II 

117TH CONGRESS 

1ST SESSION H. R. 3537 

IN THE SENATE OF THE UNITED STATES 

DECEMBER 9, 2021 

Received 

AN ACT 

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 THERAPIES FOR ALS. 

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

6 Human Services (referred to in this section as the ‘‘Sec7 retary’’) shall award grants to participating entities for 

8 purposes of scientific research utilizing data from ex9 panded access to investigational drugs for individuals who 

10 are not otherwise eligible for clinical trials for the preven11 tion, diagnosis, mitigation, treatment, or cure of 

12 amyotrophic lateral sclerosis. In the case of a participating 

13 entity seeking such a grant, an expanded access request 

14 must be submitted, and allowed to proceed by the Sec15 retary, under section 561 of the Federal Food, Drug, and 

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

17 21, Code of Federal Regulations (or any successor regula18 tions), before the application for such grant is submitted. 

19 (b) APPLICATION.— 

20 (1) IN GENERAL.—A participating entity seek21 ing a grant under this section shall submit to the 

22 Secretary an application at such time, in such man23 ner, and containing such information as the Sec24 retary shall specify. 

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

26 under paragraph (1) shall include a description of 


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1 how data generated through an expanded access re2 quest under section 561 of the Federal Food, Drug, 

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

4 to the investigational drug involved will be used to 

5 support research or development related to the pre6 vention, diagnosis, mitigation, treatment, or cure of 

7 amyotrophic lateral sclerosis. 

8 (3) NONINTERFERENCE WITH CLINICAL

9 TRIALS.—An application submitted under paragraph 

10 (1) shall include a description of how the proposed 

11 expanded access program will be designed so as not 

12 to interfere with patient enrollment in ongoing clin13 ical trials for investigational therapies for the pre14 vention, diagnosis, mitigation, treatment, or cure of 

15 amyotrophic lateral sclerosis. 

16 (c) SELECTION.—Consistent with sections 406 and 

17 492 of the Public Health Service Act (42 U.S.C. 284a, 

18 289a), the Secretary shall, in determining whether to 

19 award a grant under this section, confirm that— 

20 (1) such grant will be used to support a sci21 entific research objective relating to the prevention, 

22 diagnosis, mitigation, treatment, or cure of 

23 amyotrophic lateral sclerosis (as described in sub24 section (a)); 


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1 (2) such grant shall not have the effect of di2 minishing eligibility for, or impeding enrollment of, 

3 ongoing clinical trials for the prevention, diagnosis, 

4 mitigation, treatment, or cure of amyotrophic lateral 

5 sclerosis by determining that individuals who receive 

6 expanded access to investigational drugs through 

7 such a grant are not eligible for enrollment in— 

8 (A) ongoing clinical trials that are reg9 istered on ClinicalTrials.gov (or successor 

10 website), with respect to a drug for the preven11 tion, diagnosis, mitigation, treatment, or cure of 

12 amyotrophic lateral sclerosis; or 

13 (B) clinical trials for the prevention, diag14 nosis, mitigation, treatment, or cure of 

15 amyotrophic lateral sclerosis for which an ex16 emption under section 505(i) of the Federal 

17 Food, Drug, and Cosmetic Act (21 U.S.C. 

18 355(i)) has been granted by the Food and Drug 

19 Administration and which are expected to begin 

20 enrollment within one year; and 

21 (3) the resulting project funded by such grant 

22 will allow for equitable access to investigational 

23 drugs by minority and underserved populations. 

24 (d) USE OF FUNDS.—A participating entity shall use 

25 funds received through the grant— 


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

2 direct costs of the investigational drug, as author3 ized under section 312.8(d) of title 21, Code of Fed4 eral Regulations (or successor regulations), to pre5 vent, diagnose, mitigate, treat, or cure amyotrophic 

6 lateral sclerosis that is the subject of an expanded 

7 access request described in subsection (a), if such 

8 costs are justified as part of peer review of the 

9 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 such drug. 

15 (e) DEFINITIONS.—In this section: 

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

17 participating clinical trial site or sites sponsored by 

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

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

20 is the sponsor of a drug that is the subject of an in21 vestigational new drug application under section 

22 505(i) of the Federal Food, Drug, and Cosmetic Act 

23 (21 U.S.C. 355(i)) to prevent, diagnose, mitigate, 

24 treat, or cure amyotrophic lateral sclerosis. 


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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 health care facility, or network of facilities, 

11 at which patients participating in a participating 

12 clinical trial receive an investigational drug through 

13 such trial. 

14 (f) SUNSET.—The Secretary may not award grants 

15 under this section on or after September 30, 2026. 

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

17 NEURODEGENERATIVE DISEASES. 

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

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

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

21 ‘‘Secretary’’) shall establish and implement a Public-Pri22 vate Partnership for Neurodegenerative Diseases between 

23 the National Institutes of Health, the Food and Drug Ad24 ministration, and one or more eligible entities (to be 

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1 ship’’) through cooperative agreements, contracts, or other 

2 appropriate mechanisms with such eligible entities, for the 

3 purpose of advancing the understanding of 

4 neurodegenerative diseases and fostering the development 

5 of treatments for amytrophic lateral sclerosis and other 

6 rare neurodegenerative diseases. The Partnership shall— 

7 (1) establish partnerships and consortia with 

8 other public and private entities and individuals with 

9 expertise in amyotrophic lateral sclerosis and other 

10 rare neurodegenerative diseases for the purposes de11 scribed in this subsection; 

12 (2) focus on advancing regulatory science and 

13 scientific research that will support and accelerate 

14 the development and review of drugs for patients 

15 with amyotrophic lateral sclerosis and other rare 

16 neurodegenerative diseases; and 

17 (3) foster the development of effective drugs 

18 that improve the lives of people that suffer from 

19 amyotrophic lateral sclerosis and other rare 

20 neurodegenerative diseases. 

21 (b) ELIGIBLE ENTITY.—In this section, the term ‘‘el22 igible entity’’ means an entity that— 

23 (1) is— 

24 (A) an institution of higher education (as 

25 such term is defined in section 1001 of the 


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

2 1001)) or a consortium of such institutions; or 

3 (B) an organization described in section 

4 501(c)(3) of the Internal Revenue Code of 1986 

5 and exempt from tax under subsection (a) of 

6 such section; 

7 (2) has experienced personnel with clinical and 

8 other technical expertise in the field of biomedical 

9 sciences and demonstrated connection to the patient 

10 population; 

11 (3) demonstrates to the Secretary’s satisfaction 

12 that the entity is capable of identifying and estab13 lishing collaborations between public and private en14 tities and individuals with expertise in 

15 neurodegenerative diseases, including patients, in 

16 order to facilitate— 

17 (A) development and critical evaluation of 

18 tools, methods, and processes— 

19 (i) to characterize neurodegenerative 

20 diseases and their natural history; 

21 (ii) to identify molecular targets for 

22 neurodegenerative diseases; and 

23 (iii) to increase efficiency, predict24 ability, and productivity of clinical develop25 ment of therapies, including advancement 


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1 of rational therapeutic development and es2 tablishment of clinical trial networks; and 

3 (B) securing funding for the Partnership 

4 from Federal and non-Federal governmental 

5 sources, foundations, and private individuals; 

6 and 

7 (4) provides an assurance that the entity will 

8 not accept funding for a Partnership project from 

9 any organization that manufactures or distributes 

10 products regulated by the Food and Drug Adminis11 tration unless the entity provides assurances in its 

12 agreement with the Secretary that the results of the 

13 project will not be influenced by any source of fund14 ing. 

15 (c) GIFTS.— 

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

17 and accept gifts, grants, and other donations, estab18 lish accounts, and invest and expend funds in sup19 port of basic research and research associated with 

20 phase 3 clinical trials conducted with respect to in21 vestigational drugs that are the subjects of expanded 

22 access requests under section 561 of the Federal 

23 Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb). 

24 (2) USE.—In addition to any amounts appro25 priated for purposes of carrying out this section, the 


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1 Partnership may use, without further appropriation, 

2 any funds derived from a gift, grant, or other dona3 tion accepted pursuant to paragraph (1). 

4 SEC. 4. ALS AND OTHER RARE NEURODEGENERATIVE DIS5 EASE ACTION PLAN. 

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

7 date of enactment of this Act, the Commissioner of Food 

8 and Drugs shall publish on the website of the Food and 

9 Drug Administration an action plan describing actions the 

10 Food and Drug Administration intends to take during the 

11 5-year period following publication of the plan with respect 

12 to program enhancements, policy development, regulatory 

13 science initiatives, and other appropriate initiatives to— 

14 (1) foster the development of safe and effective 

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

16 people living with amyotrophic lateral sclerosis and 

17 other rare neurodegenerative diseases; and 

18 (2) facilitate access to investigational drugs for 

19 amyotrophic lateral sclerosis and other rare 

20 neurodegenerative diseases. 

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

22 under subsection (a) shall— 

23 (1) identify appropriate representation from 

24 within the Food and Drug Administration to be re25 sponsible for implementation of such action plan; 


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1 (2) include elements to facilitate— 

2 (A) interactions and collaboration between 

3 the Food and Drug Administration, including 

4 the review centers thereof, and stakeholders in5 cluding patients, sponsors, and the external bio6 medical research community; 

7 (B) consideration of cross-cutting clinical 

8 and regulatory policy issues, including consist9 ency of regulatory advice and decisionmaking; 

10 (C) identification of key regulatory science 

11 and policy issues critical to advancing develop12 ment of safe and effective drugs; and 

13 (D) enhancement of collaboration and en14 gagement of the relevant centers and offices of 

15 the Food and Drug Administration with other 

16 operating divisions within the Department of 

17 Health and Human Services, the Partnership, 

18 and the broader neurodegenerative disease com19 munity; 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, acting 

4 through the Commissioner of Food and Drugs, shall 

5 award grants and contracts to public and private entities 

6 to cover the costs of research on, and development of inter7 ventions intended to prevent, diagnose, mitigate, treat, or 

8 cure, amyotrophic lateral sclerosis and other rare 

9 neurodegenerative diseases in adults and children, includ10 ing costs incurred with respect to the development and 

11 critical evaluation of tools, methods, and processes— 

12 (1) to characterize such neurodegenerative dis13 eases and their natural history; 

14 (2) to identify molecular targets for such 

15 neurodegenerative diseases; and 

16 (3) to increase efficiency and productivity of 

17 clinical development of therapies, including 

18 through— 

19 (A) the use of master protocols and adapt20 ive and add-on clinical trial designs; and 

21 (B) efforts to establish new or leverage ex22 isting clinical trial networks. 

23 SEC. 6. GAO REPORT. 

24 Not later than 4 years after the date of the enact25 ment of this Act, the Comptroller General of the United 

26 States shall submit to the Committee on Energy and ComVerDate Sep 11 2014 02:45 Dec 17, 2021 Jkt 029200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6201 E:\BILLS\H3537.RDS H3537

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1 merce of the House of Representatives and the Committee 

2 on Health, Education, Labor, and Pensions of the Senate 

3 a report containing— 

4 (1) with respect to grants awarded under the 

5 program established under section 2— 

6 (A) an analysis of what is known about the 

7 impact of such grants on research or develop8 ment related to the prevention, diagnosis, miti9 gation, treatment, or cure of amyotrophic lat10 eral sclerosis; and 

11 (B) data concerning such grants, includ12 ing— 

13 (i) the number of grants awarded; 

14 (ii) the participating entities to whom 

15 grants were awarded; 

16 (iii) the value of each such grant; 

17 (iv) a description of the research each 

18 such grant was used to further; 

19 (v) the number of patients who re20 ceived expanded access to an investiga21 tional drug to prevent, diagnose, mitigate, 

22 treat, or cure amyotrophic lateral sclerosis 

23 under each grant; 

24 (vi) whether the investigational drug 

25 that was the subject of such a grant was 


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1 approved by the Food and Drug Adminis2 tration; and 

3 (vii) the average number of days be4 tween when a grant application is sub5 mitted and when a grant is awarded; and 

6 (2) with respect to grants awarded under the 

7 program established under section 5— 

8 (A) an analysis of what is known about the 

9 impact of such grants on research or develop10 ment related to the prevention, diagnosis, miti11 gation, treatment, or cure of amyotrophic lat12 eral sclerosis; 

13 (B) an analysis of what is known about 

14 how such grants increased efficiency and pro15 ductivity of the clinical development of thera16 pies, including through the use of clinical trials 

17 that operated with common master protocols, or 

18 had adaptive or add-on clinical trial designs; 

19 and 

20 (C) data concerning such grants, includ21 ing— 

22 (i) the number of grants awarded; 

23 (ii) the participating entities to whom 

24 grants were awarded; 

25 (iii) the value of each such grant; 


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1 (iv) a description of the research each 

2 such grant was used to further; and 

3 (v) whether the investigational drug 

4 that was the subject of such a grant re5 ceived approval by the Food and Drug Ad6 ministration. 

7 SEC. 7. AUTHORIZATION OF APPROPRIATIONS. 

8 For purposes of carrying out this Act, there are au9 thorized to be appropriated $100,000,000 for each of fis10 cal years 2022 through 2026. 

Passed the House of Representatives December 8, 

2021. 

Attest: CHERYL L. JOHNSON, 

Clerk. 


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