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. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Data for Pediatric 
Brain Cancer Act of 2023’’. 
SEC. 2. FINDINGS. 
Congress finds the following: 
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(1) Randomized clinical trials require the use of 
control groups to serve as the baseline for deter-
mining the effectiveness of a study treatment. 
(2) Because of the rarity of some diseases, it is 
often hard to find enough study participants in a 
sufficient amount of time, making randomized clin-
ical trials for these treatments infeasible. Couple this 
with the fact that the current standard of care for 
many rare diseases is ineffective (often leading to 
death), making administering the current standard 
of care unethical. 
(3) In these cases, data reflecting patient treat-
ment in routine clinical practice can be used to de-
velop external control groups for single-arm trials. 
Clinical trials using external control cohorts can pro-
vide valuable benchmark results on potential com-
parator treatment efficacy. 
(4) The Food and Drug Administration (in this 
section referred to as the ‘‘FDA’’) has distributed 
draft guidance on this issue commenting on consid-
erations for the use of real-world data for the design 
and conduct of externally controlled trials. 
(5) Through collaboration with community ad-
vocates and industry partners with input from the 
FDA, researchers focused on atypical teratoid 
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rhabdoid tumor (in this section referred to as 
‘‘ATRT’’), an ultra-rare and poor prognosis pedi-
atric brain tumor, have taken the lead in the design 
of real-world data sets. 
(6) Because of the advancements in the re-
search, ATRT has presented itself as the ideal can-
didate to pilot the development of real-world data 
sets for use in external control cohorts. 
SEC. 3. PEDIATRIC BRAIN TUMOR REAL-WORLD DATA REG-
ISTRY PROGRAM. 
Chapter X of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 391 et seq.) is amended by adding at the 
end the following: 
‘‘SEC. 1015. PEDIATRIC BRAIN TUMOR REAL-WORLD DATA 
REGISTRY PROGRAM. 
‘‘(a) IN GENERAL.—The Secretary shall carry out a 
program, to be known as the Pediatric Brain Tumor Real- 
World Data Registry Program, to strengthen and expand 
activities related to the collection, sharing, and use of real- 
world data for children with brain tumors. 
‘‘(b) REQUIREMENTS.—In carrying out the program 
under this section, the Secretary shall— 
‘‘(1) develop and maintain (or support the de-
velopment and maintenance of) a registry of real- 
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world data for children with atypical teratoid 
rhabdoid tumors; 
‘‘(2) consider new and innovative approaches 
and technology for data collection, integration, and 
analysis; 
‘‘(3) continue and expand activities, which may 
include existing data collection activities, to establish 
real-world database infrastructure; 
‘‘(4) provide support for data integration, 
bioinformatics, and statistical analyses; and 
‘‘(5) identify potential uses of real-world data 
registries as external control cohorts for pediatric 
brain tumor clinical trial design. 
‘‘(c) COLLABORATION AND CONSULTATION.—In car-
rying out the program under this section, the Secretary 
shall collaborate and consult, as appropriate, with public 
and private entities, including relevant departments and 
agencies, academic institutions, and industry. 
‘‘(d) GRANTS, CONTRACTS, 
AND
COOPERATIVE 
AGREEMENTS.— 
‘‘(1) AWARDS.—In carrying out the program 
under this section, the Secretary shall award grants, 
contracts, and cooperative agreements to academic 
institutions and other entities with relevant expertise 
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