I 

116TH CONGRESS 

2D SESSION H. R. 8662 

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 

OCTOBER 23, 2020 

Mr. FORTENBERRY (for himself, Mr. QUIGLEY, Mr. KING of New York, Mr. 

DIAZ-BALART, Mr. KEVIN HERN of Oklahoma, Mr. KATKO, Mr. 

GIANFORTE, Mr. HAGEDORN, Mr. BACON, Mr. FLEISCHMANN, Mrs. RODGERS of Washington, Mr. WESTERMAN, Mr. SMITH of Nebraska, Mr. 

BIGGS, Mr. MCKINLEY, Mr. SCHWEIKERT, Mr. UPTON, Mr. RUTHERFORD, Mr. MULLIN, Mr. KING of Iowa, Mr. AMODEI, Mr. MARSHALL, 

Mr. STIVERS, Mr. TIMMONS, Ms. VELA ́ZQUEZ, Ms. NORTON, Mr. COSTA, 

Mr. RUSH, Mr. ROUDA, Ms. SEWELL of Alabama, Mrs. BEATTY, Mr. 

O’HALLERAN, Mr. CISNEROS, Mr. LOWENTHAL, Mr. DAVID SCOTT of 

Georgia, Mr. CALVERT, Mr. WELCH, Mr. PETERSON, Mrs. DEMINGS, Ms. 

SCHAKOWSKY, Mr. SCHIFF, and Ms. BARRAGA ́N) introduced the following 

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

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, 


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1 SECTION 1. SHORT TITLE. 

2 This Act may be cited as the ‘‘Accelerating Access 

3 to Critical Therapies for ALS Act’’. 

4 SEC. 2. GRANTS FOR RESEARCH ON AND EXPANDED AC5 CESS TO EXPERIMENTAL THERAPIES FOR 

6 ALS. 

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

8 Human Services shall award grants to eligible entities for 

9 purposes of supporting research on, and expanded access 

10 for individuals to, investigational drugs for the prevention, 

11 diagnosis, mitigation, treatment, or cure of amyotrophic 

12 lateral sclerosis pursuant to an expanded access request 

13 submitted, and allowed to proceed by the Secretary, under 

14 section 561 of the Federal Food, Drug, and Cosmetic Act 

15 (21 U.S.C. 360bbb). 

16 (b) APPLICATION.—An eligible entity seeking a grant 

17 under this section shall submit to the Secretary an appli18 cation at such time, in such manner, and containing such 

19 information as the Secretary shall specify. Such applica20 tion shall specify a research objective relating to expand21 ing access to investigational drugs (as described in sub22 section (a)) that would be supported by the award of such 

23 grant. 

24 (c) SELECTION.—Not later than 90 days after the 

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

26 section, the Secretary shall determine whether to award 


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1 the grant, taking into consideration whether awarding 

2 such grant will support a research objective relating to ex3 panding access to investigational drugs (as described in 

4 subsection (a)), consistent with the mission of the Na5 tional Institutes of Health. 

6 (d) USE OF FUNDS.—An eligible entity may use 

7 funds received through the grant— 

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

9 direct costs of such drug (as authorized under sec10 tion 312.8(d) of title 21, Code of Federal Regula11 tions (or successor regulations)), if such costs are 

12 justified as part of peer review of the grant; 

13 (2) for the entity’s direct costs incurred in pro14 viding such drug consistent with the research mis15 sion of the grant; or 

16 (3) for the direct and indirect costs of the enti17 ty in conducting research with respect to the drug 

18 involved. 

19 (e) DEFINITIONS.—In this section: 

20 (1) The term ‘‘eligible entity’’ means a partici21 pating clinical trial site or sites sponsored by a small 

22 business concern (as defined in section 3(a) of the 

23 Small Business Act (15 U.S.C. 632(a)) that is the 

24 sponsor of a drug that is the subject of an investiga25 tional new drug application under section 505(i) of 


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

2 U.S.C. 355(i)). 

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

4 means a phase 3 clinical trial conducted pursuant to 

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

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

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

8 U.S.C. 262(a)) to investigate a drug intended to pre9 vent, diagnose, mitigate, treat, or cure amyotrophic 

10 lateral sclerosis. 

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

12 means a nonprofit or public health care facility, or 

13 network of facilities, at which patients participating 

14 in a participating clinical trial receive an investiga15 tional drug through such trial. 

16 SEC. 3. HHS COLLABORATIVE FOR NEURODEGENERATIVE 

17 DISEASES. 

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

19 the date of the enactment of this Act, the Secretary of 

20 Health and Human Services shall establish and implement 

21 a Collaborative for Neurodegenerative Diseases between 

22 the National Institutes of Health and the Food and Drug 

23 Administration (to be known and referred to in this sec24 tion as the ‘‘Collaborative’’), which shall— 


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1 (1) enter into a cooperative agreement, con2 tract, or other instrument with a private entity or 

3 entities under which such private entity or entities 

4 will operate the Collaborative; 

5 (2) focus on advancing regulatory improvements 

6 and scientific research that will support and accel7 erate the development and approval of drugs for pa8 tients with amyotrophic lateral sclerosis; 

9 (3) foster the development of effective drugs 

10 that improve the lives of people that suffer from rare 

11 neurodegenerative diseases; and 

12 (4) share information with the Secretary to ad13 vance the purposes specified in paragraph (3), such 

14 as through carrying out the grant programs under 

15 sections 2 and 5 and developing the action plan 

16 under section 4. 

17 (b) GIFTS.— 

18 (1) IN GENERAL.—The Collaborative may so19 licit and accept gifts, grants, and other donations, 

20 establish accounts, and invest and expend funds in 

21 support of pre-competitive research and research as22 sociated with phase 3 and phase 4 clinical trials con23 ducted with respect to investigational drugs that are 

24 the subjects of expanded access applications under 


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1 section 561 of the Federal Food, Drug, and Cos2 metic Act (21 U.S.C. 360bbb). 

3 (2) USE.—In addition to any amounts appro4 priated for purposes of carrying out this section, the 

5 Collaborative may use, without further appropria6 tion, any funds derived from a gift, grant, or other 

7 donation accepted pursuant to paragraph (1). 

8 (c) ADVISORY PANEL.— 

9 (1) IN GENERAL.—The Collaborative shall con10 vene an advisory panel to conduct a review of the de11 sign and implementation of the programs authorized 

12 by this Act. Such panel shall include representatives 

13 of patients, treating physicians, national organiza14 tions that facilitate provision of care services, re15 searchers, drug sponsors, drug manufacturers, and 

16 Federal agencies. 

17 (2) REPORT.—The advisory panel convened 

18 under paragraph (1) shall, not later than 18 months 

19 after the date of the enactment of this Act, submit 

20 to the Committee on Energy and Commerce of the 

21 House of Representatives and the Committee on 

22 Health, Education, Labor, and Pensions of the Sen23 ate a report that contains— 

24 (A) the findings and conclusions of the re25 view conducted under paragraph (1); and 


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1 (B) recommendations for carrying out the 

2 programs under this Act during the 2-year pe3 riod following the submission of such report, in4 cluding recommendations relating to the inclu5 sion of additional neurodegenerative diseases or 

6 disease areas within the grant programs under 

7 sections 2 and 5. 

8 (d) DUTIES AND AUTHORITIES.—The Collaborative 

9 shall identify and implement strategies for the Secretary— 

10 (1) for purposes of expediting the approval of 

11 drugs to treat amyotrophic lateral sclerosis, includ12 ing through coordination among the centers of the 

13 Food and Drug Administration to achieve the goals 

14 specified in the draft guidance for drug sponsors en15 titled ‘‘Amyotrophic Lateral Sclerosis: Developing 

16 Drugs for Treatment Guidance for Industry’’ pub17 lished in September 2019; 

18 (2) to facilitate access to investigational drugs 

19 for amyotrophic lateral sclerosis pursuant to an ex20 panded access request under section 561 of the Fed21 eral Food, Drug, and Cosmetic Act (21 U.S.C. 

22 360bbb) in a similar manner as investigational drugs 

23 for cancer are provided through Project Facilitate of 

24 the Center of Excellence for Oncology; 


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1 (3) with respect to the rare neurodegenerative 

2 disease grant program established under section 5; 

3 (4) to define or develop the regulatory and 

4 translational pathway for emerging therapeutic cat5 egories; 

6 (5) to share, within the Collaborative, findings 

7 and insights related to pre-competitive research and 

8 research associated with phase 3 and phase 4 clin9 ical trials conducted with respect to investigational 

10 drugs that are the subjects of expanded access appli11 cations under section 561 of the Federal Food, 

12 Drug, and Cosmetic Act (21 U.S.C. 360bbb); and 

13 (6) to develop and implement an ongoing mech14 anism to share feedback and information and de15 velop strategies with the neurodegenerative disease 

16 community, including patients, treating physicians, 

17 national organizations that facilitate provision of 

18 care services, access, and research, researchers, drug 

19 sponsors, drug manufacturers, and Federal agencies. 

20 SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN. 

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

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

23 and Human Services shall publish an action plan describ24 ing actions the Department of Health and Human Serv25 ices, through the National Institutes of Health and Food 


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1 and Drug Administration, intend to take during the 5-year 

2 period following publication of the plan with respect to 

3 program enhancements, policy development, regulatory 

4 science initiatives, and other appropriate initiatives to— 

5 (1) foster the development of safe and effective 

6 drugs that improve the lives of people living with 

7 rare neurodegenerative diseases as quickly as pos8 sible; and 

9 (2) facilitate access to investigational drugs. 

10 (b) CONTENTS.—The action plan published under 

11 subsection (a) shall— 

12 (1) identify appropriate representation from 

13 within the Food and Drug Administration and Na14 tional Institutes of Health to be responsible for im15 plementation of such action plan; and 

16 (2) include elements to facilitate— 

17 (A) interactions and collaboration between 

18 the Food and Drug Administration, including 

19 the review centers thereof, and stakeholders in20 cluding patients, sponsors, and external bio21 medical community; 

22 (B) consideration of cross-cutting clinical 

23 and regulatory policy issues, including consist24 ency of regulatory advice and decisionmaking; 


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1 (C) identification of key regulatory science 

2 and policy issues critical to advancing develop3 ment of safe and effective drugs; and 

4 (D) engagement by staff of the relevant 

5 centers of the Food and Drug Administration 

6 and other relevant offices of the Food and Drug 

7 Administration and National Institutes of 

8 Health with the designated leadership of the 

9 Collaborative. 

10 SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PRO11 GRAM. 

12 The Secretary of Health and Human Services, acting 

13 through the Commissioner of Food and Drugs, shall carry 

14 out a program of awarding grants to, and contracts en15 tered into with, public and private entities to cover the 

16 costs of research on and development of interventions in17 tended to prevent, diagnose, mitigate, treat, or cure 

18 amyotrophic lateral sclerosis and other life-threatening or 

19 severely debilitating neurodegenerative diseases, including 

20 costs incurred with respect to the development and critical 

21 evaluation of tools, methods, and processes— 

22 (1) to characterize such neurodegenerative dis23 eases and their natural history; 

24 (2) to identify molecular targets for such 

25 neurodegenerative diseases; and 


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1 (3) to increase efficiency and productivity of 

2 clinical development of therapies, including advanc3 ing rational therapeutic development and working to 

4 establish clinical trial networks. 

5 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 

6 For purposes of carrying out this Act, there are au7 thorized to be appropriated $100,000,000 for each of fis8 cal years 2022 through 2026. 

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