I 

117TH CONGRESS 

1ST SESSION H. R. 3537 

To direct the Secretary of Health and Human Services to support research 

on, and expanded access to, investigational drugs for amyotrophic lateral 

sclerosis, and for other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

MAY 25, 2021 

Mr. QUIGLEY (for himself, Mr. FORTENBERRY, Mr. BRENDAN F. BOYLE of 

Pennsylvania, Mr. MOULTON, Mr. GARCIA of California, Mr. CARBAJAL, 

Mr. LARSON of Connecticut, Ms. JACKSON LEE, Ms. DEAN, Mr. SUOZZI, 

Ms. VELA ́ZQUEZ, Mr. LEVIN of California, Mr. DEUTCH, Ms. NORTON, 

Mr. TIMMONS, Mr. BRADY, Mr. MCKINLEY, Mr. VAN DREW, Mr. CALVERT, Mr. KEATING, Mr. DIAZ-BALART, Mr. CARTER of Georgia, Mrs. 

MCBATH, Mr. SMITH of Missouri, Mr. TURNER, Mr. DUNCAN, Mr. HICE

of Georgia, Mr. YOUNG, Mr. SMITH of Nebraska, Mr. GROTHMAN, Mr. 

RUPPERSBERGER, Mr. RUTHERFORD, Mr. SCHWEIKERT, Mr. RYAN, Mr. 

CROW, Mr. GUTHRIE, Mr. FITZPATRICK, Ms. MCCOLLUM, Mr. AUSTIN

SCOTT of Georgia, Mr. BAIRD, Mr. RODNEY DAVIS of Illinois, Mr. 

VALADAO, Mr. MOOLENAAR, Mr. MALINOWSKI, Ms. ROYBAL-ALLARD, 

Mr. PAYNE, Mr. LYNCH, Ms. HERRERA BEUTLER, Mr. BUCK, Mr. 

MULLIN, Mr. GRIJALVA, Mr. COOPER, Mr. PANETTA, Mr. KIM of New 

Jersey, Mr. SIRES, Ms. LEE of California, Ms. MOORE of Wisconsin, Ms. 

SCHAKOWSKY, Mr. THOMPSON of California, Mr. GALLEGO, Mrs. AXNE, 

Mrs. NAPOLITANO, Mr. ESPAILLAT, Ms. PRESSLEY, Mr. FLEISCHMANN, 

Mr. RESCHENTHALER, Mr. CICILLINE, Ms. DEGETTE, Mr. BURCHETT, 

Mr. LAMALFA, Ms. MENG, Ms. BROWNLEY, Mr. TRONE, Ms. KUSTER, 

Mr. CONNOLLY, Mr. MEEKS, Mrs. KIRKPATRICK, Mrs. DEMINGS, Mr. 

O’HALLERAN, Mr. LIEU, Mr. DESAULNIER, Mr. GARAMENDI, Mr. KILMER, Mr. RUSH, Mr. MCCAUL, Mr. MCCLINTOCK, Mr. MFUME, Mr. 

LAMB, Mr. GREEN of Texas, Mr. SWALWELL, Mr. GOTTHEIMER, Ms. 

PINGREE, Ms. KAPTUR, Mr. FERGUSON, Ms. SCANLON, Mr. BACON, Mr. 

WITTMAN, Mr. MORELLE, Mr. AMODEI, and Mr. WALTZ) introduced the 

following bill; which was referred to the Committee on Energy and Commerce 


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A BILL 

To direct the Secretary of Health and Human Services to 

support research on, and expanded access to, investigational drugs for amyotrophic lateral sclerosis, 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 ‘‘Accelerating Access 

5 to Critical Therapies for ALS Act’’. 

6 SEC. 2. GRANTS FOR RESEARCH ON THERAPIES FOR ALS. 

7 (a) IN GENERAL.—The Secretary of Health and 

8 Human Services (referred to in this section as the Sec9 retary) shall award grants to participating entities for pur10 poses of expanded access for individuals to investigational 

11 drugs for the prevention, diagnosis, mitigation, treatment, 

12 or cure of amyotrophic lateral sclerosis. In the case of an 

13 applicant seeking such a grant, an expanded access re14 quest must be submitted, and allowed to proceed by the 

15 Secretary, under section 561 of the Federal Food, Drug, 

16 and Cosmetic Act (21 U.S.C. 360bbb) and part 312 of 

17 title 21, Code of Federal Regulations (or any successor 

18 regulations), before the application for such grant is sub19 mitted. 

20 (b) APPLICATION.— 


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1 (1) IN GENERAL.—A participating entity seek2 ing a grant under this section shall submit to the 

3 Secretary an application at such time, in such man4 ner, and containing such information as the Sec5 retary shall specify. 

6 (2) USE OF DATA.—An application submitted 

7 under paragraph (1) shall include a description of 

8 how data generated through an expanded access re9 quest under section 561 of the Federal Food, Drug, 

10 and Cosmetic Act (21 U.S.C. 360bbb) with respect 

11 to the investigational drug involved may be used by 

12 the Secretary to support research or development re13 lated to the prevention, diagnosis, mitigation, treat14 ment, or cure of amyotrophic lateral sclerosis or 

15 other rare neurodegenerative diseases. 

16 (c) SELECTION.—Not later than 120 days after the 

17 date of submission of an application for a grant under this 

18 section, the Secretary shall determine whether to award 

19 the grant, taking into consideration— 

20 (1) whether awarding such grant will support a 

21 research objective relating to expanding access to in22 vestigational drugs (as described in subsection (a)); 

23 and 


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1 (2) whether awarding such a grant may have 

2 the effect of diminishing eligibility for, or impeding 

3 enrollment of, ongoing clinical investigations. 

4 (d) USE OF FUNDS.—A participating entity may use 

5 funds received through the grant— 

6 (1) to pay the manufacturer or sponsor for the 

7 direct costs of such drug (as authorized under sec8 tion 312.8(d) of title 21, Code of Federal Regula9 tions (or successor regulations)), if such costs are 

10 justified as part of peer review of the grant; 

11 (2) for the entity’s direct costs incurred in pro12 viding such drug consistent with the research mis13 sion of the grant; or 

14 (3) for the direct and indirect costs of the enti15 ty in conducting research with respect to the drug 

16 involved. 

17 (e) DEFINITIONS.—In this section: 

18 (1) The term ‘‘participating entity’’ means a 

19 participating clinical trial site or sites sponsored by 

20 a small business concern (as defined in section 3(a) 

21 of the Small Business Act (15 U.S.C. 632(a)) that 

22 is the sponsor of a drug that is the subject of an in23 vestigational new drug application under section 

24 505(i) of the Federal Food, Drug, and Cosmetic Act 

25 (21 U.S.C. 355(i)). 


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1 (2) The term ‘‘participating clinical trial’’ 

2 means a phase 3 clinical trial conducted pursuant to 

3 an exemption under section 505(i) of the Federal 

4 Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or 

5 section 351(a) of the Public Health Service Act (42 

6 U.S.C. 262(a)) to investigate a drug intended to pre7 vent, diagnose, mitigate, treat, or cure amyotrophic 

8 lateral sclerosis. 

9 (3) The term ‘‘participating clinical trial site’’ 

10 means a nonprofit or public health care facility, or 

11 network of facilities, at which patients participating 

12 in a participating clinical trial receive an investiga13 tional drug through such trial. 

14 SEC. 3. HHS PUBLIC-PRIVATE PARTNERSHIP FOR RARE 

15 NEURODEGENERATIVE DISEASES. 

16 (a) ESTABLISHMENT.—Not later than one year after 

17 the date of enactment of this Act, the Secretary of Health 

18 and Human Services (referred to in this section as the 

19 ‘‘Secretary’’) shall establish and implement a Public-Pri20 vate Partnership for Neurodegenerative Diseases between 

21 the National Institutes of Health, the Food and Drug Ad22 ministration, and one or more eligible entities (to be 

23 known and referred to in this section as the ‘‘Partner24 ship’’) through cooperative agreements, contracts, or other 

25 appropriate instruments with such eligible entities, for the 


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1 purpose of developing treatments for amytrophic lateral 

2 sclerosis and other rare neurodegenerative diseases. The 

3 Partnership shall— 

4 (1) establish partnerships, consortia, and col5 laborations with other public and private entities 

6 and individuals with expertise in amyotrophic lateral 

7 sclerosis and other rare neurodegenerative diseases 

8 for the purposes described in this subsection; 

9 (2) focus on advancing regulatory science and 

10 scientific research that will support and accelerate 

11 the development and review of drugs for patients 

12 with amyotrophic lateral sclerosis and other rare 

13 neurodegenerative diseases; and 

14 (3) foster the development of effective drugs 

15 that improve the lives of people that suffer from 

16 amyotrophic lateral sclerosis and other rare 

17 neurodegenerative diseases. 

18 (b) ELIGIBLE ENTITY.—In this section, the term ‘‘el19 igible entity’’ means an entity that— 

20 (1) is— 

21 (A) an institution of higher education (as 

22 such term is defined in section 1001 of the 

23 Higher Education Act of 1965 (20 U.S.C. 

24 1001)) or a consortium of such institutions; or 


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1 (B) an organization described in section 

2 501(c)(3) of the Internal Revenue Code of 1986 

3 and exempt from tax under subsection (a) of 

4 such section; 

5 (2) has experienced personnel and demonstrated 

6 connection to the patient population; 

7 (3) demonstrates to the Secretary’s satisfaction 

8 that the entity is capable of identifying and estab9 lishing collaborations between public and private en10 tities and individuals with expertise in 

11 neurodegenerative diseases, including patients, in 

12 order to facilitate— 

13 (A) development and critical evaluation of 

14 tools, methods, and processes— 

15 (i) to characterize neurodegenerative 

16 diseases and their natural history; 

17 (ii) to identify drug targets for 

18 neurodegenerative diseases; and 

19 (iii) to increase efficiency, predict20 ability, and productivity of clinical develop21 ment of therapies, including advancement 

22 of rational therapeutic development and es23 tablishment of clinical trial networks; and 

24 (B) securing funding for the Partnership 

25 from Federal and non-Federal governmental 


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1 sources, foundations, and private individuals; 

2 and 

3 (4) provides an assurance that the entity will 

4 not accept funding for a Partnership project from 

5 any organization that manufactures or distributes 

6 products regulated by the Food and Drug Adminis7 tration unless the entity provides assurances in its 

8 agreement with the Secretary that the results of the 

9 project will not be influenced by any source of fund10 ing. 

11 (c) GIFTS.— 

12 (1) IN GENERAL.—The Partnership may solicit 

13 and accept gifts, grants, and other donations, estab14 lish accounts, and invest and expend funds in sup15 port of pre-competitive research and research associ16 ated with phase 3 clinical trials conducted with re17 spect to investigational drugs that are the subjects 

18 of expanded access applications under section 561 of 

19 the Federal Food, Drug, and Cosmetic Act (21 

20 U.S.C. 360bbb). 

21 (2) USE.—In addition to any amounts appro22 priated for purposes of carrying out this section, the 

23 Partnership may use, without further appropriation, 

24 any funds derived from a gift, grant, or other dona25 tion accepted pursuant to paragraph (1). 


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1 SEC. 4. ALS AND OTHER RARE NEURODEGENERATIVE DIS2 EASE ACTION PLAN. 

3 (a) IN GENERAL.—Not later than 6 months after the 

4 date of enactment of this Act, the Secretary of Health and 

5 Human Services shall publish on the website of the De6 partment of Health and Human Services an action plan 

7 describing actions the Food and Drug Administration in8 tends to take during the 5-year period following publica9 tion of the plan with respect to program enhancements, 

10 policy development, regulatory science initiatives, and 

11 other appropriate initiatives to— 

12 (1) foster the development of safe and effective 

13 drugs that improve or extend, or both, the lives of 

14 people living with amyotrophic lateral sclerosis and 

15 other rare neurodegenerative diseases as quickly as 

16 possible; and 

17 (2) facilitate access to investigational drugs for 

18 amyotrophic lateral sclerosis and other rare 

19 neurodegenerative diseases. 

20 (b) CONTENTS.—The initial action plan published 

21 under subsection (a) shall— 

22 (1) identify appropriate representation from 

23 within the Food and Drug Administration to be re24 sponsible for implementation of such action plan; 

25 (2) include elements to facilitate— 


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1 (A) interactions and collaboration between 

2 the Food and Drug Administration, including 

3 the review centers thereof, and stakeholders in4 cluding patients, sponsors, and the external bio5 medical research community; 

6 (B) consideration of cross-cutting clinical 

7 and regulatory policy issues, including consist8 ency of regulatory advice and decision making; 

9 (C) identification of key regulatory science 

10 and policy issues critical to advancing develop11 ment of safe and effective drugs; and 

12 (D) enhancement of collaboration and en13 gagement by staff of the relevant centers of the 

14 Food and Drug Administration and other rel15 evant offices of the Food and Drug Administra16 tion with other operating divisions within the 

17 Department of Health and Human Services, the 

18 Partnership, and the broader neurodegenerative 

19 disease community; and 

20 (3) be subject to revision, as determined appro21 priate by the Secretary of Health and Human Serv22 ices. 


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1 SEC. 5. FDA RARE NEURODEGENERATIVE DISEASE GRANT 

2 PROGRAM. 

3 The Secretary of Health and Human Services shall 

4 use funds made available under section 6 to award grants 

5 and contracts to public and private entities to cover the 

6 costs of research on, and development of interventions in7 tended to prevent, diagnose, mitigate, treat, or cure, 

8 amyotrophic lateral sclerosis and other rare life-threat9 ening or severely debilitating neurodegenerative diseases 

10 in adults and children, including costs incurred with re11 spect to the development and critical evaluation of tools, 

12 methods, and processes— 

13 (1) to characterize such neurodegenerative dis14 eases and their natural history; 

15 (2) to identify molecular targets for such 

16 neurodegenerative diseases; and 

17 (3) to increase efficiency and productivity of 

18 clinical development of therapies, including advanc19 ing rational therapeutic development and working to 

20 establish new or leverage existing clinical trial net21 works. 

22 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 

23 For purposes of carrying out this Act, there are au24 thorized to be appropriated $100,000,000 for each of fis25 cal years 2022 through 2026. 

Æ 


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