Accelerating Access to Critical Therapies for ALS Act
This bill establishes grant programs to address neurodegenerative diseases, such as amyotrophic lateral sclerosis (also known as ALS or Lou Gehrig's disease), and contains other related provisions.
The Department of Health and Human Services (HHS) shall award grants to eligible entities to support research on and facilitate access to investigational drugs that diagnose or treat ALS. The Food and Drug Administration shall award grants to public and private entities to cover the costs of research and development of drugs that diagnose or treat ALS and other severely debilitating neurodegenerative diseases.
HHS shall also establish the Collaborative for Neurodegenerative Diseases, which shall support the development and regulatory approval of drugs that address ALS and other rare neurodegenerative diseases.
[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[S. 4867 Introduced in Senate (IS)]
<DOC>
116th CONGRESS
2d Session
S. 4867
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 SENATE OF THE UNITED STATES
October 26 (legislative day, October 19), 2020
Mr. Coons (for himself and Ms. Murkowski) introduced the following
bill; which was read twice and referred to the Committee on Health,
Education, Labor, and Pensions
_______________________________________________________________________
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 AND EXPANDED ACCESS 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 application at such time, in
such manner, and containing such information as the Secretary shall
specify. Such application shall specify a research objective relating
to expanding access to investigational drugs (as described in
subsection (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 the grant, taking into consideration whether
awarding such grant will support a research objective relating to
expanding access to investigational drugs (as described in subsection
(a)), consistent with the mission of the National 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 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 ``eligible 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 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 section as the
``Collaborative''), which shall--
(1) enter into a cooperative agreement, contract, 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 accelerate the
development and approval of drugs for patients 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 advance 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 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 and phase 4 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 Collaborative may
use, without further appropriation, any funds derived from a
gift, grant, or other donation accepted pursuant to paragraph
(1).
(c) Advisory Panel.--
(1) In general.--The Collaborative shall convene an
advisory panel to conduct a review of the design and
implementation of the programs authorized by this Act. Such
panel shall include representatives of patients, treating
physicians, national organizations that facilitate provision of
care services, researchers, 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 Senate a report
that contains--
(A) the findings and conclusions of the review
conducted under paragraph (1); and
(B) recommendations for carrying out the programs
under this Act during the 2-year period following the
submission of such report, including recommendations
relating to the inclusion 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, including through
coordination among the centers of the Food and Drug
Administration to achieve the goals specified in the draft
guidance for drug sponsors entitled ``Amyotrophic Lateral
Sclerosis: Developing Drugs for Treatment Guidance for
Industry'' published in September 2019;
(2) to facilitate access to investigational drugs for
amyotrophic lateral sclerosis pursuant to an expanded access
request under section 561 of the Federal 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;
(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 categories;
(5) to share, within the Collaborative, findings and
insights related to pre-competitive research and research
associated with phase 3 and phase 4 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); and
(6) to develop and implement an ongoing mechanism to share
feedback and information and develop 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 describing actions the Department of Health and
Human Services, through the National Institutes of Health and Food 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 possible; 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 National Institutes of Health
to be responsible for implementation 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 including patients, sponsors,
and external biomedical 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) 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 PROGRAM.
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 entered into with, 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 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 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 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.
<all>
Introduced in Senate
Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
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