HR 3537 RDS
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 ‘‘Sec-
retary’’) shall award grants to participating entities for 
purposes of scientific research utilizing data from ex-
panded access to investigational drugs for individuals who 
are not otherwise eligible for clinical trials for the preven-
tion, 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 Sec-
retary, 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 regula-
tions), before the application for such grant is submitted. 
(b) APPLICATION.— 
(1) IN GENERAL.—A participating entity seek-
ing a grant under this section shall submit to the 
Secretary an application at such time, in such man-
ner, and containing such information as the Sec-
retary shall specify. 
(2) USE OF DATA.—An application submitted 
under paragraph (1) shall include a description of 
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how data generated through an expanded access re-
quest 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 pre-
vention, 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 clin-
ical trials for investigational therapies for the pre-
vention, 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 shall, in determining whether to 
award a grant under this section, confirm that— 
(1) such grant will be used to support a sci-
entific research objective relating to the prevention, 
diagnosis, 
mitigation, 
treatment, 
or 
cure 
of 
amyotrophic lateral sclerosis (as described in sub-
section (a)); 
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(2) such grant shall not have the effect of di-
minishing 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 reg-
istered on ClinicalTrials.gov (or successor 
website), with respect to a drug for the preven-
tion, diagnosis, mitigation, treatment, or cure of 
amyotrophic lateral sclerosis; or 
(B) clinical trials for the prevention, diag-
nosis, 
mitigation, 
treatment, 
or 
cure 
of 
amyotrophic lateral sclerosis for which an ex-
emption 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— 
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(1) to pay the manufacturer or sponsor for the 
direct costs of the investigational drug, as author-
ized under section 312.8(d) of title 21, Code of Fed-
eral Regulations (or successor regulations), to pre-
vent, 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 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 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 in-
vestigational 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. 
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(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 health care facility, or network of facilities, 
at which patients participating in a participating 
clinical trial receive an investigational drug through 
such trial. 
(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-Pri-
vate Partnership for Neurodegenerative Diseases between 
the National Institutes of Health, the Food and Drug Ad-
ministration, and one or more eligible entities (to be 
known and referred to in this section as the ‘‘Partner-
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of rational therapeutic development and es-
tablishment of clinical trial networks; and 
(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 Adminis-
tration 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 fund-
ing. 
(c) GIFTS.— 
(1) IN GENERAL.—The Partnership may solicit 
and accept gifts, grants, and other donations, estab-
lish accounts, and invest and expend funds in sup-
port of basic research and research associated with 
phase 3 clinical trials conducted with respect to in-
vestigational 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 appro-
priated for purposes of carrying out this section, the 
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Partnership may use, without further appropriation, 
any funds derived from a gift, grant, or other dona-
tion accepted pursuant to paragraph (1). 
SEC. 4. ALS AND OTHER RARE NEURODEGENERATIVE DIS-
EASE 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 re-
sponsible for implementation of such action plan; 
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(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 the external bio-
medical research community; 
(B) consideration of cross-cutting clinical 
and regulatory policy issues, including consist-
ency of regulatory advice and decisionmaking; 
(C) identification of key regulatory science 
and policy issues critical to advancing develop-
ment of safe and effective drugs; and 
(D) enhancement of collaboration and en-
gagement of the relevant centers and offices of 
the Food and Drug Administration with other 
operating divisions within the Department of 
Health and Human Services, the Partnership, 
and the broader neurodegenerative disease com-
munity; and 
(3) be subject to revision, as determined appro-
priate by the Secretary of Health and Human Serv-
ices. 
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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 inter-
ventions intended to prevent, diagnose, mitigate, treat, or 
cure, amyotrophic lateral sclerosis and other rare 
neurodegenerative diseases in adults and children, includ-
ing 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 
(3) to increase efficiency and productivity of 
clinical 
development 
of 
therapies, 
including 
through— 
(A) the use of master protocols and adapt-
ive and add-on clinical trial designs; and 
(B) efforts to establish new or leverage ex-
isting clinical trial networks. 
SEC. 6. GAO REPORT. 
Not later than 4 years after the date of the enact-
ment of this Act, the Comptroller General of the United 
States shall submit to the Committee on Energy and Com-
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merce 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 develop-
ment related to the prevention, diagnosis, miti-
gation, treatment, or cure of amyotrophic lat-
eral sclerosis; and 
(B) data concerning such grants, includ-
ing— 
(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 re-
ceived expanded access to an investiga-
tional 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 
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