[117th Congress Public Law 79]
[From the U.S. Government Publishing Office]
<DOC>
[[Page 135 STAT. 1533]]
Public Law 117-79
117th Congress
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. <<NOTE: Dec. 23,
2021 - [H.R. 3537]>>
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled, <<NOTE: Accelerating
Access to Critical Therapies for ALS Act.>>
SECTION 1. <<NOTE: 21 USC 301 note.>> SHORT TITLE.
This Act may be cited as the ``Accelerating Access to Critical
Therapies for ALS Act''.
SEC. 2. <<NOTE: 21 USC 360ee note.>> 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) <<NOTE: Determinations.>> Selection.--Consistent with sections
406 and 492 of the Public Health Service Act (42 U.S.C. 284a, 289a), the
Secretary
[[Page 135 STAT. 1534]]
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) <<NOTE: Time period.>> 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.
[[Page 135 STAT. 1535]]
(f) Sunset.--The Secretary may not award grants under this section
on or after September 30, 2026.
SEC. 3. <<NOTE: 42 USC 280g-7b.>> HHS PUBLIC-PRIVATE PARTNERSHIP
FOR RARE NEURODEGENERATIVE DISEASES.
(a) <<NOTE: Deadline. Contracts.>> 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) <<NOTE: Definition.>> 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) <<NOTE: Evaluations.>> 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
[[Page 135 STAT. 1536]]
(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. <<NOTE: 21 USC 360aa note.>> ALS AND OTHER RARE
NEURODEGENERATIVE DISEASE ACTION PLAN.
(a) <<NOTE: Deadline. Web posting. Time period.>> 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
[[Page 135 STAT. 1537]]
Department of Health and Human Services, the Partner-
ship, and the broader neurodegenerative disease community; and
(3) be subject to revision, as determined appropriate by the Secretary
Determination of Health and Human Services.
SEC. 5. FDA RARE NEURODEGENERATIVE DISEASE GRANT PROGRAM. 21 USC 360ee-1
The <<NOTE: Contracts. Evaluations.>> 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. <<NOTE: Analyses. Data.>> 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--
[[Page 135 STAT. 1538]]
(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. <<NOTE: Time period.>> 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.
Approved December 23, 2021.
LEGISLATIVE HISTORY--H.R. 3537:
---------------------------------------------------------------------------
HOUSE REPORTS: No. 117-207 (Comm. on Energy and Commerce).
CONGRESSIONAL RECORD, Vol. 167 (2021):
Dec. 8, considered and passed House.
Dec. 16, considered and passed Senate.
DAILY COMPILATION OF PRESIDENTIAL DOCUMENTS (2021):
Dec. 23, Presidential remarks.
<all>