[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 4071 Introduced in Senate (IS)]
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117th CONGRESS
2d Session
S. 4071
To amend the Federal Food, Drug, and Cosmetic Act to improve the
treatment of rare diseases and conditions, and for other purposes.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
April 7, 2022
Mr. Casey (for himself and Mr. Scott of South Carolina) introduced the
following bill; which was read twice and referred to the Committee on
Health, Education, Labor, and Pensions
_______________________________________________________________________
A BILL
To amend the Federal Food, Drug, and Cosmetic Act to improve the
treatment of rare diseases and conditions, and for other purposes.
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Helping Experts Accelerate Rare
Treatments Act of 2022''.
SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES AND CONDITIONS.
(a) Annual Report on Orphan Drug Program.--Subchapter B of chapter
V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.)
is amended by adding at the end the following new section:
``SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM.
``(a) In General.--Not later than 1 year after the date of
enactment of the Helping Experts Accelerate Rare Treatments Act of
2022, and not less frequently than annually thereafter, the Secretary
shall submit to Congress a report summarizing the activities of the
Food and Drug Administration related to designating drugs under section
526 for a rare disease or condition and approving such drugs under
section 505 of this Act or licensing such drugs under section 351 of
the Public Health Service Act, including--
``(1) the number of applications for such drugs under
section 505 of this Act and section 351 of the Public Health
Service Act received by the Food and Drug Administration, the
number of such applications accepted for filing, the number of
such applications rejected for filing, and the numbers of such
applications pending, approved, and disapproved by the Food and
Drug Administration, arrayed by the review division assigned to
the application;
``(2) the size of the affected population in the United
States of each disease or condition addressed by an application
described in paragraph (1), assessed taking into consideration
the documentation required by section 316.20 of title 21, Code
of Federal Regulations (or any successor regulation) and other
relevant information available to the Secretary; and
``(3) the extent of consultation with stakeholders and
external experts pursuant to section 569.
``(b) Manner of Submission.--The information required to be
reported under this section may be submitted as part of another report
to Congress related to the regulation of drugs.
``(c) Public Availability.--The Secretary shall make each report
under subsection (a) available to the public, including by posting the
report on the website of the Food and Drug Administration.''.
(b) Study on European Union Safety and Efficacy Reviews of Drugs
for Rare Diseases and Conditions.--
(1) In general.--Not later than 1 year after the date of
enactment of this Act, the Secretary of Health and Human
Services shall seek to enter into an agreement with the
National Academies of Sciences, Engineering, and Medicine
(referred to in this section as the ``National Academies'') to
examine and report on European Union safety and efficacy
reviews of drugs for rare diseases and conditions, the use and
sufficiency of existing mechanisms and tools of the Food and
Drug Administration in ensuring that patient and physician
perspectives are considered throughout such reviews, and
opportunities to improve such reviews in the United States.
(2) Content.--The report developed under paragraph (1)
shall--
(A) assess and evaluate, with respect to drugs for
rare diseases and conditions--
(i) any flexibilities, authorities, or
mechanisms available in the European Union;
(ii) consideration and use by the European
Medicines Agency of supplemental data submitted
during the orphan drug application review
process, including data associated with open
label extension studies and expanded access
programs; and
(iii) each formal or informal process that
the Food and Drug Administration has utilized
to gather external expertise on orphan drug
applications, separately considering orphan
drugs for diseases or conditions that affect
fewer than 20,000 individuals in the United
States, compared to orphan drugs for other rare
diseases or conditions; and
(B) provide recommendations for changes to the
processes and authorities of the Food and Drug
Administration to facilitate development of, and access
to, orphan drugs, which may include--
(i) new tools or mechanisms to improve
efforts and initiatives of the Food and Drug
Administration to collect and consider external
expertise on orphan drug applications,
separately considering treatments for diseases
or conditions that affect fewer than 20,000
individuals in the United States, including
with respect to processes related to
application review, including structured
benefit-risk assessments, advisory committee
deliberations, and postapproval safety
monitoring; and
(ii) in the case of a need for input from
external experts where there are limited
clinical and research experts available with
respect to a rare disease or condition, setting
forth alternative processes to address or
resolve any conflicts of interest that would
otherwise impede inclusion of input from such
external experts.
(3) Input.--In conducting the study and developing the
report under paragraph (2), the National Academies shall--
(A) consider input from the Department of Health
and Human Services, and any other Federal agencies, as
appropriate; and
(B) consult with relevant stakeholders, which may
include conducting public meetings and other forms of
engagement, as appropriate, with patient groups, health
care providers, medical professional societies, public
health experts, medical product manufacturers, and
other entities with relevant experience.
(4) Definitions.--In this subsection--
(A) the term ``orphan drug'' means a drug
designated as a drug for a rare disease or condition
under section 526 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360bb); and
(B) the term ``rare disease or condition'' has the
meaning given such term in section 526 of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 360bb).
(c) Review Process.--
(1) Consultation with stakeholders.--Section 569(a)(1) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-
8(a)(1)) is amended--
(A) by striking ``at a time'' and inserting ``at
any time'';
(B) by striking ``Consistent with sections'' and
inserting the following:
``(A) In general.--Consistent with sections''; and
(C) by adding at the end the following:
``(B) Consultation with patients and patient
groups.--
``(i) In general.--The Secretary may, as
appropriate, consult with patients and relevant
patient groups impacted by the rare disease or
condition, together with at least one expert
included on the list under paragraph (2)(A) and
selected by such groups--
``(I) during the review process of
an application for a new drug or
biological product for a rare disease
or condition or a drug or biological
product that is genetically targeted;
and
``(II) as applicable, during
meetings between the Food and Drug
Administration and sponsors prior to
the submission of an application for a
drug described in subclause (I).
``(ii) Conflicts of interest.--For purposes
of clause (i), to be eligible for consultation
pursuant to clause (i), patients and relevant
patient groups may not have any financial
interest in the applicable drug or biological
product, and external experts shall be in
compliance with applicable law, including
section 208 of title 18, United States Code.
``(C) Consultation with disproportionately affected
communities.--To the extent an application for a new
drug or biological product relates to a rare disease or
condition that disproportionately affects communities
of color or other historically underrepresented and
vulnerable populations, the Secretary is encouraged to
consult with patients of that subpopulation, or one or
more patient groups that represent that
subpopulation.''.
(2) Requiring appropriate expert consultation.--Section
569(a)(2) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360bbb-8(a)(2)) is amended--
(A) in subparagraph (A), by striking the second
sentence; and
(B) by striking subparagraph (B) and inserting the
following:
``(B) Consultation.--With respect to any
application under section 505 of this Act or section
351 of the Public Health Service Act for a drug
designated under section 526 for a rare disease or
condition or a drug or biological product that is
genetically targeted, the Secretary may, as
appropriate, consult--
``(i) with an expert with respect to the
disease or condition referenced in the
application who appears on the list described
in subparagraph (A); or
``(ii) if no such expert is available,
including because of conflicts of interest,
with an expert on the list described in
subparagraph (A) in the science of small
population studies.
``(C) Availability at meetings.--In connection with
each drug product advisory committee meeting concerning
a drug or biological product for a rare disease or
condition, the Secretary may, as appropriate--
``(i) include--
``(I) an expert in the rare disease
or condition; or
``(II) if no such expert is
available, including because of
conflicts of interest, an expert in the
science of small population studies;
and
``(ii) invite at least one disease or
condition expert identified by the relevant
patient groups to participate as a nonvoting
member of the advisory committee.''.
(3) Additional topic for consultation.--Section 569(b) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-
8(b)) is amended--
(A) in paragraph (6), by striking ``; and'' and
inserting ``;'';
(B) in paragraph (7), by striking the period and
inserting ``; and''; and
(C) by adding at the end the following:
``(8) the science of small population studies.''.
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