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using appropriate formulations for each 
age group for which the study is required, 
regarding dosing, safety, and preliminary 
efficacy to inform potential pediatric label-
ing. 
‘‘(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 ingre-
dient; or 
‘‘(II) more than one active ingre-
dient, if an application for the com-
bination of active ingredients has not 
previously been approved but each ac-
tive ingredient has been previously ap-
proved to treat an adult cancer. 
‘‘(iii) RESULTS
OF
ALREADY-COM-
PLETED PRECLINICAL STUDIES OF APPLI-
CATION
DRUG.—The Secretary may re-
quire that reports on an investigation re-
quired pursuant to paragraph (1)(B) in-
clude the results of all preclinical studies 
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on which the decision to conduct such in-
vestigation was based. 
‘‘(iv) RULE
OF
CONSTRUCTION
RE-
GARDING
INACTIVE
INGREDIENTS.—With 
respect to a combination of active ingredi-
ents referred to in subparagraph (A)(ii), 
such subparagraph shall not be construed 
as addressing the use of inactive ingredi-
ents with such combination.’’. 
(2) DETERMINATION OF APPLICABLE REQUIRE-
MENTS.—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) shall apply with respect 
to an application before the date on which the appli-
cant 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 Cos-
metic Act (21 U.S.C. 355c(a)(1)) is amended by 
adding at the end the following: 
‘‘(C) RULE OF CONSTRUCTION.—No appli-
cation that is subject to the requirements of 
subparagraph (B) shall be subject to the re-
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quirements of subparagraph (A), and no appli-
cation (or supplement to an application) that is 
subject to the requirements of subparagraph 
(A) shall be subject to the requirements of sub-
paragraph (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 para-
graph’’ 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 re-
quired 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 sub-
section (a); and 
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(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 SERV-
ICES.—Not later than 2 years after the date of en-
actment of this Act, the Secretary of Health and 
Human Services shall submit to the Committee on 
Energy and Commerce of the House of Representa-
tives 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 3 years after 
the date of enactment of this Act, the Comp-
troller General of the United States shall con-
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duct a study of the effectiveness of requiring 
assessments and investigations described in sec-
tion 505B of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C.355c), as amended by sub-
section (a), in the development of drugs and bi-
ological products for pediatric cancer indica-
tions. 
(B) FINDINGS.—Not later than 7 years 
after the date of enactment of this Act, the 
Comptroller General shall submit to the Com-
mittee 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. 102. 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 amend-
ed— 
(1) in paragraph (1), by striking ‘‘Beginning 
270’’ and inserting ‘‘NONCOMPLIANCE
LETTER.— 
Beginning 270’’; 
(2) in paragraph (2)— 
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(A) by striking ‘‘The drug or’’ and insert-
ing ‘‘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 satis-
fying 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 fail-
ures 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 Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)), 
as amended by subsection (a), is further amended by add-
ing at the end the following: 
‘‘(4) DUE
DILIGENCE.—Before the Secretary 
may conclude that a person failed to submit or oth-
erwise meet a requirement as described in the mat-
ter preceding paragraph (1), the Secretary shall— 
‘‘(A) issue a noncompliance letter pursuant 
to paragraph (1); 
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‘‘(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 require-
ment.’’. 
(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 enact-
ment of this Act. 
SEC. 103. 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 
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of compliance with deadlines provided for in defer-
rals 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 pay-
ments 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 im-
posed; and’’. 
SEC. 104. EXTENSION OF AUTHORITY TO ISSUE PRIORITY 
REVIEW VOUCHERS TO ENCOURAGE TREAT-
MENTS 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 ‘‘September 30, 2024, 
unless’’ and all that follows through the period at the end 
and inserting ‘‘September 30, 2029.’’. 
(b) GAO REPORT ON EFFECTIVENESS OF RARE PE-
DIATRIC
DISEASE
PRIORITY
VOUCHER
AWARDS
IN 
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INCENTIVIZING RARE PEDIATRIC DISEASE DRUG DEVEL-
OPMENT.— 
(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 Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 
360ff), as amended by subsection (a), in the de-
velopment 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 Comp-
troller General shall examine the following: 
(i) The indications for each drug or 
biological product that— 
(I) is the subject of a rare pedi-
atric 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 re-
view voucher was awarded; and 
(II) was approved under section 
505 of the Federal Food, Drug, and 
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Cosmetic Act (42 U.S.C. 355) or li-
censed under section 351 of the Pub-
lic 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 biological product. 
(iv) The value of such priority review 
voucher if transferred. 
(v) Identification of each drug for 
which a priority review voucher awarded 
under such section 529 was used. 
(vi) The size of the company using 
each priority review voucher awarded 
under such section 529. 
(vii) The length of the period of time 
between the date on which a priority re-
view voucher was awarded under such sec-
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tion 529 and the date on which it was 
used. 
(viii) Whether, and to what extent, an 
unmet need related to the treatment or 
prevention of a rare pediatric disease was 
met through the approval under section 
505 of the Federal Food, Drug, and Cos-
metic Act (42 U.S.C. 355) or licensure 
under section 351 of the Public Health 
Service Act (42 U.S.C. 262) of a drug for 
which a priority review voucher was used. 
(ix) Whether, and to what extent, 
companies were motivated by the avail-
ability of priority review vouchers under 
section 529 of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 360ff) to at-
tempt to develop a drug for a rare pedi-
atric disease. 
(x) Whether, and to what extent, pedi-
atric review vouchers awarded under such 
section were successful in stimulating de-
velopment and expedited patient access to 
drug products for treatment or prevention 
of a rare pediatric disease that wouldn’t 
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otherwise take place without the incentive 
provided by such vouchers. 
(xi) The impact of such priority re-
view vouchers on the workload, review 
process, and public health prioritization ef-
forts of the Food and Drug Administra-
tion. 
(xii) Any other incentives in Federal 
law that exist for companies developing 
drugs or biological products described in 
clause (i). 
(2) REPORT ON FINDINGS.—Not later than 5 
years after the date of the enactment of this Act, the 
Comptroller General of the United States shall sub-
mit to the Committee on Energy and Commerce of 
the House of Representatives and the Committee on 
Health, Education, Labor, and Pensions of the Sen-
ate a report containing the findings of the study 
conducted under paragraph (1). 
SEC. 105. LIMITATIONS ON EXCLUSIVE APPROVAL OR LI-
CENSURE OF ORPHAN DRUGS. 
(a) IN GENERAL.—Section 527 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360cc) is amended— 
(1) in subsection (a), in the matter following 
paragraph (2), by striking ‘‘same disease or condi-
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