I 

118TH CONGRESS 

1ST SESSION H. R. 6288 

To amend the Federal Food, Drug, and Cosmetic Act to provide for a 

Pediatric Brain Tumor Real-World Data Registry Program, and for 

other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

NOVEMBER 8, 2023 

Mr. BERA (for himself and Mr. KELLY of Pennsylvania) introduced the 

following bill; which was referred to the Committee on Energy and Commerce 

A BILL 

To amend the Federal Food, Drug, and Cosmetic Act to 

provide for a Pediatric Brain Tumor Real-World Data 

Registry Program, 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 ‘‘Data for Pediatric 

5 Brain Cancer Act of 2023’’. 

6 SEC. 2. FINDINGS. 

7 Congress finds the following: 


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1 (1) Randomized clinical trials require the use of 

2 control groups to serve as the baseline for deter3 mining the effectiveness of a study treatment. 

4 (2) Because of the rarity of some diseases, it is 

5 often hard to find enough study participants in a 

6 sufficient amount of time, making randomized clin7 ical trials for these treatments infeasible. Couple this 

8 with the fact that the current standard of care for 

9 many rare diseases is ineffective (often leading to 

10 death), making administering the current standard 

11 of care unethical. 

12 (3) In these cases, data reflecting patient treat13 ment in routine clinical practice can be used to de14 velop external control groups for single-arm trials. 

15 Clinical trials using external control cohorts can pro16 vide valuable benchmark results on potential com17 parator treatment efficacy. 

18 (4) The Food and Drug Administration (in this 

19 section referred to as the ‘‘FDA’’) has distributed 

20 draft guidance on this issue commenting on consid21 erations for the use of real-world data for the design 

22 and conduct of externally controlled trials. 

23 (5) Through collaboration with community ad24 vocates and industry partners with input from the 

25 FDA, researchers focused on atypical teratoid 


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1 rhabdoid tumor (in this section referred to as 

2 ‘‘ATRT’’), an ultra-rare and poor prognosis pedi3 atric brain tumor, have taken the lead in the design 

4 of real-world data sets. 

5 (6) Because of the advancements in the re6 search, ATRT has presented itself as the ideal can7 didate to pilot the development of real-world data 

8 sets for use in external control cohorts. 

9 SEC. 3. PEDIATRIC BRAIN TUMOR REAL-WORLD DATA REG10 ISTRY PROGRAM. 

11 Chapter X of the Federal Food, Drug, and Cosmetic 

12 Act (21 U.S.C. 391 et seq.) is amended by adding at the 

13 end the following: 

14 ‘‘SEC. 1015. PEDIATRIC BRAIN TUMOR REAL-WORLD DATA 

15 REGISTRY PROGRAM. 

16 ‘‘(a) IN GENERAL.—The Secretary shall carry out a 

17 program, to be known as the Pediatric Brain Tumor Real18 World Data Registry Program, to strengthen and expand 

19 activities related to the collection, sharing, and use of real20 world data for children with brain tumors. 

21 ‘‘(b) REQUIREMENTS.—In carrying out the program 

22 under this section, the Secretary shall— 

23 ‘‘(1) develop and maintain (or support the de24 velopment and maintenance of) a registry of realVerDate Sep 11 2014 07:09 Nov 15, 2023 Jkt 049200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H6288.IH H6288

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1 world data for children with atypical teratoid 

2 rhabdoid tumors; 

3 ‘‘(2) consider new and innovative approaches 

4 and technology for data collection, integration, and 

5 analysis; 

6 ‘‘(3) continue and expand activities, which may 

7 include existing data collection activities, to establish 

8 real-world database infrastructure; 

9 ‘‘(4) provide support for data integration, 

10 bioinformatics, and statistical analyses; and 

11 ‘‘(5) identify potential uses of real-world data 

12 registries as external control cohorts for pediatric 

13 brain tumor clinical trial design. 

14 ‘‘(c) COLLABORATION AND CONSULTATION.—In car15 rying out the program under this section, the Secretary 

16 shall collaborate and consult, as appropriate, with public 

17 and private entities, including relevant departments and 

18 agencies, academic institutions, and industry. 

19 ‘‘(d) GRANTS, CONTRACTS, AND COOPERATIVE

20 AGREEMENTS.— 

21 ‘‘(1) AWARDS.—In carrying out the program 

22 under this section, the Secretary shall award grants, 

23 contracts, and cooperative agreements to academic 

24 institutions and other entities with relevant expertise 


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1 in pediatric neuro-oncology or the collection, integra2 tion, and analysis of real-word data. 

3 ‘‘(2) APPLICATION.— 

4 ‘‘(A) IN GENERAL.—To seek an award 

5 under paragraph (1), an entity described in 

6 such paragraph shall submit to the Secretary 

7 an application at such time, in such manner, 

8 and containing such information as the Sec9 retary may require. 

10 ‘‘(B) CONTENTS.—An application under 

11 subparagraph (A) shall include a description of 

12 how the applicant will partner, as applicable, 

13 with academic institutions or a consortium of 

14 academic institutions that have relevant exper15 tise, such as expertise in pediatric neuro-oncol16 ogy, clinical research, or the application of 

17 bioinformatics or statistics. 

18 ‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—For 

19 carrying out this section, there are authorized to be appro20 priated $2,000,000 for fiscal year 2025.’’. 

Æ 


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