Accelerating Access to Critical Therapies for ALS Act

II 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, 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 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 2 •S 4867 IS SEC. 2. GRANTS FOR RESEARCH ON AND EXPANDED AC- 1 CESS TO EXPERIMENTAL THERAPIES FOR 2 ALS. 3 (a) IN GENERAL.—The Secretary of Health and 4 Human Services shall award grants to eligible entities for 5 purposes of supporting research on, and expanded access 6 for individuals to, investigational drugs for the prevention, 7 diagnosis, mitigation, treatment, or cure of amyotrophic 8 lateral sclerosis pursuant to an expanded access request 9 submitted, and allowed to proceed by the Secretary, under 10 section 561 of the Federal Food, Drug, and Cosmetic Act 11 (21 U.S.C. 360bbb). 12 (b) APPLICATION.—An eligible entity seeking a grant 13 under this section shall submit to the Secretary an appli- 14 cation at such time, in such manner, and containing such 15 information as the Secretary shall specify. Such applica- 16 tion shall specify a research objective relating to expand- 17 ing access to investigational drugs (as described in sub- 18 section (a)) that would be supported by the award of such 19 grant. 20 (c) SELECTION.—Not later than 90 days after the 21 date of submission of an application for a grant under this 22 section, the Secretary shall determine whether to award 23 the grant, taking into consideration whether awarding 24 such grant will support a research objective relating to ex- 25 panding access to investigational drugs (as described in 26 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 3 •S 4867 IS subsection (a)), consistent with the mission of the Na- 1 tional Institutes of Health. 2 (d) USE OF FUNDS.—An eligible entity may use 3 funds received through the grant— 4 (1) to pay the manufacturer or sponsor for the 5 direct costs of such drug (as authorized under sec- 6 tion 312.8(d) of title 21, Code of Federal Regula- 7 tions (or successor regulations)), if such costs are 8 justified as part of peer review of the grant; 9 (2) for the entity’s direct costs incurred in pro- 10 viding such drug consistent with the research mis- 11 sion of the grant; or 12 (3) for the direct and indirect costs of the enti- 13 ty in conducting research with respect to the drug 14 involved. 15 (e) DEFINITIONS.—In this section: 16 (1) The term ‘‘eligible entity’’ means a partici- 17 pating clinical trial site or sites sponsored by a small 18 business concern (as defined in section 3(a) of the 19 Small Business Act (15 U.S.C. 632(a))) that is the 20 sponsor of a drug that is the subject of an investiga- 21 tional new drug application under section 505(i) of 22 the Federal Food, Drug, and Cosmetic Act (21 23 U.S.C. 355(i)). 24 VerDate Sep 11 2014 20:10 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 4 •S 4867 IS (2) The term ‘‘participating clinical trial’’ 1 means a phase 3 clinical trial conducted pursuant to 2 an exemption under section 505(i) of the Federal 3 Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or 4 section 351(a) of the Public Health Service Act (42 5 U.S.C. 262(a)) to investigate a drug intended to pre- 6 vent, diagnose, mitigate, treat, or cure amyotrophic 7 lateral sclerosis. 8 (3) The term ‘‘participating clinical trial site’’ 9 means a nonprofit or public health care facility, or 10 network of facilities, at which patients participating 11 in a participating clinical trial receive an investiga- 12 tional drug through such trial. 13 SEC. 3. HHS COLLABORATIVE FOR NEURODEGENERATIVE 14 DISEASES. 15 (a) ESTABLISHMENT.—Not later than one year after 16 the date of the enactment of this Act, the Secretary of 17 Health and Human Services shall establish and implement 18 a Collaborative for Neurodegenerative Diseases between 19 the National Institutes of Health and the Food and Drug 20 Administration (to be known and referred to in this sec- 21 tion as the ‘‘Collaborative’’), which shall— 22 (1) enter into a cooperative agreement, con- 23 tract, or other instrument with a private entity or 24 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 5 •S 4867 IS entities under which such private entity or entities 1 will operate the Collaborative; 2 (2) focus on advancing regulatory improvements 3 and scientific research that will support and accel- 4 erate the development and approval of drugs for pa- 5 tients with amyotrophic lateral sclerosis; 6 (3) foster the development of effective drugs 7 that improve the lives of people that suffer from rare 8 neurodegenerative diseases; and 9 (4) share information with the Secretary to ad- 10 vance the purposes specified in paragraph (3), such 11 as through carrying out the grant programs under 12 sections 2 and 5 and developing the action plan 13 under section 4. 14 (b) GIFTS.— 15 (1) IN GENERAL.—The Collaborative may so- 16 licit and accept gifts, grants, and other donations, 17 establish accounts, and invest and expend funds in 18 support of pre-competitive research and research as- 19 sociated with phase 3 and phase 4 clinical trials con- 20 ducted with respect to investigational drugs that are 21 the subjects of expanded access applications under 22 section 561 of the Federal Food, Drug, and Cos- 23 metic Act (21 U.S.C. 360bbb). 24 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 6 •S 4867 IS (2) USE.—In addition to any amounts appro- 1 priated for purposes of carrying out this section, the 2 Collaborative may use, without further appropria- 3 tion, any funds derived from a gift, grant, or other 4 donation accepted pursuant to paragraph (1). 5 (c) ADVISORY PANEL.— 6 (1) IN GENERAL.—The Collaborative shall con- 7 vene an advisory panel to conduct a review of the de- 8 sign and implementation of the programs authorized 9 by this Act. Such panel shall include representatives 10 of patients, treating physicians, national organiza- 11 tions that facilitate provision of care services, re- 12 searchers, drug sponsors, drug manufacturers, and 13 Federal agencies. 14 (2) REPORT.—The advisory panel convened 15 under paragraph (1) shall, not later than 18 months 16 after the date of the enactment of this Act, submit 17 to the Committee on Energy and Commerce of the 18 House of Representatives and the Committee on 19 Health, Education, Labor, and Pensions of the Sen- 20 ate a report that contains— 21 (A) the findings and conclusions of the re- 22 view conducted under paragraph (1); and 23 (B) recommendations for carrying out the 24 programs under this Act during the 2-year pe- 25 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 7 •S 4867 IS riod following the submission of such report, in- 1 cluding recommendations relating to the inclu- 2 sion of additional neurodegenerative diseases or 3 disease areas within the grant programs under 4 sections 2 and 5. 5 (d) DUTIES AND AUTHORITIES.—The Collaborative 6 shall identify and implement strategies for the Secretary— 7 (1) for purposes of expediting the approval of 8 drugs to treat amyotrophic lateral sclerosis, includ- 9 ing through coordination among the centers of the 10 Food and Drug Administration to achieve the goals 11 specified in the draft guidance for drug sponsors en- 12 titled ‘‘Amyotrophic Lateral Sclerosis: Developing 13 Drugs for Treatment Guidance for Industry’’ pub- 14 lished in September 2019; 15 (2) to facilitate access to investigational drugs 16 for amyotrophic lateral sclerosis pursuant to an ex- 17 panded access request under section 561 of the Fed- 18 eral Food, Drug, and Cosmetic Act (21 U.S.C. 19 360bbb) in a similar manner as investigational drugs 20 for cancer are provided through Project Facilitate of 21 the Center of Excellence for Oncology; 22 (3) with respect to the rare neurodegenerative 23 disease grant program established under section 5; 24 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 8 •S 4867 IS (4) to define or develop the regulatory and 1 translational pathway for emerging therapeutic cat- 2 egories; 3 (5) to share, within the Collaborative, findings 4 and insights related to pre-competitive research and 5 research associated with phase 3 and phase 4 clin- 6 ical trials conducted with respect to investigational 7 drugs that are the subjects of expanded access appli- 8 cations under section 561 of the Federal Food, 9 Drug, and Cosmetic Act (21 U.S.C. 360bbb); and 10 (6) to develop and implement an ongoing mech- 11 anism to share feedback and information and de- 12 velop strategies with the neurodegenerative disease 13 community, including patients, treating physicians, 14 national organizations that facilitate provision of 15 care services, access, and research, researchers, drug 16 sponsors, drug manufacturers, and Federal agencies. 17 SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN. 18 (a) IN GENERAL.—Not later than 6 months after the 19 date of the enactment of this Act, the Secretary of Health 20 and Human Services shall publish an action plan describ- 21 ing actions the Department of Health and Human Serv- 22 ices, through the National Institutes of Health and Food 23 and Drug Administration, intend to take during the 5-year 24 period following publication of the plan with respect to 25 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 9 •S 4867 IS program enhancements, policy development, regulatory 1 science initiatives, and other appropriate initiatives to— 2 (1) foster the development of safe and effective 3 drugs that improve the lives of people living with 4 rare neurodegenerative diseases as quickly as pos- 5 sible; and 6 (2) facilitate access to investigational drugs. 7 (b) CONTENTS.—The action plan published under 8 subsection (a) shall— 9 (1) identify appropriate representation from 10 within the Food and Drug Administration and Na- 11 tional Institutes of Health to be responsible for im- 12 plementation of such action plan; and 13 (2) include elements to facilitate— 14 (A) interactions and collaboration between 15 the Food and Drug Administration, including 16 the review centers thereof, and stakeholders in- 17 cluding patients, sponsors, and external bio- 18 medical community; 19 (B) consideration of cross-cutting clinical 20 and regulatory policy issues, including consist- 21 ency of regulatory advice and decision making; 22 (C) identification of key regulatory science 23 and policy issues critical to advancing develop- 24 ment of safe and effective drugs; and 25 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 10 •S 4867 IS (D) engagement by staff of the relevant 1 centers of the Food and Drug Administration 2 and other relevant offices of the Food and Drug 3 Administration and National Institutes of 4 Health with the designated leadership of the 5 Collaborative. 6 SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PRO- 7 GRAM. 8 The Secretary of Health and Human Services, acting 9 through the Commissioner of Food and Drugs, shall carry 10 out a program of awarding grants to, and contracts en- 11 tered into with, public and private entities to cover the 12 costs of research on and development of interventions in- 13 tended to prevent, diagnose, mitigate, treat, or cure 14 amyotrophic lateral sclerosis and other life-threatening or 15 severely debilitating neurodegenerative diseases, including 16 costs incurred with respect to the development and critical 17 evaluation of tools, methods, and processes— 18 (1) to characterize such neurodegenerative dis- 19 eases and their natural history; 20 (2) to identify molecular targets for such 21 neurodegenerative diseases; and 22 (3) to increase efficiency and productivity of 23 clinical development of therapies, including advanc- 24 VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS 11 •S 4867 IS ing rational therapeutic development and working to 1 establish clinical trial networks. 2 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 3 For purposes of carrying out this Act, there are au- 4 thorized to be appropriated $100,000,000 for each of fis- 5 cal years 2022 through 2026. 6 Æ VerDate Sep 11 2014 00:53 Nov 06, 2020 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6301 E:\BILLS\S4867.IS S4867 pamtmann on DSKBC07HB2PROD with BILLS