[Congressional Record Volume 167, Number 194 (Thursday, November 4, 2021)]
[Senate]
[Pages S7979-S7981]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

  SA 4474. Mr. COONS (for himself and Ms. Murkowski) submitted an 
amendment intended to be proposed to amendment SA 3867 submitted by Mr. 
Reed and intended to be proposed to the bill H.R. 4350, to authorize 
appropriations for fiscal year 2022 for military activities of the 
Department of Defense, for military construction, and for defense 
activities of the Department of Energy, to prescribe military personnel 
strengths for such fiscal year, and for other purposes; which was 
ordered to lie on the table; as follows:

        At the end of title X, add the following:

     Subtitle H--Accelerating Access to Critical Therapies for ALS

     SEC. 1071. 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

[[Page S7980]]

     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
       (3) the resulting project funded by such grant will allow 
     for equitable access to investigational drugs by minority and 
     underserved populations.
       (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;
       (2) for the entity's direct costs incurred in providing 
     such drug consistent with the research mission of the grant; 
     or
       (3) for the direct and indirect costs of the entity in 
     conducting research with respect to such drug.
       (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.
       (3) The term ``participating clinical trial site'' means a 
     health care facility, or network of facilities, at which 
     patients participating in a participating clinical trial 
     receive an investigational drug through such trial.
       (f) Sunset.--The Secretary may not award grants under this 
     section on or after September 30, 2026.

     SEC. 1072. 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 amyotrophic 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;
       (2) focus on advancing regulatory science and scientific 
     research that will support and accelerate the development and 
     review of drugs for patients with amyotrophic lateral 
     sclerosis and other rare neurodegenerative diseases; and
       (3) foster the development of effective drugs that improve 
     the lives of people that suffer from amyotrophic lateral 
     sclerosis and other rare neurodegenerative diseases.
       (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--
       (A) development and critical evaluation of tools, methods, 
     and processes--
       (i) to characterize neurodegenerative diseases and their 
     natural history;
       (ii) to identify molecular targets for neurodegenerative 
     diseases; and
       (iii) to increase efficiency, predictability, and 
     productivity of clinical development of therapies, including 
     advancement of rational therapeutic development and 
     establishment of clinical trial networks; and
       (B) securing funding for the Partnership from Federal and 
     non-Federal governmental sources, foundations, and private 
     individuals; and
       (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).
       (2) Use.--In addition to any amounts appropriated for 
     purposes of carrying out this section, the Partnership may 
     use, without further appropriation, any funds derived from a 
     gift, grant, or other donation accepted pursuant to paragraph 
     (1).

     SEC. 1073. 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--
       (1) foster the development of safe and effective drugs that 
     improve or extend, or both, the lives of people living with 
     amyotrophic lateral sclerosis and other rare 
     neurodegenerative diseases; and
       (2) facilitate access to investigational drugs for 
     amyotrophic lateral sclerosis and other rare 
     neurodegenerative diseases.
       (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 
     decision making;
       (C) identification of key regulatory science and policy 
     issues critical to advancing development of safe and 
     effective drugs; and
       (D) enhancement of collaboration and engagement of the 
     relevant centers and offices of the Food and Drug 
     Administration with other operating divisions within the 
     Department of Health and Human Services, the Partnership, and 
     the broader neurodegenerative disease community; and
       (3) be subject to revision, as determined appropriate by 
     the Secretary of Health and Human Services.

     SEC. 1074. 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--
       (1) to characterize such neurodegenerative diseases and 
     their natural history;
       (2) to identify molecular targets for such 
     neurodegenerative diseases; and
       (3) to increase efficiency and productivity of clinical 
     development of therapies, including through--
       (A) the use of master protocols and adaptive and add-on 
     clinical trial designs; and
       (B) efforts to establish new or leverage existing clinical 
     trial networks.

     SEC. 1075. GAO REPORT.

       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 1071--
       (A) an analysis of what is known about the impact of such 
     grants on research or development related to the prevention, 
     diagnosis,

[[Page S7981]]

     mitigation, treatment, or cure of amyotrophic lateral 
     sclerosis; and
       (B) data concerning such grants, including--
       (i) the number of grants awarded;
       (ii) the participating entities to whom grants were 
     awarded;
       (iii) the value of each such grant;
       (iv) a description of the research each such grant was used 
     to further;
       (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;
       (vi) whether the investigational drug that was the subject 
     of such a grant was approved by the Food and Drug 
     Administration; and
       (vii) the average number of days between when a grant 
     application is submitted and when a grant is awarded; and
       (2) with respect to grants awarded under the program 
     established under section 1074--
       (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
       (C) data concerning such grants, including--
       (i) the number of grants awarded;
       (ii) the participating entities to whom grants were 
     awarded;
       (iii) the value of each such grant;
       (iv) a description of the research each such grant was used 
     to further; and
       (v) whether the investigational drug that was the subject 
     of such a grant received approval by the Food and Drug 
     Administration.

     SEC. 1076. AUTHORIZATION OF APPROPRIATIONS.

       For purposes of carrying out this subtitle, there are 
     authorized to be appropriated $100,000,000 for each of fiscal 
     years 2022 through 2026.
                                 ______