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[Congressional Bills 118th Congress]
[From the U.S. Government Publishing Office]
[H.R. 9979 Introduced in House (IH)]

<DOC>

118th CONGRESS
  2d Session
                                H. R. 9979

   To amend the Federal Food, Drug, and Cosmetic Act to establish a 
process for externally led, science-focused drug development meetings, 
                        and for other purposes.

_______________________________________________________________________

                    IN THE HOUSE OF REPRESENTATIVES

                            October 11, 2024

  Ms. Matsui (for herself and Mr. Bilirakis) 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 establish a 
process for externally led, science-focused drug development meetings, 
                        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 ``Scientific External Process for 
Educated Review of Therapeutics Act of 2024'' or the ``Scientific 
EXPERT Act of 2024''.

SEC. 2. SCIENCE-FOCUSED DRUG DEVELOPMENT MEETINGS.

    The Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.) is 
amended by inserting after section 770 (21 U.S.C. 379dd) the following:

``SEC. 770A. SCIENCE-FOCUSED DRUG DEVELOPMENT MEETINGS.

    ``(a) In General.--The Secretary shall develop and implement a 
process for externally led, science-focused drug development meetings 
to provide an opportunity for medical experts, drug sponsors, 
scientific organizations, and patient organizations to--
            ``(1) discuss science-related challenges impacting the 
        development of drugs for rare diseases and conditions;
            ``(2) identify scientific approaches and opportunities to 
        facilitate the development, review, and approval of such drugs; 
        and
            ``(3) align on novel approaches for the development of 
        drugs for particular diseases, including appropriate clinical 
        trial designs and metrics, manufacturing standards, patient 
        populations, clinical endpoints, the use of biomarkers as 
        surrogate endpoints, and natural history as a control, to 
        advance treatment options to address unmet medical needs.
    ``(b) Arrangement.--
            ``(1) Qualified third party convenor.--The Secretary shall 
        enter into an arrangement with the Reagan-Udall Foundation for 
        the Food and Drug Administration (in this section referred to 
        as the `Foundation') under which the Foundation agrees to 
        convene EL-SFDD meetings in accordance with this section.
            ``(2) Minimum number of meetings.--The Foundation shall 
        convene no fewer than four EL-SFDD meetings each year, with 
        each such meeting focused on addressing a different rare 
        disease or condition or a different group of rare diseases and 
        conditions.
            ``(3) Steering committee.--
                    ``(A) In general.--The Foundation shall establish 
                and maintain a permanent steering committee, to be 
                known as the Science-Focused Drug Development 
                Multistakeholder Steering Committee, to advise the 
                Foundation on implementation of this section, including 
                by--
                            ``(i) establishing a process by which 
                        medical experts, drug sponsors, scientific 
                        organizations, patient organizations, and other 
                        entities can provide suggested meeting topics 
                        to the Foundation;
                            ``(ii) reviewing such suggested meeting 
                        topics for EL-SFDD meetings; and
                            ``(iii) based on the criteria under 
                        subparagraph (B), recommending to the 
                        Foundation topics for EL-SFDD meetings.
                    ``(B) Criteria for meetings.--In formulating 
                recommendations under subparagraph (A), the Foundation 
                shall consider--
                            ``(i) unmet therapeutic needs;
                            ``(ii) the size of the patient population 
                        of the rare disease or condition;
                            ``(iii) whether there are multiple products 
                        in development to prevent or treat the rare 
                        disease or condition involved;
                            ``(iv) whether there is a need for 
                        increased regulatory flexibility to facilitate 
                        the development of products;
                            ``(v) whether the disease or condition 
                        involved would benefit from clarity and 
                        alignment on drug development questions (such 
                        as clinical trial design, natural history as a 
                        control, appropriate clinical endpoints, 
                        biomarkers that may serve as surrogate 
                        endpoints, and other approaches) to expedite 
                        drug development for such disease or condition; 
                        and
                            ``(vi) whether the discussions about such 
                        rare disease or condition may have broader 
                        impact on other rare diseases and conditions.
                    ``(C) Membership.--The members of the Steering 
                Committee shall include--
                            ``(i) representatives of the Center for 
                        Drug Evaluation and Research, the Center for 
                        Biologics Evaluation and Research, and the 
                        Center for Devices and Radiological Health;
                            ``(ii) academic and medical experts;
                            ``(iii) patient representatives;
                            ``(iv) industry experts engaged in the 
                        development of drugs for rare diseases and 
                        conditions.
            ``(4) Planning process.--In planning an EL-SFDD meeting 
        under this section, the Foundation, in consultation with the 
        stakeholders listed in paragraph (5), shall develop--
                    ``(A) a list of the specific objectives of the 
                meeting related to key drug development issues for the 
                rare disease or condition, or group of rare diseases 
                and conditions, with a goal of expediting drug 
                development;
                    ``(B) a proposed agenda for the meeting; and
                    ``(C) a list of medical experts, drug sponsors, 
                scientific organizations, patient organizations, and 
                other entities to be invited to participate in the 
                meeting.
            ``(5) Agency and stakeholder engagement.--Throughout the 
        process of planning an EL-SFDD meeting, the Foundation shall 
        consult with--
                    ``(A) appropriate staff of the Food and Drug 
                Administration;
                    ``(B) the Steering Committee established under this 
                subsection;
                    ``(C) industry representatives engaged in the 
                development of products for rare diseases and 
                conditions to be discussed at such EL-SFDD meeting;
                    ``(D) patient representatives of rare diseases and 
                conditions under discussion in such EL-SFDD meeting; 
                and
                    ``(E) other appropriate stakeholders.
            ``(6) Post-meeting reports.--
                    ``(A) In general.--Within 180 days after an EL-SFDD 
                meeting, the Foundation, in consultation with the 
                stakeholders listed in paragraph (5), shall make 
                publicly available on the website of the Food and Drug 
                Administration--
                            ``(i) a transcript and recording of the 
                        meeting; and
                            ``(ii) a summary analysis of the input 
                        received during the meeting that is relevant to 
                        approval or licensing of drugs for the rare 
                        disease or condition involved.
                    ``(B) Contents.--Each publication under 
                subparagraph (A) shall include a clear identification 
                of--
                            ``(i) areas of consensus;
                            ``(ii) areas where additional clarification 
                        or information is needed to reach consensus; 
                        and
                            ``(iii) next steps agreed upon with the 
                        Food and Drug Administration.
    ``(c) Representatives of FDA Review Divisions.--The Secretary shall 
require appropriate representatives of the review divisions of the Food 
and Drug Administration to participate in each EL-SFDD meeting under 
this section.
    ``(d) Rules of Construction.--Nothing in this section shall be 
construed--
            ``(1) to prevent other third-party organizations from 
        organizing similarly structured EL-SFDD-like meetings to 
        discuss challenges in rare disease drug development;
            ``(2) to require the Food and Drug Administration to 
        participate in additional meetings described in paragraph (1);
            ``(3) to alter the protections offered by laws, 
        regulations, or policies governing disclosure of confidential 
        commercial or trade secret information and any other 
        information exempt from disclosure pursuant to section 552(b) 
        of title 5, United States Code;
            ``(4) to limit the ability of the Secretary to consult with 
        individuals and organizations;
            ``(5) to create a legal right for consultation on any 
        matter or require the Secretary to meet with any particular 
        expert or stakeholder;
            ``(6) to alter agreed-upon goals and procedures identified 
        in the letters described in section 1001(b) of the FDA User Fee 
        Reauthorization Act of 2022; or
            ``(7) to increase the number of review cycles for drugs.
    ``(e) Definitions.--In this section:
            ``(1) The term `EL-SFDD meeting' means an externally led, 
        science-focused drug development meeting.
            ``(2) The terms `rare diseases and conditions' and `rare 
        disease or condition' refer to a rare disease or condition as 
        that term is defined in section 526.
            ``(3) The term `Steering Committee' means the Science-
        Focused Drug Development Multistakeholder Steering Committee 
        established under subsection (b)(3).
    ``(f) Authorization of Appropriations.--
            ``(1) In general.--To carry out this section, there is 
        authorized to be appropriated $1,000,000 for each of fiscal 
        years 2025 through 2029.
            ``(2) Rule of construction.--Nothing in this section shall 
        be construed to prohibit the Foundation from soliciting or 
        accepting funds pursuant to section 770(i) for the purposes of 
        planning or operating an EL-SFDD meeting authorized by this 
        section.

``SEC. 770B. REQUIRED ACTIONS FOLLOWING EL-SFDD MEETINGS.

    ``(a) Incorporation of Input Into Risk-Benefit Assessments.--In 
approving or licensing a drug under subsection (c) or (j) of section 
505 of this Act or subsection (a) or (k) of section 351 of the Public 
Health Service Act, the Secretary shall make public a brief statement--
            ``(1) stating whether any EL-SFDD meeting under section 
        770A was held that was relevant to such approval or licensure; 
        and
            ``(2) if so, including a description of how the Secretary 
        incorporated input from such meeting in the risk-benefit 
        assessment described in section 505(d).
    ``(b) Annual Report.--On an annual basis, the Secretary shall 
submit a report to the Congress summarizing--
            ``(1) the number and topics of EL-SFDD meetings held during 
        the reporting period;
            ``(2) the extent of participation in such meetings from the 
        review divisions of the Food and Drug Administration;
            ``(3) the impact of EL-SFDD meetings on the workload and 
        resources of the Food and Drug Administration; and
            ``(4) an assessment of how the input received during such 
        meetings was used in--
                    ``(A) deliberations throughout the drug development 
                lifecycle;
                    ``(B) regulatory decisionmaking; and
                    ``(C) formulating recommendations for future 
                meetings.
    ``(c) Definition.--In this section, the term `EL-SFDD meeting' has 
the meaning given to that term in section 770A.
    ``(d) Authorization of Appropriations.--To carry out this section, 
there is authorized to be appropriated $1,000,000 for each of fiscal 
years 2025 through 2029.''.
                                 <all>
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