II 

116TH CONGRESS 

2D SESSION S. 4867 

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 SENATE OF THE UNITED STATES 

OCTOBER 26 (legislative day, OCTOBER 19), 2020 

Mr. COONS (for himself and Ms. MURKOWSKI) introduced the following bill; 

which was read twice and referred to the Committee on Health, Education, Labor, and Pensions 

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’’. 


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1 SEC. 2. GRANTS FOR RESEARCH ON AND EXPANDED AC2 CESS TO EXPERIMENTAL THERAPIES FOR 

3 ALS. 

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

5 Human Services shall award grants to eligible entities for 

6 purposes of supporting research on, and expanded access 

7 for individuals to, investigational drugs for the prevention, 

8 diagnosis, mitigation, treatment, or cure of amyotrophic 

9 lateral sclerosis pursuant to an expanded access request 

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

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

12 (21 U.S.C. 360bbb). 

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

14 under this section shall submit to the Secretary an appli15 cation at such time, in such manner, and containing such 

16 information as the Secretary shall specify. Such applica17 tion shall specify a research objective relating to expand18 ing access to investigational drugs (as described in sub19 section (a)) that would be supported by the award of such 

20 grant. 

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

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

23 section, the Secretary shall determine whether to award 

24 the grant, taking into consideration whether awarding 

25 such grant will support a research objective relating to ex26 panding access to investigational drugs (as described in 


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1 subsection (a)), consistent with the mission of the Na2 tional Institutes of Health. 

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

4 funds received through the grant— 

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

6 direct costs of such drug (as authorized under sec7 tion 312.8(d) of title 21, Code of Federal Regula8 tions (or successor regulations)), if such costs are 

9 justified as part of peer review of the grant; 

10 (2) for the entity’s direct costs incurred in pro11 viding such drug consistent with the research mis12 sion of the grant; or 

13 (3) for the direct and indirect costs of the enti14 ty in conducting research with respect to the drug 

15 involved. 

16 (e) DEFINITIONS.—In this section: 

17 (1) The term ‘‘eligible entity’’ means a partici18 pating clinical trial site or sites sponsored by a small 

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

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

21 sponsor of a drug that is the subject of an investiga22 tional new drug application under section 505(i) of 

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

24 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 COLLABORATIVE FOR NEURODEGENERATIVE 

15 DISEASES. 

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

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

18 Health and Human Services shall establish and implement 

19 a Collaborative for Neurodegenerative Diseases between 

20 the National Institutes of Health and the Food and Drug 

21 Administration (to be known and referred to in this sec22 tion as the ‘‘Collaborative’’), which shall— 

23 (1) enter into a cooperative agreement, con24 tract, or other instrument with a private entity or 


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1 entities under which such private entity or entities 

2 will operate the Collaborative; 

3 (2) focus on advancing regulatory improvements 

4 and scientific research that will support and accel5 erate the development and approval of drugs for pa6 tients with amyotrophic lateral sclerosis; 

7 (3) foster the development of effective drugs 

8 that improve the lives of people that suffer from rare 

9 neurodegenerative diseases; and 

10 (4) share information with the Secretary to ad11 vance the purposes specified in paragraph (3), such 

12 as through carrying out the grant programs under 

13 sections 2 and 5 and developing the action plan 

14 under section 4. 

15 (b) GIFTS.— 

16 (1) IN GENERAL.—The Collaborative may so17 licit and accept gifts, grants, and other donations, 

18 establish accounts, and invest and expend funds in 

19 support of pre-competitive research and research as20 sociated with phase 3 and phase 4 clinical trials con21 ducted with respect to investigational drugs that are 

22 the subjects of expanded access applications under 

23 section 561 of the Federal Food, Drug, and Cos24 metic Act (21 U.S.C. 360bbb). 


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1 (2) USE.—In addition to any amounts appro2 priated for purposes of carrying out this section, the 

3 Collaborative may use, without further appropria4 tion, any funds derived from a gift, grant, or other 

5 donation accepted pursuant to paragraph (1). 

6 (c) ADVISORY PANEL.— 

7 (1) IN GENERAL.—The Collaborative shall con8 vene an advisory panel to conduct a review of the de9 sign and implementation of the programs authorized 

10 by this Act. Such panel shall include representatives 

11 of patients, treating physicians, national organiza12 tions that facilitate provision of care services, re13 searchers, drug sponsors, drug manufacturers, and 

14 Federal agencies. 

15 (2) REPORT.—The advisory panel convened 

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

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

18 to the Committee on Energy and Commerce of the 

19 House of Representatives and the Committee on 

20 Health, Education, Labor, and Pensions of the Sen21 ate a report that contains— 

22 (A) the findings and conclusions of the re23 view conducted under paragraph (1); and 

24 (B) recommendations for carrying out the 

25 programs under this Act during the 2-year peVerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867

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1 riod following the submission of such report, in2 cluding recommendations relating to the inclu3 sion of additional neurodegenerative diseases or 

4 disease areas within the grant programs under 

5 sections 2 and 5. 

6 (d) DUTIES AND AUTHORITIES.—The Collaborative 

7 shall identify and implement strategies for the Secretary— 

8 (1) for purposes of expediting the approval of 

9 drugs to treat amyotrophic lateral sclerosis, includ10 ing through coordination among the centers of the 

11 Food and Drug Administration to achieve the goals 

12 specified in the draft guidance for drug sponsors en13 titled ‘‘Amyotrophic Lateral Sclerosis: Developing 

14 Drugs for Treatment Guidance for Industry’’ pub15 lished in September 2019; 

16 (2) to facilitate access to investigational drugs 

17 for amyotrophic lateral sclerosis pursuant to an ex18 panded access request under section 561 of the Fed19 eral Food, Drug, and Cosmetic Act (21 U.S.C. 

20 360bbb) in a similar manner as investigational drugs 

21 for cancer are provided through Project Facilitate of 

22 the Center of Excellence for Oncology; 

23 (3) with respect to the rare neurodegenerative 

24 disease grant program established under section 5; 


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1 (4) to define or develop the regulatory and 

2 translational pathway for emerging therapeutic cat3 egories; 

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

5 and insights related to pre-competitive research and 

6 research associated with phase 3 and phase 4 clin7 ical trials conducted with respect to investigational 

8 drugs that are the subjects of expanded access appli9 cations under section 561 of the Federal Food, 

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

11 (6) to develop and implement an ongoing mech12 anism to share feedback and information and de13 velop strategies with the neurodegenerative disease 

14 community, including patients, treating physicians, 

15 national organizations that facilitate provision of 

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

17 sponsors, drug manufacturers, and Federal agencies. 

18 SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN. 

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

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

21 and Human Services shall publish an action plan describ22 ing actions the Department of Health and Human Serv23 ices, through the National Institutes of Health and Food 

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

25 period following publication of the plan with respect to 


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1 program enhancements, policy development, regulatory 

2 science initiatives, and other appropriate initiatives to— 

3 (1) foster the development of safe and effective 

4 drugs that improve the lives of people living with 

5 rare neurodegenerative diseases as quickly as pos6 sible; and 

7 (2) facilitate access to investigational drugs. 

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

9 subsection (a) shall— 

10 (1) identify appropriate representation from 

11 within the Food and Drug Administration and Na12 tional Institutes of Health to be responsible for im13 plementation of such action plan; and 

14 (2) include elements to facilitate— 

15 (A) interactions and collaboration between 

16 the Food and Drug Administration, including 

17 the review centers thereof, and stakeholders in18 cluding patients, sponsors, and external bio19 medical community; 

20 (B) consideration of cross-cutting clinical 

21 and regulatory policy issues, including consist22 ency of regulatory advice and decision making; 

23 (C) identification of key regulatory science 

24 and policy issues critical to advancing develop25 ment of safe and effective drugs; and 


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1 (D) engagement by staff of the relevant 

2 centers of the Food and Drug Administration 

3 and other relevant offices of the Food and Drug 

4 Administration and National Institutes of 

5 Health with the designated leadership of the 

6 Collaborative. 

7 SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PRO8 GRAM. 

9 The Secretary of Health and Human Services, acting 

10 through the Commissioner of Food and Drugs, shall carry 

11 out a program of awarding grants to, and contracts en12 tered into with, public and private entities to cover the 

13 costs of research on and development of interventions in14 tended to prevent, diagnose, mitigate, treat, or cure 

15 amyotrophic lateral sclerosis and other life-threatening or 

16 severely debilitating neurodegenerative diseases, including 

17 costs incurred with respect to the development and critical 

18 evaluation of tools, methods, and processes— 

19 (1) to characterize such neurodegenerative dis20 eases and their natural history; 

21 (2) to identify molecular targets for such 

22 neurodegenerative diseases; and 

23 (3) to increase efficiency and productivity of 

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1 ing rational therapeutic development and working to 

2 establish clinical trial networks. 

3 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 

4 For purposes of carrying out this Act, there are au5 thorized to be appropriated $100,000,000 for each of fis6 cal years 2022 through 2026. 

Æ 


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