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[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[S. 4867 Introduced in Senate (IS)]

<DOC>

116th CONGRESS
  2d Session
                                S. 4867

    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.

_______________________________________________________________________

                   IN THE SENATE OF THE UNITED STATES

             October 26 (legislative day, October 19), 2020

  Mr. Coons (for himself and Ms. Murkowski) introduced the following 
  bill; which was read twice and referred to the Committee on Health, 
                     Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL

 
    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 AND EXPANDED ACCESS TO EXPERIMENTAL 
              THERAPIES FOR ALS.

    (a) In General.--The Secretary of Health and Human Services shall 
award grants to eligible entities for purposes of supporting research 
on, and expanded access for individuals to, investigational drugs for 
the prevention, diagnosis, mitigation, treatment, or cure of 
amyotrophic lateral sclerosis pursuant to an expanded access request 
submitted, and allowed to proceed by the Secretary, under section 561 
of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb).
    (b) Application.--An eligible 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. Such application shall specify a research objective relating 
to expanding access to investigational drugs (as described in 
subsection (a)) that would be supported by the award of such grant.
    (c) Selection.--Not later than 90 days after the date of submission 
of an application for a grant under this section, the Secretary shall 
determine whether to award the grant, taking into consideration whether 
awarding such grant will support a research objective relating to 
expanding access to investigational drugs (as described in subsection 
(a)), consistent with the mission of the National Institutes of Health.
    (d) Use of Funds.--An eligible entity may use funds received 
through the grant--
            (1) to pay the manufacturer or sponsor for the direct costs 
        of such drug (as authorized under section 312.8(d) of title 21, 
        Code of Federal Regulations (or successor regulations)), 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 the drug involved.
    (e) Definitions.--In this section:
            (1) The term ``eligible 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)).
            (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 
        nonprofit or public health care facility, or network of 
        facilities, at which patients participating in a participating 
        clinical trial receive an investigational drug through such 
        trial.

SEC. 3. HHS COLLABORATIVE FOR NEURODEGENERATIVE DISEASES.

    (a) Establishment.--Not later than one year after the date of the 
enactment of this Act, the Secretary of Health and Human Services shall 
establish and implement a Collaborative for Neurodegenerative Diseases 
between the National Institutes of Health and the Food and Drug 
Administration (to be known and referred to in this section as the 
``Collaborative''), which shall--
            (1) enter into a cooperative agreement, contract, or other 
        instrument with a private entity or entities under which such 
        private entity or entities will operate the Collaborative;
            (2) focus on advancing regulatory improvements and 
        scientific research that will support and accelerate the 
        development and approval of drugs for patients with amyotrophic 
        lateral sclerosis;
            (3) foster the development of effective drugs that improve 
        the lives of people that suffer from rare neurodegenerative 
        diseases; and
            (4) share information with the Secretary to advance the 
        purposes specified in paragraph (3), such as through carrying 
        out the grant programs under sections 2 and 5 and developing 
        the action plan under section 4.
    (b) Gifts.--
            (1) In general.--The Collaborative may solicit and accept 
        gifts, grants, and other donations, establish accounts, and 
        invest and expend funds in support of pre-competitive research 
        and research associated with phase 3 and phase 4 clinical 
        trials conducted with respect to investigational drugs that are 
        the subjects of expanded access applications 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 Collaborative may 
        use, without further appropriation, any funds derived from a 
        gift, grant, or other donation accepted pursuant to paragraph 
        (1).
    (c) Advisory Panel.--
            (1) In general.--The Collaborative shall convene an 
        advisory panel to conduct a review of the design and 
        implementation of the programs authorized by this Act. Such 
        panel shall include representatives of patients, treating 
        physicians, national organizations that facilitate provision of 
        care services, researchers, drug sponsors, drug manufacturers, 
        and Federal agencies.
            (2) Report.--The advisory panel convened under paragraph 
        (1) shall, not later than 18 months after the date of the 
        enactment of this Act, 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 
        that contains--
                    (A) the findings and conclusions of the review 
                conducted under paragraph (1); and
                    (B) recommendations for carrying out the programs 
                under this Act during the 2-year period following the 
                submission of such report, including recommendations 
                relating to the inclusion of additional 
                neurodegenerative diseases or disease areas within the 
                grant programs under sections 2 and 5.
    (d) Duties and Authorities.--The Collaborative shall identify and 
implement strategies for the Secretary--
            (1) for purposes of expediting the approval of drugs to 
        treat amyotrophic lateral sclerosis, including through 
        coordination among the centers of the Food and Drug 
        Administration to achieve the goals specified in the draft 
        guidance for drug sponsors entitled ``Amyotrophic Lateral 
        Sclerosis: Developing Drugs for Treatment Guidance for 
        Industry'' published in September 2019;
            (2) to facilitate access to investigational drugs for 
        amyotrophic lateral sclerosis pursuant to an expanded access 
        request under section 561 of the Federal Food, Drug, and 
        Cosmetic Act (21 U.S.C. 360bbb) in a similar manner as 
        investigational drugs for cancer are provided through Project 
        Facilitate of the Center of Excellence for Oncology;
            (3) with respect to the rare neurodegenerative disease 
        grant program established under section 5;
            (4) to define or develop the regulatory and translational 
        pathway for emerging therapeutic categories;
            (5) to share, within the Collaborative, findings and 
        insights related to pre-competitive research and research 
        associated with phase 3 and phase 4 clinical trials conducted 
        with respect to investigational drugs that are the subjects of 
        expanded access applications under section 561 of the Federal 
        Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb); and
            (6) to develop and implement an ongoing mechanism to share 
        feedback and information and develop strategies with the 
        neurodegenerative disease community, including patients, 
        treating physicians, national organizations that facilitate 
        provision of care services, access, and research, researchers, 
        drug sponsors, drug manufacturers, and Federal agencies.

SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN.

    (a) In General.--Not later than 6 months after the date of the 
enactment of this Act, the Secretary of Health and Human Services shall 
publish an action plan describing actions the Department of Health and 
Human Services, through the National Institutes of Health and Food and 
Drug Administration, intend 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 the lives of people living with rare neurodegenerative 
        diseases as quickly as possible; and
            (2) facilitate access to investigational drugs.
    (b) Contents.--The action plan published under subsection (a) 
shall--
            (1) identify appropriate representation from within the 
        Food and Drug Administration and National Institutes of Health 
        to be responsible for implementation of such action plan; and
            (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 external biomedical community;
                    (B) consideration of cross-cutting clinical and 
                regulatory policy issues, including consistency of 
                regulatory advice and decision making;
                    (C) identification of key regulatory science and 
                policy issues critical to advancing development of safe 
                and effective drugs; and
                    (D) engagement by staff of the relevant centers of 
                the Food and Drug Administration and other relevant 
                offices of the Food and Drug Administration and 
                National Institutes of Health with the designated 
                leadership of the Collaborative.

SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PROGRAM.

    The Secretary of Health and Human Services, acting through the 
Commissioner of Food and Drugs, shall carry out a program of awarding 
grants to, and contracts entered into with, 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 life-threatening or severely debilitating 
neurodegenerative diseases, 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 advancing rational 
        therapeutic development and working to establish clinical trial 
        networks.

SEC. 6. 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.
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