[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[H.R. 1262 Introduced in House (IH)]
<DOC>
119th CONGRESS
1st Session
H. R. 1262
To amend the Federal Food, Drug, and Cosmetic Act with respect to
molecularly targeted pediatric cancer investigations, and for other
purposes.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
February 12, 2025
Mr. McCaul (for himself, Mr. Bilirakis, Mrs. Dingell, Ms. Schrier, Mrs.
Harshbarger, Ms. Matsui, Mr. Crenshaw, Ms. Castor of Florida, Mr. Kelly
of Pennsylvania, Mrs. Trahan, and Mr. Weber of Texas) introduced the
following bill; which was referred to the Committee on Energy and
Commerce
_______________________________________________________________________
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; TABLE OF CONTENTS.
(a) Short Title.--This Act may be cited as the ``Give Kids a Chance
Act of 2025''.
(b) Table of Contents.--The table of contents for this Act is as
follows:
Sec. 1. Short title; table of contents.
Sec. 2. Research into pediatric uses of drugs; additional authorities
of Food and Drug Administration regarding
molecularly targeted cancer drugs.
Sec. 3. Ensuring completion of pediatric study requirements.
Sec. 4. FDA report on PREA enforcement.
Sec. 5. Extension of authority to issue priority review vouchers to
encourage treatments for rare pediatric
diseases.
Sec. 6. Limitations on exclusive approval or licensure of orphan drugs.
Sec. 7. Program for pediatric studies of drugs.
Sec. 8. Organ Procurement and Transplantation Network.
Sec. 9. Establishment of Abraham Accords Office within Food and Drug
Administration.
SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDITIONAL AUTHORITIES
OF FOOD AND DRUG ADMINISTRATION REGARDING MOLECULARLY
TARGETED CANCER DRUGS.
(a) In General.--
(1) Additional active ingredient for application drug;
limitation regarding novel-combination application drug.--
Section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 355c(a)(3)) is amended--
(A) by redesignating subparagraphs (B) and (C) as
subparagraphs (C) and (D), respectively; and
(B) by striking subparagraph (A) and inserting the
following:
``(A) In general.--For purposes of paragraph
(1)(B), the investigation described in this paragraph
is a molecularly targeted pediatric cancer
investigation of--
``(i) the drug or biological product for
which the application referred to in such
paragraph is submitted; or
``(ii) such drug or biological product used
in combination with--
``(I) an active ingredient of a
drug or biological product--
``(aa) for which an
approved application under
section 505(j) under this Act
or under section 351(k) of the
Public Health Service Act is in
effect; and
``(bb) that is determined
by the Secretary, after
consultation with the
applicant, to be part of the
standard of care for treating a
pediatric cancer; or
``(II) an active ingredient of a
drug or biological product--
``(aa) for which an
approved application under
section 505(b) of this Act or
section 351(a) of the Public
Health Service Act to treat an
adult cancer is in effect and
is held by the same person
submitting the application
under paragraph (1)(B); and
``(bb) that is directed at
a molecular target that the
Secretary determines to be
substantially relevant to the
growth or progression of a
pediatric cancer.
``(B) Additional requirements.--
``(i) Design of investigation.--A
molecularly targeted pediatric cancer
investigation referred to in subparagraph (A)
shall be designed to yield clinically
meaningful pediatric study data that is
gathered using appropriate formulations for
each age group for which the study is required,
regarding dosing, safety, and preliminary
efficacy to inform potential pediatric
labeling.
``(ii) Limitation.--An investigation
described in subparagraph (A)(ii) may be
required only if the drug or biological product
for which the application referred to in
paragraph (1)(B) contains either--
``(I) a single new active
ingredient; or
``(II) more than one active
ingredient, if an application for the
combination of active ingredients has
not previously been approved but each
active ingredient is in a drug product
that has been previously approved to
treat an adult cancer.
``(iii) Results of already-completed
preclinical studies of application drug.--With
respect to an investigation required pursuant
to paragraph (1)(B), the Secretary may require
the results of any completed preclinical
studies relevant to the initial pediatric study
plan be submitted to the Secretary at the same
time that the initial pediatric study plan
required under subsection (e)(1) is submitted.
``(iv) Rule of construction regarding
inactive ingredients.--With respect to a
combination of active ingredients referred to
in subparagraph (A)(ii), such subparagraph
shall not be construed as addressing the use of
inactive ingredients with such combination.''.
(2) Determination of applicable requirements.--Section
505B(e)(1) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355c(e)(1)) is amended by adding at the end the
following: ``The Secretary shall determine whether subparagraph
(A) or (B) of subsection (a)(1) applies with respect to an
application before the date on which the applicant is required
to submit the initial pediatric study plan under paragraph
(2)(A).''.
(3) Clarifying applicability.--Section 505B(a)(1) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1)) is
amended by adding at the end the following:
``(C) Rule of construction.--No application that is
subject to the requirements of subparagraph (B) shall
be subject to the requirements of subparagraph (A), and
no application (or supplement to an application) that
is subject to the requirements of subparagraph (A)
shall be subject to the requirements of subparagraph
(B).''.
(4) Conforming amendments.--Section 505B(a) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)) is amended--
(A) in paragraph (3)(C), as redesignated by
paragraph (1)(A) of this subsection, by striking
``investigations described in this paragraph'' and
inserting ``investigations referred to in subparagraph
(A)''; and
(B) in paragraph (3)(D), as redesignated by
paragraph (1)(A) of this subsection, by striking ``the
assessments under paragraph (2)(B)'' and inserting
``the assessments required under paragraph (1)(A)''.
(b) Guidance.--The Secretary of Health and Human Services, acting
through the Commissioner of Food and Drugs, shall--
(1) not later than 12 months after the date of enactment of
this Act, issue draft guidance on the implementation of the
amendments made by subsection (a); and
(2) not later than 12 months after closing the comment
period on such draft guidance, finalize such guidance.
(c) Applicability.--The amendments made by this section apply with
respect to any application under section 505(b) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355(b)) and any application under
section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)),
that is submitted on or after the date that is 3 years after the date
of enactment of this Act.
(d) Reports to Congress.--
(1) Secretary of health and human services.--Not later than
6 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 Secretary's efforts, in coordination
with industry, to ensure implementation of the amendments made
by subsection (a).
(2) GAO study and report.--
(A) Study.--Not later than 8 years after the date
of enactment of this Act, the Comptroller General of
the United States shall conduct a study of the
effectiveness of requiring assessments and
investigations described in section 505B of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355c), as
amended by subsection (a), in the development of drugs
and biological products for pediatric cancer
indications, including consideration of any benefits
to, or burdens on, pediatric cancer drug development.
(B) Findings.--Not later than 10 years after the
date of enactment of this Act, the Comptroller General
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 findings of the study conducted
under subparagraph (A).
SEC. 3. 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) only for failures described in
section 505B(d) of such Act (21 U.S.C. 355c(d)) that occur on or after
the date that is 180 days after the date of enactment of this Act.
SEC. 4. 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; and''.
SEC. 5. EXTENSION OF AUTHORITY TO ISSUE PRIORITY REVIEW VOUCHERS TO
ENCOURAGE TREATMENTS FOR RARE PEDIATRIC DISEASES.
(a) Extension.--Paragraph (5) of section 529(b) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(b)) is amended by
striking ``December 20, 2024, unless'' and all that follows through the
period at the end and inserting ``September 30, 2029.''.
(b) User Fee Payment.--Section 529(c)(4) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 360ff(c)(4)) is amended by striking
subparagraph (A) and inserting the following:
``(A) In general.--The priority review user fee
required by this subsection shall be due upon the
submission of a human drug application under section
505(b)(1) or section 351(a) of the Public Health
Service Act for which the priority review voucher is
used. All other user fees associated with the human
drug application shall be due as required by the
Secretary or under applicable law.''.
(c) GAO Report on Effectiveness of Rare Pediatric Disease Priority
Voucher Awards in Incentivizing Rare Pediatric Disease Drug
Development.--
(1) GAO study.--
(A) Study.--The Comptroller General of the United
States shall conduct a study of the effectiveness of
awarding rare pediatric disease priority vouchers under
section 529 of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360ff), as amended by subsection (a), in the
development of human drug products that treat or
prevent rare pediatric diseases (as defined in such
section 529).
(B) Contents of study.--In conducting the study
under subparagraph (A), the Comptroller General shall
examine the following:
(i) The indications for each drug or
biological product that--
(I) is the subject of a rare
pediatric disease product application
(as defined in section 529 of the
Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360ff)) for which a priority
review voucher was awarded; and
(II) was approved under section 505
of the Federal Food, Drug, and Cosmetic
Act (42 U.S.C. 355) or licensed under
section 351 of the Public Health
Service Act (42 U.S.C. 262).
(ii) Whether, and to what extent, an unmet
need related to the treatment or prevention of
a rare pediatric disease was met through the
approval or licensure of such a drug or
biological product.
(iii) The size of the company to which a
priority review voucher was awarded under
section 529 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360ff) for such a drug
or biologi