[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3537 Introduced in House (IH)]
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117th CONGRESS
1st Session
H. R. 3537
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
May 25, 2021
Mr. Quigley (for himself, Mr. Fortenberry, Mr. Brendan F. Boyle of
Pennsylvania, Mr. Moulton, Mr. Garcia of California, Mr. Carbajal, Mr.
Larson of Connecticut, Ms. Jackson Lee, Ms. Dean, Mr. Suozzi, Ms.
Velazquez, Mr. Levin of California, Mr. Deutch, Ms. Norton, Mr.
Timmons, Mr. Brady, Mr. McKinley, Mr. Van Drew, Mr. Calvert, Mr.
Keating, Mr. Diaz-Balart, Mr. Carter of Georgia, Mrs. McBath, Mr. Smith
of Missouri, Mr. Turner, Mr. Duncan, Mr. Hice of Georgia, Mr. Young,
Mr. Smith of Nebraska, Mr. Grothman, Mr. Ruppersberger, Mr. Rutherford,
Mr. Schweikert, Mr. Ryan, Mr. Crow, Mr. Guthrie, Mr. Fitzpatrick, Ms.
McCollum, Mr. Austin Scott of Georgia, Mr. Baird, Mr. Rodney Davis of
Illinois, Mr. Valadao, Mr. Moolenaar, Mr. Malinowski, Ms. Roybal-
Allard, Mr. Payne, Mr. Lynch, Ms. Herrera Beutler, Mr. Buck, Mr.
Mullin, Mr. Grijalva, Mr. Cooper, Mr. Panetta, Mr. Kim of New Jersey,
Mr. Sires, Ms. Lee of California, Ms. Moore of Wisconsin, Ms.
Schakowsky, Mr. Thompson of California, Mr. Gallego, Mrs. Axne, Mrs.
Napolitano, Mr. Espaillat, Ms. Pressley, Mr. Fleischmann, Mr.
Reschenthaler, Mr. Cicilline, Ms. DeGette, Mr. Burchett, Mr. LaMalfa,
Ms. Meng, Ms. Brownley, Mr. Trone, Ms. Kuster, Mr. Connolly, Mr. Meeks,
Mrs. Kirkpatrick, Mrs. Demings, Mr. O'Halleran, Mr. Lieu, Mr.
DeSaulnier, Mr. Garamendi, Mr. Kilmer, Mr. Rush, Mr. McCaul, Mr.
McClintock, Mr. Mfume, Mr. Lamb, Mr. Green of Texas, Mr. Swalwell, Mr.
Gottheimer, Ms. Pingree, Ms. Kaptur, Mr. Ferguson, Ms. Scanlon, Mr.
Bacon, Mr. Wittman, Mr. Morelle, Mr. Amodei, and Mr. Waltz) 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, 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 expanded access for individuals
to investigational drugs for the prevention, diagnosis, mitigation,
treatment, or cure of amyotrophic lateral sclerosis. In the case of an
applicant 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 may be used by the Secretary
to support research or development related to the prevention,
diagnosis, mitigation, treatment, or cure of amyotrophic
lateral sclerosis or other rare neurodegenerative diseases.
(c) Selection.--Not later than 120 days after the date of
submission of an application for a grant under this section, the
Secretary shall determine whether to award the grant, taking into
consideration--
(1) whether awarding such grant will support a research
objective relating to expanding access to investigational drugs
(as described in subsection (a)); and
(2) whether awarding such a grant may have the effect of
diminishing eligibility for, or impeding enrollment of, ongoing
clinical investigations.
(d) Use of Funds.--A participating 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 section 312.8(d) of title 21,
Code of Federal Regulations (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 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 the drug involved.
(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)).
(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
nonprofit or public health care facility, or network of
facilities, at which patients participating in a participating
clinical trial receive an investigational drug through such
trial.
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 instruments with such
eligible entities, for the purpose of developing treatments for
amytrophic lateral sclerosis and other rare neurodegenerative diseases.
The Partnership shall--
(1) establish partnerships, consortia, and collaborations
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 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 drug 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 pre-competitive research
and research associated with phase 3 clinical trials conducted
with respect to investigational drugs that are the subjects of
expanded access applications 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 Secretary of Health and Human Services shall
publish on the website of the Department of Health and Human Services
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 as quickly as possible; 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 decision making;
(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 by
staff of the relevant centers of the Food and Drug
Administration and other relevant 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 shall use funds made
available under section 6 to 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 life-threatening or
severely debilitating 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 advancing rational
therapeutic development and working to establish new or
leverage existing clinical trial networks.
SEC. 6. 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.
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