Accelerating Access to Critical Therapies for ALS Act

II 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, Edu- cation, Labor, and Pensions A BILL To direct the Secretary of Health and Human Services to support research on, and expanded access to, investiga- tional drugs for amyotrophic lateral sclerosis, and for other purposes. Be it enacted by the Senate and House of Representa- 1 tives of the United States of America in Congress assembled, 2 SECTION 1. SHORT TITLE. 3 This Act may be cited as the ‘‘Accelerating Access 4 to Critical Therapies for ALS Act’’. 5 SEC. 2. GRANTS FOR RESEARCH ON THERAPIES FOR ALS. 6 (a) IN GENERAL.—The Secretary of Health and 7 Human Services (referred to in this section as the Sec- 8 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 2 •S 1813 IS retary) shall award grants to participating entities for pur- 1 poses of expanded access for individuals to investigational 2 drugs for the prevention, diagnosis, mitigation, treatment, 3 or cure of amyotrophic lateral sclerosis. In the case of an 4 applicant seeking such a grant, an expanded access re- 5 quest must be submitted, and allowed to proceed by the 6 Secretary, under section 561 of the Federal Food, Drug, 7 and Cosmetic Act (21 U.S.C. 360bbb) and part 312 of 8 title 21, Code of Federal Regulations (or any successor 9 regulations), before the application for such grant is sub- 10 mitted. 11 (b) APPLICATION.— 12 (1) IN GENERAL.—A participating entity seek- 13 ing a grant under this section shall submit to the 14 Secretary an application at such time, in such man- 15 ner, and containing such information as the Sec- 16 retary shall specify. 17 (2) USE OF DATA.—An application submitted 18 under paragraph (1) shall include a description of 19 how data generated through an expanded access re- 20 quest under section 561 of the Federal Food, Drug, 21 and Cosmetic Act (21 U.S.C. 360bbb) with respect 22 to the investigational drug involved may be used by 23 the Secretary to support research or development re- 24 lated to the prevention, diagnosis, mitigation, treat- 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 3 •S 1813 IS ment, or cure of amyotrophic lateral sclerosis or 1 other rare neurodegenerative diseases. 2 (c) SELECTION.—Not later than 120 days after the 3 date of submission of an application for a grant under this 4 section, the Secretary shall determine whether to award 5 the grant, taking into consideration— 6 (1) whether awarding such grant will support a 7 research objective relating to expanding access to in- 8 vestigational drugs (as described in subsection (a)); 9 and 10 (2) whether awarding such a grant may have 11 the effect of diminishing eligibility for, or impeding 12 enrollment of, ongoing clinical investigations. 13 (d) USE OF FUNDS.—A participating entity may use 14 funds received through the grant— 15 (1) to pay the manufacturer or sponsor for the 16 direct costs of such drug (as authorized under sec- 17 tion 312.8(d) of title 21, Code of Federal Regula- 18 tions (or successor regulations)), if such costs are 19 justified as part of peer review of the grant; 20 (2) for the entity’s direct costs incurred in pro- 21 viding such drug consistent with the research mis- 22 sion of the grant; or 23 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 4 •S 1813 IS (3) for the direct and indirect costs of the enti- 1 ty in conducting research with respect to the drug 2 involved. 3 (e) DEFINITIONS.—In this section: 4 (1) The term ‘‘participating entity’’ means a 5 participating clinical trial site or sites sponsored by 6 a small business concern (as defined in section 3(a) 7 of the Small Business Act (15 U.S.C. 632(a)) that 8 is the sponsor of a drug that is the subject of an in- 9 vestigational new drug application under section 10 505(i) of the Federal Food, Drug, and Cosmetic Act 11 (21 U.S.C. 355(i)). 12 (2) The term ‘‘participating clinical trial’’ 13 means a phase 3 clinical trial conducted pursuant to 14 an exemption under section 505(i) of the Federal 15 Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or 16 section 351(a) of the Public Health Service Act (42 17 U.S.C. 262(a)) to investigate a drug intended to pre- 18 vent, diagnose, mitigate, treat, or cure amyotrophic 19 lateral sclerosis. 20 (3) The term ‘‘participating clinical trial site’’ 21 means a nonprofit or public health care facility, or 22 network of facilities, at which patients participating 23 in a participating clinical trial receive an investiga- 24 tional drug through such trial. 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 5 •S 1813 IS SEC. 3. HHS PUBLIC-PRIVATE PARTNERSHIP FOR RARE 1 NEURODEGENERATIVE DISEASES. 2 (a) ESTABLISHMENT.—Not later than one year after 3 the date of enactment of this Act, the Secretary of Health 4 and Human Services (referred to in this section as the 5 ‘‘Secretary’’) shall establish and implement a Public-Pri- 6 vate Partnership for Neurodegenerative Diseases between 7 the National Institutes of Health, the Food and Drug Ad- 8 ministration, and one or more eligible entities (to be 9 known and referred to in this section as the ‘‘Partner- 10 ship’’) through cooperative agreements, contracts, or other 11 appropriate instruments with such eligible entities, for the 12 purpose of developing treatments for amytrophic lateral 13 sclerosis and other rare neurodegenerative diseases. The 14 Partnership shall— 15 (1) establish partnerships, consortia, and col- 16 laborations with other public and private entities 17 and individuals with expertise in amyotrophic lateral 18 sclerosis and other rare neurodegenerative diseases 19 for the purposes described in this subsection; 20 (2) focus on advancing regulatory science and 21 scientific research that will support and accelerate 22 the development and review of drugs for patients 23 with amyotrophic lateral sclerosis and other rare 24 neurodegenerative diseases; and 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 6 •S 1813 IS (3) foster the development of effective drugs 1 that improve the lives of people that suffer from 2 amyotrophic lateral sclerosis and other rare 3 neurodegenerative diseases. 4 (b) ELIGIBLE ENTITY.—In this section, the term ‘‘el- 5 igible entity’’ means an entity that— 6 (1) is— 7 (A) an institution of higher education (as 8 such term is defined in section 1001 of the 9 Higher Education Act of 1965 (20 U.S.C. 10 1001)) or a consortium of such institutions; or 11 (B) an organization described in section 12 501(c)(3) of the Internal Revenue Code of 1986 13 and exempt from tax under subsection (a) of 14 such section; 15 (2) has experienced personnel and demonstrated 16 connection to the patient population; 17 (3) demonstrates to the Secretary’s satisfaction 18 that the entity is capable of identifying and estab- 19 lishing collaborations between public and private en- 20 tities and individuals with expertise in 21 neurodegenerative diseases, including patients, in 22 order to facilitate— 23 (A) development and critical evaluation of 24 tools, methods, and processes— 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 7 •S 1813 IS (i) to characterize neurodegenerative 1 diseases and their natural history; 2 (ii) to identify drug targets for 3 neurodegenerative diseases; and 4 (iii) to increase efficiency, predict- 5 ability, and productivity of clinical develop- 6 ment of therapies, including advancement 7 of rational therapeutic development and es- 8 tablishment of clinical trial networks; and 9 (B) securing funding for the Partnership 10 from Federal and non-Federal governmental 11 sources, foundations, and private individuals; 12 and 13 (4) provides an assurance that the entity will 14 not accept funding for a Partnership project from 15 any organization that manufactures or distributes 16 products regulated by the Food and Drug Adminis- 17 tration unless the entity provides assurances in its 18 agreement with the Secretary that the results of the 19 project will not be influenced by any source of fund- 20 ing. 21 (c) GIFTS.— 22 (1) IN GENERAL.—The Partnership may solicit 23 and accept gifts, grants, and other donations, estab- 24 lish accounts, and invest and expend funds in sup- 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 8 •S 1813 IS port of pre-competitive research and research associ- 1 ated with phase 3 clinical trials conducted with re- 2 spect to investigational drugs that are the subjects 3 of expanded access applications under section 561 of 4 the Federal Food, Drug, and Cosmetic Act (21 5 U.S.C. 360bbb). 6 (2) USE.—In addition to any amounts appro- 7 priated for purposes of carrying out this section, the 8 Partnership may use, without further appropriation, 9 any funds derived from a gift, grant, or other dona- 10 tion accepted pursuant to paragraph (1). 11 SEC. 4. ALS AND OTHER RARE NEURODEGENERATIVE DIS- 12 EASE ACTION PLAN. 13 (a) IN GENERAL.—Not later than 6 months after the 14 date of enactment of this Act, the Secretary of Health and 15 Human Services shall publish on the website of the De- 16 partment of Health and Human Services an action plan 17 describing actions the Food and Drug Administration in- 18 tends to take during the 5-year period following publica- 19 tion of the plan with respect to program enhancements, 20 policy development, regulatory science initiatives, and 21 other appropriate initiatives to— 22 (1) foster the development of safe and effective 23 drugs that improve or extend, or both, the lives of 24 people living with amyotrophic lateral sclerosis and 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 9 •S 1813 IS other rare neurodegenerative diseases as quickly as 1 possible; and 2 (2) facilitate access to investigational drugs for 3 amyotrophic lateral sclerosis and other rare 4 neurodegenerative diseases. 5 (b) CONTENTS.—The initial action plan published 6 under subsection (a) shall— 7 (1) identify appropriate representation from 8 within the Food and Drug Administration to be re- 9 sponsible for implementation of such action plan; 10 (2) include elements to facilitate— 11 (A) interactions and collaboration between 12 the Food and Drug Administration, including 13 the review centers thereof, and stakeholders in- 14 cluding patients, sponsors, and the external bio- 15 medical research community; 16 (B) consideration of cross-cutting clinical 17 and regulatory policy issues, including consist- 18 ency of regulatory advice and decision making; 19 (C) identification of key regulatory science 20 and policy issues critical to advancing develop- 21 ment of safe and effective drugs; and 22 (D) enhancement of collaboration and en- 23 gagement by staff of the relevant centers of the 24 Food and Drug Administration and other rel- 25 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 10 •S 1813 IS evant offices of the Food and Drug Administra- 1 tion with other operating divisions within the 2 Department of Health and Human Services, the 3 Partnership, and the broader neurodegenerative 4 disease community; and 5 (3) be subject to revision, as determined appro- 6 priate by the Secretary of Health and Human Serv- 7 ices. 8 SEC. 5. FDA RARE NEURODEGENERATIVE DISEASE GRANT 9 PROGRAM. 10 The Secretary of Health and Human Services shall 11 use funds made available under section 6 to award grants 12 and contracts to public and private entities to cover the 13 costs of research on, and development of interventions in- 14 tended to prevent, diagnose, mitigate, treat, or cure, 15 amyotrophic lateral sclerosis and other rare life-threat- 16 ening or severely debilitating neurodegenerative diseases 17 in adults and children, including costs incurred with re- 18 spect to the development and critical evaluation of tools, 19 methods, and processes— 20 (1) to characterize such neurodegenerative dis- 21 eases and their natural history; 22 (2) to identify molecular targets for such 23 neurodegenerative diseases; and 24 VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS 11 •S 1813 IS (3) to increase efficiency and productivity of 1 clinical development of therapies, including advanc- 2 ing rational therapeutic development and working to 3 establish new or leverage existing clinical trial net- 4 works. 5 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 6 For purposes of carrying out this Act, there are au- 7 thorized to be appropriated $100,000,000 for each of fis- 8 cal years 2022 through 2026. 9 Æ VerDate Sep 11 2014 04:27 Jun 12, 2021 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6301 E:\BILLS\S1813.IS S1813 pbinns on DSKJLVW7X2PROD with BILLS