[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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