H.R. 3537 — Accelerating Access to Critical Therapies for ALS Act

[117th Congress Public Law 79]
[From the U.S. Government Publishing Office]


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[[Page 135 STAT. 1533]]

Public Law 117-79
117th Congress

                                 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. <<NOTE: Dec. 23, 
                         2021 -  [H.R. 3537]>> 

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled, <<NOTE: Accelerating 
Access to Critical Therapies for ALS Act.>> 
SECTION 1. <<NOTE: 21 USC 301 note.>>  SHORT TITLE.

    This Act may be cited as the ``Accelerating Access to Critical 
Therapies for ALS Act''.
SEC. 2. <<NOTE: 21 USC 360ee note.>>  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) <<NOTE: Determinations.>>  Selection.--Consistent with sections 
406 and 492 of the Public Health Service Act (42 U.S.C. 284a, 289a), the 
Secretary

[[Page 135 STAT. 1534]]

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) <<NOTE: Time period.>>  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.

[[Page 135 STAT. 1535]]

    (f) Sunset.--The Secretary may not award grants under this section 
on or after September 30, 2026.
SEC. 3. <<NOTE: 42 USC 280g-7b.>>  HHS PUBLIC-PRIVATE PARTNERSHIP 
                    FOR RARE NEURODEGENERATIVE DISEASES.

    (a) <<NOTE: Deadline. Contracts.>>  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) <<NOTE: Definition.>>  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) <<NOTE: Evaluations.>>  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

[[Page 135 STAT. 1536]]

                    (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. <<NOTE: 21 USC 360aa note.>>  ALS AND OTHER RARE 
                    NEURODEGENERATIVE DISEASE ACTION PLAN.

    (a) <<NOTE: Deadline. Web posting. Time period.>>  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

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               Department of Health and Human Services, the Partner-
       ship, and the broader neurodegenerative disease community; and
      (3) be subject to revision, as determined appropriate by the Secretary 
           Determination of Health and Human Services.
           
    SEC. 5. FDA RARE NEURODEGENERATIVE DISEASE GRANT PROGRAM. 21 USC 360ee-1                                             

    The <<NOTE: Contracts. Evaluations.>>  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. <<NOTE: Analyses. Data.>>  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--

[[Page 135 STAT. 1538]]

                    (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. <<NOTE: Time period.>>  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.

    Approved December 23, 2021.

LEGISLATIVE HISTORY--H.R. 3537:
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HOUSE REPORTS: No. 117-207 (Comm. on Energy and Commerce).
CONGRESSIONAL RECORD, Vol. 167 (2021):
            Dec. 8, considered and passed House.
            Dec. 16, considered and passed Senate.
DAILY COMPILATION OF PRESIDENTIAL DOCUMENTS (2021):
            Dec. 23, Presidential remarks.
            

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Accelerating Access to Critical Therapies for ALS Act

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