating prescription drugs, biologicals,
medical devices, and health-related technology. It also would
make changes to certain surveillance activities by the CDC
relating to antimicrobials and to CDC's vaccine-related
activities. CBO's estimates reflect the expected number of
personnel and investment in information technology required to
implement the bill based on information provided by the
affected agency. (Provisions in title II primarily affect
regulatory activities by FDA.) We estimate that implementing
Title II would cost $569 million over the 2016-2020 period,
assuming the appropriation of the necessary amounts. As
discussed below, Title II would affect two regulatory areas: 1)
prescription drugs and biologics and 2) medical devices and
health-related technology.
Development and Approval of Prescription Drugs and
Biologics. Title II of H.R. 6 contains several provisions that
would modify FDA's regulatory framework for overseeing the
development and approval process of drugs and biologics. The
title also would establish a grant program for institutions of
higher education and nonprofit organizations to study
improvements in the process of continuous manufacturing and
other production-related techniques. Finally, this title would
make changes to CDC's administrative procedures involving
antibiotics and vaccines. Taken together, CBO estimates that
implementing provisions relating to drugs and biologics in
title II would cost $270 million over the 2016-2020 period.
That amount includes:
$33 million to establish a process to
qualify or validate certain drug development tools,
such as biomarkers, for use in certain applications.
That funding would also allow FDA to enter into
cooperative agreements and to award grants to assist
the agency with the review of such qualification
submissions. (The bill would authorize the
appropriation of $10 million for each of fiscal years
2016 through 2020 for such activities.);\1\
$33 million to identify and publish a list
of interpretive criteria for tests that characterize
the susceptibility of particular bacteria, fungi, or
other microorganisms to drugs;
$31 million to facilitate approval for
certain antibacterial and antifungal drugs used by a
limited population of patients;
$25 million to issue and update guidance to
industry, including documents that would assist
sponsors in the development of precision drugs and
biologics, provide guidelines on responsible
communication of certain types of information, and
clarify agency procedures regarding its review of
combination drug products;
$21 million to administer a new grant
program to study continuous drug manufacturing and
production-related technologies. (The bill would
authorize the appropriation of $5 million each of
fiscal years 2016 through 2020 for that program.);\1\
$21 million to implement a program that
would aim to provide incentives to drug companies to
develop new indications for drugs and biologics that
target rare diseases and conditions and to extend the
voucher program for rare pediatric diseases through
December 31, 2018;
$20 million to develop a regulatory
structure that would allow the use of new protocols for
statistical modeling and trial designs to support
marketing applications for drug and biological
products;
$18 million to devise a plan with sponsors
of drug and biological products eligible for
accelerated approval to agree on certain details of the
design of clinical studies in a manner that would
expedite approval of such products;
$14 million, which reflects the costs for a
range of federal programs generated by a provision that
would extend exclusivities for certain brand-name
drugs. (See discussion of the effect of that provision
on mandatory costs for federal health programs below.);
$14 million to establish a ``streamlined
data review program'' that would allow sponsors to
submit qualified summaries of clinical data to support
the approval or licensure of new indications under
certain circumstances;
$14 million to conduct pilot demonstrations
that would expand the use of FDA's existing
surveillance program (that allows the agency to query
electronic data systems and proactively evaluate safety
issues with medical products) to also capture
additional evidence of clinical experiences associated
with marketed drug products. (The bill would authorize
the appropriation of $3 million for each of fiscal
years 2016 through 2020 for such activities.);\1\
$13 million for CDC to monitor and track
usage of antibiotic and antifungal drugs; and
$14 million for miscellaneous provisions of
title II that would affect discretionary spending by
various federal agencies, primarily FDA, CDC, and the
Government Accountability Office.
Development and Regulation of Medical Devices and
Technology. Title II of the bill also contains provisions that
would modify the regulatory framework surrounding medical
devices and oversight of technology by FDA. Assuming
appropriation of the necessary amounts, CBO estimates that
implementing those provisions would cost $299 million over the
2016-2020 period, primarily for FDA's personnel-related
expenses. That amount includes:
$158 million to establish a program to
provide expedited review for certain devices that
represent breakthrough technologies where no approved
alternatives exist and that technology offers
significant advantages over existing alternatives;
$68 million to establish a new accreditation
program for third parties to expedite the approval
process for certain devices, review and recognize
national and international standards, and develop and
update several guidances and regulations;
$68 million to implement a new framework for
the regulation of medical software based on a new
definition of health software, and exempting such
software from most regulation; and
$4 million for FDA's Center for Devices and
Radiological Health to issue final guidance regarding
its review of combination products within 18 months
after the date of enactment of H.R. 6, and to update
that guidance regularly.
Title III--Delivery. CBO estimates that implementing title
III would cost $18 million over the 2016-2020 period, assuming
appropriation of the necessary funds. That amount reflects:
$10 million for contracts with organizations
that develop standards to make recommendations for new
interoperability standards for electronic health
records. (The bill would authorize the appropriation of
$10 million in 2016 for such activities.);
$5 million for the Office of the National
Coordinator of Health Information Technology to
administer the adoption of those interoperability
standards and to publish reports on interoperability;
$2 million for the Centers for Medicare and
Medicaid Services (CMS) and the Medicare Payment
Advisory Commission to provide information to the
Congress on the use and limitations of telehealth
services; and
$1 million for the Secretary of HHS to
establish a Medicare pharmaceutical and technology
ombudsman within CMS.
Title III also contains provisions that would affect direct
spending. Those provisions are discussed in the direct spending
section below.
Title IV--Medicaid, Medicare, and Other Reforms. H.R. 6
would create a fund to pay for new initiatives and
administrative costs associated with regulatory requirements
established by the bill. It would also direct the Department of
Energy (DOE) to sell 64 million barrels of oil from the SPR,
and would expand research activities on Lyme disease and other
tick-borne diseases. CBO estimates that implementing the
provisions in title IV would cost $684 million over the 2016-
2020 period, assuming appropriation of the necessary funds.
(Title IV also contains provisions that would affect direct
spending.)
Cures Innovation Fund. Section 4041 would direct the
Secretary of HHS to establish a ``Cures Innovation Fund'' in
the U.S. Treasury. The legislation would authorize the
appropriation of $110 million a year from the fund for fiscal
years 2016 through 2020. Such authorizations would be in
addition to any amounts made available from other
authorizations of appropriations identified specifically in
titles I and II for the following activities:
Participating in public-private partnerships
and awarding grants that foster the collection,
analysis, and availability of data on the natural
history of disease;
Supporting initiatives of the Council for
21st Century Cures;
Creating a regulatory framework at FDA that
incorporates information about patients' experiences
with a specific condition or disease, including the
risks and benefits of new drug treatments;
Establishing a process to qualify or
validate certain drug development tools, such as
biomarkers, for use in certain applications and
allowing FDA to enter into cooperative agreements and
to award grants to assist the agency with reviewing
such qualification submissions;
Establishing a regulatory framework at FDA
to allow information from clinical experiences to
support the approval or licensure of a new indication
for a drug or biologic, or to fulfill requirements for
post-approval study; and
Administering a new FDA grant program that
promotes the study of continuous drug manufacturing and
other production-related technologies.
CBO estimates that spending from the Cures Innovation Fund
would total $327 million over the 2016-2020 period, assuming
appropriation of the authorized amounts.
SPR Drawdown. The bill would direct the DOE to sell 64
million barrels of oil from the SPR, subject to certain
conditions. Based on information from DOE, CBO estimates that
the transaction costs associated with selling oil from the SPR
would average about 21 cents per barrel. Thus, assuming the
appropriation of the necessary amounts, CBO estimates that
implementing the sales would cost $6 million over the 2016-2020
period. That estimate includes the incremental cost of power,
storage, labor, and various other logistical expenses.
According to DOE, selling a total of 64 million barrels from
the SPR--which would reduce the current inventory by roughly 9
percent--would not require any decommissioning activities or
expenses. (See discussion of the effect of the provision on
mandatory costs in the direct spending section of the cost
estimate.)
Lyme Disease and Other Tick-borne Diseases. Section 4081
would amend the Public Health Service Act to require the
Secretary of HHS to conduct or support research on Lyme disease
and other tick-borne diseases. Currently, several federal
agencies fund research on tick-borne diseases including NIH and
CDC. The bill also would require the Secretary to establish a
permanent interagency working group on Lyme disease and other
tick-borne diseases and to periodically submit to the Congress
a strategic plan for conducting and supporting research in that
area. Based on a 2011 study by the Institute of Medicine that
reported the average annual funding level for Lyme disease and
other tick-borne diseases totaled almost $90 million, CBO
estimates that implementing section 4081 would cost $351
million over the 2016-2020 period.
Direct spending: Several provisions in H.R. 6 would affect
direct spending. Taken together, CBO estimates that enacting
H.R. 6 would reduce on-budget direct spending, on net, by about
$11.9 billion over the 2016-2025 period (see Table 3); off-
budget costs for the U.S. Postal Service would increase by $6
million over the same period.
TABLE 3. ESTIMATED CHANGES IN ON-BUDGET MANDATORY COSTS FOR H.R. 6
--------------------------------------------------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
---------------------------------------------------------------------------------------------------------------------
2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 2016-2020 2016-2025
--------------------------------------------------------------------------------------------------------------------------------------------------------
CHANGES IN ON-BUDGET DIRECT SPENDINGa
Title II--Development
Encouraging the Development and 0 0 49 60 63 66 71 72 73 81 172 535
Use of New Antimicrobial Drugs.
Extension of Exclusivity 0 0 14 47 84 114 142 147 149 172 145 869
Periods for Certain Drugs
Approved for a New Indication
for a Rare Disease or
Condition....................
Subtotal, Title II........ 0 0 63 107 147 180 213 219 222 253 317 1,404
Title III--Delivery
Treatment of Certain Items and 0 -4 -8 -11 -15 -17 -23 -27 -30 -37 -38 -172
Devices......................
Medicare Site-of-service Price 1 5 0 0 0 0 0 0 0 0 6 6
Transparency.................
Programs to Prevent 0 -8 -10 -11 -12 -13 -15 -15 -15 -16 -41 -115
Prescription Drug Abuse under
Medicare Parts C and D.......
Subtotal, Title III....... 1 -7 -18 -22 -27 -30 -38 -42 -45 -53 -73 -281
Title IV--Medicaid, Medicare, and
Other Reforms
Limiting Federal Medicaid 0 0 0 0 -274 -391 -417 -444 -473 -504 -274 -2,503
Reimbursement to States for
DME..........................
Medicare Payment for X-rays 0 -5 -16 -17 -17 -18 -19 -18 -17 -18 -55 -145
and Other Imaging Services...
Delay Certain Payments to 0 0 0 0 -3,184 -301 -4,139 -708 3,786 -417 -3,184 -4,963
Medicare Prescription Drug
Plans........................
SPR Drawdown.................. 0 0 -600 -600 -650 -650 -700 -700 -750 -750 -1,850 -5,400
Subtotal, Title IV........ 0 -5 -616 -617 -4,125 -1,360 -5,275 -1,870 2,546 -1,689 -5,363 -13,011
Total, Changes in On- -11 -12 -571 -532 -4,005 -1,210 -5,100 -1,693 2,723 -1,489 -5,119 11,888
budget Direct Spendingb..
--------------------------------------------------------------------------------------------------------------------------------------------------------
Notes: DME = durable medical equipment; SPR = Strategic Petroleum Reserve.
Numbers may not add up to totals because of rounding.
a Budget authority equals outlays for all direct spending provisions. Medicare provisions include interactions with Medicare Advantage payments, the
effect on Medicare Part A and Part B premiums, and TRICARE.
b CBO estimates that enacting H.R. 6 also would increase off-budget costs for the U.S. Postal Service by $6 million over the 2016-2025 period.
Title II--Development. Title II of the bill would require
Medicare to make additional payments to hospitals for using
qualifying antimicrobial drugs. It also would extend
exclusivity periods for certain drugs approved for a new
indication that pertains to treating a rare disease or
condition. CBO estimates that implementing such provisions
would increase on-budget direct spending for mandatory health
programs by $1.4 billion over the 2016-2025 period.\2\
---------------------------------------------------------------------------
\2\Extending exclusivity periods for certain drugs would affect
off-budget spending by the U.S. Postal Service (USPS) for health
insurance premiums for its workers and retirees who are covered under
the Federal Employees Health Benefits program. CBO estimates that the
bill would increase USPS costs (which are classified as off-budget) by
$6 million over the 2016-2025 period.
---------------------------------------------------------------------------
Encouraging the Development and Use of New Antimicrobial
Drugs. The bill would require Medicare to make an additional
payment to hospitals when Medicare beneficiaries use certain
new antimicrobial drugs during the course of their hospital
stay. To qualify for the additional payment, a drug would have
to meet certain criteria, including: the drug must be approved
to treat certain infections for which existing antibiotics are
not sufficient and the drug must receive approval for use or
for a new indication on or after December 1, 2014. A qualifying
drug would be eligible for the additional payment for a period
of five years. Each year, the Secretary would establish payment
rates for eligible drugs based on payment rates under Part B of
Medicare, subject to the pro rata reduction, if any, that the
Secretary estimates is needed to limit total payments for such
drugs to 0.03 percent of expected Medicare spending for
hospital inpatient services. Based on information about drugs
currently in the development pipeline that would be likely to
satisfy the specified criteria and data from the CDC on rates
of antibiotic-resistant infection, CBO estimates that enacting
that provision would increase direct spending by about $535
million over the 2016-2025 period.
Extension of Exclusivity Periods for Certain Drugs Approved
for a New Indication for a Rare Disease or Condition. Section
2151 of the bill would authorize the FDA to extend exclusivity
periods for certain brand-name drugs already on the market by
six months if, after enactment of the bill, the drug is
approved for a new indication that pertains to treating a rare
disease or condition. Such extensions could delay the timing of
market entry by lower-priced generic drugs or biosimilars. In
addition, the provision would add six months of exclusivity to
the patents of select drugs; such provision only would apply to
certain designated drugs previously approved under the Federal
Food, Drug, and Cosmetic Act (FDCA) and not to biologics
previously licensed under the Public Health Service Act.
In order to be eligible for the six month exclusivity
period, a drug manufacturer would have to demonstrate safety
and efficacy for treatment of a rare disease or condition for
which the drug had not been previously approved. CBO expects
that approval for the new indication would hinge on
successfully completing new clinical trials. While many
manufacturers could benefit over the next 10 years from such an
extension of exclusivity, CBO expects that only certain drugs
that meet all of the following criteria likely would receive
one. First, a drug must have the potential to treat a rare
disease or condition for which it was not originally approved.
Second, the expected value of returns from undertaking the
additional research to obtain approval for the new indication
must offset the costs. And finally, sufficient time must be
available for the manufacturer to conduct the necessary trials,
prepare a marketing application, undergo regulatory review, and
obtain approval before facing generic competition.
CBO estimates that about 15 percent of the share of brand-
name sales for drugs previously approved under the FDCA that
are expected to first experience generic competition before
2025 would have such competition delayed by 6 months under this
provision. By delaying the timing of market entry of lower
priced generics or biosimilars, CBO expects the provision would
increase the drug-related costs of federal health programs
(both mandatory and discretionary programs) that pay for
prescription drugs and biological products. CBO estimates that
the provision would increase on-budget spending on prescription
drugs by mandatory health programs by $869 million over the
2016-2025 period. Beyond 2025, the potential for the
legislation to delay the entry of generic drugs or biosimilars
is greater and the federal budgetary effect would increase in
later years.
Title III--Delivery. Title III of the bill also contains
provisions that would affect direct spending for Medicare. CBO
estimates that enacting those provisions would reduce direct
spending, on net, by $281 million over the 2016-2025 period.
Treatment of Certain Items and Devices. Under current law,
Medicare beneficiaries may receive negative pressure wound
therapy (NPWT), which uses a vacuum pump and special dressings
to promote wound healing. NPWTs are available using either a
durable pump or a disposable pump that can be used at home. If
a home health agency (HHA) furnishes a beneficiary with NPWT
using a durable form of the device, Medicare makes a payment to
the HHA for the visit and to a DME supplier for the NPWT. If
the HHA uses a disposable NPWT, Medicare does not make an
additional payment and the HHA absorbs the cost of the NPWT.
H.R. 6 would establish a new add-on payment to HHAs when they
furnish disposable NPWT; that payment would be lower than the
payment for durable NPWT. CBO estimates that there would be
some switching from durable NPWT to disposable NPWT and thus
this provision would save about $172 million over the 2016-2025
period.
Medicare Site-of-service Price Transparency. H.R. 6 would
require CMS to create a new database and website that would
enable beneficiaries to compare the estimated Medicare payment
and cost-sharing amounts for items and services provided in
hospital outpatient departments and ambulatory surgical
centers. The bill would appropriate $6 million for this purpose
and CBO estimates that all of the funding would be spent by the
end of fiscal year 2017.
Programs to Prevent Prescription Drug Abuse under Medicare
Parts C and D. The bill would permit private drug plans that
administer the Medicare Part D prescription drug benefit to
establish a program to limit the number of physicians and
pharmacies allowed to prescribe and dispense certain drugs to
enrollees identified as being at high risk for prescription
drug abuse. Under H.R. 6, plans that implement such a program
would use clinical guidelines established by the Secretary of
HHS to target certain beneficiaries who use controlled
substances the Secretary determines are frequently abused or
diverted. For example, restrictions might be placed on
beneficiaries suspected of abusing or reselling certain
controlled substances, but not placed on beneficiaries with
cancer or other conditions for which those drugs are considered
appropriate. Based on information from HHS and other
stakeholders, CBO estimates that enacting that provision would
reduce direct spending by $115 million over the 2016-2025
period.
Title IV--Medicaid, Medicare, and Other Reforms. Title IV
of the bill contains provisions that would reduce direct
spending. CBO estimates that enacting those provisions would
reduce direct spending by $13.0 billion over the 2016-2025
period.
Limiting Federal Medicaid Reimbursement to States for DME.
Under current law, states have broad flexibility to set
coverage and payment policies in their Medicaid programs.
Generally, the federal government reimburses states for a
portion of the amount they spend on Medicaid. Section 4001
would limit the amount of spending by states to purchase DME
that is eligible for federal reimbursement to the amount that
would be paid by the Medicare program. Twelve states have
adopted similar policies as of 2014. CBO estimates that
enacting DME payment limits in the remaining states beginning
January 2020 would reduce direct spending for Medicaid by
approximately $2.5 billion over the 2016-2025 period.
Medicare Payments for X-rays and Other Imaging Services.
The legislation would reduce Medicare's payment rates under the
physician fee schedule for x-ray and other imaging services
that do not use digital imaging technology, beginning in 2017.
Payment rates for imaging services that use film would be
reduced by 20 percent. The reduction for imaging services that
use computed radiography would be 7 percent in 2018 through
2022, then 10 percent in 2023 and subsequent years. Based on a
review of Medicare claims, CBO estimates that about 1 percent
of current spending for imaging services paid under the
physician fee schedule would be subject to the reductions in
2017. CBO expects that implementation of the payment reductions
would spur adoption of digital technology, and that less than
0.2 percent of spending would be subject to those reductions by
2025. This provision would reduce direct spending about $145
million over the 2016-2025 period, CBO estimates.
Delay Certain Payments to Medicare Prescription Drug Plans.
Under current law, most Medicare payments to Part D plans
(including capitated payments and reinsurance payments for
beneficiaries whose spending exceeds the threshold for the
catastrophic portion of the prescription drug benefit) are made
on either the first day of the month or the last day of the
preceding month (when the first day is a weekend or holiday).
Beginning in calendar year 2020, the legislation would delay
monthly reinsurance payments to stand-alone prescription drug
plans in Medicare Part D. Starting with a payment shift from
2020 to 2021, the provision would shift spending between fiscal
years and would shift an estimated $5.0 billion from fiscal
year 2025 to fiscal year 2026.\3\
---------------------------------------------------------------------------
\3\For example, $3.2 billion would be shifted from fiscal year 2020
to 2021, thereby reducing direct spending in 2020 by that amount. For
2021, that $3.2 billion would be shifted into 2021, but $3.5 billion
would be shifted into 2022. As a result, the net change in spending in
2021 would amount to $0.3 billion. By 2025, $4.5 billion would be
shifted in (from 2024) and almost $5.0 billion would be shifted out (to
2026); the net effect in 2025 would amount to 0.4 billion.
---------------------------------------------------------------------------
SPR Drawdown. Section 4061 would direct the DOE to sell 8
million barrels of oil from the SPR in each of the fiscal years
2018 through 2025, subject to certain conditions. Under this
bill, the proceeds from such sales would be deposited in the
general fund of the Treasury by the end of each fiscal year and
could not be spent to purchase oil for the reserve. CBO
estimates that enacting that provision would increase
offsetting receipts (which are certain collections that are
treated as reductions in direct spending) by $5.4 billion over
the 2016-2025 period.
The estimated receipts reflect CBO's March 2015 projection
of oil prices, adjusted for the technical characteristics of
the oil being sold from the SPR (those adjusted prices range
from about $75 to $96 per barrel over the sales period). Based
on information from the Energy Information Administration, CBO
estimates that the volume of crude oil in the SPR after these
sales would exceed an amount equivalent to a 90-day supply of
net imports crude oil and petroleum products, as required by
the bill.
Pay-As-You-Go considerations: The Statutory Pay-As-You-Go
Act of 2010 establishes budget-reporting and enforcement
procedures for legislation affecting direct spending or
revenues. The net changes in outlays that are subject to those
pay-as-you-go procedures are shown in the table below. Only on-
budget changes to outlays are subject to pay-as-you-go rules.
Enacting H.R. 6 would not affect revenues.
CBO ESTIMATE OF PAY-AS-YOU-GO EFFECTS FOR H.R. 6, AS ORDERED REPORTED BY THE HOUSE COMMITTEE ON ENERGY AND COMMERCE ON MAY 21, 2015
--------------------------------------------------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
------------------------------------------------------------------------------------------------------------------------
2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 2015-2020 2015-2025
--------------------------------------------------------------------------------------------------------------------------------------------------------
NET DECREASE IN THE ON-BUDGET DEFICIT
Total Changes.................. 0 1 -12 -571 -532 -4,005 -1,210 -5,100 -1,693 2,723 -1,489 -5,119 -11,888
Less:
Adjustment for Timing Shifta... 0 0 0 0 0 0 0 0 0 0 -4,963 0 -4,963
Statutory Pay-As-You-Go Impact. 0 1 -12 -571 -532 -4,005 -1,210 -5,100 -1,693 2,723 3,474 -5,119 -6,925
--------------------------------------------------------------------------------------------------------------------------------------------------------
Source: Congressional Budget Office.
aSection 4 of the Statutory Pay-As-You-Go Act of 2010 provides for adjustments related to certain shifts in the timing of spending or revenues. The
provision in H.R. 6 that would delay certain payments to Medicare prescription drug plans would create such a timing shift. That provision would shift
payments in each year beginning with 2020. The adjustment for timing shifts under pay-as-you-go procedures is only applied to the spending that is
shifted from 2025 to 2026.
Increase in long term direct spending: None.
Estimated impact on state, local, and tribal governments:
H.R. 6 contains no intergovernmental mandates as defined in
UMRA. The bill would delay entry into the market of some
generic drugs resulting in an increase of state Medicaid
spending for prescription drugs of $50 million over the 2016-
2025 period. In addition, the bill would limit the amount that
is eligible for federal matching payments to states for DME in
Medicaid to the amount that Medicare would pay. That limitation
could increase state Medicaid costs by about $2.5 billion over
the 2016-2025 period. However, because states have flexibility
in Medicaid to adjust their financial and programmatic
responsibilities, including the ability to reduce the amounts
they would pay vendors for DME, those costs would not result
from an intergovernmental mandate.
Estimated impact to the private sector: H.R. 6 would impose
private-sector mandates, as defined in UMRA, on drug
manufacturers. CBO estimates that the aggregate cost of the
mandates would fall below the annual threshold established in
UMRA ($154 million in 2015, adjusted annually for inflation) in
each of the first five years that the mandates are in effect.
The bill would impose a mandate on manufacturers of generic
drugs and biosimilars by extending by six months the periods of
marketing exclusivity for products that receive a new
indication for the treatment of a rare disease. Granting drugs
additional marketing exclusivity would delay the entry of
lower-priced versions of products in those markets. The cost of
the mandate for manufacturers of generic products and
biosimilars would be the annual net loss of income resulting
from the delay, which could be significant depending on the
drugs granted an extension. However, based on information about
the sales of drugs that could be affected in the first five
years that the mandate is in effect, CBO estimates that the
cost of the mandate would amount to about $50 million or less
in each of those years.
The bill would impose two additional mandates, and CBO
estimates that the cost of each of those mandates would be
small. The bill would require manufacturers of investigational
drugs to make public their policy for reviewing and responding
to requests for access to those drugs under compassionate use
policies. The bill would allow manufacturers to comply with the
mandate by posting a general policy applicable to all its
investigational drugs. The bill also would require
manufacturers of antimicrobial drugs to submit an application
to FDA for changes to the product's label sooner than they
would need to under current law. The bill would require that
labels for all antimicrobial drugs include a reference to an
FDA website which would contain the updated criteria for
determining the effectiveness of such drugs. The mandate would
result in savings to the affected manufacturers in later years
because they would not need to change a product's label each
time those criteria are updated.
Estimate prepared by: Federal Costs: National Institutes of
Health--Ellen Werble; Food and Drug Administration--Julia
Christensen, Anna Cook, and Ellen Werble; Centers for Disease
Control and Prevention--Rebecca Yip; Health Information
Technology--Zoe Williams; Medicaid--Daniel Hoople; Medicare--
Lori Housman, Kevin McNellis, Jamease Miles, Andrea Noda, Lara
Robillard, and Rebecca Yip; Strategic Petroleum Reserve--Kathy
Gramp. Impact on State, Local, and Tribal Governments: J'nell
Blanco Suchy. Impact on the Private Sector: Amy Petz.
Estimate approved by: Holly Harvey, Deputy Assistant
Director for Budget Analysis.
Federal Mandates Statement
The Committee adopts as its own the estimate of Federal
mandates prepared by the Director of the Congressional Budget
Office pursuant to section 423 of the Unfunded Mandates Reform
Act.
Duplication of Federal Programs
No provision of H.R. 6 establishes or reauthorizes a
program of the Federal Government known to be duplicative of
another Federal program, a program that was included in any
report from the Government Accountability Office to Congress
pursuant to section 21 of Public Law 111-139, or a program
related to a program identified in the most recent Catalog of
Federal Domestic Assistance.
Disclosure of Directed Rule Makings
The Committee estimates that enacting H.R. 6 specifically
directs to be completed 3 rule makings within the meaning of 5
U.S.C. 551.
Advisory Committee Statement
No advisory committees within the meaning of section 5(b)
of the Federal Advisory Committee Act were created by this
legislation.
Applicability to Legislative Branch
The Committee finds that the legislation does not relate to
the terms and conditions of employment or access to public
services or accommodations within the meaning of section
102(b)(3) of the Congressional Accountability Act.
Section-by-Section Analysis of the Legislation
TITLE I--DISCOVERY
Section 1001. National Institutes of Health reauthorization
Section 1001 would reauthorize the National Institutes of
Health (NIH) for three years: Fiscal Year (FY) 2016,
$31,811,000,000; FY 2017, $33,331,000,000; and FY 2018,
$34,851,000,000. The NIH authorization expired at the end of FY
2009.
Section 1002. NIH Innovation Fund
This section would establish an innovation fund at NIH for
five years, FY 2016 through FY 2020, to support biomedical
research through the funding of basic, translation, and
clinical research. The Innovation Fund is meant to spur
scientific innovation and discovery by providing an additional,
supplementary funding stream to NIH. These funds will go toward
high-risk high reward research and research performed by early
stage investigators, among other initiatives. This section
establishes an Accelerating Advancement Program at NIH that
would allow for dollar for dollar matching from the Office of
the Director for institutes and centers seeking funding from
the Innovation Fund.
NIH also is encouraged to support research into effective
treatments for areas of unmet medical needs. These areas
include, but are not limited to, biomarkers, precision
medicine, infectious diseases, and antibiotics. The infectious
diseases studied should include the pathogens listed as
qualifying pathogens by the FDA: Acinetobacter species,
Aspergillus species, Burkholderia cepacia complex,
Campylobacter species, Candida species, Clostridium difficile,
Enterobacteriaceae, Enterococcus species, Mycobacterium
tuberculosis complex, Neisseria gonorrhoeae, Neisseria
meningitidis, Non-tuberculous mycobacteria species, Pseudomonas
species, Staphylococcus aureus, Streptococcus agalactiae,
Streptococcus pneumoniae, Streptococcus pyogenes, Vibrio
cholera, Coccidioides species, Crytococcus species, and
Helicobacter pylori. These also should include those pathogens
listed by FDA in the future.
The Committee also recognizes that several tropical
diseases have been identified in the United States over the
last decade, including West Nile virus, Chagas disease,
cysticerosis, and toxocariasis. While NIH currently supports
tropical medicine research centers located in endemic areas
outside the U.S., similar support is necessary in parts of the
U.S. that are emerging as highly endemic as well, such as the
Gulf Coast and Southwest regions. Considering the urgent need
for diagnostics and vaccines, the Committee expects NIH to
consider tropical diseases endemic to the U.S. to be ``unmet
medical needs in the United States'' for purposes of
identifying strategic focus areas for the Innovation Fund, in
addition to the tropical diseases listed as eligible for the
priority review voucher program established in the Food and
Drug Administration Amendments Act of 2007.
Section 1021. NIH research strategic plan
Section 1021 directs NIH to create a scientifically based
five-year strategic plan to support research priorities
determined by NIH. The plan would be used by the Director of
NIH to identify research opportunities and strategic focus
areas in which the resources of the national research
institutes and national centers can best contribute to the goal
of expanding knowledge of human health in the U.S. The plan
also would contain overarching and trans-NIH Mission Priority
Focus Areas that have the goal of preventing or eliminating the
burden of a disease or condition. NIH would have to update the
plan every five years. The strategic plan also would ensure
that NIH prioritizes maintaining the biomedical workforce of
the future.
Currently, individual institutes and centers compile their
own strategic plans. NIH as a whole does not have an
overarching strategic plan to inform decisions on where to
focus research efforts. At this time, no plan shows how inter-
institute research is occurring or coordinated. With increased
funding, taxpayers should understand the overarching vision of
NIH and the priorities it is setting.
Section 1022. Increasing accountability at the National Institutes of
Health
This section would provide the Director of NIH with
increased authority and address duplication in Federal
biomedical research. Under this section, the Director of NIH
would have the authority to appoint directors of the national
research institutes, excluding the Director of the National
Cancer Institute, as well as the directors of the national
centers for five-year terms. There would be no limit on the
number of terms a director may serve. If the office of a
director of a national research institute or national center is
vacant before the end of a five-year term, the Director of NIH
could appoint a new director for a five year term starting at
the date of appointment. Currently, the Director of NIH makes
recommendations to the Secretary of Health and Human Services
(HHS), and the Secretary makes the final decision on the
appointment of the directors.
Section 1022 also would increase accountability by
including a review of all ``R-series'' grants by directors of
the national research institutes or national centers. Directors
would have to consider the NIH strategic plan when awarding
these grants and whether other agencies are funding programs or
projects to accomplish the same goal.
This section would instruct the Institute of Medicine (IOM)
to conduct a report on duplication in Federal biomedical
research.
This section, in conjunction with the strategic plan, is
intended to increase accountability and ownership of decision-
making and prioritization at NIH.
Section 1023. Reducing administrative burdens of researchers
This section would help reduce the administrative burden on
researchers funded by NIH by implementing findings from various
sources. The Scientific Management Review Board, the National
Academy of Sciences, and the 2007 and 2012 Faculty Burden
Surveys conducted by the Federal Demonstration Partnership have
examined this issue extensively. Administrative burden takes
time away from researchers focusing on their scientific
pursuits.
Section 1024. Exemption for the National Institutes of Health from the
Paper Work Reduction Act requirements
This section would exempt the NIH from the Paperwork
Reduction Act (PRA) requirements surrounding proposed
information collection. Currently, two rounds of public comment
and Office of Management and Budget (OMB) approval are required
for each proposed information collection. The complete review
and approval process takes between six and nine months. The PRA
was designed to maximize the utility of information collected
by the Federal government. However, this review is burdensome
on NIH investigators and delays important research efforts.
Section 1025. NIH travel
This section would set forth a sense of Congress to
reiterate the importance of scientific conferences and meetings
to the mission of NIH.
Section 1026. Other transactions authority
This section would provide the National Center for
Advancing Translational Science (NCATS) at NIH with more
flexibility on the use of Other Transaction Authority (OTA) so
that it can operate like the Defense Advanced Research Projects
Agency (DARPA).
Section 1027. NCATS phase IIB restriction
This section would remove a restriction NCATS' conduct of,
or grants for, phase II and III clinical trials.
Section 1028. High-risk, high-reward research
This section would support research that pursues innovative
approaches to major challenges in biomedical research that
involve inherent high risk, but has the potential to lead to
breakthroughs.
Robert Lefkowitz, the 2012 Nobel Prize winner for Chemistry
said:
There's a current problem in biomedical research. The
emphasis is on doing things which are not risky. To
have a grant proposal funded, you have to propose
something and then present what is called preliminary
data, which is basically evidence that you've already
done what you're proposing to do. If there's any risk
involved, then your proposal won't be funded. So the
entire system tends to encourage not particularly
creative research, relatively descriptive and
incremental changes which are incremental advances
which you are certain to make but not change things
very much.
According to the most recent budget congressional
justification, NIH states:
Since the High-Risk High-Reward program tests new
ways of supporting innovation, NIH commissioned a
rigorous external evaluation of the most mature of
these initiatives, the Pioneer Awards. Comparison of
research from Pioneer Awards, R01s (NIH's most common
project-based grant mechanism), and research funded by
the Howard Hughes Medical Institute (HHMI) showed that
the Pioneer program has been successful in attracting
and supporting research that is more innovative and has
greater impact than R01s, and it is comparable to HHMI-
supported research.
The language would build on actions already occurring at
NIH and require each institute, as appropriate, to fund high
risk, high impact science. The NIH Director would establish the
percentage of funding an individual institute will be required
to spend in this area. Institute Directors in collaboration
with the NIH Director would establish programs to accomplish
this goal. This language does not state what funding mechanism
must be used to accomplish this goal. While NIH's Pioneer and
Innovator Awards would be logical types of programs, this
section would provide NIH with flexibility to accomplish the
goal. The NIH Director would be given the authority to
establish the percentage of funding because not all Institute
Directors have embraced funding this type of research.
Section 1029. Sense of Congress on increased inclusion of
underrepresented communities in clinical trials
Over the past twenty-five years, there have been national
efforts in the U.S. to increase the representation of
minorities in clinical trials. Although current participation
rates do not fully represent the overall population of
minorities in the U.S., some progress has been made. This
section would set forth a sense of Congress to reiterate the
importance of increasing the representation of underrepresented
communities in clinical trials. This would help ensure that
adequate information is available to assess the safety and
effectiveness of drugs in all populations.
Section 1041. Improvement of loan repayment programs of National
Institutes of Health
The NIH loan repayment programs help attract and retain
early career health professionals by assisting in their
repayment of debt associated with medical education and
training. NIH wants to encourage outstanding health
professionals to pursue careers in biomedical, behavioral,
social and clinical research. Since 1998, the NIH Loan
Repayment Programs have encouraged promising researchers and
scientists to pursue research careers by repaying up to $35,000
of their qualified student loan debt each year if they commit
at least two years to conducting qualified research.
Participation in the NIH Loan Repayment Programs has had a
major impact on the careers of thousands of research
scientists. Participants in the NIH Loan Repayment Programs
stay in research longer, apply for and receive more research
grants, and become independent investigators more frequently
than their colleagues who do not receive loan repayment program
funding.
This section would increase the loan repayment cap from
$35,000 to $50,000 for the loan repayment programs administered
at NIH.
Section 1042. Report
The bulk of the money received by NIH goes to researchers
who are esteemed in their fields--but there are concerns that
this may limit funding for early or mid-career scientists who
may have innovative and potentially transformational ideas. A
study for the National Bureau of Economic Research from 2005
examined the age at which over 2,000 Nobel Prize winners and
other notable scientists in the 20th century came up with the
idea that led to their breakthrough. Most were between thirty-
five and thirty-nine. Yet the median age of first-time
recipients of R01 grants, the most common and sought-after form
of NIH funding, is forty-two, while the median age of all
recipients is fifty-two. More people over sixty-five are funded
with research grants than those under age thirty-five.
The most innovative thinking frequently comes from younger
scientists. NIH is aware of the disparity; NIH Director Francis
S. Collins has spoken out about not investing in young
scientists and taken steps to target younger researchers. As a
result, the average age of first-time grant recipients has
stopped rising, but the problem still exists, and NIH can do
more.
The language in this section would require NIH to submit a
report to Congress within eighteen months of enactment on the
actions taken by NIH to attract, retain, and develop emerging
scientists.
Section 1061. Capstone Award
The reasons for the age increase of NIH grantees are
diverse and complicated. There are not enough faculty positions
for the number of graduates with PhDs. When mandatory
retirement at age 65 was removed from academia, the number of
scientists remaining on the faculty after age sixty-five and
even after age seventy dramatically increased. Since so few
openings in academia become available, individuals often pursue
multiple postdoctoral fellowships. This increases the age at
which they start applying for R01 or R01-equivalent grants
higher and higher. The current structure actually encourages
individuals to get multiple postdoctoral fellowships. Another
reason academic institutions encourage multiple postdoctoral
fellowships is because postdoctoral fellows cost academic
centers and labs less.
The language would create a new ``Capstone Award'' at NIH.
The award could be used to facilitate the transition or
conclusion of research programs for senior investigators. This
award would allow researchers a new funding mechanism should
senior researchers choose to transition their research to
pursue other opportunities. Once a researcher receives a
capstone award, he or she may not receive another grant from
NIH. The Howard Hughes Medical Institute created a similar
program, which has seen significant success.
Section 1081. National Pediatric Research Network
The National Pediatric Research Network Act of 2013 passed
as part of the PREEMIE Reauthorization Act and became law on
November 27, 2013.
The bill amended the Public Health Service Act to authorize
the Director of NIH to establish a National Pediatric Research
Network.
It authorized the Director of NIH, in part, to make awards
for not more than twenty pediatric research consortia,
disseminate scientific findings, and meet requirements
prescribed by the Director.
This provision would amend the law to require NIH to
establish new pediatric research consortia, and clarify that
the Office of the Director can work with any other research
institutes or centers to implement the National Pediatric
Research Network Act.
Section 1082. Global pediatric clinical study network sense of Congress
Clinical trials are one of the most challenging, resource
intensive, and time consuming components of medical product
development. Clinical trials for pediatric products prove even
more challenging due to limited populations and a limited
infrastructure. According to the discussion paper, Developing a
Clinical Trials Infrastructure, by Paul Eisenberg, Amgen, Inc.;
Petra Kaufmann, National Institute of Neurological Disorders
and Stroke; Ellen Sigal, Friends of Cancer Research; and Janet
Woodcock, U.S. Food and Drug Administration, ``clinical trials
in the United States have become too expensive, difficult to
enroll, inefficient to implement, and ineffective to support
the development of new medical products using modern
evidentiary standards.'' The cost and time associated with
clinical trials are symptoms of dysfunction within the clinical
trial system and infrastructure. Establishing a pediatric
clinical study network would create a mechanism to facilitate
clinical trials for pediatrics in a more efficient and cost
effective way.
This sense of Congress encourages NIH and FDA to work with
external stakeholders to build on the success of the Enro-EMA
pediatric network to establish a larger and stronger
infrastructure to facilitate more pediatric research in order
to increase the number of medical products for pediatric
populations.
Section 1083. Appropriate age groupings in clinical research
This section would ensure appropriate age groups are
included in research studies involving human subjects.
Section 1101. Sharing of data generated through NIH-funded research
There is an increasing expectation from the scientific
community and the public that data generated with Federal
funds, particularly scientific data, should be shared to
enhance transparency in Federal research spending, improve the
return on investment in research, and help enhance the quality
of research. In particular, the President has directed Federal
agencies to ensure that peer-
reviewed publications and digital scientific data resulting
from Federally funded scientific research are accessible to the
public, the scientific community, and industry.
NIH has long-standing policies that expect data to be
shared, such as the 2003 Data Sharing Policy and the 2014
Genomic Data Sharing Policy, as well as specific program
policies in place at NIH Institutes and Centers. The NIH
Director has explicit statutory authority (section 217 of the
HHS Omnibus Appropriations Act of 2009) to require NIH-funded
investigators to make their final, peer-reviewed manuscripts
publicly available through submission to PubMed Central.
NIH promotes data sharing among awardees in a number of
ways. For example, consistent with the Final NIH Statement on
Sharing Research Data (Data Sharing Policy) issued in 2003, NIH
generally expects applicants to address data sharing in their
applications. Through the applicants' submission of data
sharing plans, which are incorporated into the terms and
conditions of grant awards, sharing consistent with those plans
becomes a requirement.
This section would make explicit in statute that the
Director has the authority to require researchers to share data
that results from NIH funded research.
Section 1102. Standardization of data in clinical trial registry data
bank on eligibility for clinical trials
This section would enhance patient searches for ongoing
trials by requiring NIH to standardize certain patient
inclusion and exclusion information across all trials housed in
ClinicalTrials.gov.
The Committee recognizes the important role
ClinicalTrials.gov can and does play in helping to facilitate
patient matching with ongoing clinical trials. While the
current database does afford opportunities for patients and
providers to identify such trials, the process is cumbersome
and can be difficult to navigate.
During hearings that preceded the legislative process of
21st Century Cures, various patient advocates and other
stakeholders noted that increased standardization of data
elements across the various trials would help ease the time and
effort required to search the database. Such standardization
would serve as a foundation upon which health information
technology developers might develop applications that
facilitate searches from a physician's office through platforms
such as electronic health records. Such innovation would help
support patients in search of treatment who have run out of
FDA-approved treatment options.
The Committee agrees that increased standardization would
be of immense benefit to those seeking to search
ClinicalTrials.gov, but recognizes that too much
standardization could result in valuable data being excluded.
Section 1121. Clinical trial data system
This section would create a pilot third-party scientific
research sharing system for trials solely funded by the
agencies of HHS in order to allow the use and analysis of de-
identified data beyond each individual research project.
Currently, the U.S. is the world leader in medical and
scientific research. The Committee commends NIH for its
leadership and as supports increased funding through the 21st
Century Cures legislation. However, the Committee recognizes
that the data generated from such research can play a role in
the development of future cures well beyond the conclusion of
the specific project being funded.
The NIH has taken steps to increase access to participant-
level data from clinical trials. For example, a National
Institute of Allergy and Infectious Diseases program provided
de-identified participant level information from a network of
clinical trials studying autoimmune disorders. Additionally,
the National Institute of Mental Health is establishing a
common informatics platform for exchanging data from clinical
trials of novel interventions for the treatment of mental
illness. The creation of the clinical trial data system would
build upon those efforts.
The creation of a database designed to store data
originating from HHS funded research projects for use and
analysis beyond the initial research project holds much promise
for the future research. Consider the implications of granting
researchers access to de-identified, patient-specific clinical
trial data in order to identify ways to improve subsequent
trials and reduce the potential for bad outcomes, such as
adverse events or even death. Or, consider the potential that
research projects in one area could help inform successful
solutions in others. The Committee is also cognizant of the
paramount importance of protecting patient privacy and
confidential commercial information and is confident that these
benefits can be obtained while simultaneously protecting these
important concerns.
The Committee finds that the creation of a clinical trial
database for the purposes of sharing HHS funded data with
researchers to support the development of new cures for
patients in need allows the Federal government to maximize the
benefit of such data and support our global authority in the
area of new medical product development.
Section 1122. National neurological diseases surveillance system
Currently, there is limited infrastructure to advance data
collection regarding neurological diseases. This lack of data
has hindered research and understanding of neurological
diseases and new disease targets.
Section 1122 would require the Centers for Disease Control
and Prevention (CDC) to set up a surveillance system for
neurological diseases like Parkinson's and MS. Other diseases
could include Alzheimer's disease among others. The
neurological disease surveillance system would enhance and
expand the infrastructure and activities to track the
epidemiology of neurological diseases. This system will help
foster the collection of information on neurological diseases,
such as demographics, risk factors, diagnosis, progression
markers, and other relevant information.
This provision would authorize $5 million for FY 2016 to FY
2020 for the activities authorized in section 1122. Recognizing
the scope of this program will be governed by the availability
of resources, the CDC should include multiple sclerosis and
Parkinson's disease, and others as practicable.
Section 1123. Data on natural history of diseases
Section 1123 would add section 229A to Part A of title II
of the Public Health Service Act, authorizing the Secretary to
participate in public-private partnerships and award grants to
patient advocacy organizations or other organizations to
establish or facilitate the collection, maintenance, analysis,
and interpretation of data regarding the natural history of
diseases, with a particular focus on rare diseases. Such data
would be made publicly available to help facilitate and
expedite medical product development programs.
Section 1124. Accessing, sharing, and using health data for research
purposes
Section 1124 would make several clarifications and
revisions to the regulations implementing the Health Insurance
Portability and Accountability Act (HIPAA).
First, it would permit, but not require, covered entities
to use protected health information for research purposes by
including research within the definition of health care
operations. The Committee intends for the covered entity to be
able to elect for an activity meeting the requirements of this
section to constitute health care operations, for the purposes
of compliance with HIPAA, even if it constitutes research under
other applicable law. The Committee also intends that this bill
creates no changes with respect to the handling of information
protected under the Uniform Trade Secrets Act or other
commercial or financial information that is privileged or
confidential.
Further, section 1124 would permit covered entities to
provide access to data for research purposes in the same
controlled ways as they currently do for public health
activities. In addition, it would permit remote access to
protected health information maintained by a covered entity or
by a business associate and/or cloud provider on behalf of a
covered entity. However, a researcher authorized to access such
information may not copy or otherwise retain the protected
health information after the approved activities are concluded.
The covered entity and the researcher must maintain appropriate
privacy and security safeguards.
Finally, section 1124 would clarify the permissibility of
authorization for future research use and disclosure of an
individual's protected health information under controlled
conditions
Section 1141. Council for 21st Century Cures
This section would establish a public-private partnership
in the U.S. to accelerate the discovery, development, and
delivery of innovative cures, treatments, and preventive
measures for patients. This public-private partnership would
bring together regulators, academia, patients, providers and
others to review and recommend improvements to help accelerate
cures and treatments.
TITLE II--DEVELOPMENT
Section 2001. Development and use of patient experience data to enhance
structured risk-benefit framework
Section 2001 would amend section 505 of the Federal Food,
Drug and Cosmetic Act (FFDCA), requiring the Secretary, in the
context of the new drug review process, to implement a
structured framework to facilitate the incorporation of patient
experiences in the consideration of a drug's benefits and
risks. Within two years of the date of enactment of this bill,
the Secretary would be required to establish processes under
which an entity seeking to develop patient experience data,
such as data relating to the impact of a disease or a therapy
on patients' lives could submit research concepts, proposed
guidance documents, completed data, and summaries and analyses
of such data, among other things. Further, within three years
of enactment and after convening workshops to obtain input, the
Secretary would be required to publish draft guidance on, among
other things, methodological considerations and approaches for
the collection of patient experience data, including patient-
reported outcomes. Final guidance would be published after a
public meeting is convened and not later than one year after
the comment period for the draft guidance closes.
Section 2021. Qualification of drug development tools
The Committee believes that collaboratively improving the
development and qualification of drug development tools,
including biomarkers, is critical and that biomedical research
consortia and other outside entities can play an important role
throughout the process.
Section 2021 would add section 507 to the FFDCA, codifying
a structured framework for the submission, review, and
qualification of biomarkers and other drug development tools
for specific contexts of use that, if qualified, can be relied
on by any person for such purposes. The Secretary may consult
with outside experts and consider their recommendations
throughout the review of a qualification package submitted
under this framework.
In addition, section 2021 would require the Secretary to
make public information related to each submission, all drug
development tools qualified, and all surrogate endpoints that
were the basis of approval or licensure of a drug or biological
product, while maintaining the protections currently in place
for confidential commercial or trade secret information
contained in an application submitted outside this
legislation's public qualification process and under section
505 of the FFDCA or section 351 of the Public Health Service
Act.
The Secretary, in consultation with biomedical research
consortia and other interested parties, through a collaborative
public process, is required under section 2021 to issue draft
guidance on the implementation of section 507 no later than two
years after enactment. The development of this guidance is to
be informed by the Secretary's publication of a taxonomy for
the classification of biomarkers, which is required to be
published in draft form no later than one year after enactment.
Section 2022. Accelerated approval development plan
Section 2022 would amend section 506 of the FFDCA to enable
the sponsor of a drug that the Secretary determines may be
eligible for accelerated approval (i.e., a drug that the
Secretary determines could meet all of the statutory and
regulatory criteria for approval pursuant section 506(c)) to
request voluntarily that the Secretary agree to an accelerated
approval development plan, including the surrogate endpoint to
be assessed; the design of the study that will utilize the
surrogate endpoint; and the magnitude of the effect of the
surrogate endpoint that would be sufficient to form the primary
basis of a claim that the drug is effective. The Secretary may
require the sponsor to modify or terminate such plan if
additional data or information indicates the plan as originally
agreed upon is no longer sufficient to demonstrate the safety
and effectiveness of the drug, or the drug is no longer
eligible for accelerated approval under section 506(c). Prior
to the effective date of the modification or termination, the
sponsor shall be granted a meeting to discuss the Secretary's
basis for the modification or termination.
Section 2041. Precision medicine guidance and other programs of Food
and Drug Administration
Section 2041 would require the Secretary to issue and
periodically update guidance defining the term ``precision drug
or biological product'' and assisting sponsors in their
development. Further, section 2041 would add section 592 to the
FFDCA authorizing the Secretary, in the case of a drug or
biological product for the treatment of a serious or life-
threatening disease or condition designated under section 526
as a drug for a rare disease or condition, to rely upon data or
information previously submitted by the sponsor for a different
drug or biological product that incorporates or utilizes the
same or similar underlying approach. This provision does not
permit FDA or any applicant to rely upon data or information of
another sponsor unless the applicant has provided the agency
with an effective contractual right of reference permitting
such reliance.
Section 2061. Broader application of Bayesian statistics and adaptive
trial designs
Section 2061 would require FDA to hold a public meeting and
issue guidance documents that would assist sponsors in
incorporating adaptive designs and Bayesian statistical
modeling into their proposed clinical protocols and
applications for new drugs and biological products.
Section 2062. Utilizing evidence from clinical experience
Section 2062 would amend the FFDCA by adding section 505F,
which would require the Secretary to establish a program to
evaluate the potential use of evidence from clinical experience
to help support the approval of a new indication for a drug
approved under section 505(b) and to help support or satisfy
post-approval study requirements. Such program shall be
implemented no later than two years after enactment. In
parallel, FDA would identify and execute pilot demonstrations
to extend existing use of the Sentinel System to, among other
things, support these efforts. The Committee does not intend
for anything in this section to be construed as prohibiting the
Secretary from using evidence from clinical experience for
purposes not specified in this section or in any way altering
the standards of evidence required by the FFDCA or the PHS Act,
or the Secretary's authority to require post-approval studies
or clinical trials. Furthermore, the Committee believes that
FDA's current safety surveillance activities using Sentinel are
exempt under existing law, and that no inference should be
drawn that the activities set forth in the provision, or
similar activities, are not exempt under existing law.
Section 2063. Streamlined data review program
Section 2063 would amend the FFDCA by adding section 505H,
which would require the Secretary to establish a program
authorizing the holder of an approved application to submit a
summary of clinical data intended to support the approval or
licensure of the drug for a new indication for the treatment of
cancer or other types of indications as determined appropriate
by the Secretary. The Committee believes that this program
should enable FDA to make approval decisions for certain
supplemental applications based on such qualified data
summaries. In implementing the program, the Secretary is
required to post on the FDA website and update annually the
number of applications reviewed under the program, the average
time for completion of reviews, and the number of applications
for which FDA made use of full data sets in addition to the
qualified data summary. Further, FDA is required to issue final
guidance on implementing the program within two years of
enactment, including the process for expanding the types of
indications to be considered.
Section 2081. Sense of Congress
Section 2081 reaffirms the Committee's belief that FDA
should continue to expedite the approval of drugs designated as
breakthrough therapies as early as possible in the clinical
development process, provided that the Secretary determines
that an application for a drug meets the required evidentiary
standards of safety and effectiveness under the FFDCA.
Section 2082. Expanded access policy
Section 2083. Finalizing draft guidance on expanded access
Expanded access programs provide a pathway for patients to
gain access to investigational new drugs that have not been
approved by the FDA. However, some patients encounter
challenges navigating that. There are also scientific
challenges regarding how adverse events experienced by patients
should be used during FDA's review of a drug for safety and
efficacy.
Section 2082 would require certain manufacturers or
distributors of an investigational drug to make publicly
available, such as through a manufacturer's or distributor's
web site, basic information on their expanded access policy.
Such policy may apply generally to all of such manufacturer's
or distributor's investigational drugs. The provision would not
require such companies to offer a drug through an expanded
access program or serve as a right to an expanded access
program.
Section 2083 would require the FDA to expedite guidance
providing clarity to sponsors seeking drug approval regarding
the consideration during the drug review process of adverse
events experienced by patients receiving a drug through an
expanded access program.
Section 2101. Facilitating dissemination of health care economic
information
Section 2101 would amend section 502(a) of the FFDCA to
clarify the scope of health care economic information drug
manufacturers can permissibly disseminate to payors, formulary
committees, or other similar entities.
Section 2102. Facilitating responsible communication of scientific and
medical developments
Section 2102 would require the Secretary to issue draft
guidance, no later than eighteen months from the date of
enactment, to clarify how drug and device manufacturers can
permissibly disseminate truthful and non-misleading scientific
and medical information about a drug or device that is not
included in the approved labeling for the product.
Section 2121. Approval of certain drugs for use in a limited population
of patients
Section 2122. Susceptibility test interpretive criteria for
microorganisms
Section 2123. Encouraging the development and use of antimicrobial
drugs
This section would build off of the progress Congress made
with the passage of the GAIN Act as a part of the Food and Drug
Safety and Innovation Act (FDASIA) in 2012 by facilitating the
development of new antibacterial or antifungal drugs through a
new FDA approval pathway and creating economic incentives for
new drug development.
The Committee believes that antibiotic resistance remains a
real and material public threat to the U.S. The unique nature
of resistance means that every antimicrobial drug currently on
the market is susceptible to drug resistance. The development
of new and important treatments for patients with unmet medical
needs therefore needs to continue if we are to stay ahead of
the pace of antimicrobial resistance. The Committee thinks such
development can benefit from regulatory improvements and
increased Federal investments. Furthermore, the Committee
recognizes that the prudent and appropriate use of
antimicrobial drugs can help to prevent the development of
resistance and maintain the effectiveness of these drugs.
The Committee supports a limited population approval
pathway that would allow the FDA, in partnership with
developers, to develop a regulatory process that takes into
account the nature of the product and the population of
patients it is intended to treat as a means of reducing the
time and investment needed to approve such drugs.
In order for such a pathway to function appropriately,
choosing such an approach over the traditional development
pathway must be voluntary for both the sponsor and Agency. In
addition, pursuit of approval for a limited population of
patients under this pathway, with its commensurate
requirements, should only begin upon completion of a formal
written agreement between the sponsor and FDA.
Products approved under this pathway are intended for use
in limited populations of patients with infections for which
there are few or no satisfactory treatment options available,
most likely infections caused by multi-drug resistant
organisms. The new tools that will be available to the Agency
include labeling requirements that stipulate that the words
``Limited Population'' be placed in a prominent manner and
adjacent to the brand name on the product labeling. This
branding element will assist in alerting the healthcare
community about the additional uncertainty and risk associated
with these products as a result of their streamlined
development program. It also will assist FDA, CDC, and
professional groups by educating healthcare providers that the
risk-benefit profile for these drugs is different than for
antimicrobial products approved under a traditional pathway and
that these products should be used judiciously and only for
patients with serious or life-threatening infections where
other suitable, alternative therapies are not available.
Section 2122 provides a streamlined mechanism for the
incorporation of updated susceptibility interpretive criteria
(`breakpoints') in antimicrobial susceptibility testing (AST)
devices, and in their labeling. It does this by establishing an
efficient and transparent process for FDA recognition of
susceptibility test interpretive criteria, while maintaining
FDA's rigorous review standards. AST devices use breakpoints to
identify whether a pathogen is susceptible (likely to respond),
intermediate, or resistant (not likely to respond) to the
tested antimicrobial drugs. These breakpoints change over time
because of the evolving nature of antimicrobial resistance.
Given the urgent public health crisis caused by antimicrobial
resistance, and the need to support the prudent and appropriate
use of antimicrobial drugs, the Committee believes that AST
devices must provide information based on up-to-date
breakpoints for the wide range of pathogens seen in clinical
practice, while directing healthcare providers to the labeling
of antimicrobial drugs for information about their approved
uses.
The Committee also has put forward a new economic incentive
through Medicare-payment policy for antimicrobial drugs
developed to treat highly-resistant infections. In combination,
the Committee intends that such efforts support the development
of new and lifesaving antibiotic treatments.
Section 2141. Timely review of vaccines by the Advisory Committee on
Immunization Practices
In addition to the challenging development environment for
vaccines, vaccine manufacturers have an additional step in the
development process; the Advisory Committee on Immunization
Practices (ACIP) Recommendation process, which takes place
following FDA licensure. ACIP working groups ``carefully review
data available on the vaccine in order to make recommendations
to ACIP'' and the 15 voting members of ACIP vote on their
working groups recommendations before forwarding them to the
CDC's Director for approval.\1\
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\1\http://www.cdc.gov/vaccines/hcp/patient-ed/conservations/
downloads/vacsafe-acip-color-office.pdf.
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The Committee believes the working groups should have
enough time to review the data and information to make an
informed recommendation to ACIP, and that ACIP vote on such
recommendations in a timely way to ensure patient access to
these lifesaving vaccines. Under this section, ACIP would have
to provide a decision or update the working groups' status at
each scheduled meeting so manufacturers will be able to plan
for vaccine distribution. If the Advisory Committee does not
make recommendations regarding a vaccine at the first regularly
scheduled meeting after licensure, the sponsor can request that
the Advisory Committee make such recommendations on an
expedited basis.
Section 2142. Review of processes and consistency of ACIP
recommendations
This section would require the Director of the CDC to
conduct a review of the process used by ACIP in order to
evaluate ACIP's consistency in formulating and issuing
recommendations pertaining to vaccines. Following such review,
the CDC Director shall publish a report on the results of the
review, including recommendations on improving the consistency
of process.
Section 2143. Meetings between CDC and vaccine developers
Currently, there is no formalized process for stakeholders
to meet with CDC to understand the process for developing
vaccines. This section would create and formalize processes for
the making of vaccination scheduling recommendations by ACIP,
for CDC review of ACIP recommendations, and for meetings
between CDC and vaccine developers. These meetings will provide
companies investing in vaccines more certainty and
understanding when establishing investment and development
plans.
Section 2151. Extension of exclusivity periods for a drug approved for
a new indication for a rare disease or condition
Despite the success of the Orphan Drug Act in bringing
drugs to market to patients facing diseases with smaller
patient populations, most of the thirty million patients
suffering from 7,000 rare diseases still have no treatment
options. Ninety-five percent of rare diseases have no FDA-
approved treatment.
Section 2151 would incentivize the repurposing of major
market drugs for rare diseases--advancing safe and effective
treatments and cures to patients facing these rare diseases.
The provision would provide a one-time, six month extension of
certain exclusivity periods and patent protection for an
already-approved drug if the drug's sponsor obtains approval of
a new indication for the drug for a rare disease or condition.
Section 2152. Reauthorization of rare pediatric disease priority review
voucher incentive program
This section would reauthorize the rare pediatric disease
priority review voucher (PRV) program through December 31,
2018. The rare pediatric disease PRV program was established in
FDASIA in order to encourage companies to invest in rare
pediatric diseases. There are many challenges associated with
pediatric drug development and even greater hurdles for rare
pediatric diseases with small patient populations. The ability
to sell such priority review vouchers was intended to provide
an incentive to foster drug development for rare pediatric
diseases.
This section also would broaden the definition of a rare
pediatric disease to ensure that pediatric oncology drugs and
treatments for sickle cell disease are eligible for
designation. The original legislation intended for these
products to be included; but FDA found that it could not
interpret the statutory language to include them. The draft
guidance that FDA released on November 17, 2014, entitled
``Rare Pediatric Disease Priority Review Vouchers,''
interpreted the definition of rare pediatric disease to mean
greater than 50 percent of the affected population with the
rare disease in the U.S. is aged 0 through 18 years. While many
drugs have qualified for this program under this
interpretation, including some drugs for pediatric cancers,
this section broadens the definition to include more drugs for
pediatric rare diseases, including drugs for a broader range of
pediatric cancers and sickle cell disease.
Finally, this provision requires the U.S. Government
Accounting Office to complete a report evaluating the
effectiveness of the program for encouraging drug development
for rare pediatric diseases.
Section 2161. Grants for studying the process of continuous drug
manufacturing
This section would allow FDA to award grants to higher
education and non-profit organizations to study and recommend
improvements to the process of continuous manufacturing (and
other similar innovative monitoring and control techniques) of
drugs and biologics.
The Committee believes that continuous manufacturing
advancements will make drug development less expensive and
manufacturing more flexible, and improve the quality of drugs.
Section 2162. Re-exportation among members of the European Economic
Area
Under current law, U.S. companies and manufacturers are
limited in their ability to re-export controlled substances
products within the European Economic Area. Section 2162 would
allow U.S. pharmaceutical companies to re-export controlled
substances similar to foreign pharmaceutical manufacturers,
providing a level-playing field regarding controlled substances
exports.
Section 2181. Enhancing combination products review
Combination products are often at the cutting edge of
innovation as they combine drugs, devices, and/or biological
products. Section 2181 requires FDA to issue a final guidance
document describing the role of all agency centers when
reviewing a combination product
Section 2201. Priority review for breakthrough devices
Section 2201 would add section 515B to the FFDCA, which
would require the Secretary to establish a program to provide
priority review for qualifying medical devices.
Under this section, a sponsor would be able to request
designation at any time prior to the submission of an
application under section 515(c), a petition for classification
under section 513(f)(2), or a notification under section
510(k). No later than sixty days after the receipt of such a
request, the Secretary shall determine whether the device
qualifies based on criteria set forth in this section. For
purposes of expediting the development and review of qualifying
devices, this section would require the Secretary to take a
number of actions, including the assignment of senior agency
personnel to oversee the process.
The Committee believes that this section is intended to
encourage the Secretary and provide the Secretary with
sufficient authorities to apply efficient and flexible
approaches to expedite the development of, and prioritize the
agency's review of, devices that represent breakthrough
technologies.
Section 2221. Third-party quality system assessment
Section 2221 would amend the FFDCA by adding section 524B.
Section 524B would require the Secretary to establish a third-
party quality system assessment program to accredit persons to
assess whether a requestor's quality system, including its
design controls, can reasonably assure the safety and
effectiveness of certain devices. This section would allow
companies to make specified changes to devices or device
manufacturing processes without prior FDA approval or clearance
if their quality system has been assessed by an accredited
person and deemed certified by the Secretary as being capable
of evaluating such specified changes and ensuring the safety
and effectiveness of the devices subject to such changes.
A certification deemed accepted by the Secretary under
proposed section 524B may be revoked upon a determination that
the requestor's quality system no longer meets the
certification criteria specified in the final guidance with
respect to in-scope devices. As a result, any device-related
changes made to an in-scope device subsequent to such a
determination are subject to the applicable submission
requirements. Any device that incorporates device-related
changes implemented subsequent to the determination would be
considered unapproved or uncleared, and thus may not serve as a
legally marketed predicate until the Secretary has approved or
cleared the changed device for marketing.
Section 2222. Valid scientific evidence
Section 2222 would amend section 513(a)(3)(B) of the FFDCA
to clarify that the Secretary, in the context of reviewing
device submissions, may rely on registry data, studies
published in peer-reviewed journals, and data collected in
countries other than the U.S.
Section 2223. Training and oversight in least-burdensome appropriate
means concept
Section 2223 would amend section 513 of the FFDCA and
require that each FDA employee involved in the review of
premarket device submissions under section 515 or section
510(k) receive training on the ``least burdensome'' concept,
which has been in statute since 1997. Further, FDA would be
required to update its guidance and have an audit by the FDA
ombudsman conducted on such training.
Section 2224. Recognition of standards
Section 2224 would amend section 514(c) of the FFDCA to
establish a process by which any person can request that the
Secretary recognize all or part of an appropriate standard
relating to devices that has been established by a nationally
or internationally recognized standard setting organization.
This section would require the Secretary, within sixty days of
receiving such a request, to make a determination and issue a
response in writing that explains the Secretary's rationale for
such determination, including the scientific, technical,
regulatory, or other basis, and to make such response publicly
available as the Secretary determines appropriate. Further,
section 2224 would require the Secretary to provide training on
the use of such standards in device reviews and to issue
guidance identifying principles for recognizing standards.
Section 2225. Easing regulatory burden with respect to certain Class I
and Class II devices
Section 2225 would amend section 510(l) and (m) of the
FFDCA to provide a process for the Secretary to identify,
through publication in the Federal Register, certain types of
class I and II devices the Secretary determines no longer
require reports under section 510(k) to provide reasonable
assurance of safety and effectiveness.
Section 2226. Advisory committee process
Section 2226 would amend section 513(b) of the FFDCA to
enable the more active participation of the person whose device
is specifically the subject of the panel review. It would
provide the sponsor the opportunity to make recommendations on
the expertise needed among voting members of the panel and
codify the Secretary's existing practice for giving due
consideration to such recommendations. Further, the Secretary
would be required to provide adequate time for initial
presentations by the person whose device is specifically the
subject of the classification panel review. The provisions in
section 2226 apply only to panel meetings where a specific
device is the subject, not panel meetings where an entire
device type is the subject.
For purposes of 513(b)(5)(B) and 513(b)(6)(A) and (B), as
amended by section 2226, a device that is specifically the
subject of review of a classification panel means a device that
is the subject a premarket approval application, premarket
notification (510(k)), de novo classification request,
humanitarian device exemption application, or investigational
device exemption application, or such other type of premarket
submission as the Secretary in its sole discretion may specify,
and which was referred to the panel for review.
Section 2227. Humanitarian device exemption application
Section 2227 would amend section 520(m) of the FFDCA to
provide the Secretary with the authority to apply the
humanitarian device exemption to diseases and conditions that
affect up to 8,000 individuals in the U.S.
Section 2228. CLIA waiver study design guidance for in vitro
diagnostics
Section 2228 would require the Secretary to publish draft
guidance within one year of enactment on the appropriate use of
comparable performance between a waived user and a moderately
complex laboratory user to demonstrate accuracy under the
Clinical Laboratory Improvement Amendments of 1988 (CLIA). The
Secretary would be required to finalize such guidance no later
than one year after the comment period closes.
Section 2241. Health software
Section 2242. Applicability and inapplicability of regulation
Section 2243. Exclusion from definition of device
Technological innovation around health software and
wireless platforms such as smartphones hold great promise for
the health care system. However, there are concerns related to
the current regulatory approach for health technologies.
Sections 2241, 2242, and 2243 would support further development
in this field by updating the regulatory laws around software
and creating clarity for developers and reviewers alike.
The Committee believes that regulatory discretion is an
important tool for FDA to keep up with the pace of innovation.
The Committee believes that the ability to bring certainty
and clarity to developers of software products will encourage
further innovation in the health care sector. Software and
hardware products are unique platforms and distinct enough from
each other to warrant review of the ways in which software is
approved by FDA to ensure such innovation in this sector
continues long into the future.
Further, the Committee believes that software-provided
health-related information, in and of itself, is not a threat
to the health and welfare of the public. As such, it should not
on its own warrant FDA regulation. Save for those products
designed to replace the judgment of a clinician, the Committee
believes that the Federal government should take a cautious
approach to regulation. The Committee believes that the clarity
for developers that these provisions provide will support
continued health information technology development in the
health care sector for decades to come.
Section 2261. Protection of human subjects in research; applicability
of rules
Section 2261 would require the Secretary to harmonize
differences between the HHS Human Subject Regulations and the
FDA Human Subject Regulations to simplify and facilitate
researchers' compliance with applicable regulations for the
protection of human subjects in research. Specifically, the
Secretary would be required to make such modifications as may
be necessary to reduce regulatory duplication and unnecessary
delays, to modernize such provisions in the context of
multisite and cooperative research projects, and to incorporate
local considerations, community values, and mechanisms to
protect vulnerable populations. Further, the Secretary would be
required to issue such regulations and guidance and take such
other actions as may be necessary to implement this section and
help facilitate the broader use of single, central, or lead
institutional review boards.
This provision seeks to reduce regulatory duplication and
delays for clinical trials by harmonizing the clinical trial
rules imposed by HHS's Office of Human Research Protection
(OHRP) and FDA, to the extent possible and consistent with
other statutory provisions.
Section 2262. Use of non-local institutional review boards for review
of investigational device exemptions and human device
exemptions
Section 2262 would amend section 520 of the FFDCA to remove
the requirement to use local institutional review boards (IRB)
for clinical testing of medical devices and the use of approved
humanitarian use devices. By permitting the use of a central
IRB in multi-site studies, when appropriate, such research can
be facilitated and costs may be reduced, while still protecting
the rights, safety, and welfare of study subjects. The ability
to have non-local IRB review for approved humanitarian use
devices should improve accessibility to these devices in
facilities without an associated IRB, such as outpatient
surgical clinics or small hospitals.
Section 2263. Alteration or waiver of informed consent for clinical
investigations
Section 2263 would amend sections 520(g)(3) and 505(i)(4)
of the FFDCA to specify that informed consent is not required
for clinical testing of devices and drugs that pose no more
than minimal risk to the human subjects and includes
appropriate safeguards as prescribed by the Secretary to
protect the rights, safety, and welfare of the participants.
This provision will enable the FDA to further harmonize its
regulations with the current Federal Policy for the Protection
of Human Subjects (the Common Rule), by allowing a waiver of
informed consent to conduct minimal-risk research under certain
conditions, and in the absence of an emergency or serious or
life-threatening risk to subjects, consistent with the Common
Rule.
Section 2281. Silvio O. Conte Senior Biomedical Research Service
This section would enable FDA to hire more efficiently and
ensure that the agency has the staff required to ensure they
keep up with the pace of innovation. One of the biggest
challenges that FDA faces is hiring qualified and competitive
individuals, and this provision enables the Agency to attract
scientists that are designated as ``outstanding in their field
of biomedical research, clinical research evaluation, and
biomedical product assessment.'' Increasing the number of
scientists in the biomedical research service will ensure that
FDA can utilize the service to hire and retain experts in the
biomedical field.
Section 2282. Enabling FDA scientific engagement
This section expresses congressional support for
eliminating barriers that prevent agency staff from attending
scientific conferences and meetings. Allowing staff travel for
their continued training and education will help the agency
keep pace with the latest scientific developments. Such travel
has been discouraged due to the resource intensive approval
processes, which has negatively affected FDA's ability to
review applications with the latest scientific knowledge.
Section 2283. Reagan-Udall Foundation for the Food and Drug
Administration
The changes to the Reagan Udall Foundation (RUF) statute
relate to issues of internal Foundation governance and
management to ensure that RUF has access to the expertise and
human capital it needs to fulfill its statutory mission of
advancing FDA's scientific priorities. The section would
clarify the ability of the RUF Board of Directors to expand the
Board's size and add to the Board members of FDA Advisory
Committees. It also would leave the matter of compensation for
the RUF Executive Director to the discretion of the Board, as
is the case with the Foundation for the NIH. These changes
would help ensure that RUF has, both on its Board and in its
senior management, persons who have the experience and skills
to collaborate effectively with FDA in identifying and
advancing RUF's core mission areas and who can be effective in
locating the public and private resources needed to achieve
those shared goals.
RUF's purpose is to assist FDA in fulfilling its mission.
Ensuring staff are educated and informed of what outside groups
are doing and of the latest scientific innovation is essential
to achieving that purpose.
Section 2284. Collection of certain voluntary information exempted from
Paperwork Reduction Act
This section would exempt FDA from the PRA with respect to
the collection from patients, industry, academia, and other
stakeholders of voluntary information such as through voluntary
surveys and questionnaires. This would enable FDA to more
easily and efficiently receive patient input. The PRA has
increased administrative burden at FDA, drained resources, and
prohibited FDA from communicating effectively and efficiently
with outside parties. Removing these burdens from FDA should
enable the agency to utilize these resources for other
important work.
Section 2285. Hiring authority for scientific, technical, and
professional personnel
The Committee finds that, in order to have a modern and
efficient regulatory system, FDA needs to have scientists
trained and educated in the latest science and medical
research. During the Subcommittee on Health hearing on H.R. 6,
Janet Woodcock, M.D., Director of the Center for Drug
Evaluation and Research at FDA, noted that hiring is a problem
across FDA:
The science right now is exploding; the new products
are extremely innovative. That is wonderful, but we
need to have some good scientists who can go toe-to-toe
with the best in industry, and industry can afford the
best scientists . . . we have extreme difficulty hiring
senior people who have worked outside the
government.\2\
---------------------------------------------------------------------------
\2\http://docs.house.gov/neetings/IF/IF14/20150430/103400/HHRG-
1140IF14-Transcript020150430-U23.pdf.
This provision would enable FDA to hire more efficiently by
giving the agency broad and flexible new authority to recruit
and retain the staff required to ensure that the agency keeps
up with the pace of innovation. It also includes the ability to
offer salaries competitive with those in the private sector and
in academia. This authority is essential for FDA be able to
hire in an effective manner and to avoid existing inefficient
processes.
Section 2301. Exempting from sequestration certain user fees
Section 2301 would permanently exempt the following FDA
user fees from sequestration: fees for medical devices,
prescription drugs, generics drugs, biosimilars, animal drugs,
and generic animal drugs. This provision would provide
certainty regarding access to FDA user fees and provide funding
for drug and device review and other critical agency functions.
TITLE III--DELIVERY
Section 3001. Ensuring interoperability
As evidenced by statements from numerous 21st Century Cures
roundtable participants, the ability to share research and
clinical data is a cornerstone of our drive for new cures. The
Office of the National Coordinator (ONC) for Health Information
Technology has led the charge, but recently has identified
barriers to nationwide interoperability of health technology.
Section 3001 would refocus national efforts on making systems
interoperable and holding individuals responsible for blocking
or otherwise inhibiting the flow of patient information
throughout our healthcare system.
Section 3021. Telehealth service under the Medicare program
The Energy and Commerce Bipartisan Telemedicine Member
Working Group has been working to find a solution that has
plagued Congress and our health system for decades: how to
adopt new technologies into our delivery system in ways that
promote greater quality care and fiscal integrity. Section 3021
would support the efforts of the working group by requiring
specific actions of government bodies identified as critical to
developing a long-term solution to this problem.
The Committee believes that continuing efforts to identify
and adopt solutions that promote increased utilization of
telemedicine services and technologies in the Medicare program
is beneficial for Medicare beneficiaries. The Committee
therefore will continue its efforts and those of the Bipartisan
Working Group to effectuate such change.
Section 3041. Exempting from manufacturer transparency reporting
certain transfers used for educational purposes
Section 3041 would amend section 1128G(e)(10)(B) of the
Social Security Act to exempt certain transfers of value from
reporting requirements that providers have noted had a chilling
effect on their engagement in important continuing medical
education activities.
Section 3061. Treatment of certain items and devices
Today, seniors who receive their care in a home setting are
not able to access certain services afforded others because of
the nature of the durable medical equipment (DME) payment
system. This section would ensure that those seniors receiving
care in the home setting are not denied access to certain
treatments that would otherwise be available to them based
simply on the location in which they seek care.
Section 3081. Improvements in the Medicare local coverage determination
(LCD) process
The local coverage determination (LCD) process is an
important means by which seniors can access treatments that
would not otherwise be covered by Medicare due to the length of
time it takes for the national process to conclude its work.
However, improvements are needed. Section 3081 would increase
transparency around the LCD process and begin the process of
bringing greater accountability to the actions of those
contracting with the Centers for Medicare and Medicaid Services
to manage the operation of the Medicare program.
Section 3101. Medicare pharmaceutical and technology ombudsman
This section would create a new technology ombudsman within
Medicare to address problems relating to coverage of new and
life-saving technologies.
Section 3121. Medicare site-of-service price transparency
The Medicare benefit currently pays varying rates for the
same services depending on where they are delivered. As a
result, seniors' out of pocket costs can be higher or lower for
a given procedure based upon where the service is provided.
This section would give seniors the ability to shop among
certain sites of service for certain services so that they can
identify the most cost-effective treatments. The Committee
believes this is an important step in supporting seniors who
wish to shop for the best value at the best cost.
Section 3141. Programs to prevent prescription drug abuse under
Medicare parts C and D
This section would allow prescription drug plans in
Medicare Part D to develop a safe prescribing and dispensing
program for beneficiaries that are prescribed a high volume of
controlled substances.
TITLE IV--MEDICAID, MEDICARE, AND OTHER REFORMS
Section 4001. Limiting Federal Medicaid reimbursement to States for
durable medical equipment (DME) to Medicare payment rates
Modeled after the policy in the President's FY 2016 budget
and previous budget proposals from the Administration, this
section would limit Federal matching funds for Medicaid
payments for certain durable medical equipment, prosthetics,
orthotics, and supplies (DMEPOS) to Medicare fee schedule
rates, and where applicable, the market-based rates paid by
Medicare under its competitive bidding program (CBP). The
Federal upper payment limit established by this policy is not
specific to payment for an individual DMEPOS claim, but would
apply in the aggregate to classes of DMEPOS, as defined by the
Secretary.
This policy would take effect beginning January 1, 2020.
The provision also directs the Medicare Ombudsman established
by this legislation to evaluate the impact of this limit on
Federal matching funds on beneficiary health status and
outcomes. This policy should not be construed as limiting a
State from covering or continuing to cover DME items that are
not listed on the Medicare fee schedule. Rather, this policy
establishes a Federal upper limit for DME in Medicaid to the
applicable Medicare rate.
Section 4002. Medicare payment incentive for the transition from
traditional x-ray imaging to digital radiography and other
Medicare imaging payment provision
Consistent with the Committee's goals of improving patient
care and encouraging the adoption of innovative medical
technologies, this section would implement differential
Medicare reimbursement for film x-ray and computed radiography
(CR) to incentivize the transition to digital radiography (DR).
This policy would encourage providers' transition to modern
digital technologies and improve patient safety and care as
providers transition away from older technology, which may
produce less detailed images, thus requiring another image to
be taken which exposes patients to the negative clinical
effects of additional scans. Additionally, as a matter of basic
fairness and transparency, this policy would eliminate
application of the multiple procedure payment reduction unless
the Secretary conducts and publishes empirical analysis within
the Medicare Physician Fee Schedule Proposed Rule in the prior
year.
Section 4003. Implementation of Office of Inspector General
recommendations to delay certain Medicare prescription drug
plan prepayments
Section 4003 would delay monthly reinsurance prepayments
for prescription drug plans, beginning in January 2020. This
policy implements an Office of Inspector General recommendation
to adjust the timing of Medicare's prepayments to Medicare
Advantage organizations to better reflect when payments are
made to providers, as is currently the policy in the Federal
Employees Health Benefits Program. However, this policy only
applies to the reinsurance portion of the payment.
Section 4041. Cures Innovation Fund
This section would establish a $110 million annual fund
from FY 2016 through FY 2020 for research, regulatory
modernization, and other activities authorized by the 21st
Century Cures Act, including activities of FDA.
Section 4061. SPR drawdown
This section would direct the Department of Energy to draw
down and sell crude oil from the Strategic Petroleum Reserve
(SPR).
Section 4081. Lyme disease and other tick-borne diseases
This section would help to accelerate improved methods for
prevention, diagnosis, and treatment of Lyme disease. It would
establish a working group to prepare a report that would
summarize Federal research efforts related to Lyme disease and
other tick-borne diseases. Informed by the report prepared by
the working group, the Secretary would develop a strategic plan
to improve health outcomes.
Changes in Existing Law Made by the Bill, as Reported
In compliance with clause 3(e) of rule XIII of the Rules of
the House of Representatives, changes in existing law made by
the bill, as reported, are shown as follows (existing law
proposed to be omitted is enclosed in black brackets, new
matter is printed in italic, and existing law in which no
change is proposed is shown in roman):
PUBLIC HEALTH SERVICE ACT
* * * * * * *
TITLE II--ADMINISTRATION AND MISCELLANEOUS PROVISIONS
Part A--Administration
* * * * * * *
silvio o. conte senior biomedical research and biomedical product
assessment service
Sec. 228. (a)(1) There shall be in the Public Health Service
a [Silvio O. Conte Senior Biomedical Research Service, not to
exceed 500 members] Silvio O. Conte Senior Biomedical Research
and Biomedical Product Assessment Service (in this section
referred to as the ``Service''), the purpose of which is to
recruit and retain competitive and qualified scientific and
technical experts outstanding in the field of biomedical
research, clinical research evaluation, and biomedical product
assessment.
[(2) The authority established in paragraph (1) regarding the
number of members in the Silvio O. Conte Senior Biomedical
Research Service is in addition to any authority established
regarding the number of members in the commissioned Regular
Corps, in the Reserve Corps, and in the Senior Executive
Service. Such paragraph may not be construed to require that
the number of members in the commissioned Regular Corps, in the
Reserve Corps, or in the Senior Executive Service be reduced to
offset the number of members serving in the Silvio O. Conte
Senior Biomedical Research Service (in this section referred to
as the ``Service'').]
(2) The authority established in paragraph (1) may not be
construed to require the Secretary to reduce the number of
employees serving under any other employment system in order to
offset the number of members serving in the Service.
(b) The Service shall be appointed by the Secretary without
regard to the provisions of title 5, United States Code,
regarding appointment, and shall consist of individuals
outstanding in the field of biomedical research [or clinical
research evaluation], clinical research evaluation or
biomedical product assessment. No individual may be appointed
to the Service unless such individual (1) has earned a doctoral
level degree in biomedicine or a related field, or a master's
level degree in engineering, bioinformatics, or a related or
emerging field, and (2) meets the qualification standards
prescribed by the Office of Personnel Management for
appointment to a position at GS-15 of the General Schedule.
Notwithstanding any previous applicability to an individual who
is a member of the Service, the provisions of subchapter I of
chapter 35 (relating to retention preference), chapter 43
(relating to performance appraisal and performance actions),
chapter 51 (relating to classification), subchapter III of
chapter 53 (relating to General Schedule pay rates), and
chapter 75 (relating to adverse actions) of title 5, United
States Code, shall not apply to any member of the Service.
(c) The Secretary shall develop a performance appraisal
system designed to--
(1) provide for the systematic appraisal of the
performance of members, and
(2) encourage excellence in performance by members.
(d)(1) The Secretary shall determine, subject to the
provisions of this subsection, the pay of members of the
Service.
(2) The pay of a member of the Service shall not be less than
the minimum rate payable for GS-15 of the General Schedule [and
shall not exceed the rate payable for level I of the Executive
Schedule unless approved by the President under section
5377(d)(2) of title 5, United States Code] and shall not exceed
the rate payable for the President.
[(e) The Secretary may, upon the request of a member who--
[(1) performed service in the employ of an
institution of higher education immediately prior to
his appointment as a member of the Service, and
[(2) retains the right to continue to make
contributions to the retirement system of such
institution,
contribute an amount not to exceed 10 percent per annum of the
member's basic pay to such institution's retirement system on
behalf of such member. A member who requests that such
contribution be made shall not be covered by, or earn service
credit under, any retirement system established for employees
of the United States under title 5, United States Code, but
such service shall be creditable for determining years of
service under section 6303(a) of such title.]
[(f)] (e) Subject to the following sentence, the Secretary
may, notwithstanding the provisions of title 5, United States
Code, regarding appointment, appoint an individual who is
separated from the Service involuntarily and without cause to a
position in the competitive civil service at GS-15 of the
General Schedule, and such appointment shall be a career
appointment. In the case of such an individual who immediately
prior to his appointment to the Service was not a career
appointee in the civil service or the Senior Executive Service,
such appointment shall be in the excepted civil service and may
not exceed a period of 2 years.
[(g)] (f) The Secretary shall promulgate such rules and
regulations, not inconsistent with this section, as may be
necessary for the efficient administration of the Service.
* * * * * * *
SEC. 229A. DATA ON NATURAL HISTORY OF DISEASES.
(a) In General.--The Secretary may, for the purposes
described in subsection (b)--
(1) participate in public-private partnerships
engaged in one or more activities specified in
subsection (c); and
(2) award grants to patient advocacy groups or other
organizations determined appropriate by the Secretary.
(b) Purposes Described.--The purposes described in this
subsection are to establish or facilitate the collection,
maintenance, analysis, and interpretation of data regarding the
natural history of diseases, with a particular focus on rare
diseases.
(c) Activities of Public-Private Partnerships.--The
activities of public-private partnerships in which the
Secretary may participate for purposes of this section
include--
(1) cooperating with other entities that sponsor or
maintain disease registries, including disease
registries and disease registry platforms for rare
diseases;
(2) developing or enhancing a secure information
technology system that--
(A) has the capacity to support data needs
across a wide range of disease studies;
(B) is easily modified as knowledge is gained
during such studies; and
(C) is capable of handling increasing amounts
of data as more studies are carried out; and
(3) providing advice to clinical researchers, patient
advocacy groups, and other entities with respect to--
(A) the design and conduct of disease
studies;
(B) the modification of any such ongoing
studies; and
(C) addressing associated patient privacy
issues.
(d) Availability of Data on Natural History of Diseases.--
Data relating to the natural history of diseases obtained,
aggregated, or otherwise maintained by a public-private
partnership in which the Secretary participates under
subsection (a) shall be made available, consistent with
otherwise applicable Federal and State privacy laws, to the
public (including patient advocacy groups, researchers, and
drug developers) to help to facilitate and expedite medical
product development programs.
(e) Confidentiality.--Notwithstanding subsection (d), nothing
in this section authorizes the disclosure of any information
that is a trade secret or commercial or financial information
that is privileged or confidential and subject to section
552(b)(4) of title 5, United States Code, or section 1905 of
title 18, United States Code.
(f) Authorization of Appropriations.--There is authorized to
be appropriated to carry out this section $5,000,000 for each
of fiscal years 2016 through 2020.
* * * * * * *
PART E--COUNCIL FOR 21ST CENTURY CURES
SEC. 281. ESTABLISHMENT.
A nonprofit corporation to be known as the Council for 21st
Century Cures (referred to in this part as the ``Council'')
shall be established in accordance with this section. The
Council shall be a public-private partnership headed by an
Executive Director (referred to in this part as the ``Executive
Director''), appointed by the members of the Board of
Directors. The Council shall not be an agency or
instrumentality of the United States Government.
SEC. 281A. PURPOSE.
The purpose of the Council is to accelerate the discovery,
development, and delivery in the United States of innovative
cures, treatments, and preventive measures for patients.
SEC. 281B. DUTIES.
For the purpose described in section 281A, the Council
shall--
(1) foster collaboration and coordination among the
entities that comprise the Council, including academia,
government agencies, industry, health care payors and
providers, patient advocates, and others engaged in the
cycle of discovery, development, and delivery of life-
saving and health-enhancing innovative interventions;
(2) undertake communication and dissemination
activities;
(3) publish information on the activities funded
under section 281D;
(4) establish a strategic agenda for accelerating the
discovery, development, and delivery in the United
States of innovative cures, treatments, and preventive
measures for patients;
(5) identify gaps and opportunities within and across
the discovery, development, and delivery cycle;
(6) develop and propose recommendations based on the
gaps and opportunities so identified;
(7) facilitate the interoperability of the components
of the discovery, development, and delivery cycle;
(8) propose recommendations that will facilitate
precompetitive collaboration;
(9) identify opportunities to work with, but not
duplicate the efforts of, nonprofit organizations and
other public-private partnerships; and
(10) identify opportunities for collaboration with
organizations operating outside of the United States,
such as the Innovative Medicines Initiative of the
European Union.
SEC. 281C. ORGANIZATION; ADMINISTRATION.
(a) Board of Directors.--
(1) Establishment.--
(A) In general.--The Council shall have a
Board of Directors (in this part referred to as
the ``Board of Directors''), which shall be
composed of the ex officio members under
subparagraph (B) and the appointed members
under subparagraph (C). All members of the
Board shall be voting members.
(B) Ex officio members.--The ex officio
members of the Board shall be the following
individuals or their designees:
(i) The Director of the National
Institutes of Health.
(ii) The Commissioner of Food and
Drugs.
(iii) The Administrator of the
Centers for Medicare & Medicaid
Services.
(iv) The heads of five other Federal
agencies deemed by the Secretary to be
engaged in biomedical research and
development.
(C) Appointed members.--The appointed members
of the Board shall consist of 17 individuals,
of whom--
(i) 8 shall be appointed by the
Comptroller General of the United
States from a list of nominations
submitted by leading trade
associations--
(I) 4 of whom shall be
representatives of the
biopharmaceutical industry;
(II) 2 of whom shall be
representatives of the medical
device industry; and
(III) 2 of whom shall be
representatives of the
information and digital
technology industry; and
(ii) 9 shall be appointed by the
Comptroller General of the United
States, after soliciting nominations--
(I) 2 of whom shall be
representatives of academic
researchers;
(II) 3 of whom shall be
representatives of patients;
(III) 2 of whom shall be
representatives of health care
providers; and
(IV) 2 of whom shall be
representatives of health care
plans and insurers.
(D) Chair.--The Chair of the Board shall be
selected by the members of the Board by
majority vote from among the members of the
Board.
(2) Terms and vacancies.--
(A) In general.--The term of office of each
member of the Board appointed under paragraph
(1)(C) shall be 5 years.
(B) Vacancy.--Any vacancy in the membership
of the Board--
(i) shall not affect the power of the
remaining members to execute the duties
of the Board; and
(ii) shall be filled by appointment
by the appointed members described in
paragraph (1)(C) by majority vote.
(C) Partial term.--If a member of the Board
does not serve the full term applicable under
subparagraph (A), the individual appointed
under subparagraph (B) to fill the resulting
vacancy shall be appointed for the remainder of
the term of the predecessor of the individual.
(3) Responsibilities.--Not later than 90 days after
the date on which the Council is incorporated and its
Board of Directors is fully constituted, the Board of
Directors shall establish bylaws and policies for the
Council that--
(A) are published in the Federal Register and
available for public comment;
(B) establish policies for the selection and,
as applicable, appointment of--
(i) the officers, employees, agents,
and contractors of the Council; and
(ii) the members of any committees of
the Council;
(C) establish policies, including ethical
standards, for the conduct of programs and
other activities under section 281D; and
(D) establish specific duties of the
Executive Director.
(4) Meetings.--
(A) In general.--The Board of Directors
shall--
(i) meet on a quarterly basis; and
(ii) submit to Congress, and make
publicly available, the minutes of such
meetings.
(B) Agenda.--The Board of Directors shall,
not later than 3 months after the incorporation
of the Council--
(i) issue an agenda (in this part
referred to as the ``agenda'')
outlining how the Council will achieve
the purpose described in section 281A;
and
(ii) annually thereafter, in
consultation with the Executive
Director, review and update such
agenda.
(b) Appointment and Incorporation.--Not later than 6 months
after the date of enactment of the 21st Century Cures Act--
(1) the Comptroller General of the United States
shall appoint the appointed members of the Board of
Directors under subsection (a)(1)(C); and
(2) the ex officio members of the Board of Directors
under subsection (a)(1)(B) shall serve as incorporators
and shall take whatever actions are necessary to
incorporate the Council.
(c) Nonprofit Status.--In carrying out this part, the Board
of Directors shall establish such policies and bylaws, and the
Executive Director shall carry out such activities, as may be
necessary to ensure that the Council maintains status as an
organization that--
(1) is described in subsection (c)(3) of section 501
of the Internal Revenue Code of 1986; and
(2) is, under subsection (a) of such section, exempt
from taxation.
(d) Executive Director.--The Executive Director shall--
(1) be the chief executive officer of the Council;
and
(2) subject to the oversight of the Board of
Directors, be responsible for the day-to-day management
of the Council.
SEC. 281D. OPERATIONAL ACTIVITIES AND ASSISTANCE.
(a) In General.--The Council shall establish a sufficient
operational infrastructure to fulfill the duties specified in
section 281B.
(b) Private Sector Matching Funds.--The Council may accept
financial or in-kind support from participating entities or
private foundations or organizations when such support is
deemed appropriate.
SEC. 281E. TERMINATION; REPORT.
(a) In General.--The Council shall terminate on September 30,
2023.
(b) Report.--Not later than one year after the date on which
the Council is established and each year thereafter, the
Executive Director shall submit to the appropriate
congressional committees a report on the performance of the
Council. In preparing such report, the Council shall consult
with a nongovernmental consultant with appropriate expertise.
SEC. 281F. FUNDING.
For the each of fiscal years 2016 through 2023, there is
authorized to be appropriated $10,000,000 to the Council for
purposes of carrying out the duties of the Council under this
part.
TITLE III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE
Part A--Research and Investigation
* * * * * * *
health conferences and health education information
Sec. 310. (a) A conference of the health authorities in and
among the several States shall be called annually by the
Secretary. Whenever in his opinion the interests of the public
health would be promoted by a conference, the Secretary may
invite as many of such health authorities and officials of
other State or local public or private agencies, institutions,
or organizations to confer as he deems necessary or proper.
Upon the application of health authorities of five or more
States it shall be the duty of the Secretary to call a
conference of all State health authorities joining in the
request. Each State represented at any conference shall be
entitled to a single vote. Whenever at any such conference
matters relating to mental health are to be discussed, the
mental health authorities of the respective States shall be
invited to attend.
(b) From time to time the Secretary shall issue information
related to public health, in the form of publications or
otherwise, for the use of the public, and shall publish weekly
reports of health conditions in the United States and other
countries and other pertinent health information for the use of
persons and institutions concerned with health services.
(c)(1) In this subsection, the term ``vaccine developer''
means a nongovernmental entity engaged in--
(A)(i) the development of a vaccine with the intent
to pursue licensing of the vaccine by the Food and Drug
Administration; or
(ii) the production of a vaccine licensed by the Food
and Drug Administration; and
(B) vaccine research.
(2)(A) Upon the submission of a written request for a meeting
by a vaccine developer, that includes a justification for the
meeting, the Secretary, acting through the Director of the
Centers for Disease Control and Prevention, shall convene a
meeting of representatives of the vaccine developer and experts
from the Centers for Disease Control and Prevention in
immunization programs, epidemiology, and other relevant areas
at which the Director (or the Director's designee), for the
purpose of informing the vaccine developer's understanding of
public health needs and priorities, shall provide the
perspectives of the Centers for Disease Control and Prevention
and other relevant Federal agencies regarding--
(i) public health needs, epidemiology, and
implementation considerations with regard to a vaccine
developer's potential vaccine profile; and
(ii) potential implications of such perspectives for
the vaccine developer's vaccine research and
development planning.
(B) In addition to the representatives specified in
subparagraph (A), the Secretary may, with the agreement of the
vaccine developer requesting a meeting under such subparagraph,
include in such meeting representatives of--
(i) the Food and Drug Administration; and
(ii) the National Vaccine Program.
(C) The Secretary shall convene a meeting requested under
subparagraph (A) not later than 120 days after receipt of the
request for the meeting.
(3)(A) Upon the submission of a written request by a vaccine
developer, the Secretary, acting through the Director of the
Centers for Disease Control and Prevention, shall provide to
the vaccine developer any age-based or other demographically
assessed disease epidemiological analyses or data that--
(i) are specified in the request;
(ii) have been published;
(iii) have been performed by or are in the possession
of the Centers;
(iv) are not a trade secret or commercial or
financial information that is privileged or
confidential and subject to section 552(b)(4) of title
5, United States Code, or section 1905 of title 18,
United States Code; and
(v) do not contain individually identifiable
information.
(B) The Secretary shall provide analyses requested by a
vaccine manufacturer under subparagraph (A) not later than 120
calendar days after receipt of the request for the analyses.
(4) The Secretary shall promptly notify a vaccine developer
if--
(A) the Secretary becomes aware of any change to
information that was--
(i) shared by the Secretary with the vaccine
developer during a meeting under paragraph (2);
or
(ii) provided by the Secretary to the vaccine
developer in one or more analyses under
paragraph (3); and
(B) the change to such information may have
implications for the vaccine developer's vaccine
research and development.
* * * * * * *
Part B--Federal-State Cooperation
* * * * * * *
SEC. 317U. MONITORING ANTIBACTERIAL AND ANTIFUNGAL DRUG USE AND
RESISTANCE.
(a) Monitoring.--The Secretary shall use an appropriate
monitoring system to monitor--
(1) the use of antibacterial and antifungal drugs,
including those receiving approval or licensure for a
limited population pursuant to section 505(z) of the
Federal Food, Drug, and Cosmetic Act; and
(2) changes in bacterial and fungal resistance to
drugs.
(b) Public Availability of Data.--The Secretary shall make
summaries of the data derived from monitoring under this
section publicly available for the purposes of--
(1) improving the monitoring of important trends in
antibacterial and antifungal resistance; and
(2) ensuring appropriate stewardship of antibacterial
and antifungal drugs, including those receiving
approval or licensure for a limited population pursuant
to section 505(z) of the Federal Food, Drug, and
Cosmetic Act.
* * * * * * *
Part F--Licensing--Biological Products and Clinical Laboratories
Subpart 1--Biological Products
regulation of biological products
Sec. 351. (a)(1) No person shall introduce or deliver for
introduction into interstate commerce any biological product
unless--
(A) a biologics license under this subsection or
subsection (k) is in effect for the biological product;
and
(B) each package of the biological product is plainly
marked with--
(i) the proper name of the biological product
contained in the package;
(ii) the name, address, and applicable
license number of the manufacturer of the
biological product; and
(iii) the expiration date of the biological
product.
(2)(A) The Secretary shall establish, by regulation,
requirements for the approval, suspension, and revocation of
biologics licenses.
(B) Pediatric studies.--A person that submits an
application for a license under this paragraph shall
submit to the Secretary as part of the application any
assessments required under section 505B of the Federal
Food, Drug, and Cosmetic Act.
(C) The Secretary shall approve a biologics license
application--
(i) on the basis of a demonstration that--
(I) the biological product that is the
subject of the application is safe, pure, and
potent; and
(II) the facility in which the biological
product is manufactured, processed, packed, or
held meets standards designed to assure that
the biological product continues to be safe,
pure, and potent; and
(ii) if the applicant (or other appropriate person)
consents to the inspection of the facility that is the
subject of the application, in accordance with
subsection (c).
(D) Postmarket Studies and Clinical Trials; Labeling; Risk
Evaluation and Mitigation Strategy.--A person that submits an
application for a license under this paragraph is subject to
sections 505(o), 505(p), and 505-1 of the Federal Food, Drug,
and Cosmetic Act.
(3) The Secretary shall prescribe requirements under which a
biological product undergoing investigation shall be exempt
from the requirements of paragraph (1).
(b) No person shall falsely label or mark any package or
container of any biological product or alter any label or mark
on the package or container of the biological product so as to
falsify the label or mark.
(c) Any officer, agent, or employee of the Department of
Health and Human Services, authorized by the Secretary for the
purpose, may during all reasonable hours enter and inspect any
establishment for the propagation or manufacture and
preparation of any biological product.
(d)(1) Upon a determination that a batch, lot, or other
quantity of a product licensed under this section presents an
imminent or substantial hazard to the public health, the
Secretary shall issue an order immediately ordering the recall
of such batch, lot, or other quantity of such product. An order
under this paragraph shall be issued in accordance with section
554 of title 5, United States Code.
(2) Any violation of paragraph (1) shall subject the violator
to a civil penalty of up to $100,000 per day of violation. The
amount of a civil penalty under this paragraph shall, effective
December 1 of each year beginning 1 year after the effective
date of this paragraph, be increased by the percent change in
the Consumer Price Index for the base quarter of such year over
the Consumer Price Index for the base quarter of the preceding
year, adjusted to the nearest \1/10\ of 1 percent. For purposes
of this paragraph, the term ``base quarter'', as used with
respect to a year, means the calendar quarter ending on
September 30 of such year and the price index for a base
quarter is the arithmetical mean of such index for the 3 months
comprising such quarter.
(e) No person shall interfere with any officer, agent, or
employee of the Service in the performance of any duty imposed
upon him by this section or by regulations made by authority
thereof.
(f) Any person who shall violate, or aid or abet in
violating, any of the provisions of this section shall be
punished upon conviction by a fine not exceeding $500 or by
imprisonment not exceeding one year, or by both such fine and
imprisonment, in the discretion of the court.
(g) Nothing contained in this Act shall be construed as in
any way affecting, modifying, repealing, or superseding the
provisions of the Federal Food, Drug, and Cosmetic Act (U.S.C.,
1940 edition, title 21, ch. 9).
(h) A partially processed biological product which--
(1) is not in a form applicable to the prevention,
treatment, or cure of diseases or injuries of man;
(2) is not intended for sale in the United States;
and
(3) is intended for further manufacture into final
dosage form outside the United States,
shall be subject to no restriction on the export of the product
under this Act or the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 321 et. seq.) if the product is manufactured, processed,
packaged, and held in conformity with current good
manufacturing practice requirements or meets international
manufacturing standards as certified by an international
standards organization recognized by the Secretary and meets
the requirements of section 801(e)(1) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 381(e)).
(i) In this section:
(1) The term ``biological product'' means a virus,
therapeutic serum, toxin, antitoxin, vaccine, blood,
blood component or derivative, allergenic product,
protein (except any chemically synthesized
polypeptide), or analogous product, or arsphenamine or
derivative of arsphenamine (or any other trivalent
organic arsenic compound), applicable to the
prevention, treatment, or cure of a disease or
condition of human beings.
(2) The term ``biosimilar'' or ``biosimilarity'', in
reference to a biological product that is the subject
of an application under subsection (k), means--
(A) that the biological product is highly
similar to the reference product
notwithstanding minor differences in clinically
inactive components; and
(B) there are no clinically meaningful
differences between the biological product and
the reference product in terms of the safety,
purity, and potency of the product.
(3) The term ``interchangeable'' or
``interchangeability'', in reference to a biological
product that is shown to meet the standards described
in subsection (k)(4), means that the biological product
may be substituted for the reference product without
the intervention of the health care provider who
prescribed the reference product.
(4) The term ``reference product'' means the single
biological product licensed under subsection (a)
against which a biological product is evaluated in an
application submitted under subsection (k).
(j)(1) The Federal Food, Drug, and Cosmetic Act, including
the requirements under sections 505(o), 505(p), 505(z), and
505-1 of such Act, applies to a biological product subject to
regulation under this section, except that a product for which
a license has been approved under subsection (a) shall not be
required to have an approved application under section 505 of
such Act.
(2) In applying section 505(z) of the Federal Food, Drug, and
Cosmetic Act to the licensure of biological products under this
section--
(A) references to an antibacterial or antifungal drug
that is intended to treat a serious or life-threatening
infection shall be construed to refer to a biological
product intended to treat a serious or life-threatening
bacterial or fungal infection; and
(B) references to approval of a drug under section
505(c) of such Act shall be construed to refer to a
licensure of a biological product under subsection (a)
of this section.
(k) Licensure of Biological Products as Biosimilar or
Interchangeable.--
(1) In general.--Any person may submit an application
for licensure of a biological product under this
subsection.
(2) Content.--
(A) In general.--
(i) Required information.--An
application submitted under this
subsection shall include information
demonstrating that--
(I) the biological product is
biosimilar to a reference
product based upon data derived
from--
(aa) analytical
studies that
demonstrate that the
biological product is
highly similar to the
reference product
notwithstanding minor
differences in
clinically inactive
components;
(bb) animal studies
(including the
assessment of
toxicity); and
(cc) a clinical study
or studies (including
the assessment of
immunogenicity and
pharmacokinetics or
pharmacodynamics) that
are sufficient to
demonstrate safety,
purity, and potency in
1 or more appropriate
conditions of use for
which the reference
product is licensed and
intended to be used and
for which licensure is
sought for the
biological product;
(II) the biological product
and reference product utilize
the same mechanism or
mechanisms of action for the
condition or conditions of use
prescribed, recommended, or
suggested in the proposed
labeling, but only to the
extent the mechanism or
mechanisms of action are known
for the reference product;
(III) the condition or
conditions of use prescribed,
recommended, or suggested in
the labeling proposed for the
biological product have been
previously approved for the
reference product;
(IV) the route of
administration, the dosage
form, and the strength of the
biological product are the same
as those of the reference
product; and
(V) the facility in which the
biological product is
manufactured, processed,
packed, or held meets standards
designed to assure that the
biological product continues to
be safe, pure, and potent.
(ii) Determination by secretary.--The
Secretary may determine, in the
Secretary's discretion, that an element
described in clause (i)(I) is
unnecessary in an application submitted
under this subsection.
(iii) Additional information.--An
application submitted under this
subsection--
(I) shall include publicly-
available information regarding
the Secretary's previous
determination that the
reference product is safe,
pure, and potent; and
(II) may include any
additional information in
support of the application,
including publicly-available
information with respect to the
reference product or another
biological product.
(B) Interchangeability.--An application (or a
supplement to an application) submitted under
this subsection may include information
demonstrating that the biological product meets
the standards described in paragraph (4).
(3) Evaluation by secretary.--Upon review of an
application (or a supplement to an application)
submitted under this subsection, the Secretary shall
license the biological product under this subsection
if--
(A) the Secretary determines that the
information submitted in the application (or
the supplement) is sufficient to show that the
biological product--
(i) is biosimilar to the reference
product; or
(ii) meets the standards described in
paragraph (4), and therefore is
interchangeable with the reference
product; and
(B) the applicant (or other appropriate
person) consents to the inspection of the
facility that is the subject of the
application, in accordance with subsection (c).
(4) Safety standards for determining
interchangeability.--Upon review of an application
submitted under this subsection or any supplement to
such application, the Secretary shall determine the
biological product to be interchangeable with the
reference product if the Secretary determines that the
information submitted in the application (or a
supplement to such application) is sufficient to show
that--
(A) the biological product--
(i) is biosimilar to the reference
product; and
(ii) can be expected to produce the
same clinical result as the reference
product in any given patient; and
(B) for a biological product that is
administered more than once to an individual,
the risk in terms of safety or diminished
efficacy of alternating or switching between
use of the biological product and the reference
product is not greater than the risk of using
the reference product without such alternation
or switch.
(5) General rules.--
(A) One reference product per application.--A
biological product, in an application submitted
under this subsection, may not be evaluated
against more than 1 reference product.
(B) Review.--An application submitted under
this subsection shall be reviewed by the
division within the Food and Drug
Administration that is responsible for the
review and approval of the application under
which the reference product is licensed.
(C) Risk evaluation and mitigation
strategies.--The authority of the Secretary
with respect to risk evaluation and mitigation
strategies under the Federal Food, Drug, and
Cosmetic Act shall apply to biological products
licensed under this subsection in the same
manner as such authority applies to biological
products licensed under subsection (a).
(6) Exclusivity for first interchangeable biological
product.--Upon review of an application submitted under
this subsection relying on the same reference product
for which a prior biological product has received a
determination of interchangeability for any condition
of use, the Secretary shall not make a determination
under paragraph (4) that the second or subsequent
biological product is interchangeable for any condition
of use until the earlier of--
(A) 1 year after the first commercial
marketing of the first interchangeable
biosimilar biological product to be approved as
interchangeable for that reference product;
(B) 18 months after--
(i) a final court decision on all
patents in suit in an action instituted
under subsection (l)(6) against the
applicant that submitted the
application for the first approved
interchangeable biosimilar biological
product; or
(ii) the dismissal with or without
prejudice of an action instituted under
subsection (l)(6) against the applicant
that submitted the application for the
first approved interchangeable
biosimilar biological product; or
(C)(i) 42 months after approval of the first
interchangeable biosimilar biological product
if the applicant that submitted such
application has been sued under subsection
(l)(6) and such litigation is still ongoing
within such 42-month period; or
(ii) 18 months after approval of the first
interchangeable biosimilar biological product
if the applicant that submitted such
application has not been sued under subsection
(l)(6).
For purposes of this paragraph, the term ``final court
decision'' means a final decision of a court from which
no appeal (other than a petition to the United States
Supreme Court for a writ of certiorari) has been or can
be taken.
(7) Exclusivity for reference product.--
(A) Effective date of biosimilar application
approval.--Approval of an application under
this subsection may not be made effective by
the Secretary until the date that is 12 years
after the date on which the reference product
was first licensed under subsection (a).
(B) Filing period.--An application under this
subsection may not be submitted to the
Secretary until the date that is 4 years after
the date on which the reference product was
first licensed under subsection (a).
(C) First licensure.--Subparagraphs (A) and
(B) shall not apply to a license for or
approval of--
(i) a supplement for the biological
product that is the reference product;
or
(ii) a subsequent application filed
by the same sponsor or manufacturer of
the biological product that is the
reference product (or a licensor,
predecessor in interest, or other
related entity) for--
(I) a change (not including a
modification to the structure
of the biological product) that
results in a new indication,
route of administration, dosing
schedule, dosage form, delivery
system, delivery device, or
strength; or
(II) a modification to the
structure of the biological
product that does not result in
a change in safety, purity, or
potency.
(8) Guidance documents.--
(A) In general.--The Secretary may, after
opportunity for public comment, issue guidance
in accordance, except as provided in
subparagraph (B)(i), with section 701(h) of the
Federal Food, Drug, and Cosmetic Act with
respect to the licensure of a biological
product under this subsection. Any such
guidance may be general or specific.
(B) Public comment.--
(i) In general.--The Secretary shall
provide the public an opportunity to
comment on any proposed guidance issued
under subparagraph (A) before issuing
final guidance.
(ii) Input regarding most valuable
guidance.--The Secretary shall
establish a process through which the
public may provide the Secretary with
input regarding priorities for issuing
guidance.
(C) No requirement for application
consideration.--The issuance (or non-issuance)
of guidance under subparagraph (A) shall not
preclude the review of, or action on, an
application submitted under this subsection.
(D) Requirement for product class-specific
guidance.--If the Secretary issues product
class-specific guidance under subparagraph (A),
such guidance shall include a description of--
(i) the criteria that the Secretary
will use to determine whether a
biological product is highly similar to
a reference product in such product
class; and
(ii) the criteria, if available, that
the Secretary will use to determine
whether a biological product meets the
standards described in paragraph (4).
(E) Certain product classes.--
(i) Guidance.--The Secretary may
indicate in a guidance document that
the science and experience, as of the
date of such guidance, with respect to
a product or product class (not
including any recombinant protein) does
not allow approval of an application
for a license as provided under this
subsection for such product or product
class.
(ii) Modification or reversal.--The
Secretary may issue a subsequent
guidance document under subparagraph
(A) to modify or reverse a guidance
document under clause (i).
(iii) No effect on ability to deny
license.--Clause (i) shall not be
construed to require the Secretary to
approve a product with respect to which
the Secretary has not indicated in a
guidance document that the science and
experience, as described in clause (i),
does not allow approval of such an
application.
(l) Patents.--
(1) Confidential access to subsection (k)
application.--
(A) Application of paragraph.--Unless
otherwise agreed to by a person that submits an
application under subsection (k) (referred to
in this subsection as the ``subsection (k)
applicant'') and the sponsor of the application
for the reference product (referred to in this
subsection as the ``reference product
sponsor''), the provisions of this paragraph
shall apply to the exchange of information
described in this subsection.
(B) In general.--
(i) Provision of confidential
information.--When a subsection (k)
applicant submits an application under
subsection (k), such applicant shall
provide to the persons described in
clause (ii), subject to the terms of
this paragraph, confidential access to
the information required to be produced
pursuant to paragraph (2) and any other
information that the subsection (k)
applicant determines, in its sole
discretion, to be appropriate (referred
to in this subsection as the
``confidential information'').
(ii) Recipients of information.--The
persons described in this clause are
the following:
(I) Outside counsel.--One or
more attorneys designated by
the reference product sponsor
who are employees of an entity
other than the reference
product sponsor (referred to in
this paragraph as the ``outside
counsel''), provided that such
attorneys do not engage,
formally or informally, in
patent prosecution relevant or
related to the reference
product.
(II) In-house counsel.--One
attorney that represents the
reference product sponsor who
is an employee of the reference
product sponsor, provided that
such attorney does not engage,
formally or informally, in
patent prosecution relevant or
related to the reference
product.
(iii) Patent owner access.--A
representative of the owner of a patent
exclusively licensed to a reference
product sponsor with respect to the
reference product and who has retained
a right to assert the patent or
participate in litigation concerning
the patent may be provided the
confidential information, provided that
the representative informs the
reference product sponsor and the
subsection (k) applicant of his or her
agreement to be subject to the
confidentiality provisions set forth in
this paragraph, including those under
clause (ii).
(C) Limitation on disclosure.--No person that
receives confidential information pursuant to
subparagraph (B) shall disclose any
confidential information to any other person or
entity, including the reference product sponsor
employees, outside scientific consultants, or
other outside counsel retained by the reference
product sponsor, without the prior written
consent of the subsection (k) applicant, which
shall not be unreasonably withheld.
(D) Use of confidential information.--
Confidential information shall be used for the
sole and exclusive purpose of determining, with
respect to each patent assigned to or
exclusively licensed by the reference product
sponsor, whether a claim of patent infringement
could reasonably be asserted if the subsection
(k) applicant engaged in the manufacture, use,
offering for sale, sale, or importation into
the United States of the biological product
that is the subject of the application under
subsection (k).
(E) Ownership of confidential information.--
The confidential information disclosed under
this paragraph is, and shall remain, the
property of the subsection (k) applicant. By
providing the confidential information pursuant
to this paragraph, the subsection (k) applicant
does not provide the reference product sponsor
or the outside counsel any interest in or
license to use the confidential information,
for purposes other than those specified in
subparagraph (D).
(F) Effect of infringement action.--In the
event that the reference product sponsor files
a patent infringement suit, the use of
confidential information shall continue to be
governed by the terms of this paragraph until
such time as a court enters a protective order
regarding the information. Upon entry of such
order, the subsection (k) applicant may
redesignate confidential information in
accordance with the terms of that order. No
confidential information shall be included in
any publicly-available complaint or other
pleading. In the event that the reference
product sponsor does not file an infringement
action by the date specified in paragraph (6),
the reference product sponsor shall return or
destroy all confidential information received
under this paragraph, provided that if the
reference product sponsor opts to destroy such
information, it will confirm destruction in
writing to the subsection (k) applicant.
(G) Rule of construction.--Nothing in this
paragraph shall be construed--
(i) as an admission by the subsection
(k) applicant regarding the validity,
enforceability, or infringement of any
patent; or
(ii) as an agreement or admission by
the subsection (k) applicant with
respect to the competency, relevance,
or materiality of any confidential
information.
(H) Effect of violation.--The disclosure of
any confidential information in violation of
this paragraph shall be deemed to cause the
subsection (k) applicant to suffer irreparable
harm for which there is no adequate legal
remedy and the court shall consider immediate
injunctive relief to be an appropriate and
necessary remedy for any violation or
threatened violation of this paragraph.
(2) Subsection (k) application information.--Not
later than 20 days after the Secretary notifies the
subsection (k) applicant that the application has been
accepted for review, the subsection (k) applicant--
(A) shall provide to the reference product
sponsor a copy of the application submitted to
the Secretary under subsection (k), and such
other information that describes the process or
processes used to manufacture the biological
product that is the subject of such
application; and
(B) may provide to the reference product
sponsor additional information requested by or
on behalf of the reference product sponsor.
(3) List and description of patents.--
(A) List by reference product sponsor.--Not
later than 60 days after the receipt of the
application and information under paragraph
(2), the reference product sponsor shall
provide to the subsection (k) applicant--
(i) a list of patents for which the
reference product sponsor believes a
claim of patent infringement could
reasonably be asserted by the reference
product sponsor, or by a patent owner
that has granted an exclusive license
to the reference product sponsor with
respect to the reference product, if a
person not licensed by the reference
product sponsor engaged in the making,
using, offering to sell, selling, or
importing into the United States of the
biological product that is the subject
of the subsection (k) application; and
(ii) an identification of the patents
on such list that the reference product
sponsor would be prepared to license to
the subsection (k) applicant.
(B) List and description by subsection (k)
applicant.--Not later than 60 days after
receipt of the list under subparagraph (A), the
subsection (k) applicant--
(i) may provide to the reference
product sponsor a list of patents to
which the subsection (k) applicant
believes a claim of patent infringement
could reasonably be asserted by the
reference product sponsor if a person
not licensed by the reference product
sponsor engaged in the making, using,
offering to sell, selling, or importing
into the United States of the
biological product that is the subject
of the subsection (k) application;
(ii) shall provide to the reference
product sponsor, with respect to each
patent listed by the reference product
sponsor under subparagraph (A) or
listed by the subsection (k) applicant
under clause (i)--
(I) a detailed statement that
describes, on a claim by claim
basis, the factual and legal
basis of the opinion of the
subsection (k) applicant that
such patent is invalid,
unenforceable, or will not be
infringed by the commercial
marketing of the biological
product that is the subject of
the subsection (k) application;
or
(II) a statement that the
subsection (k) applicant does
not intend to begin commercial
marketing of the biological
product before the date that
such patent expires; and
(iii) shall provide to the reference
product sponsor a response regarding
each patent identified by the reference
product sponsor under subparagraph
(A)(ii).
(C) Description by reference product
sponsor.--Not later than 60 days after receipt
of the list and statement under subparagraph
(B), the reference product sponsor shall
provide to the subsection (k) applicant a
detailed statement that describes, with respect
to each patent described in subparagraph
(B)(ii)(I), on a claim by claim basis, the
factual and legal basis of the opinion of the
reference product sponsor that such patent will
be infringed by the commercial marketing of the
biological product that is the subject of the
subsection (k) application and a response to
the statement concerning validity and
enforceability provided under subparagraph
(B)(ii)(I).
(4) Patent resolution negotiations.--
(A) In general.--After receipt by the
subsection (k) applicant of the statement under
paragraph (3)(C), the reference product sponsor
and the subsection (k) applicant shall engage
in good faith negotiations to agree on which,
if any, patents listed under paragraph (3) by
the subsection (k) applicant or the reference
product sponsor shall be the subject of an
action for patent infringement under paragraph
(6).
(B) Failure to reach agreement.--If, within
15 days of beginning negotiations under
subparagraph (A), the subsection (k) applicant
and the reference product sponsor fail to agree
on a final and complete list of which, if any,
patents listed under paragraph (3) by the
subsection (k) applicant or the reference
product sponsor shall be the subject of an
action for patent infringement under paragraph
(6), the provisions of paragraph (5) shall
apply to the parties.
(5) Patent resolution if no agreement.--
(A) Number of patents.--The subsection (k)
applicant shall notify the reference product
sponsor of the number of patents that such
applicant will provide to the reference product
sponsor under subparagraph (B)(i)(I).
(B) Exchange of patent lists.--
(i) In general.--On a date agreed to
by the subsection (k) applicant and the
reference product sponsor, but in no
case later than 5 days after the
subsection (k) applicant notifies the
reference product sponsor under
subparagraph (A), the subsection (k)
applicant and the reference product
sponsor shall simultaneously exchange--
(I) the list of patents that
the subsection (k) applicant
believes should be the subject
of an action for patent
infringement under paragraph
(6); and
(II) the list of patents, in
accordance with clause (ii),
that the reference product
sponsor believes should be the
subject of an action for patent
infringement under paragraph
(6).
(ii) Number of patents listed by
reference product sponsor.--
(I) In general.--Subject to
subclause (II), the number of
patents listed by the reference
product sponsor under clause
(i)(II) may not exceed the
number of patents listed by the
subsection (k) applicant under
clause (i)(I).
(II) Exception.--If a
subsection (k) applicant does
not list any patent under
clause (i)(I), the reference
product sponsor may list 1
patent under clause (i)(II).
(6) Immediate patent infringement action.--
(A) Action if agreement on patent list.--If
the subsection (k) applicant and the reference
product sponsor agree on patents as described
in paragraph (4), not later than 30 days after
such agreement, the reference product sponsor
shall bring an action for patent infringement
with respect to each such patent.
(B) Action if no agreement on patent list.--
If the provisions of paragraph (5) apply to the
parties as described in paragraph (4)(B), not
later than 30 days after the exchange of lists
under paragraph (5)(B), the reference product
sponsor shall bring an action for patent
infringement with respect to each patent that
is included on such lists.
(C) Notification and publication of
complaint.--
(i) Notification to secretary.--Not
later than 30 days after a complaint is
served to a subsection (k) applicant in
an action for patent infringement
described under this paragraph, the
subsection (k) applicant shall provide
the Secretary with notice and a copy of
such complaint.
(ii) Publication by secretary.--The
Secretary shall publish in the Federal
Register notice of a complaint received
under clause (i).
(7) Newly issued or licensed patents.--In the case of
a patent that--
(A) is issued to, or exclusively licensed by,
the reference product sponsor after the date
that the reference product sponsor provided the
list to the subsection (k) applicant under
paragraph (3)(A); and
(B) the reference product sponsor reasonably
believes that, due to the issuance of such
patent, a claim of patent infringement could
reasonably be asserted by the reference product
sponsor if a person not licensed by the
reference product sponsor engaged in the
making, using, offering to sell, selling, or
importing into the United States of the
biological product that is the subject of the
subsection (k) application,
not later than 30 days after such issuance or
licensing, the reference product sponsor shall provide
to the subsection (k) applicant a supplement to the
list provided by the reference product sponsor under
paragraph (3)(A) that includes such patent, not later
than 30 days after such supplement is provided, the
subsection (k) applicant shall provide a statement to
the reference product sponsor in accordance with
paragraph (3)(B), and such patent shall be subject to
paragraph (8).
(8) Notice of commercial marketing and preliminary
injunction.--
(A) Notice of commercial marketing.--The
subsection (k) applicant shall provide notice
to the reference product sponsor not later than
180 days before the date of the first
commercial marketing of the biological product
licensed under subsection (k).
(B) Preliminary injunction.--After receiving
the notice under subparagraph (A) and before
such date of the first commercial marketing of
such biological product, the reference product
sponsor may seek a preliminary injunction
prohibiting the subsection (k) applicant from
engaging in the commercial manufacture or sale
of such biological product until the court
decides the issue of patent validity,
enforcement, and infringement with respect to
any patent that is--
(i) included in the list provided by
the reference product sponsor under
paragraph (3)(A) or in the list
provided by the subsection (k)
applicant under paragraph (3)(B); and
(ii) not included, as applicable,
on--
(I) the list of patents
described in paragraph (4); or
(II) the lists of patents
described in paragraph (5)(B).
(C) Reasonable cooperation.--If the reference
product sponsor has sought a preliminary
injunction under subparagraph (B), the
reference product sponsor and the subsection
(k) applicant shall reasonably cooperate to
expedite such further discovery as is needed in
connection with the preliminary injunction
motion.
(9) Limitation on declaratory judgment action.--
(A) Subsection (k) application provided.--If
a subsection (k) applicant provides the
application and information required under
paragraph (2)(A), neither the reference product
sponsor nor the subsection (k) applicant may,
prior to the date notice is received under
paragraph (8)(A), bring any action under
section 2201 of title 28, United States Code,
for a declaration of infringement, validity, or
enforceability of any patent that is described
in clauses (i) and (ii) of paragraph (8)(B).
(B) Subsequent failure to act by subsection
(k) applicant.--If a subsection (k) applicant
fails to complete an action required of the
subsection (k) applicant under paragraph
(3)(B)(ii), paragraph (5), paragraph (6)(C)(i),
paragraph (7), or paragraph (8)(A), the
reference product sponsor, but not the
subsection (k) applicant, may bring an action
under section 2201 of title 28, United States
Code, for a declaration of infringement,
validity, or enforceability of any patent
included in the list described in paragraph
(3)(A), including as provided under paragraph
(7).
(C) Subsection (k) application not
provided.--If a subsection (k) applicant fails
to provide the application and information
required under paragraph (2)(A), the reference
product sponsor, but not the subsection (k)
applicant, may bring an action under section
2201 of title 28, United States Code, for a
declaration of infringement, validity, or
enforceability of any patent that claims the
biological product or a use of the biological
product.
(m) Pediatric Studies.--
(1) Application of certain provisions.--The
provisions of subsections (a), (d), (e), (f), (h), (i),
(j), (k), (l), (n), and (p) of section 505A of the
Federal Food, Drug, and Cosmetic Act shall apply with
respect to the extension of a period under paragraphs
(2) and (3) to the same extent and in the same manner
as such provisions apply with respect to the extension
of a period under subsection (b) or (c) of section 505A
of the Federal Food, Drug, and Cosmetic Act.
(2) Market exclusivity for new biological products.--
If, prior to approval of an application that is
submitted under subsection (a), the Secretary
determines that information relating to the use of a
new biological product in the pediatric population may
produce health benefits in that population, the
Secretary makes a written request for pediatric studies
(which shall include a timeframe for completing such
studies), the applicant agrees to the request, such
studies are completed using appropriate formulations
for each age group for which the study is requested
within any such timeframe, and the reports thereof are
submitted and accepted in accordance with section
505A(d)(3) of the Federal Food, Drug, and Cosmetic
Act--
(A) the periods for such biological product
referred to in subsection (k)(7) are deemed to
be 4 years and 6 months rather than 4 years and
12 years and 6 months rather than 12 years; and
(B) if the biological product is designated
under section 526 for a rare disease or
condition, the period for such biological
product referred to in section 527(a) is deemed
to be 7 years and 6 months rather than 7 years.
(3) Market exclusivity for already-marketed
biological products.--If the Secretary determines that
information relating to the use of a licensed
biological product in the pediatric population may
produce health benefits in that population and makes a
written request to the holder of an approved
application under subsection (a) for pediatric studies
(which shall include a timeframe for completing such
studies), the holder agrees to the request, such
studies are completed using appropriate formulations
for each age group for which the study is requested
within any such timeframe, and the reports thereof are
submitted and accepted in accordance with section
505A(d)(3) of the Federal Food, Drug, and Cosmetic
Act--
(A) the periods for such biological product
referred to in subsection (k)(7) are deemed to
be 4 years and 6 months rather than 4 years and
12 years and 6 months rather than 12 years; and
(B) if the biological product is designated
under section 526 for a rare disease or
condition, the period for such biological
product referred to in section 527(a) is deemed
to be 7 years and 6 months rather than 7 years.
(4) Exception.--The Secretary shall not extend a
period referred to in paragraph (2)(A), (2)(B), (3)(A),
or (3)(B) if the determination under section 505A(d)(3)
is made later than 9 months prior to the expiration of
such period.
(5) Relation to exclusivity for a biological product
approved for a new indication for a rare disease or
condition.--Notwithstanding the references in
paragraphs (2)(A), (2)(B), (3)(A), and (3)(B) to the
lengths of the exclusivity periods after application of
pediatric exclusivity, the 6-month extensions described
in such paragraphs shall be in addition to any
extensions under section 505G.
* * * * * * *
PART P--ADDITIONAL PROGRAMS
* * * * * * *
SEC. 399V-6 SURVEILLANCE OF NEUROLOGICAL DISEASES.
(a) In General.--The Secretary, acting through the Director
of the Centers for Disease Control and Prevention and in
coordination with other agencies as determined appropriate by
the Secretary, shall--
(1) enhance and expand infrastructure and activities
to track the epidemiology of neurological diseases,
including multiple sclerosis and Parkinson's disease;
and
(2) incorporate information obtained through such
activities into a statistically sound, scientifically
credible, integrated surveillance system, to be known
as the National Neurological Diseases Surveillance
System.
(b) Research.--The Secretary shall ensure that the National
Neurological Diseases Surveillance System is designed in a
manner that facilitates further research on neurological
diseases.
(c) Content.--In carrying out subsection (a), the Secretary--
(1) shall provide for the collection and storage of
information on the incidence and prevalence of
neurological diseases in the United States;
(2) to the extent practicable, shall provide for the
collection and storage of other available information
on neurological diseases, such as information
concerning--
(A) demographics and other information
associated or possibly associated with
neurological diseases, such as age, race,
ethnicity, sex, geographic location, and family
history;
(B) risk factors associated or possibly
associated with neurological diseases,
including genetic and environmental risk
factors; and
(C) diagnosis and progression markers;
(3) may provide for the collection and storage of
information relevant to analysis on neurological
diseases, such as information concerning--
(A) the epidemiology of the diseases;
(B) the natural history of the diseases;
(C) the prevention of the diseases;
(D) the detection, management, and treatment
approaches for the diseases; and
(E) the development of outcomes measures; and
(4) may address issues identified during the
consultation process under subsection (d).
(d) Consultation.--In carrying out this section, the
Secretary shall consult with individuals with appropriate
expertise, including--
(1) epidemiologists with experience in disease
surveillance or registries;
(2) representatives of national voluntary health
associations that--
(A) focus on neurological diseases, including
multiple sclerosis and Parkinson's disease; and
(B) have demonstrated experience in research,
care, or patient services;
(3) health information technology experts or other
information management specialists;
(4) clinicians with expertise in neurological
diseases; and
(5) research scientists with experience conducting
translational research or utilizing surveillance
systems for scientific research purposes.
(e) Grants.--The Secretary may award grants to, or enter into
contracts or cooperative agreements with, public or private
nonprofit entities to carry out activities under this section.
(f) Coordination With Other Federal, State, and Local
Agencies.--Subject to subsection (h), the Secretary shall make
information and analysis in the National Neurological Diseases
Surveillance System available, as appropriate--
(1) to Federal departments and agencies, such as the
National Institutes of Health, the Food and Drug
Administration, the Centers for Medicare & Medicaid
Services, the Agency for Healthcare Research and
Quality, the Department of Veterans Affairs, and the
Department of Defense; and
(2) to State and local agencies.
(g) Public Access.--Subject to subsection (h), the Secretary
shall make information and analysis in the National
Neurological Diseases Surveillance System available, as
appropriate, to the public, including researchers.
(h) Privacy.--The Secretary shall ensure that privacy and
security protections applicable to the National Neurological
Diseases Surveillance System are at least as stringent as the
privacy and security protections under HIPAA privacy and
security law (as defined in section 3009(a)(2)).
(i) Report.--Not later than 4 years after the date of the
enactment of this section, the Secretary shall submit a report
to the Congress concerning the implementation of this section.
Such report shall include information on--
(1) the development and maintenance of the National
Neurological Diseases Surveillance System;
(2) the type of information collected and stored in
the System;
(3) the use and availability of such information,
including guidelines for such use; and
(4) the use and coordination of databases that
collect or maintain information on neurological
diseases.
(j) Definition.--In this section, the term ``national
voluntary health association'' means a national nonprofit
organization with chapters, other affiliated organizations, or
networks in States throughout the United States.
(k) Authorization of Appropriations.--To carry out this
section, there is authorized to be appropriated $5,000,000 for
each of fiscal years 2016 through 2020.
* * * * * * *
PART W--LYME DISEASE AND OTHER TICK-BORNE DISEASES
SEC. 399OO. RESEARCH.
(a) In General.--The Secretary shall conduct or support
epidemiological, basic, translational, and clinical research
regarding Lyme disease and other tick-borne diseases.
(b) Biennial Reports.--The Secretary shall ensure that each
biennial report under section 403 includes information on
actions undertaken by the National Institutes of Health to
carry out subsection (a) with respect to Lyme disease and other
tick-borne diseases, including an assessment of the progress
made in improving the outcomes of Lyme disease and such other
tick-borne diseases.
SEC. 399OO-1. WORKING GROUP.
(a) Establishment.--The Secretary shall establish a permanent
working group, to be known as the Interagency Lyme and Tick-
Borne Disease Working Group (in this section and section 399OO-
2 referred to as the ``Working Group''), to review all efforts
within the Department of Health and Human Services concerning
Lyme disease and other tick-borne diseases to ensure
interagency coordination, minimize overlap, and examine
research priorities.
(b) Responsibilities.--The Working Group shall--
(1) not later than 24 months after the date of
enactment of this part, and every 24 months thereafter,
develop or update a summary of--
(A) ongoing Lyme disease and other tick-borne
disease research related to causes, prevention,
treatment, surveillance, diagnosis,
diagnostics, duration of illness, intervention,
and access to services and supports for
individuals with Lyme disease or other tick-
borne diseases;
(B) advances made pursuant to such research;
(C) the engagement of the Department of
Health and Human Services with persons that
participate at the public meetings required by
paragraph (5); and
(D) the comments received by the Working
Group at such public meetings and the
Secretary's response to such comments;
(2) ensure that a broad spectrum of scientific
viewpoints is represented in each such summary;
(3) monitor Federal activities with respect to Lyme
disease and other tick-borne diseases;
(4) make recommendations to the Secretary regarding
any appropriate changes to such activities; and
(5) ensure public input by holding annual public
meetings that address scientific advances, research
questions, surveillance activities, and emerging
strains in species of pathogenic organisms.
(c) Membership.--
(1) In general.--The Working Group shall be composed
of a total of 14 members as follows:
(A) Federal members.--Seven Federal members,
consisting of one or more representatives of
each of--
(i) the Office of the Assistant
Secretary for Health;
(ii) the Food and Drug
Administration;
(iii) the Centers for Disease Control
and Prevention;
(iv) the National Institutes of
Health; and
(v) such other agencies and offices
of the Department of Health and Human
Services as the Secretary determines
appropriate.
(B) Non-federal public members.--Seven non-
Federal public members, consisting of
representatives of the following categories:
(i) Physicians and other medical
providers with experience in diagnosing
and treating Lyme disease and other
tick-borne diseases.
(ii) Scientists or researchers with
expertise.
(iii) Patients and their family
members.
(iv) Nonprofit organizations that
advocate for patients with respect to
Lyme disease and other tick-borne
diseases.
(v) Other individuals whose expertise
is determined by the Secretary to be
beneficial to the functioning of the
Working Group.
(2) Appointment.--The members of the Working Group
shall be appointed by the Secretary, except that of the
non-Federal public members under paragraph (1)(B)--
(A) one shall be appointed by the Speaker of
the House of Representatives; and
(B) one shall be appointed by the majority
leader of the Senate.
(3) Diversity of scientific perspectives.--In making
appointments under paragraph (2), the Secretary, the
Speaker of the House of Representatives, and the
majority leader of the Senate shall ensure that the
non-Federal public members of the Working Group
represent a diversity of scientific perspectives.
(4) Terms.--The non-Federal public members of the
Working Group shall each be appointed to serve a 4-year
term and may be reappointed at the end of such term.
(d) Meetings.--The Working Group shall meet as often as
necessary, as determined by the Secretary, but not less than
twice each year.
(e) Applicability of FACA.--The Working Group shall be
treated as an advisory committee subject to the Federal
Advisory Committee Act.
(f) Reporting.--Not later than 24 months after the date of
enactment of this part, and every 24 months thereafter, the
Working Group--
(1) shall submit a report on its activities,
including an up-to-date summary under subsection (b)(1)
and any recommendations under subsection (b)(4), to the
Secretary, the Committee on Energy and Commerce of the
House of Representatives, and the Committee on Health,
Education, Labor and Pensions of the Senate;
(2) shall make each such report publicly available on
the website of the Department of Health and Human
Services; and
(3) shall allow any member of the Working Group to
include in any such report minority views.
SEC. 399OO-2. STRATEGIC PLAN.
Not later than 3 years after the date of enactment of this
section, and every 5 years thereafter, the Secretary shall
submit to the Congress a strategic plan, informed by the most
recent summary under section 399OO-1(b)(1), for the conduct and
support of Lyme disease and tick-borne disease research,
including--
(1) proposed budgetary requirements;
(2) a plan for improving outcomes of Lyme disease and
other tick-borne diseases, including progress related
to chronic or persistent symptoms and chronic or
persistent infection and co-infections;
(3) a plan for improving diagnosis, treatment, and
prevention;
(4) appropriate benchmarks to measure progress on
achieving the improvements described in paragraphs (2)
and (3); and
(5) a plan to disseminate each summary under section
399OO-1(b)(1) and other relevant information developed
by the Working Group to the public, including health
care providers, public health departments, and other
relevant medical groups.
TITLE IV--NATIONAL RESEARCH INSTITUTES
Part A--National Institutes of Health
* * * * * * *
appointment and authority of director of nih
Sec. 402. (a) The National Institutes of Health shall be
headed by the Director of NIH who shall be appointed by the
President by and with the advice and consent of the Senate. The
Director of NIH shall perform functions as provided under
subsection (b) and as the Secretary may otherwise prescribe.
(b) In carrying out the purposes of section 301, the
Secretary, acting through the Director of NIH--
(1) shall carry out this title, including being
responsible for the overall direction of the National
Institutes of Health and for the establishment and
implementation of general policies respecting the
management and operation of programs and activities
within the National Institutes of Health;
(2) shall coordinate and oversee the operation of the
national research institutes, national centers, and
administrative entities within the National Institutes
of Health;
(3) shall, in consultation with the heads of the
national research institutes and national centers, be
responsible for program coordination across the
national research institutes and national centers,
including conducting priority-setting reviews, to
ensure that the research portfolio of the National
Institutes of Health is balanced and free of
unnecessary duplication, and takes advantage of
collaborative, cross-cutting research;
(4) shall assemble accurate data to be used to assess
research priorities, including information to better
evaluate scientific opportunity, public health burdens,
and progress in reducing minority and other health
disparities;
[(5) shall ensure that scientifically based strategic
planning is implemented in support of research
priorities as determined by the agencies of the
National Institutes of Health;]
(5) shall ensure that scientifically based strategic
planning is implemented in support of research
priorities as determined by the agencies of the
National Institutes of Health, including through
development, use, and updating of the research
strategic plan under subsection (m);
(6) shall ensure that the resources of the National
Institutes of Health are sufficiently allocated for
research projects identified in strategic plans;
(7)(A) shall, through the Division of Program
Coordination, Planning, and Strategic Initiatives--
(i) identify research that represents
important areas of emerging scientific
opportunities, rising public health
challenges, or knowledge gaps that
deserve special emphasis and would
benefit from conducting or supporting
additional research that involves
collaboration between 2 or more
national research institutes or
national centers, or would otherwise
benefit from strategic coordination and
planning;
(ii) include information on such
research in reports under section 403;
and
(iii) in the case of such research
supported with funds referred to in
subparagraph (B)--
(I) require as appropriate
that proposals include
milestones and goals for the
research;
(II) require that the
proposals include timeframes
for funding of the research;
and
(III) ensure appropriate
consideration of proposals for
which the principal
investigator is an individual
who has not previously served
as the principal investigator
of research conducted or
supported by the National
Institutes of Health;
(B)(i) may, with respect to funds reserved
under section 402A(c)(1) for the Common Fund,
allocate such funds to the national research
institutes and national centers for conducting
and supporting research that is identified
under subparagraph (A); and
(ii) shall, with respect to funds appropriated to the
Common Fund pursuant to section 402A(a)(2), allocate
such funds to the national research institutes and
national centers for making grants for pediatric
research that is identified under subparagraph (A); and
(C) may assign additional functions to the
Division in support of responsibilities
identified in subparagraph (A), as determined
appropriate by the Director;
(8) shall, in coordination with the heads of the
national research institutes and national centers,
ensure that such institutes and centers--
(A) preserve an emphasis on investigator-
initiated research project grants, including
with respect to research involving
collaboration between 2 or more such institutes
or centers; and
(B) when appropriate, maximize investigator-
initiated research project grants in their
annual research portfolios;
(9) shall ensure that research conducted or supported
by the National Institutes of Health is subject to
review in accordance with section 492 and that, after
such review, the research is reviewed in accordance
with section 492A(a)(2) by the appropriate advisory
council under section 406 before the research proposals
are approved for funding;
(10) shall have authority to review and approve the
establishment of all centers of excellence recommended
by the national research institutes;
(11)(A) shall oversee research training for all of
the national research institutes and National Research
Service Awards in accordance with section 487; and
(B) may conduct and support research training--
(i) for which fellowship support is not
provided under section 487; and
(ii) that does not consist of residency
training of physicians or other health
professionals;
(12) may, from funds appropriated under section
402A(b), reserve funds to provide for research on
matters that have not received significant funding
relative to other matters, to respond to new issues and
scientific emergencies, and to act on research
opportunities of high priority;
(13) may, subject to appropriations Acts, collect and
retain registration fees obtained from third parties to
defray expenses for scientific, educational, and
research-related conferences;
(14) for the national research institutes and
administrative entities within the National Institutes
of Health--
(A) may acquire, construct, improve, repair,
operate, and maintain, at the site of such
institutes and entities, laboratories, and
other research facilities, other facilities,
equipment, and other real or personal property,
and
(B) may acquire, without regard to the Act of
March 3, 1877 (40 U.S.C. 34), by lease or
otherwise through the Administrator of General
Services, buildings or parts of buildings in
the District of Columbia or communities located
adjacent to the District of Columbia for use
for a period not to exceed ten years;
(15) may secure resources for research conducted by
or through the National Institutes of Health;
(16) may, without regard to the provisions of title
5, United States Code, governing appointments in the
competitive service, and without regard to the
provisions of chapter 51 and subchapter III of chapter
53 of such title relating to classification and General
Schedule pay rates, establish such technical and
scientific peer review groups and scientific program
advisory committees as are needed to carry out the
requirements of this title and appoint and pay the
members of such groups, except that officers and
employees of the United States shall not receive
additional compensation for service as members of such
groups;
(17) may secure for the National Institutes of Health
consultation services and advice of persons from the
United States or abroad;
(18) may use, with their consent, the services,
equipment, personnel, information, and facilities of
other Federal, State, or local public agencies, with or
without reimbursement therefor;
(19) may, for purposes of study, admit and treat at
facilities of the National Institutes of Health
individuals not otherwise eligible for such treatment;
(20) may accept voluntary and uncompensated services;
(21) may perform such other administrative functions
as the Secretary determines are needed to effectively
carry out this title;
(22) may appoint physicians, dentists, and other
health care professionals, subject to the provisions of
title 5, United States Code, relating to appointments
and classifications in the competitive service, and may
compensate such professionals subject to the provisions
of chapter 74 of title 38, United States Code;
(23) shall designate a contact point or office to
help innovators and physicians identify sources of
funding available for pediatric medical device
development; [and]
(24) implement the Cures Acceleration Network
described in section 480[.]; and
(25) shall, with respect to funds appropriated under
section 402A(e) to the NIH Innovation Fund, allocate
such funds to the national research institutes and
national centers for conducting and supporting
innovation fund initiatives identified under paragraph
(3) of such section.
The Federal Advisory Committee Act shall not apply to the
duration of a peer review group appointed under paragraph (16).
The members of such a group shall be individuals who by virtue
of their training or experience are eminently qualified to
perform the review functions of such group. Not more than one-
fourth of the members of any such group shall be officers or
employees of the United States.
(c) The Director of NIH may make available to individuals and
entities, for biomedical and behavioral research, substances
and living organisms. Such substances and organisms shall be
made available under such terms and conditions (including
payment for them) as the Secretary determines appropriate.
(d)(1) The Director of NIH may obtain (in accordance with
section 3109 of title 5, United States Code, but without regard
to the limitation in such section on the period of service) the
services of not more than 220 experts or consultants, with
scientific or other professional qualifications, for the
National Institutes of Health.
(2)(A) Except as provided in subparagraph (B), experts and
consultants whose services are obtained under paragraph (1)
shall be paid or reimbursed, in accordance with title 5, United
States Code, for their travel to and from their place of
service and for other expenses associated with their
assignment.
(B) Expenses specified in subparagraph (A) shall not be
allowed in connection with the assignment of an expert or
consultant whose services are obtained under paragraph (1)
unless the expert or consultant has agreed in writing to
complete the entire period of the assignment or one year of the
assignment, whichever is shorter, unless separated or
reassigned for reasons which are beyond the control of the
expert or consultant and which are acceptable to the Secretary.
If the expert or consultant violates the agreement, the money
spent by the United States for such expenses is recoverable
from the expert or consultant as a debt due the United States.
The Secretary may waive in whole or in part a right of recovery
under this subparagraph.
(e) The Director of NIH shall--
(1) advise the agencies of the National Institutes of
Health on medical applications of research;
(2) coordinate, review, and facilitate the systematic
identification and evaluation of, clinically relevant
information from research conducted by or through the
national research institutes;
(3) promote the effective transfer of the information
described in paragraph (2) to the health care community
and to entities that require such information;
(4) monitor the effectiveness of the activities
described in paragraph (3); and
(5) ensure that, after January 1, 1994, all new or
revised health education and promotion materials
developed or funded by the National Institutes of
Health and intended for the general public are in a
form that does not exceed a level of functional
literacy, as defined in the National Literacy Act of
1991 (Public Law 102-73).
(f) There shall be in the National Institutes of Health an
Associate Director for Prevention. The Director of NIH shall
delegate to the Associate Director for Prevention the functions
of the Director relating to the promotion of the disease
prevention research programs of the national research
institutes and the coordination of such programs among the
national research institutes and between the national research
institutes and other public and private entities, including
elementary, secondary, and post-secondary schools. The
Associate Director shall--
(1) annually review the efficacy of existing policies
and techniques used by the national research institutes
to disseminate the results of disease prevention and
behavioral research programs; and
(2) recommend, coordinate, and oversee the
modification or reconstruction of such policies and
techniques to ensure maximum dissemination, using
advanced technologies to the maximum extent
practicable, of research results to such entities.
(h) The Secretary, acting through the Director of NIH and the
Directors of the agencies of the National Institutes of Health,
shall, in conducting and supporting programs for research,
research training, recruitment, and other activities, provide
for an increase in the number of women and individuals from
disadvantaged backgrounds (including racial and ethnic
minorities) in the fields of biomedical and behavioral
research.
(i)(1)(A) The Secretary, acting through the Director of NIH,
shall establish, maintain, and operate a data bank of
information on clinical trials for drugs for serious or life-
threatening diseases and conditions (in this subsection
referred to as the ``data bank''). The activities of the data
bank shall be integrated and coordinated with related
activities of other agencies of the Department of Health and
Human Services, and to the extent practicable, coordinated with
other data banks containing similar information.
(B) The Secretary shall establish the data bank after
consultation with the Commissioner of Food and Drugs, the
directors of the appropriate agencies of the National
Institutes of Health (including the National Library of
Medicine), and the Director of the Centers for Disease Control
and Prevention.
(2) In carrying out paragraph (1), the Secretary shall
collect, catalog, store, and disseminate the information
described in such paragraph. The Secretary shall disseminate
such information through information systems, which shall
include toll-free telephone communications, available to
individuals with serious or life-threatening diseases and
conditions, to other members of the public, to health care
providers, and to researchers.
(3) The data bank shall include the following:
(A) A registry of clinical trials (whether federally
or privately funded) of experimental treatments for
serious or life-threatening diseases and conditions
under regulations promulgated pursuant to section
505(i) of the Federal Food, Drug, and Cosmetic Act,
which provides a description of the purpose of each
experimental drug, either with the consent of the
protocol sponsor, or when a trial to test effectiveness
begins. Information provided shall consist of
eligibility criteria for participation in the clinical
trials, a description of the location of trial sites,
and a point of contact for those wanting to enroll in
the trial, and shall be in a form that can be readily
understood by members of the public. Such information
shall be forwarded to the data bank by the sponsor of
the trial not later than 21 days after the approval of
the protocol.
(B) Information pertaining to experimental treatments
for serious or life-threatening diseases and conditions
that may be available--
(i) under a treatment investigational new
drug application that has been submitted to the
Secretary under section 561(c) of the Federal
Food, Drug, and Cosmetic Act; or
(ii) as a Group C cancer drug (as defined by
the National Cancer Institute).
The data bank may also include information pertaining
to the results of clinical trials of such treatments,
with the consent of the sponsor, including information
concerning potential toxicities or adverse effects
associated with the use or administration of such
experimental treatments.
(4) The data bank shall not include information relating to
an investigation if the sponsor has provided a detailed
certification to the Secretary that disclosure of such
information would substantially interfere with the timely
enrollment of subjects in the investigation, unless the
Secretary, after the receipt of the certification, provides the
sponsor with a detailed written determination that such
disclosure would not substantially interfere with such
enrollment.
(5) Fees collected under section 736 of the Federal Food,
Drug, and Cosmetic Act shall not be used in carrying out this
subsection.
(j) Expanded Clinical Trial Registry Data Bank.--
(1) Definitions; requirement.--
(A) Definitions.--In this subsection:
(i) Applicable clinical trial.--The
term ``applicable clinical trial''
means an applicable device clinical
trial or an applicable drug clinical
trial.
(ii) Applicable device clinical
trial.--The term ``applicable device
clinical trial'' means--
(I) a prospective clinical
study of health outcomes
comparing an intervention with
a device subject to section
510(k), 515, or 520(m) of the
Federal Food, Drug, and
Cosmetic Act against a control
in human subjects (other than a
small clinical trial to
determine the feasibility of a
device, or a clinical trial to
test prototype devices where
the primary outcome measure
relates to feasibility and not
to health outcomes); and
(II) a pediatric postmarket
surveillance as required under
section 522 of the Federal
Food, Drug, and Cosmetic Act.
(iii) Applicable drug clinical
trial.--
(I) In general.--The term
``applicable drug clinical
trial'' means a controlled
clinical investigation, other
than a phase I clinical
investigation, of a drug
subject to section 505 of the
Federal Food, Drug, and
Cosmetic Act or to section 351
of this Act.
(II) Clinical
investigation.--For purposes of
subclause (I), the term
``clinical investigation'' has
the meaning given that term in
section 312.3 of title 21, Code
of Federal Regulations (or any
successor regulation).
(III) Phase i.--For purposes
of subclause (I), the term
``phase I'' has the meaning
given that term in section
312.21 of title 21, Code of
Federal Regulations (or any
successor regulation).
(iv) Clinical trial information.--The
term ``clinical trial information''
means, with respect to an applicable
clinical trial, those data elements
that the responsible party is required
to submit under paragraph (2) or under
paragraph (3).
(v) Completion date.--The term
``completion date'' means, with respect
to an applicable clinical trial, the
date that the final subject was
examined or received an intervention
for the purposes of final collection of
data for the primary outcome, whether
the clinical trial concluded according
to the prespecified protocol or was
terminated.
(vi) Device.--The term ``device''
means a device as defined in section
201(h) of the Federal Food, Drug, and
Cosmetic Act.
(vii) Drug.--The term ``drug'' means
a drug as defined in section 201(g) of
the Federal Food, Drug, and Cosmetic
Act or a biological product as defined
in section 351 of this Act.
(viii) Ongoing.--The term ``ongoing''
means, with respect to a clinical trial
of a drug or a device and to a date,
that--
(I) 1 or more patients is
enrolled in the clinical trial;
and
(II) the date is before the
completion date of the clinical
trial.
(ix) Responsible party.--The term
``responsible party'', with respect to
a clinical trial of a drug or device,
means--
(I) the sponsor of the
clinical trial (as defined in
section 50.3 of title 21, Code
of Federal Regulations (or any
successor regulation)); or
(II) the principal
investigator of such clinical
trial if so designated by a
sponsor, grantee, contractor,
or awardee, so long as the
principal investigator is
responsible for conducting the
trial, has access to and
control over the data from the
clinical trial, has the right
to publish the results of the
trial, and has the ability to
meet all of the requirements
under this subsection for the
submission of clinical trial
information.
(B) Requirement.--The Secretary shall develop
a mechanism by which the responsible party for
each applicable clinical trial shall submit the
identity and contact information of such
responsible party to the Secretary at the time
of submission of clinical trial information
under paragraph (2).
(2) Expansion of clinical trial registry data bank
with respect to clinical trial information.--
(A) In general.--
(i) Expansion of data bank.--To
enhance patient enrollment and provide
a mechanism to track subsequent
progress of clinical trials, the
Secretary, acting through the Director
of NIH, shall expand, in accordance
with this subsection, the clinical
trials registry of the data bank
described under subsection (i)(1)
(referred to in this subsection as the
``registry data bank''). The Director
of NIH shall ensure that the registry
data bank is made publicly available
through the Internet.
(ii) Content.--The clinical trial
information required to be submitted
under this paragraph for an applicable
clinical trial shall include--
(I) descriptive information,
including--
(aa) a brief title,
intended for the lay
public;
(bb) a brief summary,
intended for the lay
public;
(cc) the primary
purpose;
(dd) the study
design;
(ee) for an
applicable drug
clinical trial, the
study phase;
(ff) study type;
(gg) the primary
disease or condition
being studied, or the
focus of the study;
(hh) the intervention
name and intervention
type;
(ii) the study start
date;
(jj) the expected
completion date;
(kk) the target
number of subjects; and
(ll) outcomes,
including primary and
secondary outcome
measures;
(II) recruitment information,
including--
(aa) eligibility
criteria;
(bb) gender;
(cc) age limits;
(dd) whether the
trial accepts healthy
volunteers;
(ee) overall
recruitment status;
(ff) individual site
status; and
(gg) in the case of
an applicable drug
clinical trial, if the
drug is not approved
under section 505 of
the Federal Food, Drug,
and Cosmetic Act or
licensed under section
351 of this Act,
specify whether or not
there is expanded
access to the drug
under section 561 of
the Federal Food, Drug,
and Cosmetic Act for
those who do not
qualify for enrollment
in the clinical trial
and how to obtain
information about such
access;
(III) location and contact
information, including--
(aa) the name of the
sponsor;
(bb) the responsible
party, by official
title; and
(cc) the facility
name and facility
contact information
(including the city,
State, and zip code for
each clinical trial
location, or a toll-
free number through
which such location
information may be
accessed); and
(IV) administrative data
(which the Secretary may make
publicly available as
necessary), including--
(aa) the unique
protocol identification
number;
(bb) other protocol
identification numbers,
if any; and
(cc) the Food and
Drug Administration
IND/IDE protocol number
and the record
verification date.
(iii) Modifications.--The Secretary
may by regulation modify the
requirements for clinical trial
information under this paragraph, if
the Secretary provides a rationale for
why such a modification improves and
does not reduce such clinical trial
information.
(B) Format and structure.--
(i) Searchable categories.--The
Director of NIH shall ensure that the
public may, in addition to keyword
searching, search the entries in the
registry data bank by 1 or more of the
following criteria:
(I) The disease or condition
being studied in the clinical
trial, using Medical Subject
Headers (MeSH) descriptors.
(II) The name of the
intervention, including any
drug or device being studied in
the clinical trial.
(III) The location of the
clinical trial.
(IV) The age group studied in
the clinical trial, including
pediatric subpopulations.
(V) The study phase of the
clinical trial.
(VI) The sponsor of the
clinical trial, which may be
the National Institutes of
Health or another Federal
agency, a private industry
source, or a university or
other organization.
(VII) The recruitment status
of the clinical trial.
(VIII) The National Clinical
Trial number or other study
identification for the clinical
trial.
(ii) Additional searchable
category.--Not later than 18 months
after the date of the enactment of the
Food and Drug Administration Amendments
Act of 2007, the Director of NIH shall
ensure that the public may search the
entries of the registry data bank by
the safety issue, if any, being studied
in the clinical trial as a primary or
secondary outcome.
(iii) Other elements.--The Director
of NIH shall also ensure that the
public may search the entries of the
registry data bank by such other
elements as the Director deems
necessary on an ongoing basis.
(iv) Format.--The Director of the NIH
shall ensure that the registry data
bank is easily used by the public, and
that entries are easily compared.
(C) Data submission.--The responsible party
for an applicable clinical trial, including an
applicable drug clinical trial for a serious or
life-threatening disease or condition, that is
initiated after, or is ongoing on the date that
is 90 days after, the date of the enactment of
the Food and Drug Administration Amendments Act
of 2007, shall submit to the Director of NIH
for inclusion in the registry data bank the
clinical trial information described in of
subparagraph (A)(ii) not later than the later
of--
(i) 90 days after such date of
enactment;
(ii) 21 days after the first patient
is enrolled in such clinical trial; or
(iii) in the case of a clinical trial
that is not for a serious or life-
threatening disease or condition and
that is ongoing on such date of
enactment, 1 year after such date of
enactment.
(D) Posting of data.--
(i) Applicable drug clinical trial.--
The Director of NIH shall ensure that
clinical trial information for an
applicable drug clinical trial
submitted in accordance with this
paragraph is posted in the registry
data bank not later than 30 days after
such submission.
(ii) Applicable device clinical
trial.--The Director of NIH shall
ensure that clinical trial information
for an applicable device clinical trial
submitted in accordance with this
paragraph is posted publicly in the
registry data bank--
(I) not earlier than the date
of clearance under section
510(k) of the Federal Food,
Drug, and Cosmetic Act, or
approval under section 515 or
520(m) of such Act, as
applicable, for a device that
was not previously cleared or
approved, and not later than 30
days after such date; or
(II) for a device that was
previously cleared or approved,
not later than 30 days after
the clinical trial information
under paragraph (3)(C) is
required to be posted by the
Secretary.
(3) Expansion of registry data bank to include
results of clinical trials.--
(A) Linking registry data bank to existing
results.--
(i) In general.--Beginning not later
than 90 days after the date of the
enactment of the Food and Drug
Administration Amendments Act of 2007,
for those clinical trials that form the
primary basis of an efficacy claim or
are conducted after the drug involved
is approved or after the device
involved is cleared or approved, the
Secretary shall ensure that the
registry data bank includes links to
results information as described in
clause (ii) for such clinical trial--
(I) not earlier than 30 days
after the date of the approval
of the drug involved or
clearance or approval of the
device involved; or
(II) not later than 30 days
after the results information
described in clause (ii)
becomes publicly available.
(ii) Required information.--
(I) FDA information.--The
Secretary shall ensure that the
registry data bank includes
links to the following
information:
(aa) If an advisory
committee considered at
a meeting an applicable
clinical trial, any
posted Food and Drug
Administration summary
document regarding such
applicable clinical
trial.
(bb) If an applicable
drug clinical trial was
conducted under section
505A or 505B of the
Federal Food, Drug, and
Cosmetic Act, a link to
the posted Food and
Drug Administration
assessment of the
results of such trial.
(cc) Food and Drug
Administration public
health advisories
regarding the drug or
device that is the
subject of the
applicable clinical
trial, if any.
(dd) For an
applicable drug
clinical trial, the
Food and Drug
Administration action
package for approval
document required under
section 505(l)(2) of
the Federal Food, Drug,
and Cosmetic Act.
(ee) For an
applicable device
clinical trial, in the
case of a premarket
application under
section 515 of the
Federal Food, Drug, and
Cosmetic Act, the
detailed summary of
information respecting
the safety and
effectiveness of the
device required under
section 520(h)(1) of
such Act, or, in the
case of a report under
section 510(k) of such
Act, the section 510(k)
summary of the safety
and effectiveness data
required under section
807.95(d) of title 21,
Code of Federal
Regulations (or any
successor regulation).
(II) NIH information.--The
Secretary shall ensure that the
registry data bank includes
links to the following
information:
(aa) Medline
citations to any
publications focused on
the results of an
applicable clinical
trial.
(bb) The entry for
the drug that is the
subject of an
applicable drug
clinical trial in the
National Library of
Medicine database of
structured product
labels, if available.
(iii) Results for existing data bank
entries.--The Secretary may include the
links described in clause (ii) for data
bank entries for clinical trials
submitted to the data bank prior to
enactment of the Food and Drug
Administration Amendments Act of 2007,
as available.
(B) Inclusion of results.--The Secretary,
acting through the Director of NIH, shall--
(i) expand the registry data bank to
include the results of applicable
clinical trials (referred to in this
subsection as the ``registry and
results data bank'');
(ii) ensure that such results are
made publicly available through the
Internet;
(iii) post publicly a glossary for
the lay public explaining technical
terms related to the results of
clinical trials; and
(iv) in consultation with experts on
risk communication, provide information
with the information included under
subparagraph (C) in the registry and
results data bank to help ensure that
such information does not mislead the
patients or the public.
(C) Basic results.--Not later than 1 year
after the date of the enactment of the Food and
Drug Administration Amendments Act of 2007, the
Secretary shall include in the registry and
results data bank for each applicable clinical
trial for a drug that is approved under section
505 of the Federal Food, Drug, and Cosmetic Act
or licensed under section 351 of this Act or a
device that is cleared under section 510(k) of
the Federal Food, Drug, and Cosmetic Act or
approved under section 515 or 520(m) of such
Act, the following elements:
(i) Demographic and baseline
characteristics of patient sample.--A
table of the demographic and baseline
data collected overall and for each arm
of the clinical trial to describe the
patients who participated in the
clinical trial, including the number of
patients who dropped out of the
clinical trial and the number of
patients excluded from the analysis, if
any.
(ii) Primary and secondary
outcomes.--The primary and secondary
outcome measures as submitted under
paragraph (2)(A)(ii)(I)(ll), and a
table of values for each of the primary
and secondary outcome measures for each
arm of the clinical trial, including
the results of scientifically
appropriate tests of the statistical
significance of such outcome measures.
(iii) Point of contact.--A point of
contact for scientific information
about the clinical trial results.
(iv) Certain agreements.--Whether
there exists an agreement (other than
an agreement solely to comply with
applicable provisions of law protecting
the privacy of participants) between
the sponsor or its agent and the
principal investigator (unless the
sponsor is an employer of the principal
investigator) that restricts in any
manner the ability of the principal
investigator, after the completion date
of the trial, to discuss the results of
the trial at a scientific meeting or
any other public or private forum, or
to publish in a scientific or academic
journal information concerning the
results of the trial.
(D) Expanded registry and results data
bank.--
(i) Expansion by rulemaking.--To
provide more complete results
information and to enhance patient
access to and understanding of the
results of clinical trials, not later
than 3 years after the date of the
enactment of the Food and Drug
Administration Amendments Act of 2007,
the Secretary shall by regulation
expand the registry and results data
bank as provided under this
subparagraph.
(ii) Clinical trials.--
(I) Approved products.--The
regulations under this
subparagraph shall require the
inclusion of the results
information described in clause
(iii) for--
(aa) each applicable
drug clinical trial for
a drug that is approved
under section 505 of
the Federal Food, Drug,
and Cosmetic Act or
licensed under section
351 of this Act; and
(bb) each applicable
device clinical trial
for a device that is
cleared under section
510(k) of the Federal
Food, Drug, and
Cosmetic Act or
approved under section
515 or 520(m) of such
Act.
(II) Unapproved products.--
The regulations under this
subparagraph shall establish
whether or not the results
information described in clause
(iii) shall be required for--
(aa) an applicable
drug clinical trial for
a drug that is not
approved under section
505 of the Federal
Food, Drug, and
Cosmetic Act and not
licensed under section
351 of this Act
(whether approval or
licensure was sought or
not); and
(bb) an applicable
device clinical trial
for a device that is
not cleared under
section 510(k) of the
Federal Food, Drug, and
Cosmetic Act and not
approved under section
515 or section 520(m)
of such Act (whether
clearance or approval
was sought or not).
(iii) Required elements.--The
regulations under this subparagraph
shall require, in addition to the
elements described in subparagraph (C),
information within each of the
following categories:
(I) A summary of the clinical
trial and its results that is
written in non-technical,
understandable language for
patients, if the Secretary
determines that such types of
summary can be included without
being misleading or
promotional.
(II) A summary of the
clinical trial and its results
that is technical in nature, if
the Secretary determines that
such types of summary can be
included without being
misleading or promotional.
(III) The full protocol or
such information on the
protocol for the trial as may
be necessary to help to
evaluate the results of the
trial.
(IV) Such other categories as
the Secretary determines
appropriate.
(iv) Results submission.--The results
information described in clause (iii)
shall be submitted to the Director of
NIH for inclusion in the registry and
results data bank as provided by
subparagraph (E), except that the
Secretary shall by regulation
determine--
(I) whether the 1-year period
for submission of clinical
trial information described in
subparagraph (E)(i) should be
increased from 1 year to a
period not to exceed 18 months;
(II) whether the clinical
trial information described in
clause (iii) should be required
to be submitted for an
applicable clinical trial for
which the clinical trial
information described in
subparagraph (C) is submitted
to the registry and results
data bank before the effective
date of the regulations issued
under this subparagraph; and
(III) in the case when the
clinical trial information
described in clause (iii) is
required to be submitted for
the applicable clinical trials
described in clause (ii)(II),
the date by which such clinical
trial information shall be
required to be submitted,
taking into account--
(aa) the
certification process
under subparagraph
(E)(iii) when approval,
licensure, or clearance
is sought; and
(bb) whether there
should be a delay of
submission when
approval, licensure, or
clearance will not be
sought.
(v) Additional provisions.--The
regulations under this subparagraph
shall also establish--
(I) a standard format for the
submission of clinical trial
information under this
paragraph to the registry and
results data bank;
(II) additional information
on clinical trials and results
that is written in
nontechnical, understandable
language for patients;
(III) considering the
experience under the pilot
quality control project
described in paragraph (5)(C),
procedures for quality control,
including using representative
samples, with respect to
completeness and content of
clinical trial information
under this subsection, to help
ensure that data elements are
not false or misleading and are
non-promotional;
(IV) the appropriate timing
and requirements for updates of
clinical trial information, and
whether and, if so, how such
updates should be tracked;
(V) a statement to accompany
the entry for an applicable
clinical trial when the primary
and secondary outcome measures
for such clinical trial are
submitted under paragraph
(4)(A) after the date specified
for the submission of such
information in paragraph
(2)(C); and
(VI) additions or
modifications to the manner of
reporting of the data elements
established under subparagraph
(C).
(vi) Consideration of world health
organization data set.--The Secretary
shall consider the status of the
consensus data elements set for
reporting clinical trial results of the
World Health Organization when issuing
the regulations under this
subparagraph.
(vii) Public meeting.--The Secretary
shall hold a public meeting no later
than 18 months after the date of the
enactment of the Food and Drug
Administration Amendments Act of 2007
to provide an opportunity for input
from interested parties with regard to
the regulations to be issued under this
subparagraph.
(E) Submission of results information.--
(i) In general.--Except as provided
in clauses (iii), (iv), (v), and (vi)
the responsible party for an applicable
clinical trial that is described in
clause (ii) shall submit to the
Director of NIH for inclusion in the
registry and results data bank the
clinical trial information described in
subparagraph (C) not later than 1 year,
or such other period as may be provided
by regulation under subparagraph (D),
after the earlier of--
(I) the estimated completion
date of the trial as described
in paragraph
(2)(A)(ii)(I)(jj)); or
(II) the actual date of
completion.
(ii) Clinical trials described.--An
applicable clinical trial described in
this clause is an applicable clinical
trial subject to--
(I) paragraph (2)(C); and
(II)(aa) subparagraph (C); or
(bb) the regulations issued
under subparagraph (D).
(iii) Delayed submission of results
with certification.--If the responsible
party for an applicable clinical trial
submits a certification that clause
(iv) or (v) applies to such clinical
trial, the responsible party shall
submit to the Director of NIH for
inclusion in the registry and results
data bank the clinical trial
information described in subparagraphs
(C) and (D) as required under the
applicable clause.
(iv) Seeking initial approval of a
drug or device.--With respect to an
applicable clinical trial that is
completed before the drug is initially
approved under section 505 of the
Federal Food, Drug, and Cosmetic Act or
initially licensed under section 351 of
this Act, or the device is initially
cleared under section 510(k) or
initially approved under section 515 or
520(m) of the Federal Food, Drug, and
Cosmetic Act, the responsible party
shall submit to the Director of NIH for
inclusion in the registry and results
data bank the clinical trial
information described in subparagraphs
(C) and (D) not later than 30 days
after the drug or device is approved
under such section 505, licensed under
such section 351, cleared under such
section 510(k), or approved under such
section 515 or 520(m), as applicable.
(v) Seeking approval of a new use for
the drug or device.--
(I) In general.--With respect
to an applicable clinical trial
where the manufacturer of the
drug or device is the sponsor
of an applicable clinical
trial, and such manufacturer
has filed, or will file within
1 year, an application seeking
approval under section 505 of
the Federal Food, Drug, and
Cosmetic Act, licensing under
section 351 of this Act, or
clearance under section 510(k),
or approval under section 515
or 520(m), of the Federal Food,
Drug, and Cosmetic Act for the
use studied in such clinical
trial (which use is not
included in the labeling of the
approved drug or device), then
the responsible party shall
submit to the Director of NIH
for inclusion in the registry
and results data bank the
clinical trial information
described in subparagraphs (C)
and (D) on the earlier of the
date that is 30 days after the
date--
(aa) the new use of
the drug or device is
approved under such
section 505, licensed
under such section 351,
cleared under such
section 510(k), or
approved under such
section 515 or 520(m);
(bb) the Secretary
issues a letter, such
as a complete response
letter, not approving
the submission or not
clearing the
submission, a not
approvable letter, or a
not substantially
equivalent letter for
the new use of the drug
or device under such
section 505, 351,
510(k), 515, or 520(m);
or
(cc) except as
provided in subclause
(III), the application
or premarket
notification under such
section 505, 351,
510(k), 515, or 520(m)
is withdrawn without
resubmission for no
less than 210 days.
(II) Requirement that each
clinical trial in application
be treated the same.--If a
manufacturer makes a
certification under clause
(iii) that this clause applies
with respect to a clinical
trial, the manufacturer shall
make such a certification with
respect to each applicable
clinical trial that is required
to be submitted in an
application or report for
licensure, approval, or
clearance (under section 351 of
this Act or section 505,
510(k), 515, or 520(m) of the
Federal Food, Drug, and
Cosmetic Act, as applicable) of
the use studied in the clinical
trial.
(III) Two-year limitation.--
The responsible party shall
submit to the Director of NIH
for inclusion in the registry
and results data bank the
clinical trial information
subject to subclause (I) on the
date that is 2 years after the
date a certification under
clause (iii) was made to the
Director of NIH, if an action
referred to in item (aa), (bb),
or (cc) of subclause (I) has
not occurred by such date.
(vi) Extensions.--The Director of NIH
may provide an extension of the
deadline for submission of clinical
trial information under clause (i) if
the responsible party for the trial
submits to the Director a written
request that demonstrates good cause
for the extension and provides an
estimate of the date on which the
information will be submitted. The
Director of NIH may grant more than one
such extension for a clinical trial.
(F) Notice to director of nih.--The
Commissioner of Food and Drugs shall notify the
Director of NIH when there is an action
described in subparagraph (E)(iv) or item (aa),
(bb), or (cc) of subparagraph (E)(v)(I) with
respect to an application or a report that
includes a certification required under
paragraph (5)(B) of such action not later than
30 days after such action.
(G) Posting of data.--The Director of NIH
shall ensure that the clinical trial
information described in subparagraphs (C) and
(D) for an applicable clinical trial submitted
in accordance with this paragraph is posted
publicly in the registry and results database
not later than 30 days after such submission.
(H) Waivers regarding certain clinical trial
results.--The Secretary may waive any
applicable requirements of this paragraph for
an applicable clinical trial, upon a written
request from the responsible party, if the
Secretary determines that extraordinary
circumstances justify the waiver and that
providing the waiver is consistent with the
protection of public health, or in the interest
of national security. Not later than 30 days
after any part of a waiver is granted, the
Secretary shall notify, in writing, the
appropriate committees of Congress of the
waiver and provide an explanation for why the
waiver was granted.
(I) Adverse events.--
(i) Regulations.--Not later than 18
months after the date of the enactment
of the Food and Drug Administration
Amendments Act of 2007, the Secretary
shall by regulation determine the best
method for including in the registry
and results data bank appropriate
results information on serious adverse
and frequent adverse events for
applicable clinical trials described in
subparagraph (C) in a manner and form
that is useful and not misleading to
patients, physicians, and scientists.
(ii) Default.--If the Secretary fails
to issue the regulation required by
clause (i) by the date that is 24
months after the date of the enactment
of the Food and Drug Administration
Amendments Act of 2007, clause (iii)
shall take effect.
(iii) Additional elements.--Upon the
application of clause (ii), the
Secretary shall include in the registry
and results data bank for applicable
clinical trials described in
subparagraph (C), in addition to the
clinical trial information described in
subparagraph (C), the following
elements:
(I) Serious adverse events.--
A table of anticipated and
unanticipated serious adverse
events grouped by organ system,
with number and frequency of
such event in each arm of the
clinical trial.
(II) Frequent adverse
events.--A table of anticipated
and unanticipated adverse
events that are not included in
the table described in
subclause (I) that exceed a
frequency of 5 percent within
any arm of the clinical trial,
grouped by organ system, with
number and frequency of such
event in each arm of the
clinical trial.
(iv) Posting of other information.--
In carrying out clause (iii), the
Secretary shall, in consultation with
experts in risk communication, post
with the tables information to enhance
patient understanding and to ensure
such tables do not mislead patients or
the lay public.
(v) Relation to subparagraph (c).--
Clinical trial information included in
the registry and results data bank
pursuant to this subparagraph is deemed
to be clinical trial information
included in such data bank pursuant to
subparagraph (C).
(4) Additional submissions of clinical trial
information.--
(A) Voluntary submissions.--A responsible
party for a clinical trial that is not an
applicable clinical trial, or that is an
applicable clinical trial that is not subject
to paragraph (2)(C), may submit complete
clinical trial information described in
paragraph (2) or paragraph (3) provided the
responsible party submits clinical trial
information for each applicable clinical trial
that is required to be submitted under section
351 or under section 505, 510(k), 515, or
520(m) of the Federal Food, Drug, and Cosmetic
Act in an application or report for licensure,
approval, or clearance of the drug or device
for the use studied in the clinical trial.
(B) Required submissions.--
(i) In general.--Notwithstanding
paragraphs (2) and (3) and subparagraph
(A), in any case in which the Secretary
determines for a specific clinical
trial described in clause (ii) that
posting in the registry and results
data bank of clinical trial information
for such clinical trial is necessary to
protect the public health--
(I) the Secretary may require
by notification that such
information be submitted to the
Secretary in accordance with
paragraphs (2) and (3) except
with regard to timing of
submission;
(II) unless the responsible
party submits a certification
under paragraph (3)(E)(iii),
such information shall be
submitted not later than 30
days after the date specified
by the Secretary in the
notification; and
(III) failure to comply with
the requirements under
subclauses (I) and (II) shall
be treated as a violation of
the corresponding requirement
of such paragraphs.
(ii) Clinical trials described.--A
clinical trial described in this clause
is--
(I) an applicable clinical
trial for a drug that is
approved under section 505 of
the Federal Food, Drug, and
Cosmetic Act or licensed under
section 351 of this Act or for
a device that is cleared under
section 510(k) of the Federal
Food, Drug, and Cosmetic Act or
approved under section 515 or
section 520(m) of such Act,
whose completion date is on or
after the date 10 years before
the date of the enactment of
the Food and Drug
Administration Amendments Act
of 2007; or
(II) an applicable clinical
trial that is described by both
by paragraph (2)(C) and
paragraph (3)(D)(ii)(II)).
(C) Updates to clinical trial data bank.--
(i) Submission of updates.--The
responsible party for an applicable
clinical trial shall submit to the
Director of NIH for inclusion in the
registry and results data bank updates
to reflect changes to the clinical
trial information submitted under
paragraph (2). Such updates--
(I) shall be provided not
less than once every 12 months,
unless there were no changes to
the clinical trial information
during the preceding 12-month
period;
(II) shall include
identification of the dates of
any such changes;
(III) not later than 30 days
after the recruitment status of
such clinical trial changes,
shall include an update of the
recruitment status; and
(IV) not later than 30 days
after the completion date of
the clinical trial, shall
include notification to the
Director that such clinical
trial is complete.
(ii) Public availability of
updates.--The Director of NIH shall
make updates submitted under clause (i)
publicly available in the registry data
bank. Except with regard to overall
recruitment status, individual site
status, location, and contact
information, the Director of NIH shall
ensure that updates to elements
required under subclauses (I) to (V) of
paragraph (2)(A)(ii) do not result in
the removal of any information from the
original submissions or any preceding
updates, and information in such
databases is presented in a manner that
enables users to readily access each
original element submission and to
track the changes made by the updates.
The Director of NIH shall provide a
link from the table of primary and
secondary outcomes required under
paragraph (3)(C)(ii) to the tracked
history required under this clause of
the primary and secondary outcome
measures submitted under paragraph
(2)(A)(ii)(I)(ll).
(5) Coordination and compliance.--
(A) Clinical trials supported by grants from
federal agencies.--
(i) Grants from certain federal
agencies.--If an applicable clinical
trial is funded in whole or in part by
a grant from any agency of the
Department of Health and Human
Services, including the Food and Drug
Administration, the National Institutes
of Health, or the Agency for Healthcare
Research and Quality, any grant or
progress report forms required under
such grant shall include a
certification that the responsible
party has made all required submissions
to the Director of NIH under paragraphs
(2) and (3).
(ii) Verification by federal
agencies.--The heads of the agencies
referred to in clause (i), as
applicable, shall verify that the
clinical trial information for each
applicable clinical trial for which a
grantee is the responsible party has
been submitted under paragraphs (2) and
(3) before releasing any remaining
funding for a grant or funding for a
future grant to such grantee.
(iii) Notice and opportunity to
remedy.--If the head of an agency
referred to in clause (i), as
applicable, verifies that a grantee has
not submitted clinical trial
information as described in clause
(ii), such agency head shall provide
notice to such grantee of such non-
compliance and allow such grantee 30
days to correct such non-compliance and
submit the required clinical trial
information.
(iv) Consultation with other federal
agencies.--The Secretary shall--
(I) consult with other
agencies that conduct research
involving human subjects in
accordance with any section of
part 46 of title 45, Code of
Federal Regulations (or any
successor regulations), to
determine if any such research
is an applicable clinical
trial; and
(II) develop with such
agencies procedures comparable
to those described in clauses
(i), (ii), and (iii) to ensure
that clinical trial information
for such applicable clinical
trial is submitted under
paragraphs (2) and (3).
(B) Certification to accompany drug,
biological product, and device submissions.--At
the time of submission of an application under
section 505 of the Federal Food, Drug, and
Cosmetic Act, section 515 of such Act, section
520(m) of such Act, or section 351 of this Act,
or submission of a report under section 510(k)
of such Act, such application or submission
shall be accompanied by a certification that
all applicable requirements of this subsection
have been met. Where available, such
certification shall include the appropriate
National Clinical Trial control numbers.
(C) Quality control.--
(i) Pilot quality control project.--
Until the effective date of the
regulations issued under paragraph
(3)(D), the Secretary, acting through
the Director of NIH and the
Commissioner of Food and Drugs, shall
conduct a pilot project to determine
the optimal method of verification to
help to ensure that the clinical trial
information submitted under paragraph
(3)(C) is non-promotional and is not
false or misleading in any particular
under subparagraph (D). The Secretary
shall use the publicly available
information described in paragraph
(3)(A) and any other information
available to the Secretary about
applicable clinical trials to verify
the accuracy of the clinical trial
information submitted under paragraph
(3)(C).
(ii) Notice of compliance.--If the
Secretary determines that any clinical
trial information was not submitted as
required under this subsection, or was
submitted but is false or misleading in
any particular, the Secretary shall
notify the responsible party and give
such party an opportunity to remedy
such noncompliance by submitting the
required revised clinical trial
information not later than 30 days
after such notification.
(D) Truthful clinical trial information.--
(i) In general.--The clinical trial
information submitted by a responsible
party under this subsection shall not
be false or misleading in any
particular.
(ii) Effect.--Clause (i) shall not
have the effect of--
(I) requiring clinical trial
information with respect to an
applicable clinical trial to
include information from any
source other than such clinical
trial involved; or
(II) requiring clinical trial
information described in
paragraph (3)(D) to be
submitted for purposes of
paragraph (3)(C).
(E) Public notices.--
(i) Notice of violations.--If the
responsible party for an applicable
clinical trial fails to submit clinical
trial information for such clinical
trial as required under paragraphs (2)
or (3), the Director of NIH shall
include in the registry and results
data bank entry for such clinical trial
a notice--
(I) that the responsible
party is not in compliance with
this Act by--
(aa) failing to
submit required
clinical trial
information; or
(bb) submitting false
or misleading clinical
trial information;
(II) of the penalties imposed
for the violation, if any; and
(III) whether the responsible
party has corrected the
clinical trial information in
the registry and results data
bank.
(ii) Notice of failure to submit
primary and secondary outcomes.--If the
responsible party for an applicable
clinical trial fails to submit the
primary and secondary outcomes as
required under section 2(A)(ii)(I)(ll),
the Director of NIH shall include in
the registry and results data bank
entry for such clinical trial a notice
that the responsible party is not in
compliance by failing to register the
primary and secondary outcomes in
accordance with this act, and that the
primary and secondary outcomes were not
publicly disclosed in the database
before conducting the clinical trial.
(iii) Failure to submit statement.--
The notice under clause (i) for a
violation described in clause
(i)(I)(aa) shall include the following
statement: ``The entry for this
clinical trial was not complete at the
time of submission, as required by law.
This may or may not have any bearing on
the accuracy of the information in the
entry.''.
(iv) Submission of false information
statement.--The notice under clause (i)
for a violation described in clause
(i)(I)(bb) shall include the following
statement: ``The entry for this
clinical trial was found to be false or
misleading and therefore not in
compliance with the law.''.
(v) Non-submission of statement.--The
notice under clause (ii) for a
violation described in clause (ii)
shall include the following statement:
``The entry for this clinical trial did
not contain information on the primary
and secondary outcomes at the time of
submission, as required by law. This
may or may not have any bearing on the
accuracy of the information in the
entry.''.
(vi) Compliance searches.--The
Director of NIH shall provide that the
public may easily search the registry
and results data bank for entries that
include notices required under this
subparagraph.
(6) Limitation on disclosure of clinical trial
information.--
(A) In general.--Nothing in this subsection
(or under section 552 of title 5, United States
Code) shall require the Secretary to publicly
disclose, by any means other than the registry
and results data bank, information described in
subparagraph (B).
(B) Information described.--Information
described in this subparagraph is--
(i) information submitted to the
Director of NIH under this subsection,
or information of the same general
nature as (or integrally associated
with) the information so submitted; and
(ii) information not otherwise
publicly available, including because
it is protected from disclosure under
section 552 of title 5, United States
Code.
(7) Standardization.--The Director of NIH shall--
(A) ensure that the registry and results data
bank is easily used by the public;
(B) ensure that entries in the registry and
results data bank are easily compared;
(C) ensure that information required to be
submitted to the registry and results data
bank, including recruitment information under
paragraph (2)(A)(ii)(II), is submitted by
persons and posted by the Director of NIH in a
standardized format and includes at least--
(i) the disease or indication being
studied;
(ii) inclusion criteria such as age,
gender, diagnosis or diagnoses,
laboratory values, or imaging results;
and
(iii) exclusion criteria such as
specific diagnosis or diagnoses,
laboratory values, or prohibited
medications; and
(D) to the extent possible, in carrying out
this paragraph, make use of standard health
care terminologies, such as the International
Classification of Diseases or the Current
Procedural Terminology, that facilitate
electronic matching to data in electronic
health records or other relevant health
information technologies.
[(7)] (8) Authorization of appropriations.--There are
authorized to be appropriated to carry out this
subsection $10,000,000 for each fiscal year.
(k)(1) The Director of NIH may establish a program to provide
day care services for the employees of the National Institutes
of Health similar to those services provided by other Federal
agencies (including the availability of day care service on a
24-hour-a-day basis).
(2) Any day care provider at the National Institutes of
Health shall establish a sliding scale of fees that takes into
consideration the income and needs of the employee.
(3) For purposes regarding the provision of day care
services, the Director of NIH may enter into rental or lease
purchase agreements.
(l) Council of Councils.--
(1) Establishment.--Not later than 90 days after the
date of the enactment of the National Institutes of
Health Reform Act of 2006, the Director of NIH shall
establish within the Office of the Director an advisory
council to be known as the ``Council of Councils''
(referred to in this subsection as the ``Council'') for
the purpose of advising the Director on matters related
to the policies and activities of the Division of
Program Coordination, Planning, and Strategic
Initiatives, including making recommendations with
respect to the conduct and support of research
described in subsection (b)(7).
(2) Membership.--
(A) In general.--The Council shall be
composed of 27 members selected by the Director
of NIH with approval from the Secretary from
among the list of nominees under subparagraph
(C).
(B) Certain requirements.--In selecting the
members of the Council, the Director of NIH
shall ensure--
(i) the representation of a broad
range of disciplines and perspectives;
and
(ii) the ongoing inclusion of at
least 1 representative from each
national research institute whose
budget is substantial relative to a
majority of the other institutes.
(C) Nomination.--The Director of NIH shall
maintain an updated list of individuals who
have been nominated to serve on the Council,
which list shall consist of the following:
(i) For each national research
institute and national center, 3
individuals nominated by the head of
such institute or center from among the
members of the advisory council of the
institute or center, of which--
(I) two shall be scientists;
and
(II) one shall be from the
general public or shall be a
leader in the field of public
policy, law, health policy,
economics, or management.
(ii) For each office within the
Division of Program Coordination,
Planning, and Strategic Initiatives, 1
individual nominated by the head of
such office.
(iii) Members of the Council of
Public Representatives.
(3) Terms.--
(A) In general.--The term of service for a
member of the Council shall be 6 years, except
as provided in subparagraphs (B) and (C).
(B) Terms of initial appointees.--Of the
initial members selected for the Council, the
Director of NIH shall designate--
(i) nine for a term of 6 years;
(ii) nine for a term of 4 years; and
(iii) nine for a term of 2 years.
(C) Vacancies.--Any member appointed to fill
a vacancy occurring before the expiration of
the term for which the member's predecessor was
appointed shall be appointed only for the
remainder of that term. A member may serve
after the expiration of that member's term
until a successor has taken office.
(m) Research Strategic Plan.--
(1) Five-year plans for biomedical research
strategy.--
(A) In general.--For each successive five-
year period beginning with the period of fiscal
years 2016 through 2020, the Director of NIH,
in consultation with the entities described in
subparagraph (B), shall develop and maintain a
biomedical research strategic plan that--
(i) is designed to increase the
efficient and effective focus of
biomedical research in a manner that
leverages the best scientific
opportunities through a deliberative
planning process;
(ii) identifies areas, to be known as
strategic focus areas, in which the
resources of the National Institutes of
Health can best contribute to the goal
of expanding knowledge on human health
in the United States through biomedical
research; and
(iii) includes objectives for each
such strategic focus area.
(B) Entities described.--The entities
described in this subparagraph are the
directors of the national research institutes
and national centers, researchers, patient
advocacy groups, and industry leaders.
(2) Use of plan.--The Director of NIH and the
directors of the national research institutes and
national centers shall use the strategic plan--
(A) to identify research opportunities; and
(B) to develop individual strategic plans for
the research activities of each of the national
research institutes and national centers that--
(i) have a common template; and
(ii) identify strategic focus areas
in which the resources of the national
research institutes and national
centers can best contribute to the goal
of expanding knowledge on human health
in the United States through biomedical
research.
(3) Contents of plans.--
(A) Strategic focus areas.--The strategic
focus areas identified pursuant to paragraph
(1)(A)(ii) shall--
(i) be identified in a manner that--
(I) considers the return on
investment to the United States
public through the investments
of the National Institutes of
Health in biomedical research;
and
(II) contributes to expanding
knowledge to improve the United
States public's health through
biomedical research; and
(ii) include overarching and trans-
National Institutes of Health strategic
focus areas, to be known as Mission
Priority Focus Areas, which best serve
the goals of preventing or eliminating
the burden of a disease or condition
and scientifically merit enhanced and
focused research over the next 5 years.
(B) Rare and pediatric diseases and
conditions.--In developing and maintaining a
strategic plan under this subsection, the
Director of NIH shall ensure that rare and
pediatric diseases and conditions remain a
priority.
(C) Workforce.--In developing and maintaining
a strategic plan under this subsection, the
Director of NIH shall ensure that maintaining
the biomedical workforce of the future,
including the participation by scientists from
groups traditionally underrepresented in the
scientific workforce, remains a priority.
(4) Initial plan.--Not later than 270 days after the
date of enactment of this subsection, the Director of
NIH and the directors of the national research
institutes and national centers shall--
(A) complete the initial strategic plan
required by paragraphs (1) and (2); and
(B) make such initial strategic plan publicly
available on the website of the National
Institutes of Health.
(5) Review; updates.--
(A) Progress reviews.--Not less than
annually, the Director of NIH, in consultation
with the directors of the national research
institutes and national centers, shall conduct
progress reviews for each strategic focus area
identified under paragraph (1)(A)(ii).
(B) Updates.--Not later than the end of the
5-year period covered by the initial strategic
plan under this subsection, and every 5 years
thereafter, the Director of NIH, in
consultation with the directors of the national
research institutes and national centers,
stakeholders in the scientific field,
advocates, and the public at large, shall--
(i) conduct a review of the plan,
including each strategic focus area
identified under paragraph (2)(B); and
(ii) update such plan in accordance
with this section.
(n) Sharing of Data Generated Through NIH-funded Research.--
(1) Authority.--Subject to paragraph (2), the
Director of NIH may require recipients of the award of
an NIH grant or other financial support, provided that
the research is fully funded through such grant or
other support, to share scientific data generated from
research conducted through such support for research
purposes.
(2) Limitation.--The Director of NIH shall not
require the sharing of data that is inconsistent with
applicable law and policy protecting--
(A) privacy and confidentiality;
(B) proprietary interests;
(C) business confidential information;
(D) intellectual property rights; and
(E) other relevant rights.
SEC. 402A. AUTHORIZATION OF APPROPRIATIONS.
(a) In General.--
(1) This title.--For purposes of carrying out this
title, there are authorized to be appropriated--
(A) $30,331,309,000 for fiscal year 2007;
(B) $32,831,309,000 for fiscal year 2008;
[and]
(C) such sums as may be necessary for fiscal
year 2009[.];
(D) $31,811,000,000 for fiscal year 2016;
(E) $33,331,000,000 for fiscal year 2017; and
(F) $34,851,000,000 for fiscal year 2018.
(2) Funding for 10-year pediatric research initiative
through common fund.--For the purpose of carrying out
section 402(b)(7)(B)(ii), there is authorized to be
appropriated to the Common Fund, out of the 10-Year
Pediatric Research Initiative Fund described in section
9008 of the Internal Revenue Code of 1986, and in
addition to amounts otherwise made available under
paragraph (1) of this subsection and reserved under
subsection (c)(1)(B)(i) of this section, $12,600,000
for each of fiscal years 2014 through 2023.
(b) Office of the Director.--Of the amount authorized to be
appropriated under subsection (a) for a fiscal year, there are
authorized to be appropriated for programs and activities under
this title carried out through the Office of the Director of
NIH such sums as may be necessary for each of the fiscal years
2007 through 2009.
(c) Trans-NIH Research.--
(1) Common fund.--
(A) Account.--For the purpose of allocations
under section 402(b)(7)(B) (relating to
research identified by the Division of Program
Coordination, Planning, and Strategic
Initiatives), there is established an account
to be known as the Common Fund.
(B) Reservation.--
(i) In general.--Of the total amount
appropriated under subsection (a)(1)
for fiscal year 2007 or any subsequent
fiscal year, the Director of NIH shall
reserve an amount for the Common Fund,
subject to any applicable provisions in
appropriations Acts.
(ii) Minimum amount.--For each fiscal
year, the percentage constituted by the
amount reserved under clause (i)
relative to the total amount
appropriated under subsection (a)(1)
for such year may not be less than the
percentage constituted by the amount so
reserved for the preceding fiscal year
relative to the total amount
appropriated under subsection (a)(1)
for such preceding fiscal year, subject
to any applicable provisions in
appropriations Acts.
(C) Common fund strategic planning report.--
Not later than June 1, 2007, and every 2 years
thereafter, the Secretary, acting through the
Director of NIH, shall submit a report to the
Congress containing a strategic plan for
funding research described in section
402(b)(7)(A)(i) (including personnel needs)
through the Common Fund. Each such plan shall
include the following:
(i) An estimate of the amounts
determined by the Director of NIH to be
appropriate for maximizing the
potential of such research.
(ii) An estimate of the amounts
determined by the Director of NIH to be
sufficient only for continuing to fund
research activities previously
identified by the Division of Program
Coordination, Planning, and Strategic
Initiatives.
(iii) An estimate of the amounts
determined by the Director of NIH to be
necessary to fund research described in
section 402(b)(7)(A)(i)--
(I) that is in addition to
the research activities
described in clause (ii); and
(II) for which there is the
most substantial need.
(D) Evaluation.--During the 6-month period
following the end of the first fiscal year for
which the total amount reserved under
subparagraph (B) is equal to 5 percent of the
total amount appropriated under subsection
(a)(1) for such fiscal year, the Secretary,
acting through the Director of NIH, in
consultation with the advisory council
established under section 402(k), shall submit
recommendations to the Congress for changes
regarding amounts for the Common Fund.
(2) Trans-nih research reporting.--
(A) Limitation.--With respect to the total
amount appropriated under subsection (a) for
fiscal year 2008 or any subsequent fiscal year,
if the head of a national research institute or
national center fails to submit the report
required by subparagraph (B) for the preceding
fiscal year, the amount made available for the
institute or center for the fiscal year
involved may not exceed the amount made
available for the institute or center for
fiscal year 2006.
(B) Reporting.--Not later than January 1,
2008, and each January 1st thereafter--
(i) the head of each national
research institute or national center
shall submit to the Director of NIH a
report on the amount made available by
the institute or center for conducting
or supporting research that involves
collaboration between the institute or
center and 1 or more other national
research institutes or national
centers; and
(ii) the Secretary shall submit a
report to the Congress identifying the
percentage of funds made available by
each national research institute and
national center with respect to such
fiscal year for conducting or
supporting research described in clause
(i).
(C) Determination.--For purposes of
determining the amount or percentage of funds
to be reported under subparagraph (B), any
amounts made available to an institute or
center under section 402(b)(7)(B) shall be
included.
(D) Verification of amounts.--Upon receipt of
each report submitted under subparagraph
(B)(i), the Director of NIH shall review and,
in cases of discrepancy, verify the accuracy of
the amounts specified in the report.
(E) Waiver.--At the request of any national
research institute or national center, the
Director of NIH may waive the application of
this paragraph to such institute or center if
the Director finds that the conduct or support
of research described in subparagraph (B)(i) is
inconsistent with the mission of such institute
or center.
(d) Transfer Authority.--Of the total amount appropriated
under subsection (a)(1) for a fiscal year, the Director of NIH
may (in addition to the reservation under subsection (c)(1) for
such year) transfer not more than 1 percent for programs or
activities that are authorized in this title and identified by
the Director to receive funds pursuant to this subsection. In
making such transfers, the Director may not decrease any
appropriation account under subsection (a)(1) by more than 1
percent.
(e) NIH Innovation Fund.--
(1) Establishment.--For the purpose of allocations
under section 402(b)(25), there is established a fund
to be known as the NIH Innovation Fund. The Director of
NIH shall, with respect to funds appropriated to the
NIH Innovation Fund, allocate such funds to support
biomedical research through the funding of basic,
translational, and clinical research.
(2) Amounts made available to fund.--
(A) In general.--Subject to subparagraph (B),
there is authorized to be appropriated, and
appropriated, to the NIH Innovation Fund out of
any funds in the Treasury not otherwise
appropriated, $2,000,000,000 for each of fiscal
years 2016 through 2020. The amounts
appropriated to the Fund by the preceding
sentence shall be in addition to any amounts
otherwise made available to the National
Institutes of Health.
(B) Availability subject to appropriations.--
Amounts in the Fund shall not be available
except to the extent and in such amounts as are
provided in advance in appropriation Acts.
(C) Allocation of amounts.--Of the amounts
made available from the NIH Innovation Fund for
allocations under section 402(b)(25) for a
fiscal year--
(i) not less than $500,000,000 shall
be for the Accelerating Advancement
Program under paragraph (5);
(ii) not less than 35 percent of such
amounts remaining after subtracting the
allocation for the Accelerating
Advancement Program shall be for early
stage investigators (as defined in
paragraph (7));
(iii) not less than 20 percent of
such amounts remaining after
subtracting the allocation for the
Accelerating Advancement Program shall
be for high-risk, high-reward research
under section 409K; and
(iv) not more than 10 percent of such
amounts (without subtracting the
allocation for the Accelerating
Advancement Program) shall be for
intramural research.
(D) Inapplicability of certain provisions.--
Amounts in the NIH Innovation Fund shall not be
subject to--
(i) any transfer authority of the
Secretary or the Director of NIH under
section 241, subsection (c), subsection
(d), or any other provision of law
(other than section 402(b)(25) and this
subsection); or
(ii) the Nonrecurring expenses fund
under section 223 of division G of the
Consolidated Appropriations Act, 2008
(42 U.S.C. 3514a).
(3) Authorized uses.--Amounts in the NIH Innovation
Fund established under paragraph (1) may be used only
to conduct or support innovative biomedical research
through the following:
(A) Research in which--
(i) a principal investigator has a
specific project or specific
objectives; and
(ii) funding is tied to pursuit of
such project or objectives.
(B) Research in which--
(i) a principal investigator has
shown promise in biomedical research;
and
(ii) funding is not tied to a
specific project or specific
objectives.
(C) Research to be carried out by an early
stage investigator (as defined in paragraph
(7)).
(D) Research to be carried out by a small
business concern (as defined in section 3 of
the Small Business Act).
(E) The Accelerating Advancement Program
under paragraph (5).
(F) Development and implementation of the
strategic plan under paragraph (6).
(4) Coordination.--In funding programs and activities
through the NIH Innovation Fund, the Secretary, acting
through the Director of NIH, shall--
(A) ensure coordination among the national
research institutes, the national centers, and
other departments, agencies, and offices of the
Federal Government; and
(B) minimize unnecessary duplication.
(5) Accelerating advancement program.--The Director
of NIH shall establish a program, to be known as the
Accelerating Advancement Program, under which--
(A) the Director of NIH partners with
national research institutes and national
centers to accomplish important biomedical
research objectives; and
(B) for every $1 made available by the
Director of NIH to a national research
institute or national center for a research
project, the institute or center makes $1
available for such project from funds that are
not derived from the NIH Innovation Fund.
(6) Strategic plan.--
(A) In general.--The Director of NIH shall
ensure that scientifically based strategic
planning is implemented in support of research
priorities, including through development, use,
and updating of a research strategic plan
that--
(i) is designed to increase the
efficient and effective focus of
biomedical research in a manner that
leverages the best scientific
opportunities through a deliberative
planning process;
(ii) identifies areas, to be known as
strategic focus areas, in which the
resources of the NIH Innovation Fund
can contribute to the goals of
expanding knowledge to address, and
find more effective treatments for,
unmet medical needs in the United
States, including the areas of--
(I) biomarkers;
(II) precision medicine;
(III) infectious diseases,
including pathogens listed as a
qualifying pathogen under
section 505E(f) of the Federal
Food, Drug, and Cosmetic Act or
listed or designated as a
tropical disease under section
524 of such Act; and
(IV) antibiotics;
(iii) includes objectives for each
such strategic focus area; and
(iv) ensures that basic research
remains a priority.
(B) Updates and reviews.--The Director shall
review and, as appropriate, update the research
strategic plan under subparagraph (A) not less
than every 18 months.
(7) Definition.--In this subsection, the term ``early
stage investigator'' means an investigator who--
(A) will be the principal investigator or the
program director of the proposed research;
(B) has never been awarded, or has been
awarded only once, a substantial, competing
grant by the National Institutes of Health for
independent research; and
(C) is within 10 years of having completed--
(i) the investigator's terminal
degree; or
(ii) a medical residency (or the
equivalent).
[(e)] (f) Rule of Construction.--This section may not be
construed as affecting the authorities of the Director of NIH
under section 401.
* * * * * * *
Part B--General Provisions Respecting National Research Institutes
appointment and authority of the directors of the national research
institutes
Sec. 405. [(a) The Director of the National Cancer Institute
shall be appointed by the President and the Directors of the
other national research institutes shall be appointed by the
Secretary. Each Director of a national research institute shall
report directly to the Director of NIH.] (a) Appointment;
Terms.--
(1) Appointment.--The Director of the National Cancer
Institute shall be appointed by the President and the
directors of the other national research institutes, as
well as the directors of the national centers, shall be
appointed by the Director of NIH. The directors of the
national research institutes, as well as national
centers, shall report directly to the Director of NIH.
(2) Terms.--
(A) In general.--The term of office of a
director of a national research institute or
national center shall be 5 years.
(B) Removal.--The director of a national
research institute or national center may be
removed from office by the Director of NIH
prior to the expiration of such director's 5-
year term.
(C) Reappointment.--At the end of the term of
a director of a national research institute or
national center, the director may be
reappointed. There is no limit on the number of
terms a director may serve.
(D) Vacancies.--If the office of a director
of a national research institute or national
center becomes vacant before the end of such
director's term, the director appointed to fill
the vacancy shall be appointed for a 5-year
term starting on the date of such appointment.
(E) Transitional provision.--Each director of
a national research institute or national
center serving on the date of enactment of the
21st Century Cures Act is deemed to be
appointed for a 5-year term under this
subsection starting on such date of enactment.
(b)(1) In carrying out the purposes of section 301 with
respect to human diseases or disorders or other aspects of
human health for which the national research institutes were
established, the Secretary, acting through the Director of each
national research institute--
(A) shall encourage and support research,
investigations, experiments, demonstrations, and
studies in the health sciences related to--
(i) the maintenance of health,
(ii) the detection, diagnosis, treatment, and
prevention of human diseases and disorders,
(iii) the rehabilitation of individuals with
human diseases, disorders, and disabilities,
and
(iv) the expansion of knowledge of the
processes underlying human diseases, disorders,
and disabilities, the processes underlying the
normal and pathological functioning of the body
and its organ systems, and the processes
underlying the interactions between the human
organism and the environment;
(B) may, subject to the peer review prescribed under
section 492(b) and any advisory council review under
section 406(a)(3)(A)(i), conduct the research,
investigations, experiments, demonstrations, and
studies referred to in subparagraph (A);
(C) may conduct and support research training (i) for
which fellowship support is not provided under section
487, and (ii) which is not residency training of
physicians or other health professionals;
(D) may develop, implement, and support
demonstrations and programs for the application of the
results of the activities of the institute to clinical
practice and disease prevention activities;
(E) may develop, conduct, and support public and
professional education and information programs;
(F) may secure, develop and maintain, distribute, and
support the development and maintenance of resources
needed for research;
(G) may make available the facilities of the
institute to appropriate entities and individuals
engaged in research activities and cooperate with and
assist Federal and State agencies charged with
protecting the public health;
(H) may accept unconditional gifts made to the
institute for its activities, and, in the case of gifts
of a value in excess of $50,000, establish suitable
memorials to the donor;
(I) may secure for the institute consultation
services and advice of persons from the United States
or abroad;
(J) may use, with their consent, the services,
equipment, personnel, information, and facilities of
other Federal, State, or local public agencies, with or
without reimbursement therefor;
(K) may accept voluntary and uncompensated services;
and
(L) may perform such other functions as the Secretary
determines are needed to carry out effectively the
purposes of the institute.
The indemnification provisions of section 2354, title 10,
United States Code, shall apply with respect to contracts
entered into under this subsection and section 402(b).
(2) Support for an activity or program under this subsection
may be provided through grants, contracts, and cooperative
agreements. The Secretary, acting through the Director of each
national research institute--
(A) may enter into a contract for research, training,
or demonstrations only if the contract has been
recommended after technical and scientific peer review
required by regulations under section 492;
(B) may make grants and cooperative agreements under
paragraph (1) for research, training, or
demonstrations, except that--
(i) if the direct cost of the grant or
cooperative agreement to be made does not
exceed $50,000, such grant or cooperative
agreement may be made only if such grant or
cooperative agreement has been recommended
after technical and scientific peer review
required by regulations under section 492, and
(ii) if the direct cost of the grant or
cooperative agreement to be made exceeds
$50,000, such grant or cooperative agreement
may be made only if such grant or cooperative
agreement has been recommended after technical
and scientific peer review required by
regulations under section 492 and is
recommended under section 406(a)(3)(A)(ii) by
the advisory council for the national research
institute involved; and
(C) shall, subject to section 2353(d)(2), receive
from the President and the Office of Management and
Budget directly all funds appropriated by the Congress
for obligation and expenditure by the Institute.
(3) Before an award is made by a national research institute
or by a national center for a grant for a research program or
project (commonly referred to as an ``R-series grant''), other
than an award constituting a noncompeting renewal of such
grant, or a noncompeting administrative supplement to such
grant, the director of such national research institute or
national center--
(A) shall review and approve the award; and
(B) shall take into consideration--
(i) the mission of the national research
institute or national center and the scientific
priorities identified in the strategic plan
under section 402(m); and
(ii) whether other agencies are funding
programs or projects to accomplish the same
goal.
(c) In carrying out subsection (b), each Director of a
national research institute--
(1) shall coordinate, as appropriate, the activities
of the institute with similar programs of other public
and private entities;
(2) shall cooperate with the Directors of the other
national research institutes in the development and
support of multidisciplinary research and research that
involves more than one institute;
(3) may, in consultation with the advisory council
for the Institute and with the approval of the Director
of NIH--
(A) establish technical and scientific peer
review groups in addition to those appointed
under section 402(b)(16); and
(B) appoint the members of peer review groups
established under subparagraph (A); and
(4) may publish, or arrange for the publication of,
information with respect to the purpose of the
Institute without regard to section 501 of title 44,
United States Code.
The Federal Advisory Committee Act shall not apply to the
duration of a peer review group appointed under paragraph (3).
* * * * * * *
pediatric research initiative
Sec. 409D. (a) Establishment.--The Secretary shall establish
within the Office of the Director of NIH a Pediatric Research
Initiative (referred to in this section as the ``Initiative'')
to conduct and support research that is directly related to
diseases, disorders, and other conditions in children. The
Initiative shall be headed by the Director of NIH.
(b) Purpose.--The purpose of the Initiative is to provide
funds to enable the Director of NIH--
(1) to increase support for pediatric biomedical
research within the National Institutes of Health to
realize the expanding opportunities for advancement in
scientific investigations and care for children;
(2) to enhance collaborative efforts among the
Institutes to conduct and support multidisciplinary
research in the areas that the Director deems most
promising; and
(3) in coordination with the Food and Drug
Administration, to increase the development of adequate
pediatric clinical trials and pediatric use information
to promote the safer and more effective use of
prescription drugs in the pediatric population.
(c) Duties.--In carrying out subsection (b), the Director of
NIH shall--
(1) consult with the Director of the Eunice Kennedy
Shriver National Institute of Child Health and Human
Development and the other national research institutes,
in considering their requests for new or expanded
pediatric research efforts, and consult with the
Administrator of the Health Resources and Services
Administration and other advisors as the Director
determines to be appropriate;
(2) have broad discretion in the allocation of any
Initiative assistance among the Institutes, among types
of grants, and between basic and clinical research so
long as the assistance is directly related to the
illnesses and conditions of children; and
(3) be responsible for the oversight of any newly
appropriated Initiative funds and annually report to
Congress and the public on the extent of the total
funds obligated to conduct or support pediatric
research across the National Institutes of Health,
including the specific support and research awards
allocated through the Initiative.
(d) National Pediatric Research Network.--
(1) Network.--In carrying out the Initiative, the
Director of NIH, [in consultation with the Director of
the Eunice Kennedy Shriver National Institute of Child
Health and Human Development and in collaboration with
other appropriate national research institutes and
national centers that carry out activities involving
pediatric research] in collaboration with the national
research institutes and national centers that carry out
activities involving pediatric research, [may] shall
provide for the establishment of a National Pediatric
Research Network in order to more effectively support
pediatric research and optimize the use of Federal
resources. Such National Pediatric Research Network
[may be comprised of, as appropriate--]
[(A) the pediatric research consortia] may be
comprised of, as appropriate, the pediatric
research consortia receiving awards under
paragraph (2)[; or].
[(B) other consortia, centers, or networks
focused on pediatric research that are
recognized by the Director of NIH and
established pursuant to the authorities vested
in the National Institutes of Health by other
sections of this Act.]
(2) Pediatric research consortia.--
(A) In general.--The Director of NIH [may]
shall award funding, including through grants,
contracts, or other mechanisms, to public or
private nonprofit entities for providing
support for pediatric research consortia,
including with respect to--
(i) basic, clinical, behavioral, or
translational research to meet unmet
needs for pediatric research; and
(ii) training researchers in
pediatric research techniques in order
to address unmet pediatric research
needs.
(B) Research.--The Director of NIH shall, as
appropriate, ensure that--
(i) each consortium receiving an
award under subparagraph (A) conducts
or supports at least one category of
research described in subparagraph
(A)(i) and collectively such consortia
conduct or support such categories of
research; and
(ii) one or more such consortia
provide training described in
subparagraph (A)(ii).
(C) Organization of consortium.--Each
consortium receiving an award under
subparagraph (A) shall--
(i) be formed from a collaboration of
cooperating institutions;
(ii) be coordinated by a lead
institution or institutions;
(iii) agree to disseminate scientific
findings, including from clinical
trials, rapidly and efficiently, as
appropriate, to--
(I) other consortia;
(II) the National Institutes
of Health;
(III) the Food and Drug
Administration;
(IV) and other relevant
agencies; and
(iv) meet such requirements as may be
prescribed by the Director of NIH.
(D) Supplement, not supplant.--Any support
received by a consortium under subparagraph (A)
shall be used to supplement, and not supplant,
other public or private support for activities
authorized to be supported under this
paragraph.
(E) Duration of support.--Support of a
consortium under subparagraph (A) [may] shall
be for a period of not to exceed 5 years. Such
period may be extended at the discretion of the
Director of NIH.
(3) Coordination of consortia activities.--The
Director of NIH shall, as appropriate--
(A) provide for the coordination of
activities (including the exchange of
information and regular communication) among
the consortia established pursuant to paragraph
(2); and
(B) require the periodic preparation and
submission to the Director of reports on the
activities of each such consortium.
(4) Assistance with registries.--Each consortium
receiving an award under paragraph (2)(A) may provide
assistance, as appropriate, to the Centers for Disease
Control and Prevention for activities related to
patient registries and other surveillance systems upon
request by the Director of the Centers for Disease
Control and Prevention.
(e) Research on Pediatric Rare Diseases or Conditions.--In
making awards under subsection (d)(2) for pediatric research
consortia, the Director of NIH shall ensure that an appropriate
number of such awards are awarded to such consortia that agree
to--
(1) consider pediatric rare diseases or conditions,
or those related to birth defects; and
(2) conduct or coordinate one or more multisite
clinical trials of therapies for, or approaches to, the
prevention, diagnosis, or treatment of one or more
pediatric rare diseases or conditions.
(f) Transfer of Funds.--The Director of NIH may transfer
amounts appropriated under this section to any of the
Institutes for a fiscal year to carry out the purposes of the
Initiative under this section.
* * * * * * *
SEC. 409K. HIGH-RISK, HIGH-REWARD RESEARCH PROGRAM.
The director of each national research institute shall, as
appropriate--
(1) establish programs to conduct or support research
projects that pursue innovative approaches to major
contemporary challenges in biomedical research that
involve inherent high risk, but have the potential to
lead to breakthroughs; and
(2) set aside a specific percentage of funding, to be
determined by the Director of NIH for each national
research institute, for such projects.
Part C--Specific Provisions Respecting National Research Institutes
* * * * * * *
Subpart 20--National Institute on Minority Health and Health
Disparities
* * * * * * *
SEC. 464Z-5. LOAN REPAYMENT PROGRAM FOR MINORITY HEALTH DISPARITIES
RESEARCH.
(a) In General.--The Director of the Institute shall
establish a program of entering into contracts with qualified
health professionals under which such health professionals
agree to engage in minority health disparities research or
other health disparities research in consideration of the
Federal Government agreeing to repay, for each year of engaging
in such research, not more than [$35,000] $50,000 of the
principal and interest of the educational loans of such health
professionals. Subsection (b) of section 487H shall apply with
respect to the maximum amount specified in this subsection in
the same manner as it applies to the maximum amount specified
in subsection (a) of such section.
(b) Service Provisions.--The provisions of sections 338B,
338C, and 338E shall, except as inconsistent with subsection
(a), apply to the program established in such subsection to the
same extent and in the same manner as such provisions apply to
the National Health Service Corps Loan Repayment Program
established in subpart III of part D of title III.
(c) Requirement Regarding Health Disparity Populations.--The
Director of the Institute shall ensure that not fewer than 50
percent of the contracts entered into under subsection (a) are
for appropriately qualified health professionals who are
members of a health disparity population.
(d) Priority.--With respect to minority health disparities
research and other health disparities research under subsection
(a), the Secretary shall ensure that priority is given to
conducting projects of biomedical research.
* * * * * * *
Part E--Other Agencies of NIH
Subpart 1--National Center for Advancing Translational Sciences
SEC. 479. NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES.
(a) Purpose.--The purpose of the National Center for
Advancing Translational Sciences (in this subpart referred to
as the ``Center'') is to advance translational sciences,
including by--
(1) coordinating and developing resources that
leverage basic research in support of translational
science; and
(2) developing partnerships and working cooperatively
to foster synergy in ways that do not create
duplication, redundancy, and competition with industry
activities.
(b) Clinical Trial Activities.--
(1) In general.--The Center may develop and provide
infrastructure and resources for all phases of clinical
trials research. Except as provided in paragraph (2),
the Center may support clinical trials only through the
end of phase [IIA] IIB.
(2) Exception.--The Center may support clinical trial
activities through the end of phase [IIB] III for a
treatment for a rare disease or condition (as defined
in section 526 of the Federal Food, Drug, and Cosmetic
Act) so long as--
(A) the Center gives public notice for a
period of at least 120 days of the Center's
intention to support the clinical trial
activities in phase [IIB] III;
(B) no public or private organization
provides credible written intent to the Center
that the organization has timely plans to
further the clinical trial activities or
conduct clinical trials of a similar nature
beyond phase [IIA] IIB; and
(C) the Center ensures that support of the
clinical trial activities in phase [IIB] III
will not increase the Federal Government's
liability beyond the award value of the
Center's support.
(c) Annual Report.--The Center shall publish an annual report
that, with respect to all research supported by the Center,
includes a complete list of--
(1) the molecules being studied;
(2) clinical trial activities being conducted;
(3) the methods and tools in development;
(4) ongoing partnerships, including--
(A) the rationale for each partnership;
(B) the status of each partnership;
(C) the funding provided by the Center to
other entities pursuant to each partnership,
and
(D) the activities which have been
transferred to industry pursuant to each
partnership; and
(5) known research activity of other entities that is
or will expand upon research activity of the Center.
SEC. 480. CURES ACCELERATION NETWORK.
(a) Definitions.--In this section:
(1) Biological product.--The term ``biological
product'' has the meaning given such term in section
351 of the Public Health Service Act.
(2) Drug; device.--The terms ``drug'' and ``device''
have the meanings given such terms in section 201 of
the Federal Food, Drug, and Cosmetic Act.
(3) High need cure.--The term ``high need cure''
means a drug (as that term is defined by section
201(g)(1) of the Federal Food, Drug, and Cosmetic Act,
biological product (as that term is defined by section
262(i)), or device (as that term is defined by section
201(h) of the Federal Food, Drug, and Cosmetic Act)
that, in the determination of the Director of the
Center--
(A) is a priority to diagnose, mitigate,
prevent, or treat harm from any disease or
condition; and
(B) for which the incentives of the
commercial market are unlikely to result in its
adequate or timely development.
(4) Medical product.--The term ``medical product''
means a drug, device, biological product, or product
that is a combination of drugs, devices, and biological
products.
(b) Establishment of the Cures Acceleration Network.--Subject
to [the appropriation of funds as described in subsection (g)]
the availability of funds as described in subsection (f), there
is established within the Center a program to be known as the
Cures Acceleration Network (referred to in this section as
``CAN''), which shall--
(1) be under the direction of the Director of the
Center, taking into account the recommendations of a
CAN Review Board (referred to in this section as the
``Board''), described in subsection (d); and
(2) award grants and contracts to eligible entities,
as described in subsection (e), to accelerate the
development of high need cures, including through the
development of medical products and behavioral
therapies.
(c) Functions.--The functions of the CAN are to--
(1) conduct and support revolutionary advances in
basic research, translating scientific discoveries from
bench to bedside;
(2) award grants and contracts to eligible entities
to accelerate the development of high need cures;
(3) provide the resources necessary for government
agencies, independent investigators, research
organizations, biotechnology companies, academic
research institutions, and other entities to develop
high need cures;
(4) reduce the barriers between laboratory
discoveries and clinical trials for new therapies; and
(5) facilitate review in the Food and Drug
Administration for the high need cures funded by the
CAN, through activities that may include--
(A) the facilitation of regular and ongoing
communication with the Food and Drug
Administration regarding the status of
activities conducted under this section;
(B) ensuring that such activities are
coordinated with the approval requirements of
the Food and Drug Administration, with the goal
of expediting the development and approval of
countermeasures and products; and
(C) connecting interested persons with
additional technical assistance made available
under section 565 of the Federal Food, Drug,
and Cosmetic Act.
(d) CAN Board.--
(1) Establishment.--There is established a Cures
Acceleration Network Review Board (referred to in this
section as the ``Board''), which shall advise the
Director of the Center on the conduct of the activities
of the Cures Acceleration Network.
(2) Membership.--
(A) In general.--
(i) Appointment.--The Board shall be
comprised of 24 members who are
appointed by the Secretary and who
serve at the pleasure of the Secretary.
(ii) Chairperson and vice
chairperson.--The Secretary shall
designate, from among the 24 members
appointed under clause (i), one
Chairperson of the Board (referred to
in this section as the ``Chairperson'')
and one Vice Chairperson.
(B) Terms.--
(i) In general.--Each member shall be
appointed to serve a 4-year term,
except that any member appointed to
fill a vacancy occurring prior to the
expiration of the term for which the
member's predecessor was appointed
shall be appointed for the remainder of
such term.
(ii) Consecutive appointments;
maximum terms.--A member may be
appointed to serve not more than 3
terms on the Board, and may not serve
more than 2 such terms consecutively.
(C) Qualifications.--
(i) In general.--The Secretary shall
appoint individuals to the Board based
solely upon the individual's
established record of distinguished
service in one of the areas of
expertise described in clause (ii).
Each individual appointed to the Board
shall be of distinguished achievement
and have a broad range of disciplinary
interests.
(ii) Expertise.--The Secretary shall
select individuals based upon the
following requirements:
(I) For each of the fields
of--
(aa) basic research;
(bb) medicine;
(cc)
biopharmaceuticals;
(dd) discovery and
delivery of medical
products;
(ee) bioinformatics
and gene therapy;
(ff) medical
instrumentation; and
(gg) regulatory
review and approval of
medical products,
the Secretary shall select at
least 1 individual who is
eminent in such fields.
(II) At least 4 individuals
shall be recognized leaders in
professional venture capital or
private equity organizations
and have demonstrated
experience in private equity
investing.
(III) At least 8 individuals
shall represent disease
advocacy organizations.
(3) Ex-officio members.--
(A) Appointment.--In addition to the 24 Board
members described in paragraph (2), the
Secretary shall appoint as ex-officio members
of the Board--
(i) a representative of the National
Institutes of Health, recommended by
the Secretary of the Department of
Health and Human Services;
(ii) a representative of the Office
of the Assistant Secretary of Defense
for Health Affairs, recommended by the
Secretary of Defense;
(iii) a representative of the Office
of the Under Secretary for Health for
the Veterans Health Administration,
recommended by the Secretary of
Veterans Affairs;
(iv) a representative of the National
Science Foundation, recommended by the
Chair of the National Science Board;
and
(v) a representative of the Food and
Drug Administration, recommended by the
Commissioner of Food and Drugs.
(B) Terms.--Each ex-officio member shall
serve a 3-year term on the Board, except that
the Chairperson may adjust the terms of the
initial ex-officio members in order to provide
for a staggered term of appointment for all
such members.
(4) Responsibilities of the board and the director of
the center.--
(A) Responsibilities of the board.--
(i) In general.--The Board shall
advise, and provide recommendations to,
the Director of the Center with respect
to--
(I) policies, programs, and
procedures for carrying out the
duties of the Director of the
Center under this section; and
(II) significant barriers to
successful translation of basic
science into clinical
application (including issues
under the purview of other
agencies and departments).
(ii) Report.--In the case that the
Board identifies a significant barrier,
as described in clause (i)(II), the
Board shall submit to the Secretary a
report regarding such barrier.
(B) Responsibilities of the director of the
center.--With respect to each recommendation
provided by the Board under subparagraph
(A)(i), the Director of the Center shall
respond in writing to the Board, indicating
whether such Director will implement such
recommendation. In the case that the Director
of the Center indicates a recommendation of the
Board will not be implemented, such Director
shall provide an explanation of the reasons for
not implementing such recommendation.
(5) Meetings.--
(A) In general.--The Board shall meet 4 times
per calendar year, at the call of the
Chairperson.
(B) Quorum; requirements; limitations.--
(i) Quorum.--A quorum shall consist
of a total of 13 members of the Board,
excluding ex-officio members, with
diverse representation as described in
clause (iii).
(ii) Chairperson or vice
chairperson.--Each meeting of the Board
shall be attended by either the
Chairperson or the Vice Chairperson.
(iii) Diverse representation.--At
each meeting of the Board, there shall
be not less than one scientist, one
representative of a disease advocacy
organization, and one representative of
a professional venture capital or
private equity organization.
(6) Compensation and travel expenses.--
(A) Compensation.--Members shall receive
compensation at a rate to be fixed by the
Chairperson but not to exceed a rate equal to
the daily equivalent of the annual rate of
basic pay prescribed for level IV of the
Executive Schedule under section 5315 of title
5, United States Code, for each day (including
travel time) during which the member is engaged
in the performance of the duties of the Board.
All members of the Board who are officers or
employees of the United States shall serve
without compensation in addition to that
received for their services as officers or
employees of the United States.
(B) Travel expenses.--Members of the Board
shall be allowed travel expenses, including per
diem in lieu of subsistence, at rates
authorized for persons employed intermittently
by the Federal Government under section 5703(b)
of title 5, United States Code, while away from
their homes or regular places of business in
the performance of services for the Board.
(e) Grant Program.--
(1) Supporting innovation.--To carry out the purposes
described in this section, the Director of the Center
shall award contracts, grants, or cooperative
agreements to the entities described in paragraph (2),
to--
(A) promote innovation in technologies
supporting the advanced research and
development and production of high need cures,
including through the development of medical
products and behavioral therapies.
(B) accelerate the development of high need
cures, including through the development of
medical products, behavioral therapies, and
biomarkers that demonstrate the safety or
effectiveness of medical products; or
(C) help the award recipient establish
protocols that comply with Food and Drug
Administration standards and otherwise permit
the recipient to meet regulatory requirements
at all stages of development, manufacturing,
review, approval, and safety surveillance of a
medical product.
(2) Eligible entities.--To receive assistance under
paragraph (1), an entity shall--
(A) be a public or private entity, which may
include a private or public research
institution, an institution of higher
education, a medical center, a biotechnology
company, a pharmaceutical company, a disease
advocacy organization, a patient advocacy
organization, or an academic research
institution;
(B) submit an application containing--
(i) a detailed description of the
project for which the entity seeks such
grant or contract;
(ii) a timetable for such project;
(iii) an assurance that the entity
will submit--
(I) interim reports
describing the entity's--
(aa) progress in
carrying out the
project; and
(bb) compliance with
all provisions of this
section and conditions
of receipt of such
grant or contract; and
(II) a final report at the
conclusion of the grant period,
describing the outcomes of the
project; and
(iv) a description of the protocols
the entity will follow to comply with
Food and Drug Administration standards
and regulatory requirements at all
stages of development, manufacturing,
review, approval, and safety
surveillance of a medical product; and
(C) provide such additional information as
the Director of the Center may require.
(3) Awards.--
(A) The cures acceleration partnership
awards.--
(i) Initial award amount.--Each award
under this subparagraph shall be not
more than $15,000,000 per project for
the first fiscal year for which the
project is funded, which shall be
payable in one payment.
(ii) Funding in subsequent fiscal
years.--An eligible entity receiving an
award under clause (i) may apply for
additional funding for such project by
submitting to the Director of the
Center the information required under
subparagraphs (B) and (C) of paragraph
(2). The Director may fund a project of
such eligible entity in an amount not
to exceed $15,000,000 for a fiscal year
subsequent to the initial award under
clause (i).
(iii) Matching funds.--As a condition
for receiving an award under this
subsection, an eligible entity shall
contribute to the project non-Federal
funds in the amount of $1 for every $3
awarded under clauses (i) and (ii),
except that the Director of the Center
may waive or modify such matching
requirement in any case where the
Director determines that the goals and
objectives of this section cannot
adequately be carried out unless such
requirement is waived.
(B) The cures acceleration grant awards.--
(i) Initial award amount.--Each award
under this subparagraph shall be not
more than $15,000,000 per project for
the first fiscal year for which the
project is funded, which shall be
payable in one payment.
(ii) Funding in subsequent fiscal
years.--An eligible entity receiving an
award under clause (i) may apply for
additional funding for such project by
submitting to the Board the information
required under subparagraphs (B) and
(C) of paragraph (2). The Director of
the Center may fund a project of such
eligible entity in an amount not to
exceed $15,000,000 for a fiscal year
subsequent to the initial award under
clause (i).
[(C) The cures acceleration flexible research
awards.--If the Director of the Center
determines that the goals and objectives of
this section cannot adequately be carried out
through a contract, grant, or cooperative
agreement, the Director of the Center shall
have flexible research authority to use other
transactions to fund projects in accordance
with the terms and conditions of this section.
Awards made under such flexible research
authority for
