Accelerating Access to Critical Therapies for ALS Act

To provide for the acceleration of access to clinical therapies for the treatment of amyotrophic lateral sclerosis, and for other purposes. 1. Short title This Act may be cited as the Accelerating Access to Critical Therapies for ALS Act 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 (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 (d) Definitions In this section: (1) The term Director (2) The term eligible entity (A) a small business concern (as defined in section 3(a) of the Small Business Act ( 15 U.S.C. 632(a) 21 U.S.C. 355(i) (B) a participating clinical trial site for such an applicant (3) The term participating clinical trial 21 U.S.C. 355(i) 42 U.S.C. 262(a) (4) The term participating clinical trial site (5) The term Secretary (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) (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. 3. FDA Center of Excellence for Neurodegenerative Diseases Chapter X of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 391 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. .

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.