H. R. 3537 

One Hundred Seventeenth Congress 

of the 

United States of America 

AT THE FIRST SESSION 

Begun and held at the City of Washington on Monday, 

the fourth day of January, two thousand and twenty-one 

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. 

Be it enacted by the Senate and House of Representatives of 

the United States of America in Congress assembled, 

SECTION 1. SHORT TITLE. 

This Act may be cited as the ‘‘Accelerating Access to Critical 

Therapies for ALS Act’’. 

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

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

(referred to in this section as the ‘‘Secretary’’) shall award grants 

to participating entities for purposes of scientific research utilizing 

data from expanded access to investigational drugs for individuals 

who are not otherwise eligible for clinical trials for the prevention, 

diagnosis, mitigation, treatment, or cure of amyotrophic lateral 

sclerosis. In the case of a participating entity seeking such a grant, 

an expanded access request must be submitted, and allowed to 

proceed by the Secretary, under section 561 of the Federal Food, 

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

21, Code of Federal Regulations (or any successor regulations), 

before the application for such grant is submitted. 

(b) APPLICATION.— (1) IN GENERAL.—A participating entity seeking a grant 

under this section shall submit to the Secretary an application 

at such time, in such manner, and containing such information 

as the Secretary shall specify. 

(2) USE OF DATA.—An application submitted under paragraph (1) shall include a description of how data generated 

through an expanded access request under section 561 of the 

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

respect to the investigational drug involved will be used to 

support research or development related to the prevention, 

diagnosis, mitigation, treatment, or cure of amyotrophic lateral 

sclerosis. 

(3) NONINTERFERENCE WITH CLINICAL TRIALS.—An application submitted under paragraph (1) shall include a description 

of how the proposed expanded access program will be designed 

so as not to interfere with patient enrollment in ongoing clinical 

trials for investigational therapies for the prevention, diagnosis, 

mitigation, treatment, or cure of amyotrophic lateral sclerosis. 

(c) SELECTION.—Consistent with sections 406 and 492 of the 

Public Health Service Act (42 U.S.C. 284a, 289a), the Secretary 
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shall, in determining whether to award a grant under this section, 

confirm that— 

(1) such grant will be used to support a scientific research 

objective relating to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis (as described 

in subsection (a)); 

(2) such grant shall not have the effect of diminishing 

eligibility for, or impeding enrollment of, ongoing clinical trials 

for the prevention, diagnosis, mitigation, treatment, or cure 

of amyotrophic lateral sclerosis by determining that individuals 

who receive expanded access to investigational drugs through 

such a grant are not eligible for enrollment in— 

(A) ongoing clinical trials that are registered on 

ClinicalTrials.gov (or successor website), with respect to 

a drug for the prevention, diagnosis, mitigation, treatment, 

or cure of amyotrophic lateral sclerosis; or 

(B) clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis 

for which an exemption under section 505(i) of the Federal 

Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) has been 

granted by the Food and Drug Administration and which 

are expected to begin enrollment within one year; and 

(3) the resulting project funded by such grant will allow 

for equitable access to investigational drugs by minority and 

underserved populations. 

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

received through the grant— 

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

of the investigational drug, as authorized under section 312.8(d) 

of title 21, Code of Federal Regulations (or successor regulations), to prevent, diagnose, mitigate, treat, or cure amyotrophic 

lateral sclerosis that is the subject of an expanded access 

request described in subsection (a), if such costs are justified 

as part of peer review of the grant; 

(2) for the entity’s direct costs incurred in providing such 

drug consistent with the research mission of the grant; or 

(3) for the direct and indirect costs of the entity in conducting research with respect to such drug. 

(e) DEFINITIONS.—In this section: 

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

clinical trial site or sites sponsored by a small business concern 

(as defined in section 3(a) of the Small Business Act (15 U.S.C. 

632(a))) that is the sponsor of a drug that is the subject of 

an investigational new drug application under section 505(i) 

of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) 

to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral 

sclerosis. 

(2) The term ‘‘participating clinical trial’’ means a phase 

3 clinical trial conducted pursuant to an exemption under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 

U.S.C. 355(i)) or section 351(a) of the Public Health Service 

Act (42 U.S.C. 262(a)) to investigate a drug intended to prevent, 

diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis. 

(3) The term ‘‘participating clinical trial site’’ means a 

health care facility, or network of facilities, at which patients 

participating in a participating clinical trial receive an investigational drug through such trial. 
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(f) SUNSET.—The Secretary may not award grants under this 

section on or after September 30, 2026. 

SEC. 3. HHS PUBLIC-PRIVATE PARTNERSHIP FOR RARE 

NEURODEGENERATIVE DISEASES. 

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

of enactment of this Act, the Secretary of Health and Human 

Services (referred to in this section as the ‘‘Secretary’’) shall establish and implement a Public-Private Partnership for 

Neurodegenerative Diseases between the National Institutes of 

Health, the Food and Drug Administration, and one or more eligible 

entities (to be known and referred to in this section as the ‘‘Partnership’’) through cooperative agreements, contracts, or other appropriate mechanisms with such eligible entities, for the purpose of 

advancing the understanding of neurodegenerative diseases and 

fostering the development of treatments for amytrophic lateral sclerosis and other rare neurodegenerative diseases. The Partnership 

shall— 

(1) establish partnerships and consortia with other public 

and private entities and individuals with expertise in 

amyotrophic lateral sclerosis and other rare neurodegenerative 

diseases for the purposes described in this subsection; 

(2) focus on advancing regulatory science and scientific 

research that will support and accelerate the development and 

review of drugs for patients with amyotrophic lateral sclerosis 

and other rare neurodegenerative diseases; and 

(3) foster the development of effective drugs that improve 

the lives of people that suffer from amyotrophic lateral sclerosis 

and other rare neurodegenerative diseases. 

(b) ELIGIBLE ENTITY.—In this section, the term ‘‘eligible entity’’ 

means an entity that— 

(1) is— 

(A) an institution of higher education (as such term 

is defined in section 1001 of the Higher Education Act 

of 1965 (20 U.S.C. 1001)) or a consortium of such institutions; or 

(B) an organization described in section 501(c)(3) of 

the Internal Revenue Code of 1986 and exempt from tax 

under subsection (a) of such section; 

(2) has experienced personnel with clinical and other technical expertise in the field of biomedical sciences and demonstrated connection to the patient population; 

(3) demonstrates to the Secretary’s satisfaction that the 

entity is capable of identifying and establishing collaborations 

between public and private entities and individuals with expertise in neurodegenerative diseases, including patients, in order 

to facilitate— 

(A) development and critical evaluation of tools, 

methods, and processes— 

(i) to characterize neurodegenerative diseases and 

their natural history; 

(ii) to identify molecular targets for 

neurodegenerative diseases; and 

(iii) to increase efficiency, predictability, and 

productivity of clinical development of therapies, 

including advancement of rational therapeutic development and establishment of clinical trial networks; and 
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(B) securing funding for the Partnership from Federal 

and non-Federal governmental sources, foundations, and 

private individuals; and 

(4) provides an assurance that the entity will not accept 

funding for a Partnership project from any organization that 

manufactures or distributes products regulated by the Food 

and Drug Administration unless the entity provides assurances 

in its agreement with the Secretary that the results of the 

project will not be influenced by any source of funding. 

(c) GIFTS.— 

(1) IN GENERAL.—The Partnership may solicit and accept 

gifts, grants, and other donations, establish accounts, and invest 

and expend funds in support of basic research and research 

associated with phase 3 clinical trials conducted with respect 

to investigational drugs that are the subjects of expanded access 

requests under section 561 of the Federal Food, Drug, and 

Cosmetic Act (21 U.S.C. 360bbb). 

(2) USE.—In addition to any amounts appropriated for purposes of carrying out this section, the Partnership may use, 

without further appropriation, any funds derived from a gift, 

grant, or other donation accepted pursuant to paragraph (1). 

SEC. 4. ALS AND OTHER RARE NEURODEGENERATIVE DISEASE ACTION 

PLAN. 

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

enactment of this Act, the Commissioner of Food and Drugs shall 

publish on the website of the Food and Drug Administration an 

action plan describing actions the Food and Drug Administration 

intends to take during the 5-year period following publication of 

the plan with respect to program enhancements, policy development, 

regulatory science initiatives, and other appropriate initiatives to— 

(1) foster the development of safe and effective drugs that 

improve or extend, or both, the lives of people living with 

amyotrophic lateral sclerosis and other rare neurodegenerative 

diseases; and 

(2) facilitate access to investigational drugs for amyotrophic 

lateral sclerosis and other rare neurodegenerative diseases. 

(b) CONTENTS.—The initial action plan published under subsection (a) shall— 

(1) identify appropriate representation from within the 

Food and Drug Administration to be responsible for 

implementation of such action plan; 

(2) include elements to facilitate— 

(A) interactions and collaboration between the Food 

and Drug Administration, including the review centers 

thereof, and stakeholders including patients, sponsors, and 

the external biomedical research community; 

(B) consideration of cross-cutting clinical and regulatory policy issues, including consistency of regulatory 

advice and decisionmaking; 

(C) identification of key regulatory science and policy 

issues critical to advancing development of safe and effective drugs; and 

(D) enhancement of collaboration and engagement of 

the relevant centers and offices of the Food and Drug 

Administration with other operating divisions within the 
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Department of Health and Human Services, the Partnership, and the broader neurodegenerative disease community; and 

(3) be subject to revision, as determined appropriate by 

the Secretary of Health and Human Services. 

SEC. 5. FDA RARE NEURODEGENERATIVE DISEASE GRANT PROGRAM. 

The Secretary of Health and Human Services, acting through 

the Commissioner of Food and Drugs, shall award grants and 

contracts to public and private entities to cover the costs of research 

on, and development of interventions intended to prevent, diagnose, 

mitigate, treat, or cure, amyotrophic lateral sclerosis and other 

rare neurodegenerative diseases in adults and children, including 

costs incurred with respect to the development and critical evaluation of tools, methods, and processes— 

(1) to characterize such neurodegenerative diseases and 

their natural history; 

(2) to identify molecular targets for such neurodegenerative 

diseases; and 

(3) to increase efficiency and productivity of clinical 

development of therapies, including through— 

(A) the use of master protocols and adaptive and addon clinical trial designs; and 

(B) efforts to establish new or leverage existing clinical 

trial networks. 

SEC. 6. GAO REPORT. 

Not later than 4 years after the date of the enactment of 

this Act, the Comptroller General of the United States shall submit 

to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and 

Pensions of the Senate a report containing— 

(1) with respect to grants awarded under the program 

established under section 2— 

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

of such grants on research or development related to the 

prevention, diagnosis, mitigation, treatment, or cure of 

amyotrophic lateral sclerosis; and 

(B) data concerning such grants, including— 

(i) the number of grants awarded; 

(ii) the participating entities to whom grants were 

awarded; 

(iii) the value of each such grant; 

(iv) a description of the research each such grant 

was used to further; 

(v) the number of patients who received expanded 

access to an investigational drug to prevent, diagnose, 

mitigate, treat, or cure amyotrophic lateral sclerosis 

under each grant; 

(vi) whether the investigational drug that was the 

subject of such a grant was approved by the Food 

and Drug Administration; and 

(vii) the average number of days between when 

a grant application is submitted and when a grant 

is awarded; and 

(2) with respect to grants awarded under the program 

established under section 5— 
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(A) an analysis of what is known about the impact 

of such grants on research or development related to the 

prevention, diagnosis, mitigation, treatment, or cure of 

amyotrophic lateral sclerosis; 

(B) an analysis of what is known about how such 

grants increased efficiency and productivity of the clinical 

development of therapies, including through the use of 

clinical trials that operated with common master protocols, 

or had adaptive or add-on clinical trial designs; and 

(C) data concerning such grants, including— 

(i) the number of grants awarded; 

(ii) the participating entities to whom grants were 

awarded; 

(iii) the value of each such grant; 

(iv) a description of the research each such grant 

was used to further; and 

(v) whether the investigational drug that was the 

subject of such a grant received approval by the Food 

and Drug Administration. 

SEC. 7. AUTHORIZATION OF APPROPRIATIONS. 

For purposes of carrying out this Act, there are authorized 

to be appropriated $100,000,000 for each of fiscal years 2022 

through 2026. 

Speaker of the House of Representatives. 

Vice President of the United States and

President of the Senate.