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[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3537 Enrolled Bill (ENR)]

        H.R.3537

                    One Hundred Seventeenth Congress

                                 of the

                        United States of America

                          AT THE FIRST SESSION

           Begun and held at the City of Washington on Monday,
         the fourth day of January, two thousand and twenty-one

                                 An Act

 
To direct the Secretary of Health and Human Services to support research 
   on, and expanded access to, investigational drugs for amyotrophic 
               lateral sclerosis, and for other purposes.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
    This Act may be cited as the ``Accelerating Access to Critical 
Therapies for ALS Act''.
SEC. 2. GRANTS FOR RESEARCH ON THERAPIES FOR ALS.
    (a) In General.--The Secretary of Health and Human Services 
(referred to in this section as the ``Secretary'') shall award grants 
to participating entities for purposes of 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.

                               Speaker of the House of Representatives.

                            Vice President of the United States and    
                                               President of the Senate.
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