[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[S. 705 Introduced in Senate (IS)]
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119th CONGRESS
1st Session
S. 705
To amend the Federal Food, Drug, and Cosmetic Act with respect to
molecularly targeted pediatric cancer investigations, and for other
purposes.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
February 25, 2025
Mr. Reed (for himself and Mrs. Capito) 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 with respect to
molecularly targeted pediatric cancer investigations, 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 ``Innovation in Pediatric Drugs Act of
2025''.
SEC. 2. ENSURING COMPLETION OF PEDIATRIC STUDY REQUIREMENTS.
(a) Equal Accountability for Pediatric Study Requirements.--Section
505B(d) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d))
is amended--
(1) in paragraph (1), by striking ``Beginning 270'' and
inserting ``Noncompliance letter.--Beginning 270'';
(2) in paragraph (2)--
(A) by striking ``The drug or'' and inserting
``Effect of noncompliance.--The drug or''; and
(B) by striking ``(except that the drug or
biological product shall not be subject to action under
section 303)'' and inserting ``(except that the drug or
biological product shall be subject to action under
section 303 only if such person demonstrated a lack of
due diligence in satisfying the applicable
requirement)''; and
(3) by adding at the end the following:
``(3) Limitation.--The Secretary shall not issue
enforcement actions under section 303 for failures under this
subsection in the case of a drug or biological product that is
no longer marketed.''.
(b) Due Diligence.--Section 505B(d) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355c(d)), as amended by subsection (a), is
further amended by adding at the end the following:
``(4) Due diligence.--Before the Secretary may conclude
that a person failed to submit or otherwise meet a requirement
as described in the matter preceding paragraph (1), the
Secretary shall--
``(A) issue a noncompliance letter pursuant to
paragraph (1);
``(B) provide such person with a 45-day period
beginning on the date of receipt of such noncompliance
letter to respond in writing as set forth in such
paragraph; and
``(C) after reviewing such written response,
determine whether the person demonstrated a lack of due
diligence in satisfying such requirement.''.
(c) Conforming Amendments.--Section 303(f)(4)(A) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 333(f)(4)(A)) is amended by
striking ``or 505-1'' and inserting ``505-1, or 505B''.
(d) Transition Rule.--The Secretary of Health and Human Services
may take enforcement action under section 303 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 333) for failures described in
section 505B(d) of such Act (21 U.S.C. 355c(d)) only if such failures
occur on or after the date that is 180 days after the date of enactment
of this Act.
SEC. 3. FDA REPORT ON PREA ENFORCEMENT.
Section 508(b) of the Food and Drug Administration Safety and
Innovation Act (21 U.S.C. 355c-1(b)) is amended--
(1) in paragraph (11), by striking the semicolon at the end
and inserting ``, including an evaluation of compliance with
deadlines provided for in deferrals and deferral extensions;'';
(2) in paragraph (15), by striking ``and'' at the end;
(3) in paragraph (16), by striking the period at the end
and inserting ``; and''; and
(4) by adding at the end the following:
``(17) a listing of penalties, settlements, or payments
under section 303 of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 353) for failure to comply with requirements under
such section 505B, including, for each penalty, settlement, or
payment, the name of the drug, the sponsor thereof, and the
amount of the penalty, settlement, or payment imposed.''.
SEC. 4. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.
Section 409I(d) of the Public Health Service Act (42 U.S.C.
284m(d)) is amended to read as follows:
``(d) Funding.--Of the amount made available for pediatric research
to each national research institute and national center under this
title for each of fiscal years 2026 through 2030, the Director of NIH
is authorized to make available up to one percent of such amount for
pediatric research under this section.''.
SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS.
(a) Application of Pediatric Research Requirements to Orphan
Drugs.--
(1) In general.--Section 505B(k) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 355c(k)) is amended to read as
follows:
``(k) Relation to Orphan Drugs.--
``(1) In general.--This section does not apply to a drug or
biological product for an indication for which orphan
designation has been granted under section 526 unless the
Secretary determines that pediatric assessments of such drug or
biological product required under this section could represent
a meaningful therapeutic benefit as described in subsection
(c).
``(2) Deferrals and waivers.--Deferrals and waivers under
subsections (a)(4) and (a)(5) shall apply to assessments
described in this subsection to the same extent and in the same
manner as such deferrals and waivers apply with respect to the
assessments under subsection (a)(1), and waivers under
subsection (b)(2) shall apply to assessments described in this
subsection to the same extent and in the same manner as such
waivers apply with respect to the assessments required pursuant
to subsection (b)(1).''.
(2) Applicability.--The amendment made by paragraph (1)
applies only to applications described in subparagraph (A) or
(B) of section 505B(a)(1) of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355c(a)(1)) that are submitted on or
after the later of--
(A) the date that is 18 months after the date of
issuance of the final guidance under subsection (b);
and
(B) such later date as may be specified by the
Secretary of Health and Human Services (referred to in
this section as the ``Secretary'') by regulation.
(b) Guidance.--
(1) Issuance.--The Secretary shall--
(A) not later than 1 year after the date of
enactment of this Act, issue draft guidance describing
how, upon the applicability of the amendment made by
subsection (a)(1), the requirements of section 505B of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355c) will apply with respect to any drug or biological
product for an indication within a disease or condition
for which orphan designation has been granted under
section 526 of such Act (21 U.S.C. 360bb); and
(B) not later than 18 months after the date of the
public meeting required by subsection (c)(1), finalize
such draft guidance.
(2) Contents.--The guidance under subsection (b) shall
address the following:
(A) Information regarding how full and partial
waivers under subsections (a)(5)(A), (a)(5)(B), and
(b)(2) of section 505B of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355c) for any drug or
biological product for an indication within a disease
or condition for which orphan designation has been
granted under section 526 of such Act (21 U.S.C. 360bb)
will be granted.
(B) Application of the requirements of section
505B(e) of such Act (21 U.S.C. 355c(e)) to drugs or
biological products for an indication within a disease
or condition for which orphan designation has been
granted under section 526 of such Act (21 U.S.C.
360bb), including submission and timing of planned
requests for full or partial waivers and responses by
the Food and Drug Administration to those requests.
(C) Rare diseases and conditions (as defined in
section 526(a)(2) of such Act (21 U.S.C. 360bb(a)(2))
that should be added to the lists under section
505B(a)(5)(E) and 505B(b)(2)(E) of such Act, as added
by subsection (f), and a process for regularly updating
such lists.
(D) Situations where the initial pediatric study
plan under section 505B(e) of such Act (21 U.S.C.
355c(e)) includes a plan to fulfill the requirements of
section 505B(a) of such Act (21 U.S.C. 355c(a)) without
requesting waivers in any age group.
(E) Consideration of how the Secretary will balance
the unique scientific challenges of rare disease drug
development with the need for improved pediatric
labeling of drugs and biological products for
indications within diseases or conditions for which
orphan designation has been granted under section 526
of such Act (21 U.S.C. 360bb).
(F) Considerations of the strengths, weaknesses,
appropriateness, and limitations of different types of
real-world evidence specific to the fulfillment of
requirements under section 505B of such Act (21 U.S.C.
355c).
(G) Consideration of input received from the public
meeting set forth in subsection (c).
(c) Public Meeting.--The Secretary shall--
(1) not later than 6 months after the date of issuance of
the draft guidance under subsection (b)(1)(A), hold a public
meeting to inform the final guidance to be issued under
subsection (b)(1)(B); and
(2) publish prior notice of such meeting in the Federal
Register.
(d) GAO Study.--Not later than 4 years after the applicability date
described in subsection (a)(2), the Comptroller General of the United
States shall submit to the Committee on Energy and Commerce and the
Committee on Ways and Means of the House of Representatives and the
Committee on Health, Education, Labor, and Pensions of the Senate a
report that--
(1) addresses the impacts of this Act on--
(A) rare disease drug development in the United
States; and
(B) the availability of pediatric information on
drugs and biological products within diseases or
conditions for indications for which orphan designation
has been granted under section 526 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360bb); and
(2) includes--
(A) the findings of a survey of companies of
varying sizes engaged in the development of orphan
drugs, which shall include questions regarding the
feasibility and other challenges of conducting
pediatric studies for such indications;
(B) input from patient groups and medical provider
associations; and
(C) an assessment of the impact changes to required
pediatric studies had on drug development for rare
diseases.
(e) Rule of Construction.--Nothing in this section shall be
construed to limit requirements for investigations, as described in
section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355c(a)(3)), of molecularly targeted pediatric cancer drugs for
which orphan designation has been granted under section 526 of such Act
(21 U.S.C. 360bb).
(f) Certainty Regarding Waivers.--Section 505B of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355c) is amended--
(1) in subsection (a)(5), by adding at the end the
following:
``(E) Automatic full waiver list.--The Secretary
shall maintain a list, posted on the website of the
Food and Drug Administration, of adult-related diseases
and conditions--
``(i) with respect to which the necessary
studies are impossible or highly impracticable,
as described in subparagraph (A)(i); or
``(ii) for which a drug or biological
product for such disease or condition otherwise
meets the criteria described in subparagraph
(A).'';
(2) in subsection (b)(2), by adding at the end the
following:
``(E) Automatic full waiver list.--The Secretary
shall maintain a list, posted on the website of the
Food and Drug Administration, of adult-related diseases
and conditions with respect to which the necessary
studies would meet the criteria for a full waiver under
subparagraph (A).''; and
(3) in subsection (e)(4), by adding at the end the
following: ``If, at the time of an applicant's submission of
the initial pediatric study plan, the disease or condition for
which the drug is intended to treat appears on the list under
subsection (a)(5)(E), then the assessments for such disease or
condition shall be waived under subsection (a)(5).''.
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