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[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H.R. 6288 Introduced in House (IH)]
<DOC>
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 Representatives 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:
(1) Randomized clinical trials require the use of control
groups to serve as the baseline for determining 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 clinical 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 treatment in
routine clinical practice can be used to develop external
control groups for single-arm trials. Clinical trials using
external control cohorts can provide valuable benchmark results
on potential comparator 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referred to as the ``FDA'') has distributed draft guidance on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 considerations for the use of real-
world data for the design and conduct of externally controlled
trials.
(5) Through collaboration with community advocates and
industry partners with input from the FDA, researchers focused
on atypical teratoid rhabdoid tumor (in this section referred
to as ``ATRT''), an ultra-rare and poor prognosis pediatric
brain tumor, have taken the lead in the design of real-world
data sets.
(6) Because of the advancements in the research, ATRT has
presented itself as the ideal candidate to pilot the
development of real-world data sets for use in external control
cohorts.
SEC. 3. PEDIATRIC BRAIN TUMOR REAL-WORLD DATA REGISTRY 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 development and
maintenance of) a registry of real-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 carrying 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 in pediatric neuro-oncology or
the collection, integration, and analysis of real-word data.
``(2) Application.--
``(A) In general.--To seek an award under paragraph
(1), an entity described in such paragraph shall submit
to the Secretary an application at such time, in such
manner, and containing such information as the
Secretary may require.
``(B) Contents.--An application under subparagraph
(A) shall include a description of how the applicant
will partner, as applicable, with academic institutions
or a consortium of academic institutions that have
relevant expertise, such as expertise in pediatric
neuro-oncology, clinical research, or the application
of bioinformatics or statistics.
``(e) Authorization of Appropriations.--For carrying out this
section, there are authorized to be appropriated $2,000,000 for fiscal
year 2025.''.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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