[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3537 Enrolled Bill (ENR)]
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 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.
(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
(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
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 add-on
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--
(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.