S.Amdt. 4474

SA 4474. Mr. COONS (for himself and Ms. Murkowski) submitted an
amendment intended to be proposed to amendment SA 3867 submitted by Mr.
Reed and intended to be proposed to the bill H.R. 4350, to authorize
appropriations for fiscal year 2022 for military activities of the
Department of Defense, for military construction, and for defense
activities of the Department of Energy, to prescribe military personnel
strengths for such fiscal year, and for other purposes; which was
ordered to lie on the table; as follows:

At the end of title X, add the following:

Subtitle H--Accelerating Access to Critical Therapies for ALS

SEC. 1071. 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

[[Page S7980]]

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. 1072. 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 amyotrophic 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. 1073. 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
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 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. 1074. 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. 1075. 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 1071--
(A) an analysis of what is known about the impact of such
grants on research or development related to the prevention,
diagnosis,

[[Page S7981]]

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 1074--
(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. 1076. AUTHORIZATION OF APPROPRIATIONS.

For purposes of carrying out this subtitle, there are
authorized to be appropriated $100,000,000 for each of fiscal
years 2022 through 2026.
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