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

Summary:
Accelerating Access to Critical Therapies for ALS Act
This bill establishes a grant program, as well as a new center within the Food and Drug Administration (FDA), to facilitate access to investigational therapies for neurodegenerative diseases such as amyotrophic lateral sclerosis (i.e., ALS, also known as Lou Gehrig's disease, a progressive and fatal disease).
Specifically, the Department of Health and Human Services (HHS) must award grants for the provision of investigational drugs for ALS and other rapidly progressing neurodegenerative diseases through an expanded access (i.e., compassionate use) program. HHS may award these grants to (1) a small business that is the sponsor of an investigational new drug application, or (2) a participating clinical trial site for such a sponsor.
Additionally, HHS must establish within the FDA a Center of Excellence for Neurodegenerative Diseases. The center shall have duties and authorities similar to those of the FDA's Oncology Center of Excellence, which helps expedite the development of medical products and assists providers in requesting access to investigational drugs.

[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[H.R. 7071 Introduced in House (IH)]

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116th CONGRESS
  2d Session
                                H. R. 7071

To provide for the acceleration of access to clinical therapies for the 
  treatment of amyotrophic lateral sclerosis, and for other purposes.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                              June 1, 2020

Mr. Fortenberry (for himself and Mr. Quigley) introduced the following 
    bill; which was referred to the Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
To provide for the acceleration of access to clinical therapies for the 
  treatment of 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 RAPID DEVELOPMENT OF THERAPIES FOR ALS AND OTHER 
              RAPIDLY PROGRESSING NEURODEGENERATIVE DISEASES.

    (a) In General.--The Secretary of Health and Human Services shall 
award grants to eligible entities for the provision of investigational 
drugs through an expanded access program pursuant to section 561 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) for individuals 
for the prevention, diagnosis, mitigation, treatment, or cure of 
amyotrophic lateral sclerosis or another rapidly progressing 
neurodegenerative disease.
    (b) Vested Authority.--For purposes of development of an 
investigational drug pursuant to subsection (a), the Secretary may vest 
authority in the participating clinical trial site or sites to make the 
determination under subsection (b)(2), (c)(6), or (c)(7), as 
applicable, of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb).
    (c) Timing.--Not later than 60 days after the date of submission of 
an application for a grant under this section--
            (1) the Secretary, acting through the Director of the 
        National Institutes of Health, shall determine whether to award 
        the grant; and
            (2) the Secretary acting through the Commissioner of Food 
        and Drugs (or by vesting authority in the participating 
        clinical trial site, as applicable) shall make the 
        determinations required of the Secretary under subsection (b) 
        or (c), as applicable, of section 561 of the Federal Food, 
        Drug, and Cosmetic Act (21 U.S.C. 360bbb) for the provision of 
        the investigational drug to occur.
    (d) Definitions.--In this section:
            (1) The term ``Director'' means the Director of the 
        National Institutes of Health.
            (2) The term ``eligible entity'' means an entity that is--
                    (A) 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)); or
                    (B) a participating clinical trial site for such an 
                applicant.
            (3) The term ``participating clinical trial'' means a phase 
        2 or 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 treat amyotrophic lateral sclerosis or another rapidly 
        progressing neurodegenerative disease.
            (4) The term ``participating clinical trial site'' means a 
        health care facility at which patients participating in a 
        participating clinical trial receive treatment through such 
        trial.
            (5) The term ``Secretary'' means the Secretary of Health 
        and Human Services.
    (e) Funding.--
            (1) Authorization of appropriations.--There are authorized 
        to be appropriated to carry out this section--
                    (A) $75,000,000 for each of fiscal years 2021 and 
                2022; and
                    (B) $150,000,000 for each of fiscal years 2023 and 
                2024.
            (2) Gifts, grants, and other donations to foundation.--
                    (A) Acceptance.--Pursuant to section 499(c) of the 
                Public Health Service Act (42 U.S.C. 290b(c)), the 
                Foundation for the National Institutes of Health may 
                solicit and accept gifts, grants, and other donations, 
                establish accounts, and invest and expend funds in 
                support of carrying out this section.
                    (B) Use.--In addition to the amounts made available 
                pursuant to the authorizations of appropriations in 
                paragraph (1), the Director may use, without further 
                appropriation, any funds derived from a gift, grant, or 
                other donation accepted pursuant to subparagraph (A).
    (f) Review and Expansion.--Not later than 18 months after the date 
of the enactment of this Act--
            (1) the Secretary of Health and Human Services shall 
        convene an independent review panel that includes 
        representatives of patients, researchers, drug sponsors, and 
        government agencies; and
            (2) the independent review panel 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 on the findings and 
        conclusions of the panel with respect to the design and 
        implementation of the program under this section for 2023 and 
        2024.

SEC. 3. FDA CENTER OF EXCELLENCE FOR NEURODEGENERATIVE DISEASES.

    Chapter X of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
391 et seq.) is amended by adding at the end the following:

``SEC. 1015. CENTER OF EXCELLENCE FOR NEURODEGENERATIVE DISEASES.

    ``(a) Establishment.--Not later than September 2021, the Secretary 
shall establish within the Food and Drug Administration a center of 
excellence, to be known as the Center of Excellence for 
Neurodegenerative Diseases (in this section referred to as the `Center 
of Excellence').
    ``(b) Duties and Authorities.--The Center of Excellence shall have 
duties and authorities similar to those of the Center of Excellence for 
Oncology established under section 1014, including the duties and 
authorities of the Center of Excellence for Oncology with respect to 
Project Facilitate.''.
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