Accelerating Access to Critical Therapies for ALS Act

I 116TH CONGRESS 2D SESSION H. R. 8662 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 HOUSE OF REPRESENTATIVES OCTOBER 23, 2020 Mr. FORTENBERRY (for himself, Mr. QUIGLEY, Mr. KING of New York, Mr. DIAZ-BALART, Mr. KEVIN HERN of Oklahoma, Mr. KATKO, Mr. GIANFORTE, Mr. HAGEDORN, Mr. BACON, Mr. FLEISCHMANN, Mrs. ROD- GERS of Washington, Mr. WESTERMAN, Mr. SMITH of Nebraska, Mr. BIGGS, Mr. MCKINLEY, Mr. SCHWEIKERT, Mr. UPTON, Mr. RUTHER- FORD, Mr. MULLIN, Mr. KING of Iowa, Mr. AMODEI, Mr. MARSHALL, Mr. STIVERS, Mr. TIMMONS, Ms. VELA´ZQUEZ, Ms. NORTON, Mr. COSTA, Mr. RUSH, Mr. ROUDA, Ms. SEWELL of Alabama, Mrs. BEATTY, Mr. O’HALLERAN, Mr. CISNEROS, Mr. LOWENTHAL, Mr. DAVID SCOTT of Georgia, Mr. CALVERT, Mr. WELCH, Mr. PETERSON, Mrs. DEMINGS, Ms. SCHAKOWSKY, Mr. SCHIFF, and Ms. BARRAGA´N) introduced the following bill; which was referred to the Committee on Energy and Commerce 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 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 2 •HR 8662 IH SECTION 1. SHORT TITLE. 1 This Act may be cited as the ‘‘Accelerating Access 2 to Critical Therapies for ALS Act’’. 3 SEC. 2. GRANTS FOR RESEARCH ON AND EXPANDED AC- 4 CESS TO EXPERIMENTAL THERAPIES FOR 5 ALS. 6 (a) IN GENERAL.—The Secretary of Health and 7 Human Services shall award grants to eligible entities for 8 purposes of supporting research on, and expanded access 9 for individuals to, investigational drugs for the prevention, 10 diagnosis, mitigation, treatment, or cure of amyotrophic 11 lateral sclerosis pursuant to an expanded access request 12 submitted, and allowed to proceed by the Secretary, under 13 section 561 of the Federal Food, Drug, and Cosmetic Act 14 (21 U.S.C. 360bbb). 15 (b) APPLICATION.—An eligible entity seeking a grant 16 under this section shall submit to the Secretary an appli- 17 cation at such time, in such manner, and containing such 18 information as the Secretary shall specify. Such applica- 19 tion shall specify a research objective relating to expand- 20 ing access to investigational drugs (as described in sub- 21 section (a)) that would be supported by the award of such 22 grant. 23 (c) SELECTION.—Not later than 90 days after the 24 date of submission of an application for a grant under this 25 section, the Secretary shall determine whether to award 26 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 3 •HR 8662 IH the grant, taking into consideration whether awarding 1 such grant will support a research objective relating to ex- 2 panding access to investigational drugs (as described in 3 subsection (a)), consistent with the mission of the Na- 4 tional Institutes of Health. 5 (d) USE OF FUNDS.—An eligible entity may use 6 funds received through the grant— 7 (1) to pay the manufacturer or sponsor for the 8 direct costs of such drug (as authorized under sec- 9 tion 312.8(d) of title 21, Code of Federal Regula- 10 tions (or successor regulations)), if such costs are 11 justified as part of peer review of the grant; 12 (2) for the entity’s direct costs incurred in pro- 13 viding such drug consistent with the research mis- 14 sion of the grant; or 15 (3) for the direct and indirect costs of the enti- 16 ty in conducting research with respect to the drug 17 involved. 18 (e) DEFINITIONS.—In this section: 19 (1) The term ‘‘eligible entity’’ means a partici- 20 pating clinical trial site or sites sponsored by a small 21 business concern (as defined in section 3(a) of the 22 Small Business Act (15 U.S.C. 632(a)) that is the 23 sponsor of a drug that is the subject of an investiga- 24 tional new drug application under section 505(i) of 25 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 4 •HR 8662 IH the Federal Food, Drug, and Cosmetic Act (21 1 U.S.C. 355(i)). 2 (2) The term ‘‘participating clinical trial’’ 3 means a phase 3 clinical trial conducted pursuant to 4 an exemption under section 505(i) of the Federal 5 Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or 6 section 351(a) of the Public Health Service Act (42 7 U.S.C. 262(a)) to investigate a drug intended to pre- 8 vent, diagnose, mitigate, treat, or cure amyotrophic 9 lateral sclerosis. 10 (3) The term ‘‘participating clinical trial site’’ 11 means a nonprofit or public health care facility, or 12 network of facilities, at which patients participating 13 in a participating clinical trial receive an investiga- 14 tional drug through such trial. 15 SEC. 3. HHS COLLABORATIVE FOR NEURODEGENERATIVE 16 DISEASES. 17 (a) ESTABLISHMENT.—Not later than one year after 18 the date of the enactment of this Act, the Secretary of 19 Health and Human Services shall establish and implement 20 a Collaborative for Neurodegenerative Diseases between 21 the National Institutes of Health and the Food and Drug 22 Administration (to be known and referred to in this sec- 23 tion as the ‘‘Collaborative’’), which shall— 24 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 5 •HR 8662 IH (1) enter into a cooperative agreement, con- 1 tract, or other instrument with a private entity or 2 entities under which such private entity or entities 3 will operate the Collaborative; 4 (2) focus on advancing regulatory improvements 5 and scientific research that will support and accel- 6 erate the development and approval of drugs for pa- 7 tients with amyotrophic lateral sclerosis; 8 (3) foster the development of effective drugs 9 that improve the lives of people that suffer from rare 10 neurodegenerative diseases; and 11 (4) share information with the Secretary to ad- 12 vance the purposes specified in paragraph (3), such 13 as through carrying out the grant programs under 14 sections 2 and 5 and developing the action plan 15 under section 4. 16 (b) GIFTS.— 17 (1) IN GENERAL.—The Collaborative may so- 18 licit and accept gifts, grants, and other donations, 19 establish accounts, and invest and expend funds in 20 support of pre-competitive research and research as- 21 sociated with phase 3 and phase 4 clinical trials con- 22 ducted with respect to investigational drugs that are 23 the subjects of expanded access applications under 24 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 6 •HR 8662 IH section 561 of the Federal Food, Drug, and Cos- 1 metic Act (21 U.S.C. 360bbb). 2 (2) USE.—In addition to any amounts appro- 3 priated for purposes of carrying out this section, the 4 Collaborative may use, without further appropria- 5 tion, any funds derived from a gift, grant, or other 6 donation accepted pursuant to paragraph (1). 7 (c) ADVISORY PANEL.— 8 (1) IN GENERAL.—The Collaborative shall con- 9 vene an advisory panel to conduct a review of the de- 10 sign and implementation of the programs authorized 11 by this Act. Such panel shall include representatives 12 of patients, treating physicians, national organiza- 13 tions that facilitate provision of care services, re- 14 searchers, drug sponsors, drug manufacturers, and 15 Federal agencies. 16 (2) REPORT.—The advisory panel convened 17 under paragraph (1) shall, not later than 18 months 18 after the date of the enactment of this Act, submit 19 to the Committee on Energy and Commerce of the 20 House of Representatives and the Committee on 21 Health, Education, Labor, and Pensions of the Sen- 22 ate a report that contains— 23 (A) the findings and conclusions of the re- 24 view conducted under paragraph (1); and 25 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 7 •HR 8662 IH (B) recommendations for carrying out the 1 programs under this Act during the 2-year pe- 2 riod following the submission of such report, in- 3 cluding recommendations relating to the inclu- 4 sion of additional neurodegenerative diseases or 5 disease areas within the grant programs under 6 sections 2 and 5. 7 (d) DUTIES AND AUTHORITIES.—The Collaborative 8 shall identify and implement strategies for the Secretary— 9 (1) for purposes of expediting the approval of 10 drugs to treat amyotrophic lateral sclerosis, includ- 11 ing through coordination among the centers of the 12 Food and Drug Administration to achieve the goals 13 specified in the draft guidance for drug sponsors en- 14 titled ‘‘Amyotrophic Lateral Sclerosis: Developing 15 Drugs for Treatment Guidance for Industry’’ pub- 16 lished in September 2019; 17 (2) to facilitate access to investigational drugs 18 for amyotrophic lateral sclerosis pursuant to an ex- 19 panded access request under section 561 of the Fed- 20 eral Food, Drug, and Cosmetic Act (21 U.S.C. 21 360bbb) in a similar manner as investigational drugs 22 for cancer are provided through Project Facilitate of 23 the Center of Excellence for Oncology; 24 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 8 •HR 8662 IH (3) with respect to the rare neurodegenerative 1 disease grant program established under section 5; 2 (4) to define or develop the regulatory and 3 translational pathway for emerging therapeutic cat- 4 egories; 5 (5) to share, within the Collaborative, findings 6 and insights related to pre-competitive research and 7 research associated with phase 3 and phase 4 clin- 8 ical trials conducted with respect to investigational 9 drugs that are the subjects of expanded access appli- 10 cations under section 561 of the Federal Food, 11 Drug, and Cosmetic Act (21 U.S.C. 360bbb); and 12 (6) to develop and implement an ongoing mech- 13 anism to share feedback and information and de- 14 velop strategies with the neurodegenerative disease 15 community, including patients, treating physicians, 16 national organizations that facilitate provision of 17 care services, access, and research, researchers, drug 18 sponsors, drug manufacturers, and Federal agencies. 19 SEC. 4. NEURODEGENERATIVE DISEASE ACTION PLAN. 20 (a) IN GENERAL.—Not later than 6 months after the 21 date of the enactment of this Act, the Secretary of Health 22 and Human Services shall publish an action plan describ- 23 ing actions the Department of Health and Human Serv- 24 ices, through the National Institutes of Health and Food 25 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 9 •HR 8662 IH and Drug Administration, intend to take during the 5-year 1 period following publication of the plan with respect to 2 program enhancements, policy development, regulatory 3 science initiatives, and other appropriate initiatives to— 4 (1) foster the development of safe and effective 5 drugs that improve the lives of people living with 6 rare neurodegenerative diseases as quickly as pos- 7 sible; and 8 (2) facilitate access to investigational drugs. 9 (b) CONTENTS.—The action plan published under 10 subsection (a) shall— 11 (1) identify appropriate representation from 12 within the Food and Drug Administration and Na- 13 tional Institutes of Health to be responsible for im- 14 plementation of such action plan; and 15 (2) include elements to facilitate— 16 (A) interactions and collaboration between 17 the Food and Drug Administration, including 18 the review centers thereof, and stakeholders in- 19 cluding patients, sponsors, and external bio- 20 medical community; 21 (B) consideration of cross-cutting clinical 22 and regulatory policy issues, including consist- 23 ency of regulatory advice and decisionmaking; 24 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 10 •HR 8662 IH (C) identification of key regulatory science 1 and policy issues critical to advancing develop- 2 ment of safe and effective drugs; and 3 (D) engagement by staff of the relevant 4 centers of the Food and Drug Administration 5 and other relevant offices of the Food and Drug 6 Administration and National Institutes of 7 Health with the designated leadership of the 8 Collaborative. 9 SEC. 5. RARE NEURODEGENERATIVE DISEASE GRANT PRO- 10 GRAM. 11 The Secretary of Health and Human Services, acting 12 through the Commissioner of Food and Drugs, shall carry 13 out a program of awarding grants to, and contracts en- 14 tered into with, public and private entities to cover the 15 costs of research on and development of interventions in- 16 tended to prevent, diagnose, mitigate, treat, or cure 17 amyotrophic lateral sclerosis and other life-threatening or 18 severely debilitating neurodegenerative diseases, including 19 costs incurred with respect to the development and critical 20 evaluation of tools, methods, and processes— 21 (1) to characterize such neurodegenerative dis- 22 eases and their natural history; 23 (2) to identify molecular targets for such 24 neurodegenerative diseases; and 25 VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS 11 •HR 8662 IH (3) to increase efficiency and productivity of 1 clinical development of therapies, including advanc- 2 ing rational therapeutic development and working to 3 establish clinical trial networks. 4 SEC. 6. AUTHORIZATION OF APPROPRIATIONS. 5 For purposes of carrying out this Act, there are au- 6 thorized to be appropriated $100,000,000 for each of fis- 7 cal years 2022 through 2026. 8 Æ VerDate Sep 11 2014 21:54 Nov 13, 2020 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6301 E:\BILLS\H8662.IH H8662 jbell on DSKJLSW7X2PROD with BILLS