I 

118TH CONGRESS 

1ST SESSION H. R. 6664 

To encourage innovation in the development of pediatric drugs, and for 

other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

DECEMBER 7, 2023 

Ms. ESHOO (for herself and Mr. MCCAUL) introduced the following bill; which 

was referred to the Committee on Energy and Commerce 

A BILL 

To encourage innovation in the development of pediatric 

drugs, 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 2023’’. 

6 SEC. 2. PEDIATRIC STUDIES OF ORPHAN DRUGS. 

7 (a) APPLICATION OF PEDIATRIC RESEARCH RE8 QUIREMENTS TO ORPHAN DRUGS.— 


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1 (1) IN GENERAL.—Section 505B of the Federal 

2 Food, Drug, and Cosmetic Act (21 U.S.C. 355c) is 

3 amended by striking subsection (k). 

4 (2) APPLICABILITY.—The amendment made by 

5 paragraph (1) applies only to applications described 

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

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

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

9 the date that is 18 months after the date of enact10 ment of this Act. 

11 (b) GUIDANCE.—The Secretary of Health and 

12 Human Services (referred to in this Act as the ‘‘Sec13 retary’’) shall— 

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

15 enactment of this Act, issue draft guidance describ16 ing how, upon the applicability of the amendment 

17 made by subsection (a)(1), the requirements of sec18 tion 505B of the Federal Food, Drug, and Cosmetic 

19 Act (21 U.S.C. 355c) will apply with respect to any 

20 drug or biological product for an indication for 

21 which orphan designation has been granted under 

22 section 526 of such Act (21 U.S.C. 360bb); and 

23 (2) not later than 18 months after the date of 

24 enactment of this Act, finalize such draft guidance. 


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1 (c) CONTENT OF GUIDANCE.—The guidance under 

2 subsection (b) shall address the following: 

3 (1) Information regarding how full and partial 

4 waivers under subsections (a)(5)(A), (a)(5)(B), and 

5 (b)(2) of section 505B of the Federal Food, Drug, 

6 and Cosmetic Act (21 U.S.C. 355c) for any drug or 

7 biological product for an indication for which orphan 

8 designation has been granted under section 526 of 

9 such Act (21 U.S.C. 360bb) will be granted (includ10 ing a description of impossible or highly impracti11 cable studies for indications for which orphan des12 ignation has been granted under such section 526). 

13 (2) Application of the requirements of section 

14 505B(e) of such Act (21 U.S.C. 355c(e)) to drugs 

15 or biological products for an indication for which or16 phan designation has been granted under section 

17 526 of such Act (21 U.S.C. 360bb), including sub18 mission and timing of planned requests for full or 

19 partial waivers and responses by the Food and Drug 

20 Administration to those requests. 

21 (3) Process for regularly updating conditions 

22 that automatically qualify for a waiver under sub23 section (a)(5)(A) or (b)(2)(A) of section 505B of 

24 such Act (21 U.S.C. 355c). 


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1 (4) Situations where the initial pediatric study 

2 plan under section 505B(e) of such Act (21 U.S.C. 

3 355c(e)) includes a plan to study the drug or bio4 logical product in all relevant pediatric age groups. 

5 (5) Consideration of how the Secretary of 

6 Health and Human Services will balance the unique 

7 scientific challenges of rare disease drug develop8 ment with the need for improved pediatric labeling 

9 of drugs and biological products for indications for 

10 which orphan designation has been granted under 

11 section 526 of such Act (21 U.S.C. 360bb). 

12 (6) Applicability of real-world evidence in the 

13 fulfillment of requirements under section 505B of 

14 such Act (21 U.S.C. 355c). 

15 (7) Consideration of input received from the 

16 public meeting set forth in subsection (d). 

17 (d) PUBLIC MEETING.—The Secretary shall— 

18 (1) not later than 1 year after the date of en19 actment of this Act, hold a public meeting to inform 

20 the final guidance to be issued under subsection 

21 (b)(2); and 

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

23 Federal Register. 

24 (e) GAO STUDY.—Not later than 4 years after the 

25 date of enactment of this Act, the Comptroller General 


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1 of the United States shall submit to the Committee on 

2 Energy and Commerce and the Committee on Ways and 

3 Means of the House of Representatives and the Committee 

4 on Health, Education, Labor, and Pensions of the Senate 

5 a report that— 

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

7 (A) rare disease drug development in the 

8 United States; and 

9 (B) the increased availability of pediatric 

10 information on drugs and biological products 

11 for indications for which orphan designation 

12 has been granted under section 526 of the Fed13 eral Food, Drug, and Cosmetic Act (21 U.S.C. 

14 360bb); and 

15 (2) includes— 

16 (A) the findings of a survey of companies 

17 of varying sizes engaged in the development of 

18 orphan drugs; and 

19 (B) input from patient groups and medical 

20 provider associations. 

21 (f) RULE OF CONSTRUCTION.—Nothing in this sec22 tion shall be construed to limit requirements for investiga23 tions, as described in section 505B(a)(3) of the Federal 

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

25 molecularly targeted pediatric cancer drugs for which orVerDate Sep 11 2014 01:53 Dec 27, 2023 Jkt 049200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H6664.IH H6664

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1 phan designation has been granted under section 526 of 

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

3 SEC. 3. REAUTHORIZING THE PROGRAM FOR PEDIATRIC 

4 STUDIES OF DRUGS. 

5 Section 409I(d)(1) of the Public Health Service Act 

6 (42 U.S.C. 284m(d)(1)) is amended in paragraph (1) by 

7 striking ‘‘$25,000,000 for each of fiscal years 2023 

8 through 2027’’ and inserting ‘‘$50,000,000 for each of fis9 cal years 2023 through 2027’’. 

10 SEC. 4. ENSURING COMPLETION OF PEDIATRIC STUDY RE11 QUIREMENTS. 

12 (a) EQUAL ACCOUNTABILITY FOR PEDIATRIC STUDY

13 REQUIREMENTS.—Section 505B(d)(2) of the Federal 

14 Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)(2)) is 

15 amended by striking ‘‘(except that the drug or biological 

16 product shall not be subject to action under section 303)’’. 

17 (b) CONFORMING AMENDMENTS.—Section 

18 303(f)(4)(A) of the Federal Food, Drug, and Cosmetic Act 

19 (21 U.S.C. 333(f)(4)(A)) is amended by striking ‘‘or 505– 

20 1’’ and inserting ‘‘505–1, or 505B’’. 

21 (c) TRANSITION RULE.—The Secretary of Health 

22 and Human Services shall take no enforcement actions 

23 under section 303 of the Federal Food, Drug, and Cos24 metic Act (21 U.S.C. 333) for failures described in section 


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1 505B(d) of such Act (21 U.S.C. 355c(d)) before the date 

2 this is 180 days after the date of enactment of this Act. 

3 SEC. 5. FDA REPORT ON PREA ENFORCEMENT. 

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

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

6 amended— 

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

8 at the end and inserting ‘‘, including an assessment 

9 of the appropriateness of deferrals and deferral ex10 tensions granted for such products and an evalua11 tion of compliance with deadlines provided for in 

12 such deferrals and deferral extensions;’’; 

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

14 end; 

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

16 the end and inserting ‘‘; and’’; and 

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

18 ‘‘(17) a listing of penalties, settlements, or pay19 ments under section 303 of the Federal Food, Drug, 

20 and Cosmetic Act for failure to comply with require21 ments under section 505B of such Act, including for 

22 each penalty, settlement, or payment the name of 

23 the drug, the sponsor thereof, and the amount of the 

24 penalty, settlement, or payment imposed.’’. 

Æ 


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