I 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Scientific External 

5 Process for Educated Review of Therapeutics Act of 

6 2024’’ or the ‘‘Scientific EXPERT Act of 2024’’. 


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1 SEC. 2. SCIENCE-FOCUSED DRUG DEVELOPMENT MEET2 INGS. 

3 The Federal Food, Drug, and Cosmetic Act (21 

4 U.S.C. 301 et seq.) is amended by inserting after section 

5 770 (21 U.S.C. 379dd) the following: 

6 ‘‘SEC. 770A. SCIENCE-FOCUSED DRUG DEVELOPMENT 

7 MEETINGS. 

8 ‘‘(a) IN GENERAL.—The Secretary shall develop and 

9 implement a process for externally led, science-focused 

10 drug development meetings to provide an opportunity for 

11 medical experts, drug sponsors, scientific organizations, 

12 and patient organizations to— 

13 ‘‘(1) discuss science-related challenges impact14 ing the development of drugs for rare diseases and 

15 conditions; 

16 ‘‘(2) identify scientific approaches and opportu17 nities to facilitate the development, review, and ap18 proval of such drugs; and 

19 ‘‘(3) align on novel approaches for the develop20 ment of drugs for particular diseases, including ap21 propriate clinical trial designs and metrics, manufac22 turing standards, patient populations, clinical 

23 endpoints, the use of biomarkers as surrogate 

24 endpoints, and natural history as a control, to ad25 vance treatment options to address unmet medical 

26 needs. 


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1 ‘‘(b) ARRANGEMENT.— 

2 ‘‘(1) QUALIFIED THIRD PARTY CONVENOR.— 

3 The Secretary shall enter into an arrangement with 

4 the Reagan-Udall Foundation for the Food and 

5 Drug Administration (in this section referred to as 

6 the ‘Foundation’) under which the Foundation 

7 agrees to convene EL–SFDD meetings in accord8 ance with this section. 

9 ‘‘(2) MINIMUM NUMBER OF MEETINGS.—The 

10 Foundation shall convene no fewer than four EL– 

11 SFDD meetings each year, with each such meeting 

12 focused on addressing a different rare disease or 

13 condition or a different group of rare diseases and 

14 conditions. 

15 ‘‘(3) STEERING COMMITTEE.— 

16 ‘‘(A) IN GENERAL.—The Foundation shall 

17 establish and maintain a permanent steering 

18 committee, to be known as the Science-Focused 

19 Drug Development Multistakeholder Steering 

20 Committee, to advise the Foundation on imple21 mentation of this section, including by— 

22 ‘‘(i) establishing a process by which 

23 medical experts, drug sponsors, scientific 

24 organizations, patient organizations, and 


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1 other entities can provide suggested meet2 ing topics to the Foundation; 

3 ‘‘(ii) reviewing such suggested meeting 

4 topics for EL–SFDD meetings; and 

5 ‘‘(iii) based on the criteria under sub6 paragraph (B), recommending to the 

7 Foundation topics for EL–SFDD meet8 ings. 

9 ‘‘(B) CRITERIA FOR MEETINGS.—In for10 mulating recommendations under subparagraph 

11 (A), the Foundation shall consider— 

12 ‘‘(i) unmet therapeutic needs; 

13 ‘‘(ii) the size of the patient population 

14 of the rare disease or condition; 

15 ‘‘(iii) whether there are multiple prod16 ucts in development to prevent or treat the 

17 rare disease or condition involved; 

18 ‘‘(iv) whether there is a need for in19 creased regulatory flexibility to facilitate 

20 the development of products; 

21 ‘‘(v) whether the disease or condition 

22 involved would benefit from clarity and 

23 alignment on drug development questions 

24 (such as clinical trial design, natural his25 tory as a control, appropriate clinical 


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1 endpoints, biomarkers that may serve as 

2 surrogate endpoints, and other approaches) 

3 to expedite drug development for such dis4 ease or condition; and 

5 ‘‘(vi) whether the discussions about 

6 such rare disease or condition may have 

7 broader impact on other rare diseases and 

8 conditions. 

9 ‘‘(C) MEMBERSHIP.—The members of the 

10 Steering Committee shall include— 

11 ‘‘(i) representatives of the Center for 

12 Drug Evaluation and Research, the Center 

13 for Biologics Evaluation and Research, and 

14 the Center for Devices and Radiological 

15 Health; 

16 ‘‘(ii) academic and medical experts; 

17 ‘‘(iii) patient representatives; 

18 ‘‘(iv) industry experts engaged in the 

19 development of drugs for rare diseases and 

20 conditions. 

21 ‘‘(4) PLANNING PROCESS.—In planning an EL– 

22 SFDD meeting under this section, the Foundation, 

23 in consultation with the stakeholders listed in para24 graph (5), shall develop— 


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1 ‘‘(A) a list of the specific objectives of the 

2 meeting related to key drug development issues 

3 for the rare disease or condition, or group of 

4 rare diseases and conditions, with a goal of ex5 pediting drug development; 

6 ‘‘(B) a proposed agenda for the meeting; 

7 and 

8 ‘‘(C) a list of medical experts, drug spon9 sors, scientific organizations, patient organiza10 tions, and other entities to be invited to partici11 pate in the meeting. 

12 ‘‘(5) AGENCY AND STAKEHOLDER ENGAGE13 MENT.—Throughout the process of planning an EL– 

14 SFDD meeting, the Foundation shall consult with— 

15 ‘‘(A) appropriate staff of the Food and 

16 Drug Administration; 

17 ‘‘(B) the Steering Committee established 

18 under this subsection; 

19 ‘‘(C) industry representatives engaged in 

20 the development of products for rare diseases 

21 and conditions to be discussed at such EL– 

22 SFDD meeting; 

23 ‘‘(D) patient representatives of rare dis24 eases and conditions under discussion in such 

25 EL–SFDD meeting; and 


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1 ‘‘(E) other appropriate stakeholders. 

2 ‘‘(6) POST-MEETING REPORTS.— 

3 ‘‘(A) IN GENERAL.—Within 180 days after 

4 an EL–SFDD meeting, the Foundation, in con5 sultation with the stakeholders listed in para6 graph (5), shall make publicly available on the 

7 website of the Food and Drug Administration— 

8 ‘‘(i) a transcript and recording of the 

9 meeting; and 

10 ‘‘(ii) a summary analysis of the input 

11 received during the meeting that is rel12 evant to approval or licensing of drugs for 

13 the rare disease or condition involved. 

14 ‘‘(B) CONTENTS.—Each publication under 

15 subparagraph (A) shall include a clear identi16 fication of— 

17 ‘‘(i) areas of consensus; 

18 ‘‘(ii) areas where additional clarifica19 tion or information is needed to reach con20 sensus; and 

21 ‘‘(iii) next steps agreed upon with the 

22 Food and Drug Administration. 

23 ‘‘(c) REPRESENTATIVES OF FDA REVIEW DIVI24 SIONS.—The Secretary shall require appropriate rep25 resentatives of the review divisions of the Food and Drug 


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1 Administration to participate in each EL–SFDD meeting 

2 under this section. 

3 ‘‘(d) RULES OF CONSTRUCTION.—Nothing in this 

4 section shall be construed— 

5 ‘‘(1) to prevent other third-party organizations 

6 from organizing similarly structured EL–SFDD-like 

7 meetings to discuss challenges in rare disease drug 

8 development; 

9 ‘‘(2) to require the Food and Drug Administra10 tion to participate in additional meetings described 

11 in paragraph (1); 

12 ‘‘(3) to alter the protections offered by laws, 

13 regulations, or policies governing disclosure of con14 fidential commercial or trade secret information and 

15 any other information exempt from disclosure pursu16 ant to section 552(b) of title 5, United States Code; 

17 ‘‘(4) to limit the ability of the Secretary to con18 sult with individuals and organizations; 

19 ‘‘(5) to create a legal right for consultation on 

20 any matter or require the Secretary to meet with 

21 any particular expert or stakeholder; 

22 ‘‘(6) to alter agreed-upon goals and procedures 

23 identified in the letters described in section 1001(b) 

24 of the FDA User Fee Reauthorization Act of 2022; 

25 or 


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1 ‘‘(7) to increase the number of review cycles for 

2 drugs. 

3 ‘‘(e) DEFINITIONS.—In this section: 

4 ‘‘(1) The term ‘EL–SFDD meeting’ means an 

5 externally led, science-focused drug development 

6 meeting. 

7 ‘‘(2) The terms ‘rare diseases and conditions’ 

8 and ‘rare disease or condition’ refer to a rare disease 

9 or condition as that term is defined in section 526. 

10 ‘‘(3) The term ‘Steering Committee’ means the 

11 Science-Focused Drug Development Multistake12 holder Steering Committee established under sub13 section (b)(3). 

14 ‘‘(f) AUTHORIZATION OF APPROPRIATIONS.— 

15 ‘‘(1) IN GENERAL.—To carry out this section, 

16 there is authorized to be appropriated $1,000,000 

17 for each of fiscal years 2025 through 2029. 

18 ‘‘(2) RULE OF CONSTRUCTION.—Nothing in 

19 this section shall be construed to prohibit the Foun20 dation from soliciting or accepting funds pursuant to 

21 section 770(i) for the purposes of planning or oper22 ating an EL–SFDD meeting authorized by this sec23 tion. 


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1 ‘‘SEC. 770B. REQUIRED ACTIONS FOLLOWING EL–SFDD 

2 MEETINGS. 

3 ‘‘(a) INCORPORATION OF INPUT INTO RISK-BENEFIT

4 ASSESSMENTS.—In approving or licensing a drug under 

5 subsection (c) or (j) of section 505 of this Act or sub6 section (a) or (k) of section 351 of the Public Health Serv7 ice Act, the Secretary shall make public a brief state8 ment— 

9 ‘‘(1) stating whether any EL–SFDD meeting 

10 under section 770A was held that was relevant to 

11 such approval or licensure; and 

12 ‘‘(2) if so, including a description of how the 

13 Secretary incorporated input from such meeting in 

14 the risk-benefit assessment described in section 

15 505(d). 

16 ‘‘(b) ANNUAL REPORT.—On an annual basis, the 

17 Secretary shall submit a report to the Congress summa18 rizing— 

19 ‘‘(1) the number and topics of EL–SFDD 

20 meetings held during the reporting period; 

21 ‘‘(2) the extent of participation in such meet22 ings from the review divisions of the Food and Drug 

23 Administration; 

24 ‘‘(3) the impact of EL–SFDD meetings on the 

25 workload and resources of the Food and Drug Ad26 ministration; and 


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1 ‘‘(4) an assessment of how the input received 

2 during such meetings was used in— 

3 ‘‘(A) deliberations throughout the drug de4 velopment lifecycle; 

5 ‘‘(B) regulatory decisionmaking; and 

6 ‘‘(C) formulating recommendations for fu7 ture meetings. 

8 ‘‘(c) DEFINITION.—In this section, the term ‘EL– 

9 SFDD meeting’ has the meaning given to that term in 

10 section 770A. 

11 ‘‘(d) AUTHORIZATION OF APPROPRIATIONS.—To 

12 carry out this section, there is authorized to be appro13 priated $1,000,000 for each of fiscal years 2025 through 

14 2029.’’. 

Æ 


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