Title: Accelerating Access to Critical Therapies for ALS Act
Policy area: Health

Summary:
Accelerating Access to Critical Therapies for ALS Act
This bill establishes grant programs to address neurodegenerative diseases, such as amyotrophic lateral sclerosis (also known as ALS or Lou Gehrig's disease), and contains other related provisions.
The Department of Health and Human Services (HHS) shall award grants to eligible entities to facilitate patients' access to investigational drugs that diagnose or treat ALS. The Food and Drug Administration (FDA) shall award grants to public and private entities to cover the costs of research and development of drugs that diagnose or treat ALS and other severely debilitating neurodegenerative diseases.
HHS shall also establish the Public-Private Partnership for Neurodegenerative Diseases between the National Institutes of Health, the FDA, and at least one eligible entity (generally, an institution of higher education or a nonprofit organization). The partnership shall support the development and regulatory review of drugs that address ALS and other rare neurodegenerative diseases.
The FDA shall publish on its website a five-year action plan for fostering the development of drugs that improve or extend the lives of people living with rare neurodegenerative diseases.

[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 1813 Introduced in Senate (IS)]

<DOC>






117th CONGRESS
  1st Session
                                S. 1813

    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

                              May 25, 2021

  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 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 expanded access for individuals 
to investigational drugs for the prevention, diagnosis, mitigation, 
treatment, or cure of amyotrophic lateral sclerosis. In the case of an 
applicant 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 may be used by the Secretary 
        to support research or development related to the prevention, 
        diagnosis, mitigation, treatment, or cure of amyotrophic 
        lateral sclerosis or other rare neurodegenerative diseases.
    (c) Selection.--Not later than 120 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--
            (1) whether awarding such grant will support a research 
        objective relating to expanding access to investigational drugs 
        (as described in subsection (a)); and
            (2) whether awarding such a grant may have the effect of 
        diminishing eligibility for, or impeding enrollment of, ongoing 
        clinical investigations.
    (d) Use of Funds.--A participating 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 ``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)).
            (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 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 instruments with such 
eligible entities, for the purpose of developing treatments for 
amytrophic lateral sclerosis and other rare neurodegenerative diseases. 
The Partnership shall--
            (1) establish partnerships, consortia, and collaborations 
        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 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 drug 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 pre-competitive research 
        and research associated with phase 3 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 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 Secretary of Health and Human Services shall 
publish on the website of the Department of Health and Human Services 
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 as quickly as possible; 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 decision making;
                    (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 by 
                staff of the relevant centers of the Food and Drug 
                Administration and other relevant 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 shall use funds made 
available under section 6 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, 
amyotrophic lateral sclerosis and other rare life-threatening or 
severely debilitating 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 advancing rational 
        therapeutic development and working to establish new or 
        leverage existing 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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