119-s822

[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[S. 822 Introduced in Senate

    (IS) ]

<DOC>

119th CONGRESS
 1st Session
 S. 822

 To amend the Federal Food, Drug, and Cosmetic Act to establish a 
 process for science-focused drug development meetings led by the 
 Reagan-Udall Foundation for the Food and Drug Administration with 
 respect to drugs for rare diseases and conditions, and for other 
 purposes.

_______________________________________________________________________

 IN THE SENATE OF THE UNITED STATES

 March 3, 2025

 Ms. Klobuchar (for herself and Mr. Wicker) introduced the following 
 bill; which was read twice and referred to the Committee on Health, 
 Education, Labor, and Pensions

_______________________________________________________________________

 A BILL

 To amend the Federal Food, Drug, and Cosmetic Act to establish a 
 process for science-focused drug development meetings led by the 
 Reagan-Udall Foundation for the Food and Drug Administration with 
 respect to drugs for rare diseases and conditions, 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 Foundation led, science-focused drug development meetings 
to provide an opportunity for academic researchers and 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 Foundation under which the 
 Foundation agrees to convene EL-SFDD meetings regarding drugs 
 for rare diseases or conditions in accordance with this 
 section.
 ``

    (2) Minimum number of meetings.--The Foundation shall 
 convene no fewer than 4 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 
 licensed and board-certified medical clinicians 
 and academics with expertise in a rare disease 
 or condition, sponsors of drugs for a rare 
 disease or condition, scientific organizations, 
 rare disease or condition 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 rare 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 licensed and board-
 certified medical clinicians;
 ``
            (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;
 ``
            (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;
 ``
        (E) academics or board-certified and licensed 
 medical clinicians with expertise in treating a rare 
 disease or condition; and
 ``
        (F) other appropriate stakeholders.
 ``

    (6) Post-meeting reports.--
 ``
        (A) In general.--Not later than 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 term `rare disease or condition' has the meaning 
 given such term 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--
 ``

    (1) a statement of whether any EL-SFDD meeting under 
 

section 770A was held that was relevant to such approval or 
 licensure; and
 ``

    (2) if a meeting described in paragraph

    (1) was held, 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 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 decision-making; 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.''.
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