Union Calendar No. 148
117th CONGRESS 1st Session |
[Report No. 117–207]
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.
May 25, 2021
Mr. Quigley (for himself, Mr. Fortenberry, Mr. Brendan F. Boyle of Pennsylvania, Mr. Moulton, Mr. Garcia of California, Mr. Carbajal, Mr. Larson of Connecticut, Ms. Jackson Lee, Ms. Dean, Mr. Suozzi, Ms. Velázquez, Mr. Levin of California, Mr. Deutch, Ms. Norton, Mr. Timmons, Mr. Brady, Mr. McKinley, Mr. Van Drew, Mr. Calvert, Mr. Keating, Mr. Diaz-Balart, Mr. Carter of Georgia, Mrs. McBath, Mr. Smith of Missouri, Mr. Turner, Mr. Duncan, Mr. Hice of Georgia, Mr. Young, Mr. Smith of Nebraska, Mr. Grothman, Mr. Ruppersberger, Mr. Rutherford, Mr. Schweikert, Mr. Ryan, Mr. Crow, Mr. Guthrie, Mr. Fitzpatrick, Ms. McCollum, Mr. Austin Scott of Georgia, Mr. Baird, Mr. Rodney Davis of Illinois, Mr. Valadao, Mr. Moolenaar, Mr. Malinowski, Ms. Roybal-Allard, Mr. Payne, Mr. Lynch, Ms. Herrera Beutler, Mr. Buck, Mr. Mullin, Mr. Grijalva, Mr. Cooper, Mr. Panetta, Mr. Kim of New Jersey, Mr. Sires, Ms. Lee of California, Ms. Moore of Wisconsin, Ms. Schakowsky, Mr. Thompson of California, Mr. Gallego, Mrs. Axne, Mrs. Napolitano, Mr. Espaillat, Ms. Pressley, Mr. Fleischmann, Mr. Reschenthaler, Mr. Cicilline, Ms. DeGette, Mr. Burchett, Mr. LaMalfa, Ms. Meng, Ms. Brownley, Mr. Trone, Ms. Kuster, Mr. Connolly, Mr. Meeks, Mrs. Kirkpatrick, Mrs. Demings, Mr. O'Halleran, Mr. Lieu, Mr. DeSaulnier, Mr. Garamendi, Mr. Kilmer, Mr. Rush, Mr. McCaul, Mr. McClintock, Mr. Mfume, Mr. Lamb, Mr. Green of Texas, Mr. Swalwell, Mr. Gottheimer, Ms. Pingree, Ms. Kaptur, Mr. Ferguson, Ms. Scanlon, Mr. Bacon, Mr. Wittman, Mr. Morelle, Mr. Amodei, and Mr. Waltz) introduced the following bill; which was referred to the Committee on Energy and Commerce
December 8, 2021
Additional sponsors: Ms. Sewell, Ms. Sherrill, Mr. Posey, Mr. Cole, Mr. Curtis, Mr. Carson, Mr. Gonzalez of Ohio, Ms. Clark of Massachusetts, Ms. Houlahan, Ms. Newman, Mr. Smith of New Jersey, Mr. LaHood, Mr. Costa, Mr. Sherman, Mr. Chabot, Mr. Aguilar, Mrs. Beatty, Ms. Wasserman Schultz, Ms. Davids of Kansas, Mr. Hill, Mrs. Carolyn B. Maloney of New York, Mr. Vicente Gonzalez of Texas, Ms. Wexton, Mr. Huffman, Mr. Tonko, Mrs. Luria, Mr. Bishop of Georgia, Mr. Correa, Mr. Katko, Mr. Foster, Ms. Wild, Mr. Womack, Ms. Ross, Ms. Lois Frankel of Florida, Mr. Keller, Mr. Stewart, Mr. Brooks, Mr. Auchincloss, Mr. Welch, Mr. Wilson of South Carolina, Ms. Salazar, Mrs. Trahan, Ms. Stefanik, Ms. Matsui, Mr. Cartwright, Mr. Yarmuth, Mr. Schneider, Mr. Delgado, Mr. Westerman, Mr. García of Illinois, Ms. Manning, Ms. Porter, Mr. Crawford, Mr. DeFazio, Mr. Langevin, Miss Rice of New York, Mr. Golden, Mr. Sessions, Mr. Harder of California, Mr. Lowenthal, Mr. Soto, Mr. Raskin, Mr. Joyce of Ohio, Mr. Lamborn, Mr. Johnson of Georgia, Ms. Barragán, Mr. Dunn, Mr. Courtney, Mr. Carter of Louisiana, Mr. Lucas, Mr. Emmer, Mr. Graves of Missouri, Ms. Omar, Mr. Peters, Ms. Craig, Mr. Danny K. Davis of Illinois, Mr. Bera, Mr. Bilirakis, Ms. Strickland, Mr. Gibbs, Mr. Meuser, Mr. Higgins of New York, Mr. Gallagher, Mr. Lawson of Florida, Mr. Owens, Mrs. Miller-Meeks, Mr. Cárdenas, Mr. Wenstrup, Ms. Jacobs of California, Mr. Perlmutter, Mrs. Hartzler, Mr. Stauber, Mr. Krishnamoorthi, Mr. Kildee, Ms. Slotkin, Mr. Sarbanes, Mr. Griffith, Ms. Spanberger, Mr. McGovern, Mr. Khanna, Mr. Mann, Mr. Biggs, Mr. Sean Patrick Maloney of New York, Mr. Thompson of Pennsylvania, Ms. DelBene, Mr. Nadler, Ms. Sánchez, Mr. McNerney, Mrs. Watson Coleman, Mrs. Fletcher, Mr. Mast, Mr. Stanton, Mr. Smith of Washington, Mr. Weber of Texas, Ms. Kelly of Illinois, Mr. Jones, Mr. Neguse, Mr. Kahele, Mr. Himes, Mr. Huizenga, Mr. Higgins of Louisiana, Mr. Bergman, Mr. Balderson, Mr. David Scott of Georgia, Mr. Aderholt, Mr. Armstrong, Mr. DesJarlais, Mr. Moore of Alabama, Mr. Schiff, Mr. Thompson of Mississippi, Mrs. Kim of California, Mr. Feenstra, Ms. Adams, Mr. Kelly of Mississippi, Mr. Allred, Mr. Cohen, Mr. Brown of Maryland, Mr. Johnson of Ohio, Mrs. Murphy of Florida, Mr. Luetkemeyer, Ms. Williams of Georgia, Mr. Gosar, Mr. Tiffany, Mr. Casten, Ms. Cheney, Mrs. Hayes, Mr. Jeffries, Mr. Smucker, Ms. Lofgren, Mr. Hern, Ms. Tenney, Ms. Bonamici, Mrs. Steel, Mr. Garbarino, Mr. Norcross, Ms. Escobar, Mrs. Cammack, Mr. Gomez, Mr. Torres of New York, Mr. Bowman, Ms. Tlaib, Ms. Bass, Ms. Wilson of Florida, Ms. Clarke of New York, Mr. Levin of Michigan, Ms. Stevens, Ms. Leger Fernandez, Ms. Waters, Mr. Kind, Mr. Cuellar, Mr. Phillips, Mr. Hagedorn, Mr. Palazzo, Ms. Blunt Rochester, Mr. Moore of Utah, Mr. Blumenauer, Ms. Granger, Ms. Jayapal, Mr. Horsford, Mr. Cline, Ms. Speier, Mr. Carl, Mr. Cawthorn, Mr. Murphy of North Carolina, Mr. Obernolte, Mr. Kelly of Pennsylvania, Mr. Scalise, Ms. Malliotakis, Mr. Evans, Mr. Zeldin, Mr. Simpson, Mr. Steube, Mr. Johnson of Louisiana, Mr. Loudermilk, Mrs. Hinson, Mr. LaTurner, Ms. Bourdeaux, Ms. Johnson of Texas, Mr. Case, Mr. Jacobs of New York, Mrs. Bice of Oklahoma, Mrs. Wagner, Mr. Gooden of Texas, Mr. Harris, Mr. Jackson, Mr. Mooney, Mr. Burgess, Ms. Letlow, Mr. Upton, Ms. Schrier, Mr. Butterfield, Mr. Bentz, Mrs. Fischbach, Mr. Graves of Louisiana, Mr. Walberg, Mr. Good of Virginia, Mrs. Torres of California, Mr. Pappas, Mrs. McClain, Mr. Guest, Mr. C. Scott Franklin of Florida, Mr. Gohmert, Mr. Rouzer, Mr. Bost, Mr. Williams of Texas, Mr. Banks, Mrs. Lawrence, Mr. Crist, Ms. Castor of Florida, Ms. Eshoo, Mr. Crenshaw, and Mr. Newhouse
December 8, 2021
Reported with an amendment, committed to the Committee of the Whole House on the State of the Union, and ordered to be printed
[Strike out all after the enacting clause and insert the part printed in italic]
[For text of introduced bill, see copy of bill as introduced on May 25, 2021]
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.
Be it enacted by the Senate and House of Representatives of the United States of America in Congress assembled,
This Act may be cited as the “Accelerating Access to Critical Therapies for ALS Act”.
SEC. 2. Grants for research on therapies for ALS.
(a) In general.—The Secretary of Health and Human Services (referred to in this section as the “Secretary”) shall award grants to participating entities for purposes of scientific research utilizing data from expanded access to investigational drugs for individuals who are not otherwise eligible for clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis. In the case of a participating entity seeking such a grant, an expanded access request must be submitted, and allowed to proceed by the Secretary, under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) and part 312 of title 21, Code of Federal Regulations (or any successor regulations), before the application for such grant is submitted.
(b) Application.—
(1) IN GENERAL.—A participating entity seeking a grant under this section shall submit to the Secretary an application at such time, in such manner, and containing such information as the Secretary shall specify.
(2) USE OF DATA.—An application submitted under paragraph (1) shall include a description of how data generated through an expanded access request under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) with respect to the investigational drug involved will be used to support research or development related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis.
(3) NONINTERFERENCE WITH CLINICAL TRIALS.—An application submitted under paragraph (1) shall include a description of how the proposed expanded access program will be designed so as not to interfere with patient enrollment in ongoing clinical trials for investigational therapies for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis.
(c) Selection.—Consistent with sections 406 and 492 of the Public Health Service Act (42 U.S.C. 284a, 289a), the Secretary shall, in determining whether to award a grant under this section, confirm that—
(1) such grant will be used to support a scientific research objective relating to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis (as described in subsection (a));
(2) such grant shall not have the effect of diminishing eligibility for, or impeding enrollment of, ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis by determining that individuals who receive expanded access to investigational drugs through such a grant are not eligible for enrollment in—
(A) ongoing clinical trials that are registered on ClinicalTrials.gov (or successor website), with respect to a drug for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis; or
(B) clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis for which an exemption under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) has been granted by the Food and Drug Administration and which are expected to begin enrollment within one year; and
(d) Use of funds.—A participating entity shall use funds received through the grant—
(1) to pay the manufacturer or sponsor for the direct costs of the investigational drug, as authorized under section 312.8(d) of title 21, Code of Federal Regulations (or successor regulations), to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis that is the subject of an expanded access request described in subsection (a), if such costs are justified as part of peer review of the grant;
(e) Definitions.—In this section:
(1) The term “participating entity” means a participating clinical trial site or sites sponsored by 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)) to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis.
(2) The term “participating clinical trial” means a 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 prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis.
SEC. 3. HHS public-private partnership for rare neurodegenerative diseases.
(a) Establishment.—Not later than one year after the date of enactment of this Act, the Secretary of Health and Human Services (referred to in this section as the “Secretary”) shall establish and implement a Public-Private Partnership for Neurodegenerative Diseases between the National Institutes of Health, the Food and Drug Administration, and one or more eligible entities (to be known and referred to in this section as the “Partnership”) through cooperative agreements, contracts, or other appropriate mechanisms with such eligible entities, for the purpose of advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amytrophic lateral sclerosis and other rare neurodegenerative diseases. The Partnership shall—
(1) establish partnerships and consortia with other public and private entities and individuals with expertise in amyotrophic lateral sclerosis and other rare neurodegenerative diseases for the purposes described in this subsection;
(b) Eligible entity.—In this section, the term “eligible entity” means an entity that—
(1) is—
(A) an institution of higher education (as such term is defined in section 1001 of the Higher Education Act of 1965 (20 U.S.C. 1001)) or a consortium of such institutions; or
(B) an organization described in section 501(c)(3) of the Internal Revenue Code of 1986 and exempt from tax under subsection (a) of such section;
(2) has experienced personnel with clinical and other technical expertise in the field of biomedical sciences and demonstrated connection to the patient population;
(3) demonstrates to the Secretary’s satisfaction that the entity is capable of identifying and establishing collaborations between public and private entities and individuals with expertise in neurodegenerative diseases, including patients, in order to facilitate—
(4) provides an assurance that the entity will not accept funding for a Partnership project from any organization that manufactures or distributes products regulated by the Food and Drug Administration unless the entity provides assurances in its agreement with the Secretary that the results of the project will not be influenced by any source of funding.
(c) Gifts.—
(1) IN GENERAL.—The Partnership may solicit and accept gifts, grants, and other donations, establish accounts, and invest and expend funds in support of basic research and research associated with phase 3 clinical trials conducted with respect to investigational drugs that are the subjects of expanded access requests under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb).
SEC. 4. ALS and other rare neurodegenerative disease action plan.
(a) In general.—Not later than 6 months after the date of enactment of this Act, the Commissioner of Food and Drugs shall publish on the website of the Food and Drug Administration an action plan describing actions the Food and Drug Administration intends to take during the 5-year period following publication of the plan with respect to program enhancements, policy development, regulatory science initiatives, and other appropriate initiatives to—
(b) Contents.—The initial action plan published under subsection (a) shall—
(1) identify appropriate representation from within the Food and Drug Administration to be responsible for implementation of such action plan;
(2) include elements to facilitate—
(A) interactions and collaboration between the Food and Drug Administration, including the review centers thereof, and stakeholders including patients, sponsors, and the external biomedical research community;
(B) consideration of cross-cutting clinical and regulatory policy issues, including consistency of regulatory advice and decisionmaking;
SEC. 5. FDA rare neurodegenerative disease grant program.
The Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, shall award grants and contracts to public and private entities to cover the costs of research on, and development of interventions intended to prevent, diagnose, mitigate, treat, or cure, amyotrophic lateral sclerosis and other rare neurodegenerative diseases in adults and children, including costs incurred with respect to the development and critical evaluation of tools, methods, and processes—
Not later than 4 years after the date of the enactment of this Act, the Comptroller General of the United States 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 containing—
(1) with respect to grants awarded under the program established under section 2—
(A) an analysis of what is known about the impact of such grants on research or development related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis; and
(B) data concerning such grants, including—
(v) the number of patients who received expanded access to an investigational drug to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis under each grant;
(2) with respect to grants awarded under the program established under section 5—
(A) an analysis of what is known about the impact of such grants on research or development related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis;
(B) an analysis of what is known about how such grants increased efficiency and productivity of the clinical development of therapies, including through the use of clinical trials that operated with common master protocols, or had adaptive or add-on clinical trial designs; and
SEC. 7. Authorization of appropriations.
For purposes of carrying out this Act, there are authorized to be appropriated $100,000,000 for each of fiscal years 2022 through 2026.
Union Calendar No. 148 | |||||
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[Report No. 117–207] | |||||
A BILL | |||||
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. | |||||
December 8, 2021 | |||||
Reported with an amendment, committed to the Committee of the Whole House on the State of the Union, and ordered to be printed |