II
119THCONGRESS
1
STSESSION 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
FEBRUARY25, 2025
Mr. R
EED(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 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 ‘‘Innovation in Pediatric 4
Drugs Act of 2025’’. 5
SEC. 2. ENSURING COMPLETION OF PEDIATRIC STUDY RE-6
QUIREMENTS. 7
(a) E
QUALACCOUNTABILITY FOR PEDIATRICSTUDY 8
R
EQUIREMENTS.—Section 505B(d) of the Federal Food, 9
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Drug, and Cosmetic Act (21 U.S.C. 355c(d)) is amend-1
ed— 2
(1) in paragraph (1), by striking ‘‘Beginning 3
270’’ and inserting ‘‘N
ONCOMPLIANCE LETTER .— 4
Beginning 270’’; 5
(2) in paragraph (2)— 6
(A) by striking ‘‘The drug or’’ and insert-7
ing ‘‘E
FFECT OF NONCOMPLIANCE .—The drug 8
or’’; and 9
(B) by striking ‘‘(except that the drug or 10
biological product shall not be subject to action 11
under section 303)’’ and inserting ‘‘(except that 12
the drug or biological product shall be subject 13
to action under section 303 only if such person 14
demonstrated a lack of due diligence in satis-15
fying the applicable requirement)’’; and 16
(3) by adding at the end the following: 17
‘‘(3) L
IMITATION.—The Secretary shall not 18
issue enforcement actions under section 303 for fail-19
ures under this subsection in the case of a drug or 20
biological product that is no longer marketed.’’. 21
(b) D
UEDILIGENCE.—Section 505B(d) of the Fed-22
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)), 23
as amended by subsection (a), is further amended by add-24
ing at the end the following: 25
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‘‘(4) DUE DILIGENCE.—Before the Secretary 1
may conclude that a person failed to submit or oth-2
erwise meet a requirement as described in the mat-3
ter preceding paragraph (1), the Secretary shall— 4
‘‘(A) issue a noncompliance letter pursuant 5
to paragraph (1); 6
‘‘(B) provide such person with a 45-day 7
period beginning on the date of receipt of such 8
noncompliance letter to respond in writing as 9
set forth in such paragraph; and 10
‘‘(C) after reviewing such written response, 11
determine whether the person demonstrated a 12
lack of due diligence in satisfying such require-13
ment.’’. 14
(c) C
ONFORMING AMENDMENTS.—Section 15
303(f)(4)(A) of the Federal Food, Drug, and Cosmetic Act 16
(21 U.S.C. 333(f)(4)(A)) is amended by striking ‘‘or 505– 17
1’’ and inserting ‘‘505–1, or 505B’’. 18
(d) T
RANSITIONRULE.—The Secretary of Health 19
and Human Services may take enforcement action under 20
section 303 of the Federal Food, Drug, and Cosmetic Act 21
(21 U.S.C. 333) for failures described in section 505B(d) 22
of such Act (21 U.S.C. 355c(d)) only if such failures occur 23
on or after the date that is 180 days after the date of 24
enactment of this Act. 25
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SEC. 3. FDA REPORT ON PREA ENFORCEMENT. 1
Section 508(b) of the Food and Drug Administration 2
Safety and Innovation Act (21 U.S.C. 355c–1(b)) is 3
amended— 4
(1) in paragraph (11), by striking the semicolon 5
at the end and inserting ‘‘, including an evaluation 6
of compliance with deadlines provided for in defer-7
rals and deferral extensions;’’; 8
(2) in paragraph (15), by striking ‘‘and’’ at the 9
end; 10
(3) in paragraph (16), by striking the period at 11
the end and inserting ‘‘; and’’; and 12
(4) by adding at the end the following: 13
‘‘(17) a listing of penalties, settlements, or pay-14
ments under section 303 of the Federal Food, Drug, 15
and Cosmetic Act (21 U.S.C. 353) for failure to 16
comply with requirements under such section 505B, 17
including, for each penalty, settlement, or payment, 18
the name of the drug, the sponsor thereof, and the 19
amount of the penalty, settlement, or payment im-20
posed.’’. 21
SEC. 4. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS. 22
Section 409I(d) of the Public Health Service Act (42 23
U.S.C. 284m(d)) is amended to read as follows: 24
‘‘(d) F
UNDING.—Of the amount made available for 25
pediatric research to each national research institute and 26
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national center under this title for each of fiscal years 1
2026 through 2030, the Director of NIH is authorized to 2
make available up to one percent of such amount for pedi-3
atric research under this section.’’. 4
SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS. 5
(a) A
PPLICATION OF PEDIATRICRESEARCHRE-6
QUIREMENTS TOORPHANDRUGS.— 7
(1) I
N GENERAL.—Section 505B(k) of the Fed-8
eral Food, Drug, and Cosmetic Act (21 U.S.C. 9
355c(k)) is amended to read as follows: 10
‘‘(k) R
ELATION TOORPHANDRUGS.— 11
‘‘(1) I
N GENERAL.—This section does not apply 12
to a drug or biological product for an indication for 13
which orphan designation has been granted under 14
section 526 unless the Secretary determines that pe-15
diatric assessments of such drug or biological prod-16
uct required under this section could represent a 17
meaningful therapeutic benefit as described in sub-18
section (c). 19
‘‘(2) D
EFERRALS AND WAIVERS .—Deferrals 20
and waivers under subsections (a)(4) and (a)(5) 21
shall apply to assessments described in this sub-22
section to the same extent and in the same manner 23
as such deferrals and waivers apply with respect to 24
the assessments under subsection (a)(1), and waiv-25
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ers under subsection (b)(2) shall apply to assess-1
ments described in this subsection to the same ex-2
tent and in the same manner as such waivers apply 3
with respect to the assessments required pursuant to 4
subsection (b)(1).’’. 5
(2) A
PPLICABILITY.—The amendment made by 6
paragraph (1) applies only to applications described 7
in subparagraph (A) or (B) of section 505B(a)(1) of 8
the Federal Food, Drug, and Cosmetic Act (21 9
U.S.C. 355c(a)(1)) that are submitted on or after 10
the later of— 11
(A) the date that is 18 months after the 12
date of issuance of the final guidance under 13
subsection (b); and 14
(B) such later date as may be specified by 15
the Secretary of Health and Human Services 16
(referred to in this section as the ‘‘Secretary’’) 17
by regulation. 18
(b) G
UIDANCE.— 19
(1) I
SSUANCE.—The Secretary shall— 20
(A) not later than 1 year after the date of 21
enactment of this Act, issue draft guidance de-22
scribing how, upon the applicability of the 23
amendment made by subsection (a)(1), the re-24
quirements of section 505B of the Federal 25
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Food, Drug, and Cosmetic Act (21 U.S.C. 1
355c) will apply with respect to any drug or bi-2
ological product for an indication within a dis-3
ease or condition for which orphan designation 4
has been granted under section 526 of such Act 5
(21 U.S.C. 360bb); and 6
(B) not later than 18 months after the 7
date of the public meeting required by sub-8
section (c)(1), finalize such draft guidance. 9
(2) C
ONTENTS.—The guidance under sub-10
section (b) shall address the following: 11
(A) Information regarding how full and 12
partial waivers under subsections (a)(5)(A), 13
(a)(5)(B), and (b)(2) of section 505B of the 14
Federal Food, Drug, and Cosmetic Act (21 15
U.S.C. 355c) for any drug or biological product 16
for an indication within a disease or condition 17
for which orphan designation has been granted 18
under section 526 of such Act (21 U.S.C. 19
360bb) will be granted. 20
(B) Application of the requirements of sec-21
tion 505B(e) of such Act (21 U.S.C. 355c(e)) 22
to drugs or biological products for an indication 23
within a disease or condition for which orphan 24
designation has been granted under section 526 25
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of such Act (21 U.S.C. 360bb), including sub-1
mission and timing of planned requests for full 2
or partial waivers and responses by the Food 3
and Drug Administration to those requests. 4
(C) Rare diseases and conditions (as de-5
fined in section 526(a)(2) of such Act (21 6
U.S.C. 360bb(a)(2)) that should be added to 7
the lists under section 505B(a)(5)(E) and 8
505B(b)(2)(E) of such Act, as added by sub-9
section (f), and a process for regularly updating 10
such lists. 11
(D) Situations where the initial pediatric 12
study plan under section 505B(e) of such Act 13
(21 U.S.C. 355c(e)) includes a plan to fulfill 14
the requirements of section 505B(a) of such 15
Act (21 U.S.C. 355c(a)) without requesting 16
waivers in any age group. 17
(E) Consideration of how the Secretary 18
will balance the unique scientific challenges of 19
rare disease drug development with the need for 20
improved pediatric labeling of drugs and bio-21
logical products for indications within diseases 22
or conditions for which orphan designation has 23
been granted under section 526 of such Act (21 24
U.S.C. 360bb). 25
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(F) Considerations of the strengths, weak-1
nesses, appropriateness, and limitations of dif-2
ferent types of real-world evidence specific to 3
the fulfillment of requirements under section 4
505B of such Act (21 U.S.C. 355c). 5
(G) Consideration of input received from 6
the public meeting set forth in subsection (c). 7
(c) P
UBLICMEETING.—The Secretary shall— 8
(1) not later than 6 months after the date of 9
issuance of the draft guidance under subsection 10
(b)(1)(A), hold a public meeting to inform the final 11
guidance to be issued under subsection (b)(1)(B); 12
and 13
(2) publish prior notice of such meeting in the 14
Federal Register. 15
(d) GAO S
TUDY.—Not later than 4 years after the 16
applicability date described in subsection (a)(2), the 17
Comptroller General of the United States shall submit to 18
the Committee on Energy and Commerce and the Com-19
mittee on Ways and Means of the House of Representa-20
tives and the Committee on Health, Education, Labor, 21
and Pensions of the Senate a report that— 22
(1) addresses the impacts of this Act on— 23
(A) rare disease drug development in the 24
United States; and 25
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(B) the availability of pediatric information 1
on drugs and biological products within diseases 2
or conditions for indications for which orphan 3
designation has been granted under section 526 4
of the Federal Food, Drug, and Cosmetic Act 5
(21 U.S.C. 360bb); and 6
(2) includes— 7
(A) the findings of a survey of companies 8
of varying sizes engaged in the development of 9
orphan drugs, which shall include questions re-10
garding the feasibility and other challenges of 11
conducting pediatric studies for such indica-12
tions; 13
(B) input from patient groups and medical 14
provider associations; and 15
(C) an assessment of the impact changes 16
to required pediatric studies had on drug devel-17
opment for rare diseases. 18
(e) R
ULE OFCONSTRUCTION.—Nothing in this sec-19
tion shall be construed to limit requirements for investiga-20
tions, as described in section 505B(a)(3) of the Federal 21
Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)), of 22
molecularly targeted pediatric cancer drugs for which or-23
phan designation has been granted under section 526 of 24
such Act (21 U.S.C. 360bb). 25
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(f) CERTAINTYREGARDING WAIVERS.—Section 1
505B of the Federal Food, Drug, and Cosmetic Act (21 2
U.S.C. 355c) is amended— 3
(1) in subsection (a)(5), by adding at the end 4
the following: 5
‘‘(E) A
UTOMATIC FULL WAIVER LIST .— 6
The Secretary shall maintain a list, posted on 7
the website of the Food and Drug Administra-8
tion, of adult-related diseases and conditions— 9
‘‘(i) with respect to which the nec-10
essary studies are impossible or highly im-11
practicable, as described in subparagraph 12
(A)(i); or 13
‘‘(ii) for which a drug or biological 14
product for such disease or condition oth-15
erwise meets the criteria described in sub-16
paragraph (A).’’; 17
(2) in subsection (b)(2), by adding at the end 18
the following: 19
‘‘(E) A
UTOMATIC FULL WAIVER LIST .— 20
The Secretary shall maintain a list, posted on 21
the website of the Food and Drug Administra-22
tion, of adult-related diseases and conditions 23
with respect to which the necessary studies 24
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would meet the criteria for a full waiver under 1
subparagraph (A).’’; and 2
(3) in subsection (e)(4), by adding at the end 3
the following: ‘‘If, at the time of an applicant’s sub-4
mission of the initial pediatric study plan, the dis-5
ease or condition for which the drug is intended to 6
treat appears on the list under subsection (a)(5)(E), 7
then the assessments for such disease or condition 8
shall be waived under subsection (a)(5).’’. 9
Æ
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