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. ROD-
GERS of Washington, Mr. WESTERMAN, Mr. SMITH of Nebraska, Mr. 
BIGGS, Mr. MCKINLEY, Mr. SCHWEIKERT, Mr. UPTON, Mr. RUTHER-
FORD, 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, investiga-
tional drugs for amyotrophic lateral sclerosis, and for 
other purposes. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
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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 AND EXPANDED AC-
CESS TO EXPERIMENTAL THERAPIES FOR 
ALS. 
(a) IN GENERAL.—The Secretary of Health and 
Human Services shall award grants to eligible entities for 
purposes of supporting research on, and expanded access 
for individuals to, investigational drugs for the prevention, 
diagnosis, mitigation, treatment, or cure of amyotrophic 
lateral sclerosis pursuant to an expanded access request 
submitted, and allowed to proceed by the Secretary, under 
section 561 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 360bbb). 
(b) APPLICATION.—An eligible entity seeking a grant 
under this section shall submit to the Secretary an appli-
cation at such time, in such manner, and containing such 
information as the Secretary shall specify. Such applica-
tion shall specify a research objective relating to expand-
ing access to investigational drugs (as described in sub-
section (a)) that would be supported by the award of such 
grant. 
(c) SELECTION.—Not later than 90 days after the 
date of submission of an application for a grant under this 
section, the Secretary shall determine whether to award 
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the grant, taking into consideration whether awarding 
such grant will support a research objective relating to ex-
panding access to investigational drugs (as described in 
subsection (a)), consistent with the mission of the Na-
tional Institutes of Health. 
(d) USE OF FUNDS.—An eligible entity may use 
funds received through the grant— 
(1) to pay the manufacturer or sponsor for the 
direct costs of such drug (as authorized under sec-
tion 312.8(d) of title 21, Code of Federal Regula-
tions (or successor regulations)), if such costs are 
justified as part of peer review of the grant; 
(2) for the entity’s direct costs incurred in pro-
viding such drug consistent with the research mis-
sion of the grant; or 
(3) for the direct and indirect costs of the enti-
ty in conducting research with respect to the drug 
involved. 
(e) DEFINITIONS.—In this section: 
(1) The term ‘‘eligible entity’’ means a partici-
pating 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 investiga-
tional new drug application under section 505(i) of 
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the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355(i)). 
(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 pre-
vent, diagnose, mitigate, treat, or cure amyotrophic 
lateral sclerosis. 
(3) The term ‘‘participating clinical trial site’’ 
means a nonprofit or public health care facility, or 
network of facilities, at which patients participating 
in a participating clinical trial receive an investiga-
tional drug through such trial. 
SEC. 3. HHS COLLABORATIVE FOR NEURODEGENERATIVE 
DISEASES. 
(a) ESTABLISHMENT.—Not later than one year after 
the date of the enactment of this Act, the Secretary of 
Health and Human Services shall establish and implement 
a Collaborative for Neurodegenerative Diseases between 
the National Institutes of Health and the Food and Drug 
Administration (to be known and referred to in this sec-
tion as the ‘‘Collaborative’’), which shall— 
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(1) enter into a cooperative agreement, con-
tract, or other instrument with a private entity or 
entities under which such private entity or entities 
will operate the Collaborative; 
(2) focus on advancing regulatory improvements 
and scientific research that will support and accel-
erate the development and approval of drugs for pa-
tients with amyotrophic lateral sclerosis; 
(3) foster the development of effective drugs 
that improve the lives of people that suffer from rare 
neurodegenerative diseases; and 
(4) share information with the Secretary to ad-
vance the purposes specified in paragraph (3), such 
as through carrying out the grant programs under 
sections 2 and 5 and developing the action plan 
under section 4. 
(b) GIFTS.— 
(1) IN GENERAL.—The Collaborative may so-
licit and accept gifts, grants, and other donations, 
establish accounts, and invest and expend funds in 
support of pre-competitive research and research as-
sociated with phase 3 and phase 4 clinical trials con-
ducted with respect to investigational drugs that are 
the subjects of expanded access applications under 
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section 561 of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 360bbb). 
(2) USE.—In addition to any amounts appro-
priated for purposes of carrying out this section, the 
Collaborative may use, without further appropria-
tion, any funds derived from a gift, grant, or other 
donation accepted pursuant to paragraph (1). 
(c) ADVISORY PANEL.— 
(1) IN GENERAL.—The Collaborative shall con-
vene an advisory panel to conduct a review of the de-
sign and implementation of the programs authorized 
by this Act. Such panel shall include representatives 
of patients, treating physicians, national organiza-
tions that facilitate provision of care services, re-
searchers, drug sponsors, drug manufacturers, and 
Federal agencies. 
(2) REPORT.—The advisory panel convened 
under paragraph (1) shall, not later than 18 months 
after the date of the enactment of this Act, submit 
to the Committee on Energy and Commerce of the 
House of Representatives and the Committee on 
Health, Education, Labor, and Pensions of the Sen-
ate a report that contains— 
(A) the findings and conclusions of the re-
view conducted under paragraph (1); and 
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(B) recommendations for carrying out the 
programs under this Act during the 2-year pe-
riod following the submission of such report, in-
cluding recommendations relating to the inclu-
sion of additional neurodegenerative diseases or 
disease areas within the grant programs under 
sections 2 and 5. 
(d) DUTIES AND AUTHORITIES.—The Collaborative 
shall identify and implement strategies for the Secretary— 
(1) for purposes of expediting the approval of 
drugs to treat amyotrophic lateral sclerosis, includ-
ing through coordination among the centers of the 
Food and Drug Administration to achieve the goals 
specified in the draft guidance for drug sponsors en-
titled ‘‘Amyotrophic Lateral Sclerosis: Developing 
Drugs for Treatment Guidance for Industry’’ pub-
lished in September 2019; 
(2) to facilitate access to investigational drugs 
for amyotrophic lateral sclerosis pursuant to an ex-
panded access request under section 561 of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb) in a similar manner as investigational drugs 
for cancer are provided through Project Facilitate of 
the Center of Excellence for Oncology; 
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(3) with respect to the rare neurodegenerative 
disease grant program established under section 5; 
(4) to define or develop the regulatory and 
translational pathway for emerging therapeutic cat-
egories; 
(5) to share, within the Collaborative, findings 
and insights related to pre-competitive research and 
research associated with phase 3 and phase 4 clin-
ical trials conducted with respect to investigational 
drugs that are the subjects of expanded access appli-
cations under section 561 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb); and 
(6) to develop and implement an ongoing mech-
anism to share feedback and information and de-
velop strategies with the neurodegenerative disease 
community, including patients, treating physicians, 
national organizations that facilitate provision of 
care services, access, and research, researchers, drug 
sponsors, drug manufacturers, and Federal agencies. 
SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN. 
(a) IN GENERAL.—Not later than 6 months after the 
date of the enactment of this Act, the Secretary of Health 
and Human Services shall publish an action plan describ-
ing actions the Department of Health and Human Serv-
ices, through the National Institutes of Health and Food 
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and Drug Administration, intend 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 the lives of people living with 
rare neurodegenerative diseases as quickly as pos-
sible; and 
(2) facilitate access to investigational drugs. 
(b) CONTENTS.—The action plan published under 
subsection (a) shall— 
(1) identify appropriate representation from 
within the Food and Drug Administration and Na-
tional Institutes of Health to be responsible for im-
plementation of such action plan; and 
(2) include elements to facilitate— 
(A) interactions and collaboration between 
the Food and Drug Administration, including 
the review centers thereof, and stakeholders in-
cluding patients, sponsors, and external bio-
medical community; 
(B) consideration of cross-cutting clinical 
and regulatory policy issues, including consist-
ency of regulatory advice and decisionmaking; 
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(C) identification of key regulatory science 
and policy issues critical to advancing develop-
ment of safe and effective drugs; and 
(D) engagement by staff of the relevant 
centers of the Food and Drug Administration 
and other relevant offices of the Food and Drug 
Administration and National Institutes of 
Health with the designated leadership of the 
Collaborative. 
SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PRO-
GRAM. 
The Secretary of Health and Human Services, acting 
through the Commissioner of Food and Drugs, shall carry 
out a program of awarding grants to, and contracts en-
tered into with, public and private entities to cover the 
costs of research on and development of interventions in-
tended to prevent, diagnose, mitigate, treat, or cure 
amyotrophic lateral sclerosis and other life-threatening or 
severely debilitating neurodegenerative diseases, including 
costs incurred with respect to the development and critical 
evaluation of tools, methods, and processes— 
(1) to characterize such neurodegenerative dis-
eases and their natural history; 
(2) to identify molecular targets for such 
neurodegenerative diseases; and 
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(3) to increase efficiency and productivity of 
clinical development of therapies, including advanc-
ing rational therapeutic development and working to 
establish clinical trial networks. 
SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 
For purposes of carrying out this Act, there are au-
thorized to be appropriated $100,000,000 for each of fis-
cal years 2022 through 2026. 
Æ 
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