[House Report 117-207]
[From the U.S. Government Publishing Office]
117th Congress } { Report
HOUSE OF REPRESENTATIVES
1st Session } { 117-207
======================================================================
ACCELERATING ACCESS TO CRITICAL THERAPIES FOR ALS ACT
_______
December 8, 2021.--Committed to the Committee of the Whole House on the
State of the Union and ordered to be printed
_______
Mr. Pallone, from the Committee on Energy and Commerce, submitted the
following
R E P O R T
[To accompany H.R. 3537]
The Committee on Energy and Commerce, to whom was referred
the bill (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, having considered the same, reports
favorably thereon with an amendment and recommends that the
bill as amended do pass.
CONTENTS
Page
I. Purpose and Summary.............................................. 5
II. Background and Need for the Legislation.......................... 5
III. Committee Hearings............................................... 7
IV. Committee Consideration.......................................... 8
V. Committee Votes.................................................. 8
VI. Oversight Findings............................................... 8
VII. New Budget Authority, Entitlement Authority, and Tax Expenditures 8
VIII.Federal Mandates Statement....................................... 8
IX. Statement of General Performance Goals and Objectives............ 9
X. Duplication of Federal Programs.................................. 9
XI. Committee Cost Estimate.......................................... 9
XII. Earmarks, Limited Tax Benefits, and Limited Tariff Benefits...... 9
XIII.Advisory Committee Statement..................................... 9
XIV. Applicability to Legislative Branch.............................. 9
XV. Section-by-Section Analysis of the Legislation................... 9
XVI. Changes in Existing Law Made by the Bill, as Reported........... 12
The amendment is as follows:
Strike all after the enacting clause and insert the
following:
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.
I. PURPOSE AND SUMMARY
H.R. 3537, the ``Accelerating Access to Critical Therapies
for ALS Act'', authorizes the Secretary of Health and Human
Services (the Secretary) to 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
preventing, diagnosing, mitigating, treating, or curing
amyotrophic lateral sclerosis (ALS), establishes a public-
private partnership for rare neurodegenerative diseases,
requires the Food and Drug Administration (FDA) to publish an
action plan related to ALS and other rare neurodegenerative
diseases, and establishes a grant program at FDA to cover
certain costs of research and development of drugs for ALS and
other rare neurodegenerative diseases.
II. BACKGROUND AND NEED FOR LEGISLATION
ALS is a rare, progressive, neurological disease that
affects nerve cells responsible for controlling voluntary
muscle movement.\1\ Approximately 5,000 people each year in the
United States are diagnosed with ALS.\2\ ALS usually presents
at first as muscle weakness or stiffness and gradually affects
all voluntary muscles, including those used to control actions
like speaking, eating, moving, and breathing.\3\ There is
considerable clinical heterogeneity among patients with ALS,
including differing rates of progression.\4\ While most people
with ALS die within three to five years after symptoms first
appear, about 10 percent of people with ALS survive for 10
years or more.\5\
---------------------------------------------------------------------------
\1\National Institutes of Health, Amyotrophic Lateral Sclerosis
(ALS) Fact Sheet (2021) (https://www.ninds.nih.gov/Disorders/Patient-
Caregiver-Education/Fact-Sheets/Amyotrophic-Lateral-Sclerosis-ALS-Fact-
Sheet).
\2\Centers for Disease Control and Prevention, What is Amyotrophic
Lateral Sclerosis? (2018) (https://www.cdc.gov/als/
WhatisAmyotrophiclateralsclerosis.html).
\3\Id.
\4\Namita A. Goyal et al., Addressing Heterogeneity in Amyotrophic
Lateral Sclerosis, Muscle and Nerve (2020).
\5\Supra note 1.
---------------------------------------------------------------------------
There is no known cure and there are very few FDA-approved
treatments for ALS, and the cause of ALS is not well
understood. There is no clearly identified and accepted ALS
biomarker, which makes it difficult to diagnose the disease,
track disease progression, and identify clinical efficacy of
investigational treatments.\6\ It can take eight to 15 months
to diagnose an individual with ALS, which can lead to invasive
diagnostic procedures, missed opportunities to receive
treatment, and missed opportunities to participate in clinical
research.\7\ Patients have reported that they are often
excluded from clinical trials after the disease has progressed
past a certain point, effectively preventing patients from
being able to access investigational treatments.\8\ While
expanded access to investigational treatments is allowed by FDA
for some patients who cannot access clinical trials, and almost
all applications are granted, many drug manufacturers choose
not to operate expanded access programs.\9\ FDA cannot require
a manufacturer to provide a drug under expanded access.\10\
---------------------------------------------------------------------------
\6\Martin R. Turner et al., Mechanisms, Models, and Biomarkers in
Amyotrophic Lateral Sclerosis, ALS and Frontotemporal Degeneration
(2013).
\7\Sabrina Paganoni et al., Diagnostic Timelines and Delays in
Diagnosing Amyotrophic Lateral Sclerosis, ALS and Frontotemporal
Degeneration (2014).
\8\Danielle Richards, John Morren, and Erik Pioro, Time to
Diagnosis and Factors Affecting Diagnostic Delay in Amyotrophic Lateral
Sclerosis (July 25, 2021).
\9\Food and Drug Administration, Expanded Access (2021) (https://
www.fda.gov/news-events/public-health-focus/expanded-access).
\10\House Committee on Energy and Commerce, Hearing on The Path
Forward: Advancing Treatments and Cures for Neurodegenerative Diseases,
117th Cong. (July 29, 2021).
---------------------------------------------------------------------------
FDA has sought to encourage additional patient
participation in ALS clinical trials through published guidance
for industry, which recommends that developers use broader
inclusion criteria in their clinical trial design.\11\ FDA has
also recommended the use of master protocols, which allow for
the simultaneous evaluation of multiple drugs, with a shared
placebo group, thereby allowing for more drugs to be studied at
once, and more individuals to receive investigational
treatments, rather than placebo.\12\ Additionally, FDA has
recommended that developers consider add-on trial designs, in
which all individuals in the trial receive a treatment
previously shown to be effective in treating ALS, so no patient
only receives placebo.\13\ FDA also said in its guidance that
it would exercise regulatory flexibility in applying statutory
standards for effectiveness for serious diseases with unmet
medical needs, like ALS, while preserving appropriate assurance
of safety and effectiveness.\14\ However, some patients have
argued that FDA has been too stringent in applying that
regulatory flexibility, noting several investigational
therapies that were not found to have met safety and efficacy
standards.\15\
---------------------------------------------------------------------------
\11\Food and Drug Administration, Amyotrophic Lateral Sclerosis:
Developing Drugs for Treatment: Guidance for Industry (Sept. 2019).
\12\Id.
\13\Id.
\14\Id.
\15\A Slow FDA is Denying ALS Patients their Only Hope, Wall Street
Journal (Apr. 26, 2021).
---------------------------------------------------------------------------
H.R. 3537 creates two new grant programs to address issues
related to research and development of therapies for ALS and
other rare neurodegenerative diseases and patient access to
investigational therapies. First, the bill authorizes a grant
program for five years for purposes of scientific research
utilizing data from expanded access to investigational drugs
for ALS. Under that program, the Secretary could provide grants
to drug manufacturers for investigational ALS drugs to cover
costs associated with expanded access for patients not eligible
for clinical trials. Additionally, a grant program at FDA would
cover costs of research and development for ALS and other rare
neurodegenerative disease therapies, including funding to
increase efficiency and productivity of clinical therapy
development through the use of master protocols and adaptive
and add-on clinical trial designs and efforts to establish new
or leverage existing clinical trial networks. The bill requires
the Government Accountability Office (GAO) to analyze the
efficacy of these grant programs and report to Congress within
four years of enactment.
To increase the understanding of neurodegenerative diseases
among researchers, developers, and regulators, and to foster
development of treatments for ALS and other rare
neurodegenerative diseases, H.R. 3537 establishes a public-
private partnership between the National Institutes of Health
(NIH), FDA, and one or more eligible entities, such as an
institution of higher education or a 501(c)(3) non-profit
organization. The bill also requires FDA to publish a plan
describing the actions the agency intends to take over the next
five years with respect to fostering development of treatments
and facilitating access to investigational drugs for ALS and
other rare neurodegenerative diseases.
To carry out these activities, the bill authorizes $100
million annually for fiscal years 2022 through 2026.
III. COMMITTEE HEARINGS
For the purposes of section 3(c) of rule XIII of the Rules
of the House of Representatives, the following hearing was used
to develop or consider H.R. 3537:
The Subcommittee on Health held a topical hearing on July
29, 2021 entitled ``The Path Forward: Advancing Treatments and
Cures for Neurodegenerative Diseases''. The Subcommittee
received testimony from the following witnesses:
Panel I
Patrizia Cavazzoni, M.D., Director, Center
for Drug Evaluation and Research, FDA;
Richard J. Hodes, M.D., Director, National
Institute on Aging, NIH; and
Walter J. Koroshetz, M.D., Director,
National Institute of Neurological Disorders and
Stroke, NIH.
Panel II
Jinsy Andrews, M.D., Director of
Neuromuscular Clinical Trials, Neurological Institute
of New York, Associate Professor of Neurology, Columbia
University Vagelos College of Physicians and Surgeons;
Kala Booth, Huntington's Disease Caregiver
and Patient;
Merit Cudkowicz, M.D., Director, Sean M.
Healy and AMG Center for ALS, Chief, Neurology
Department, Massachusetts General Hospital, Julianne
Dorn Professor of Neurology, Harvard Medical School;
Cartier Esham, Ph.D., Executive Vice
President, Emerging Companies, Senior Vice President,
Science and Regulatory Affairs, Biotechnology
Innovation Organization;
Yvonne Latty, Caregiver; and
Brian Wallach, Co-Founder, I AM ALS.
IV. COMMITTEE CONSIDERATION
Representative Mike Quigley (D-IL) and 99 original
cosponsors introduced H.R. 3537, the ``Accelerating Access to
Critical Therapies for ALS Act,'' on May 25, 2021, and it was
referred to the Committee on Energy and Commerce. Subsequently,
on May 26, 2021, H.R. 3537 was referred to the Subcommittee on
Health.
On November 4, 2021, the Subcommittee on Health met in open
markup session, pursuant to notice, to consider H.R. 3537 and 8
other bills. During consideration of the bill, an amendment in
the nature of a substitute (AINS) offered by Representative
Rodgers (R-WA) was agreed to by a voice vote. Upon conclusion
of consideration of the bill, the Subcommittee on Health agreed
to report the bill favorably to the full Committee, amended, by
a voice vote.
On November 17, 2021, the full Committee met in open markup
session, pursuant to notice, to consider H.R. 3537 and 11 other
bills. No amendments were offered to H.R. 3537. Upon conclusion
of consideration of the bill, the full Committee agreed to a
motion on final passage offered by Representative Pallone (D-
NJ), Chairman of the Committee, to order H.R. 3537 reported
favorably to the House, as amended, by a voice vote.
V. COMMITTEE VOTES
Clause 3(b) of rule XIII of the Rules of the House of
Representatives requires the Committee to list each record vote
on the motion to report legislation and amendments thereto. The
Committee advises that there were no record votes taken on H.R.
3537.
VI. OVERSIGHT FINDINGS
Pursuant to clause 3(c)(1) of rule XIII and clause 2(b)(1)
of rule X of the Rules of the House of Representatives, the
oversight findings and recommendations of the Committee are
reflected in the descriptive portion of the report.
VII. NEW BUDGET AUTHORITY, ENTITLEMENT AUTHORITY, AND TAX EXPENDITURES
Pursuant to 3(c)(2) of rule XIII of the Rules of the House
of Representatives, the Committee adopts as its own the
estimate of new budget authority, entitlement authority, or tax
expenditures or revenues contained in the cost estimate
prepared by the Director of the Congressional Budget Office
pursuant to section 402 of the Congressional Budget Act of
1974.
The Committee has requested but not received from the
Director of the Congressional Budget Office a statement as to
whether this bill contains any new budget authority, spending
authority, credit authority, or an increase or decrease in
revenues or tax expenditures.
VIII. FEDERAL MANDATES STATEMENT
The Committee adopts as its own the estimate of Federal
mandates prepared by the Director of the Congressional Budget
Office pursuant to section 423 of the Unfunded Mandates Reform
Act.
IX. STATEMENT OF GENERAL PERFORMANCE GOALS AND OBJECTIVES
Pursuant to clause 3(c)(4) of rule XIII, the general
performance goals or objectives of this legislation are to
direct the Secretary to support research related to ALS and
other rare neurodegenerative diseases, foster development of
therapies to treat ALS and such diseases, and facilitate access
to investigational drugs for appropriate individuals through
clinical trials, and in the event that an individual is not
eligible for clinical trials and otherwise qualifies, through
expanded access.
X. DUPLICATION OF FEDERAL PROGRAMS
Pursuant to clause 3(c)(5) of rule XIII, no provision of
H.R. 3537 is known to be duplicative of another Federal
program, including any program that was included in a report to
Congress pursuant to section 21 of Public Law 111-139 or the
most recent Catalog of Federal Domestic Assistance.
XI. COMMITTEE COST ESTIMATE
Pursuant to clause 3(d)(1) of rule XIII, the Committee
adopts as its own the cost estimate prepared by the Director of
the Congressional Budget Office pursuant to section 402 of the
Congressional Budget Act of 1974.
XII. EARMARKS, LIMITED TAX BENEFITS, AND LIMITED TARIFF BENEFITS
Pursuant to clause 9(e), 9(f), and 9(g) of rule XXI, the
Committee finds that H.R. 3537 contains no earmarks, limited
tax benefits, or limited tariff benefits.
XIII. ADVISORY COMMITTEE STATEMENT
No advisory committee within the meaning of section 5(b) of
the Federal Advisory Committee Act was created by this
legislation.
XIV. APPLICABILITY TO LEGISLATIVE BRANCH
The Committee finds that the legislation does not relate to
the terms and conditions of employment or access to public
services or accommodations within the meaning of section
102(b)(3) of the Congressional Accountability Act.
XV. SECTION-BY-SECTION ANALYSIS OF THE LEGISLATION
Section 1. Short title
Section 1 designates that the Act may be cited as the
``Accelerating Access to Critical Therapies for ALS Act.''
Sec. 2. Grants for research on therapies for ALS
Section 2 creates a grant program at the Department of
Health and Human Services (HHS) 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 ALS. An expanded access request must be
submitted and allowed by FDA before an application for a grant
is submitted under this section.
In applying for grants, participating entities would be
required to include a description of how data generated through
an expanded access request will be used to support research and
development related to the prevention, diagnosis, mitigation,
treatment, or cure of ALS, and must also include a description
of how the proposed expanded access program will not interfere
with patient enrollment in ongoing clinical trials for ALS.
Consistent with sections 406 and 492 of the Public Health
Service Act, which concern advisory councils at NIH and
required peer review processes, the Secretary is required to,
when making a decision about whether to award a grant, confirm
that the grant will be used to support a scientific research
objective relating to the prevention, diagnosis, mitigation,
treatment, or cure of ALS and that the grant will not diminish
eligibility for or impede enrollment of ongoing clinical trials
for ALS, by ensuring that those patients enrolled in the
program are not otherwise eligible for either a trial
registered on ClinicalTrials.gov or another trial for an
approved investigational new drug (IND) which is expected to
begin enrollment within one year. The Secretary must also
confirm that the resulting project funded by the grant will
ensure equitable access to drugs for minority and underserved
populations.
Funds in the expanded access grant program shall be used to
pay the manufacturer or sponsor for direct costs of an
investigational drug to prevent, diagnose, mitigate, treat, or
cure ALS that is the subject of the expanded access request
allowed to proceed by FDA, if such costs are justified as part
of peer review; the participating entity's direct costs
incurred in providing such drug consistent with the research
mission of the grant; or for the direct and indirect costs of
the entity in conducting research with respect to such drug.
Participating entities are defined as a participating
clinical trial site or sites sponsored by a small business
concern, as defined by section 3(a) of the Small Business Act
that is the sponsor of a drug subject to an IND application
under the Federal Food, Drug, and Cosmetic Act to prevent,
diagnose, mitigate, treat, or cure ALS. Participating clinical
trials must operate pursuant to an approved IND and be in Phase
3. Participating clinical trial site means a health care
facility or network of facilities at which patients
participating in a participating clinical trial receive
investigational drugs for ALS.
Section 2 includes a sunset clause which prohibits the
Secretary from making any grants under this section on or after
September 30, 2026.
Sec. 3. HHS public-private partnership for rare neurodegenerative
diseases
Section 3 requires the Secretary to establish a Public-
Private Partnership for Neurodegenerative Diseases (the
``Partnership'') between NIH, FDA, and one or more eligible
private entities, for the purpose of advancing the
understanding of neurodegenerative diseases and fostering
development of treatments for ALS and other rare
neurodegenerative diseases. The Partnership would be required
to establish partnerships and consortia with other public and
private entities and individuals with expertise in ALS and
other rare neurodegenerative diseases; focus on advancing
regulatory science and scientific research to support and
accelerate the development and review of drugs for patients
with ALS and other rare neurodegenerative diseases; and foster
the development of effective drugs that improve the lives of
those with ALS and other neurodegenerative diseases.
Eligible private entities in the Partnership may be an
institution of higher education, a consortium of such
institutions, or a 501(c)(3) nonprofit organization. The
eligible entities must have experienced personnel with clinical
and other technical expertise in the field of biomedical
sciences and a demonstrated connection to the patient
population, and must demonstrate to the Secretary's
satisfaction that it is capable of identifying and establishing
collaborations between public and private entities and
individuals with expertise in neurodegenerative diseases,
including patients, to facilitate the development and
evaluation of tools, methods, and processes to characterize
neurodegenerative diseases and their natural history, identify
molecular targets for neurodegenerative diseases, and to
increase efficiency, predictability, and productivity of
clinical development of therapies. Eligible entities must also
demonstrate that the entity is capable of securing funding for
the Partnership from federal and non-federal sources and
provide an assurance that it will not accept funding from any
organization that manufactures or distributes products
regulated by FDA unless the entity provides assurances that the
results of the project will not be influenced by any source of
funding.
The Partnership is authorized to solicit 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 subject to an expanded access
request, and may use such funds received without further
appropriation.
Sec. 4. ALS and other rare neurodegenerative disease action plan
Section 4 requires the Commissioner of Food and Drugs to
publish on the FDA website an action plan describing the
actions FDA intends to take in the next five years to foster
the development of safe and effective drugs that improve or
extend the lives of people living with ALS and other rare
neurodegenerative diseases and facilitate access to
investigational drugs for such diseases.
Sec. 5. FDA rare neurodegenerative disease grant program
Section 5 establishes a grant program at FDA 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 ALS and
other rare neurodegenerative diseases in adults and children.
The costs covered by grants include those incurred with respect
to development and critical evaluation of tools, methods, and
processes to characterize such neurodegenerative diseases and
their natural history, identify molecular targets for such
neurodegenerative diseases, and increase efficiency and
productivity of clinical development of therapies, including
through the use of master protocols and adaptive and add-on
clinical trial designs and efforts to establish new or leverage
existing clinical trial networks.
Sec. 6. GAO report.
Section 6 requires GAO to issue a report to the Committee
on Energy and Commerce of the House of Representatives and the
Committee on Health, Education, Labor, and Pensions of the
Senate within four years of enactment, analyzing what is known
about the impact the grant programs established in Section 2
and Section 5 had on research and development related to the
prevention, diagnosis, mitigation, treatment, or cure of ALS.
Sec. 7. Authorization of appropriations
Section 7 authorizes $100 million annually for fiscal years
2022 through 2026 to carry out this Act.
XVI. CHANGES IN EXISTING LAW MADE BY THE BILL, AS REPORTED
There are no changes to existing law made by H.R. 3537.
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