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 para-
graph (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 applica-
tion 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
H. R. 3537—2 
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, treat-
ment, 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, mitiga-
tion, 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 regula-
tions), 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 con-
ducting 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 sec-
tion 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 inves-
tigational drug through such trial.
H. R. 3537—3 
(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 estab-
lish 
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 ‘‘Partner-
ship’’) through cooperative agreements, contracts, or other appro-
priate mechanisms with such eligible entities, for the purpose of 
advancing the understanding of neurodegenerative diseases and 
fostering the development of treatments for amytrophic lateral scle-
rosis 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 institu-
tions; 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 tech-
nical expertise in the field of biomedical sciences and dem-
onstrated 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 exper-
tise 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 develop-
ment and establishment of clinical trial networks; and