[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3537 Engrossed in House (EH)]

<DOC>
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
117th CONGRESS
  1st Session
                                H. R. 3537

_______________________________________________________________________

                                 AN ACT


 
    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,

SECTION 1. SHORT TITLE.

    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
            (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. 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;
            (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. 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--
            (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 decisionmaking;
                    (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. 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--
            (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. 6. 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 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--
                            (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 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
                    (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. 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.

            Passed the House of Representatives December 8, 2021.

            Attest:

                                                                 Clerk.
117th CONGRESS

  1st Session

                               H. R. 3537

_______________________________________________________________________

                                 AN ACT

    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.