II
119THCONGRESS
1
STSESSION S. 932
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
MARCH11 (legislative day, MARCH10), 2025
Mr. M
ULLIN(for himself and Mr. BENNET) introduced the following bill;
which was read twice and referred to the Committee on Health, Edu-
cation, Labor, and Pensions
A BILL
To amend the Federal Food, Drug, and Cosmetic Act with
respect to molecularly targeted pediatric cancer investiga-
tions, and for other purposes.
Be it enacted by the Senate and House of Representa-1
tives of the United States of America in Congress assembled, 2
SECTION 1. SHORT TITLE. 3
This Act may be cited as the ‘‘Give Kids a Chance 4
Act of 2025’’. 5
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SEC. 2. RESEARCH INTO PEDIATRIC USES OF DRUGS; ADDI-1
TIONAL AUTHORITIES OF FOOD AND DRUG 2
ADMINISTRATION REGARDING MOLECU-3
LARLY TARGETED CANCER DRUGS. 4
(a) I
NGENERAL.— 5
(1) A
DDITIONAL ACTIVE INGREDIENT FOR AP -6
PLICATION DRUG; LIMITATION REGARDING NOVEL - 7
COMBINATION APPLICATION DRUG .—Section 8
505B(a)(3) of the Federal Food, Drug, and Cos-9
metic Act (21 U.S.C. 355c(a)(3)) is amended— 10
(A) by redesignating subparagraphs (B) 11
and (C) as subparagraphs (C) and (D), respec-12
tively; and 13
(B) by striking subparagraph (A) and in-14
serting the following: 15
‘‘(A) I
N GENERAL.—For purposes of para-16
graph (1)(B), the investigation described in this 17
paragraph is a molecularly targeted pediatric 18
cancer investigation of— 19
‘‘(i) the drug or biological product for 20
which the application referred to in such 21
paragraph is submitted; or 22
‘‘(ii) such drug or biological product 23
used in combination with— 24
‘‘(I) an active ingredient of a 25
drug or biological product— 26
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‘‘(aa) for which an approved 1
application under section 505(j) 2
under this Act or under section 3
351(k) of the Public Health 4
Service Act is in effect; and 5
‘‘(bb) that is determined by 6
the Secretary, after consultation 7
with the applicant, to be part of 8
the standard of care for treating 9
a pediatric cancer; or 10
‘‘(II) an active ingredient of a 11
drug or biological product— 12
‘‘(aa) for which an approved 13
application under section 505(b) 14
of this Act or section 351(a) of 15
the Public Health Service Act to 16
treat an adult cancer is in effect 17
and is held by the same person 18
submitting the application under 19
paragraph (1)(B); and 20
‘‘(bb) that is directed at a 21
molecular target that the Sec-22
retary determines to be substan-23
tially relevant to the growth or 24
progression of a pediatric cancer. 25
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‘‘(B) ADDITIONAL REQUIREMENTS .— 1
‘‘(i) D
ESIGN OF INVESTIGATION .—A 2
molecularly targeted pediatric cancer inves-3
tigation referred to in subparagraph (A) 4
shall be designed to yield clinically mean-5
ingful pediatric study data that is gathered 6
using appropriate formulations for each 7
age group for which the study is required, 8
regarding dosing, safety, and preliminary 9
efficacy to inform potential pediatric label-10
ing. 11
‘‘(ii) L
IMITATION.—An investigation 12
described in subparagraph (A)(ii) may be 13
required only if the drug or biological 14
product for which the application referred 15
to in paragraph (1)(B) contains either— 16
‘‘(I) a single new active ingre-17
dient; or 18
‘‘(II) more than one active ingre-19
dient, if an application for the com-20
bination of active ingredients has not 21
previously been approved but each ac-22
tive ingredient is in a drug product 23
that has been previously approved to 24
treat an adult cancer. 25
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‘‘(iii) RESULTS OF ALREADY -COM-1
PLETED PRECLINICAL STUDIES OF APPLI -2
CATION DRUG.—With respect to an inves-3
tigation required pursuant to paragraph 4
(1)(B), the Secretary may require the re-5
sults of any completed preclinical studies 6
relevant to the initial pediatric study plan 7
be submitted to the Secretary at the same 8
time that the initial pediatric study plan 9
required under subsection (e)(1) is sub-10
mitted. 11
‘‘(iv) R
ULE OF CONSTRUCTION RE -12
GARDING INACTIVE INGREDIENTS .—With 13
respect to a combination of active ingredi-14
ents referred to in subparagraph (A)(ii), 15
such subparagraph shall not be construed 16
as addressing the use of inactive ingredi-17
ents with such combination.’’. 18
(2) D
ETERMINATION OF APPLICABLE REQUIRE -19
MENTS.—Section 505B(e)(1) of the Federal Food, 20
Drug, and Cosmetic Act (21 U.S.C. 355c(e)(1)) is 21
amended by adding at the end the following: ‘‘The 22
Secretary shall determine whether subparagraph (A) 23
or (B) of subsection (a)(1) applies with respect to an 24
application before the date on which the applicant is 25
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required to submit the initial pediatric study plan 1
under paragraph (2)(A).’’. 2
(3) C
LARIFYING APPLICABILITY .—Section 3
505B(a)(1) of the Federal Food, Drug, and Cos-4
metic Act (21 U.S.C. 355c(a)(1)) is amended by 5
adding at the end the following: 6
‘‘(C) R
ULE OF CONSTRUCTION .—No appli-7
cation that is subject to the requirements of 8
subparagraph (B) shall be subject to the re-9
quirements of subparagraph (A), and no appli-10
cation (or supplement to an application) that is 11
subject to the requirements of subparagraph 12
(A) shall be subject to the requirements of sub-13
paragraph (B).’’. 14
(4) C
ONFORMING AMENDMENTS .—Section 15
505B(a) of the Federal Food, Drug, and Cosmetic 16
Act (21 U.S.C. 355c(a)) is amended— 17
(A) in paragraph (3)(C), as redesignated 18
by paragraph (1)(A) of this subsection, by 19
striking ‘‘investigations described in this para-20
graph’’ and inserting ‘‘investigations referred to 21
in subparagraph (A)’’; and 22
(B) in paragraph (3)(D), as redesignated 23
by paragraph (1)(A) of this subsection, by 24
striking ‘‘the assessments under paragraph 25
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(2)(B)’’ and inserting ‘‘the assessments re-1
quired under paragraph (1)(A)’’. 2
(b) G
UIDANCE.—The Secretary of Health and 3
Human Services, acting through the Commissioner of 4
Food and Drugs, shall— 5
(1) not later than 12 months after the date of 6
enactment of this Act, issue draft guidance on the 7
implementation of the amendments made by sub-8
section (a); and 9
(2) not later than 12 months after closing the 10
comment period on such draft guidance, finalize 11
such guidance. 12
(c) A
PPLICABILITY.—The amendments made by this 13
section apply with respect to any application under section 14
505(b) of the Federal Food, Drug, and Cosmetic Act (21 15
U.S.C. 355(b)) and any application under section 351(a) 16
of the Public Health Service Act (42 U.S.C. 262(a)), that 17
is submitted on or after the date that is 3 years after the 18
date of enactment of this Act. 19
(d) R
EPORTS TOCONGRESS.— 20
(1) S
ECRETARY OF HEALTH AND HUMAN SERV -21
ICES.—Not later than 6 years after the date of en-22
actment of this Act, the Secretary of Health and 23
Human Services shall submit to the Committee on 24
Energy and Commerce of the House of Representa-25
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tives and the Committee on Health, Education, 1
Labor, and Pensions of the Senate a report on the 2
Secretary’s efforts, in coordination with industry, to 3
ensure implementation of the amendments made by 4
subsection (a). 5
(2) GAO
STUDY AND REPORT.— 6
(A) S
TUDY.—Not later than 8 years after 7
the date of enactment of this Act, the Comp-8
troller General of the United States shall con-9
duct a study of the effectiveness of requiring 10
assessments and investigations described in sec-11
tion 505B of the Federal Food, Drug, and Cos-12
metic Act (21 U.S.C. 355c), as amended by 13
subsection (a), in the development of drugs and 14
biological products for pediatric cancer indica-15
tions, including consideration of any benefits to, 16
or burdens on, pediatric cancer drug develop-17
ment. 18
(B) F
INDINGS.—Not later than 10 years 19
after the date of enactment of this Act, the 20
Comptroller General shall submit to the Com-21
mittee on Energy and Commerce of the House 22
of Representatives and the Committee on 23
Health, Education, Labor, and Pensions of the 24
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Senate a report containing the findings of the 1
study conducted under subparagraph (A). 2
SEC. 3. EXTENSION OF AUTHORITY TO ISSUE PRIORITY RE-3
VIEW VOUCHERS TO ENCOURAGE TREAT-4
MENTS FOR RARE PEDIATRIC DISEASES. 5
(a) E
XTENSION.—Section 529(b)(5) of the Federal 6
Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(b)(5)) is 7
amended by striking ‘‘December 20, 2024, unless’’ and all 8
that follows through the period at the end and inserting 9
‘‘September 30, 2029.’’. 10
(b) U
SERFEEPAYMENT.—Subsection 529(c)(4) of 11
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 12
360ff(c)(4)) is amended by striking subparagraph (A) and 13
inserting the following: 14
‘‘(A) I
N GENERAL.—The priority review 15
user fee required by this subsection shall be due 16
upon the submission of a human drug applica-17
tion under section 505(b)(1) or section 351(a) 18
of the Public Health Service Act for which the 19
priority review voucher is used. All other user 20
fees associated with the human drug application 21
shall be due as required by the Secretary or 22
under applicable law.’’. 23
(c) GAO R
EPORT ONEFFECTIVENESS OFRAREPE-24
DIATRICDISEASEPRIORITYVOUCHERAWARDS IN 25
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INCENTIVIZINGRAREPEDIATRICDISEASEDRUGDEVEL-1
OPMENT.— 2
(1) GAO
STUDY.— 3
(A) S
TUDY.—The Comptroller General of 4
the United States shall conduct a study of the 5
effectiveness of awarding rare pediatric disease 6
priority vouchers under section 529 of the Fed-7
eral Food, Drug, and Cosmetic Act (21 U.S.C. 8
360ff), as amended by subsection (a), in the de-9
velopment of human drug products that treat or 10
prevent rare pediatric diseases (as defined in 11
such section 529). 12
(B) C
ONTENTS OF STUDY .—In conducting 13
the study under subparagraph (A), the Comp-14
troller General shall examine the following: 15
(i) The indications for each drug or 16
biological product that— 17
(I) is the subject of a rare pedi-18
atric disease product application (as 19
defined in section 529 of the Federal 20
Food, Drug, and Cosmetic Act (21 21
U.S.C. 360ff)) for which a priority re-22
view voucher was awarded; and 23
(II) was approved under section 24
505 of the Federal Food, Drug, and 25
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Cosmetic Act (42 U.S.C. 355) or li-1
censed under section 351 of the Pub-2
lic Health Service Act (42 U.S.C. 3
262). 4
(ii) Whether, and to what extent, an 5
unmet need related to the treatment or 6
prevention of a rare pediatric disease was 7
met through the approval or licensure of 8
such a drug or biological product. 9
(iii) The size of the company to which 10
a priority review voucher was awarded 11
under section 529 of the Federal Food, 12
Drug, and Cosmetic Act (21 U.S.C. 360ff) 13
for such a drug or biological product. 14
(iv) The value of such priority review 15
voucher if transferred. 16
(v) Identification of each drug for 17
which a priority review voucher awarded 18
under such section 529 was used. 19
(vi) The size of the company using 20
each priority review voucher awarded 21
under such section 529. 22
(vii) The length of the period of time 23
between the date on which a priority re-24
view voucher was awarded under such sec-25
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tion 529 and the date on which it was 1
used. 2
(viii) Whether, and to what extent, an 3
unmet need related to the treatment or 4
prevention of a rare pediatric disease was 5
met through the approval under section 6
505 of the Federal Food, Drug, and Cos-7
metic Act (42 U.S.C. 355) or licensure 8
under section 351 of the Public Health 9
Service Act (42 U.S.C. 262) of a drug for 10
which a priority review voucher was used. 11
(ix) Whether, and to what extent, 12
companies were motivated by the avail-13
ability of priority review vouchers under 14
section 529 of the Federal Food, Drug, 15
and Cosmetic Act (21 U.S.C. 360ff) to at-16
tempt to develop a drug for a rare pedi-17
atric disease. 18
(x) Whether, and to what extent, pedi-19
atric review vouchers awarded under such 20
section were successful in stimulating de-21
velopment and expedited patient access to 22
drug products for treatment or prevention 23
of a rare pediatric disease that wouldn’t 24
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otherwise take place without the incentive 1
provided by such vouchers. 2
(xi) The impact of such priority re-3
view vouchers on the workload, review 4
process, and public health prioritization ef-5
forts of the Food and Drug Administra-6
tion. 7
(xii) Any other incentives in Federal 8
law that exist for companies developing 9
drugs or biological products described in 10
clause (i). 11
(2) R
EPORT ON FINDINGS .—Not later than 5 12
years after the date of the enactment of this Act, the 13
Comptroller General of the United States shall sub-14
mit to the Committee on Energy and Commerce of 15
the House of Representatives and the Committee on 16
Health, Education, Labor, and Pensions of the Sen-17
ate a report containing the findings of the study 18
conducted under paragraph (1). 19
Æ
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