Scientific External Process for Educated Review of Therapeutics Act of 2025 or the Scientific EXPERT Act of 2025
This bill requires the Food and Drug Administration (FDA) to facilitate and participate in externally led, science-focused drug development meetings to discuss the development of treatments for rare diseases and conditions.
The FDA must enter into an arrangement with the Reagan-Udall Foundation for the FDA under which the foundation agrees to convene such meetings. Meetings must be held at least four times a year, and each meeting must focus on a different rare disease or condition.
The foundation must establish a permanent steering committee to review and recommend topics for each meeting. In evaluating potential topics, the committee must consider unmet therapeutic needs, patient population sizes for different diseases and conditions, and whether a disease or condition would benefit from clarity and alignment on drug development questions, among other factors.
In planning each meeting, the foundation must develop a list of medical experts, drug sponsors, scientific organizations, patient organizations, and other entities to be invited to participate. Representatives of the FDA’s review divisions must attend each meeting.
After each meeting, the foundation must make available a summary of the meeting noting areas of consensus, areas where additional clarification or information is needed, and next steps agreed upon with the FDA.
The bill also requires the FDA to indicate whether it incorporated any input from these meetings when approving a new drug or biologic.
[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[H.R. 1532 Introduced in House (IH)]
<DOC>
119th CONGRESS
1st Session
H. R. 1532
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
February 24, 2025
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 2025'' or the ``Scientific
EXPERT Act of 2025''.
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 were or 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 be subject to all relevant conflict of
interest policies of the Foundation and 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; and
``(iv) industry representatives 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 shall make publicly available
on the website of the Food and Drug Administration--
``(i) a transcript and recording of the
meeting; and
``(ii) in consultation with the
stakeholders listed in paragraph (5), 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 2026 through 2030.
``(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 2026 through 2030.''.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
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