II 

118TH CONGRESS 

2D SESSION S. 4905 

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 

JULY 31, 2024 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Innovation in Pediatric 

5 Drugs Act of 2024’’. 

6 SEC. 2. ENSURING COMPLETION OF PEDIATRIC STUDY RE7 QUIREMENTS. 

8 (a) EQUAL ACCOUNTABILITY FOR PEDIATRIC STUDY

9 REQUIREMENTS.—Section 505B(d) of the Federal Food, 


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1 Drug, and Cosmetic Act (21 U.S.C. 355c(d)) is amend2 ed— 

3 (1) in paragraph (1), by striking ‘‘Beginning 

4 270’’ and inserting ‘‘NONCOMPLIANCE LETTER.— 

5 Beginning 270’’; 

6 (2) in paragraph (2)— 

7 (A) by striking ‘‘The drug or’’ and insert8 ing ‘‘EFFECT OF NONCOMPLIANCE.—The drug 

9 or’’; and 

10 (B) by striking ‘‘(except that the drug or 

11 biological product shall not be subject to action 

12 under section 303)’’ and inserting ‘‘(except that 

13 the drug or biological product shall be subject 

14 to action under section 303 only if such person 

15 demonstrated a lack of due diligence in satis16 fying the applicable requirement)’’; and 

17 (3) by adding at the end the following: 

18 ‘‘(3) LIMITATION.—The Secretary shall not 

19 issue enforcement actions under section 303 for fail20 ures under this subsection in the case of a drug or 

21 biological product that is no longer marketed.’’. 

22 (b) DUE DILIGENCE.—Section 505B(d) of the Fed23 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)), 

24 as amended by subsection (a), is further amended by add25 ing at the end the following: 


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1 ‘‘(4) DUE DILIGENCE.—Before the Secretary 

2 may conclude that a person failed to submit or oth3 erwise meet a requirement as described in the mat4 ter preceding paragraph (1), the Secretary shall— 

5 ‘‘(A) issue a noncompliance letter pursuant 

6 to paragraph (1); 

7 ‘‘(B) provide such person with a 45-day 

8 period beginning on the date of receipt of such 

9 noncompliance letter to respond in writing as 

10 set forth in such paragraph; and 

11 ‘‘(C) after reviewing such written response, 

12 determine whether the person demonstrated a 

13 lack of due diligence in satisfying such require14 ment.’’. 

15 (c) CONFORMING AMENDMENTS.—Section 

16 303(f)(4)(A) of the Federal Food, Drug, and Cosmetic Act 

17 (21 U.S.C. 333(f)(4)(A)) is amended by striking ‘‘or 505– 

18 1’’ and inserting ‘‘505–1, or 505B’’. 

19 (d) TRANSITION RULE.—The Secretary of Health 

20 and Human Services may take enforcement action under 

21 section 303 of the Federal Food, Drug, and Cosmetic Act 

22 (21 U.S.C. 333) for failures described in section 505B(d) 

23 of such Act (21 U.S.C. 355c(d)) only if such failures occur 

24 on or after the date that is 180 days after the date of 

25 enactment of this Act. 


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1 SEC. 3. FDA REPORT ON PREA ENFORCEMENT. 

2 Section 508(b) of the Food and Drug Administration 

3 Safety and Innovation Act (21 U.S.C. 355c–1(b)) is 

4 amended— 

5 (1) in paragraph (11), by striking the semicolon 

6 at the end and inserting ‘‘, including an evaluation 

7 of compliance with deadlines provided for in defer8 rals and deferral extensions;’’; 

9 (2) in paragraph (15), by striking ‘‘and’’ at the 

10 end; 

11 (3) in paragraph (16), by striking the period at 

12 the end and inserting ‘‘; and’’; and 

13 (4) by adding at the end the following: 

14 ‘‘(17) a listing of penalties, settlements, or pay15 ments under section 303 of the Federal Food, Drug, 

16 and Cosmetic Act (21 U.S.C. 353) for failure to 

17 comply with requirements under such section 505B, 

18 including, for each penalty, settlement, or payment, 

19 the name of the drug, the sponsor thereof, and the 

20 amount of the penalty, settlement, or payment im21 posed.’’. 

22 SEC. 4. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS. 

23 Section 409I(d) of the Public Health Service Act (42 

24 U.S.C. 284m(d)) is amended to read as follows: 

25 ‘‘(d) FUNDING.—Of the amount made available for 

26 pediatric research to each national research institute and 


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1 national center under this title for each of fiscal years 

2 2025, 2026, and 2027, the Director of NIH is authorized 

3 to make available up to one percent of such amount for 

4 pediatric research under this section.’’. 

5 SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS. 

6 (a) APPLICATION OF PEDIATRIC RESEARCH RE7 QUIREMENTS TO ORPHAN DRUGS.— 

8 (1) IN GENERAL.—Section 505B(k) of the Fed9 eral Food, Drug, and Cosmetic Act (21 U.S.C. 

10 355c(k)) is amended to read as follows: 

11 ‘‘(k) RELATION TO ORPHAN DRUGS.— 

12 ‘‘(1) IN GENERAL.—This section does not apply 

13 to a drug or biological product for an indication for 

14 which orphan designation has been granted under 

15 section 526 unless the Secretary determines that pe16 diatric assessments of such drug or biological prod17 uct required under this section could represent a 

18 meaningful therapeutic benefit as described in sub19 section (c). 

20 ‘‘(2) DEFERRALS AND WAIVERS.—Deferrals 

21 and waivers under subsections (a)(4) and (a)(5) 

22 shall apply to assessments described in this sub23 section to the same extent and in the same manner 

24 as such deferrals and waivers apply with respect to 

25 the assessments under subsection (a)(1), and waivVerDate Sep 11 2014 20:04 Aug 29, 2024 Jkt 049200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S4905.IS S4905

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1 ers under subsection (b)(2) shall apply to assess2 ments described in this subsection to the same ex3 tent and in the same manner as such waivers apply 

4 with respect to the assessments required pursuant to 

5 subsection (b)(1).’’. 

6 (2) APPLICABILITY.—The amendment made by 

7 paragraph (1) applies only to applications described 

8 in subparagraph (A) or (B) of section 505B(a)(1) of 

9 the Federal Food, Drug, and Cosmetic Act (21 

10 U.S.C. 355c(a)(1)) that are submitted on or after 

11 the later of— 

12 (A) the date that is 18 months after the 

13 date of issuance of the final guidance under 

14 subsection (b); and 

15 (B) such later date as may be specified by 

16 the Secretary of Health and Human Services 

17 (referred to in this section as the ‘‘Secretary’’) 

18 by regulation. 

19 (b) GUIDANCE.— 

20 (1) ISSUANCE.—The Secretary shall— 

21 (A) not later than 1 year after the date of 

22 enactment of this Act, issue draft guidance de23 scribing how, upon the applicability of the 

24 amendment made by subsection (a)(1), the re25 quirements of section 505B of the Federal 


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1 Food, Drug, and Cosmetic Act (21 U.S.C. 

2 355c) will apply with respect to any drug or bi3 ological product for an indication within a dis4 ease or condition for which orphan designation 

5 has been granted under section 526 of such Act 

6 (21 U.S.C. 360bb); and 

7 (B) not later than 18 months after the 

8 date of the public meeting required by sub9 section (c)(1), finalize such draft guidance. 

10 (2) CONTENTS.—The guidance under sub11 section (b) shall address the following: 

12 (A) Information regarding how full and 

13 partial waivers under subsections (a)(5)(A), 

14 (a)(5)(B), and (b)(2) of section 505B of the 

15 Federal Food, Drug, and Cosmetic Act (21 

16 U.S.C. 355c) for any drug or biological product 

17 for an indication within a disease or condition 

18 for which orphan designation has been granted 

19 under section 526 of such Act (21 U.S.C. 

20 360bb) will be granted. 

21 (B) Application of the requirements of sec22 tion 505B(e) of such Act (21 U.S.C. 355c(e)) 

23 to drugs or biological products for an indication 

24 within a disease or condition for which orphan 

25 designation has been granted under section 526 


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1 of such Act (21 U.S.C. 360bb), including sub2 mission and timing of planned requests for full 

3 or partial waivers and responses by the Food 

4 and Drug Administration to those requests. 

5 (C) Rare diseases and conditions (as de6 fined in section 526(a)(2) of such Act (21 

7 U.S.C. 360bb(a)(2)) that should be added to 

8 the lists under section 505B(a)(5)(E) and 

9 505B(b)(2)(E) of such Act, as added by sub10 section (f), and a process for regularly updating 

11 such lists. 

12 (D) Situations where the initial pediatric 

13 study plan under section 505B(e) of such Act 

14 (21 U.S.C. 355c(e)) includes a plan to fulfill 

15 the requirements of section 505B(a) of such 

16 Act (21 U.S.C. 355c(a)) without requesting 

17 waivers in any age group. 

18 (E) Consideration of how the Secretary 

19 will balance the unique scientific challenges of 

20 rare disease drug development with the need for 

21 improved pediatric labeling of drugs and bio22 logical products for indications within diseases 

23 or conditions for which orphan designation has 

24 been granted under section 526 of such Act (21 

25 U.S.C. 360bb). 


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1 (F) Considerations of the strengths, weak2 nesses, appropriateness, and limitations of dif3 ferent types of real-world evidence specific to 

4 the fulfillment of requirements under section 

5 505B of such Act (21 U.S.C. 355c). 

6 (G) Consideration of input received from 

7 the public meeting set forth in subsection (c). 

8 (c) PUBLIC MEETING.—The Secretary shall— 

9 (1) not later than 6 months after the date of 

10 issuance of the draft guidance under subsection 

11 (b)(1)(A), hold a public meeting to inform the final 

12 guidance to be issued under subsection (b)(1)(B); 

13 and 

14 (2) publish prior notice of such meeting in the 

15 Federal Register. 

16 (d) GAO STUDY.—Not later than 4 years after the 

17 applicability date described in subsection (a)(2), the 

18 Comptroller General of the United States shall submit to 

19 the Committee on Energy and Commerce and the Com20 mittee on Ways and Means of the House of Representa21 tives and the Committee on Health, Education, Labor, 

22 and Pensions of the Senate a report that— 

23 (1) addresses the impacts of this Act on— 

24 (A) rare disease drug development in the 

25 United States; and 


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1 (B) the availability of pediatric information 

2 on drugs and biological products within diseases 

3 or conditions for indications for which orphan 

4 designation has been granted under section 526 

5 of the Federal Food, Drug, and Cosmetic Act 

6 (21 U.S.C. 360bb); and 

7 (2) includes— 

8 (A) the findings of a survey of companies 

9 of varying sizes engaged in the development of 

10 orphan drugs, which shall include questions re11 garding the feasibility and other challenges of 

12 conducting pediatric studies for such indica13 tions; 

14 (B) input from patient groups and medical 

15 provider associations; and 

16 (C) an assessment of the impact changes 

17 to required pediatric studies had on drug devel18 opment for rare diseases. 

19 (e) RULE OF CONSTRUCTION.—Nothing in this sec20 tion shall be construed to limit requirements for investiga21 tions, as described in section 505B(a)(3) of the Federal 

22 Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)), of 

23 molecularly targeted pediatric cancer drugs for which or24 phan designation has been granted under section 526 of 

25 such Act (21 U.S.C. 360bb). 


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1 (f) CERTAINTY REGARDING WAIVERS.—Section 

2 505B of the Federal Food, Drug, and Cosmetic Act (21 

3 U.S.C. 355c) is amended— 

4 (1) in subsection (a)(5), by adding at the end 

5 the following: 

6 ‘‘(E) AUTOMATIC FULL WAIVER LIST.— 

7 The Secretary shall maintain a list, posted on 

8 the website of the Food and Drug Administra9 tion, of adult-related diseases and conditions— 

10 ‘‘(i) with respect to which the nec11 essary studies are impossible or highly im12 practicable, as described in subparagraph 

13 (A)(i); or 

14 ‘‘(ii) for which a drug or biological 

15 product for such disease or condition oth16 erwise meets the criteria described in sub17 paragraph (A).’’; 

18 (2) in subsection (b)(2), by adding at the end 

19 the following: 

20 ‘‘(E) AUTOMATIC FULL WAIVER LIST.— 

21 The Secretary shall maintain a list, posted on 

22 the website of the Food and Drug Administra23 tion, of adult-related diseases and conditions 

24 with respect to which the necessary studies 


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1 would meet the criteria for a full waiver under 

2 subparagraph (A).’’; and 

3 (3) in subsection (e)(4), by adding at the end 

4 the following: ‘‘If, at the time of an applicant’s sub5 mission of the initial pediatric study plan, the dis6 ease or condition for which the drug is intended to 

7 treat appears on the list under subsection (a)(5)(E), 

8 then the assessments for such disease or condition 

9 shall be waived under subsection (a)(5).’’. 

Æ 


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