[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        referred to as the ``FDA'') has distributed draft guidance on 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>