Title: Speeding Therapy Access Today Act of 2021
Policy area: Health

Summary:
Speeding Therapy Access Today Act of 2021
This bill requires and authorizes various actions to accelerate the development of therapies for rare diseases.
The Food and Drug Administration (FDA) shall establish the Intercenter Institute on Rare Diseases and Conditions. The institute shall (1) coordinate engagement with relevant stakeholders, (2) build the FDA's expertise in the review of medical products to treat rare diseases, (3) coordinate regulatory science initiatives related to rare diseases, (4) establish and implement a program to make recommendations to address challenges associated with developing medical products to treat rare diseases in an individual or in very small populations, (5) convene a stakeholder meeting to consider potential amendments to labels for medical products to treat rare diseases, and (6) establish and carry out a program to facilitate voluntary communication between the sponsors of such medical products and third-party payers (e.g., insurance companies).
The bill also authorizes the FDA to make grants to assist in developing practices related to the development and production of individualized therapies or therapies to treat very small populations.
The bill also establishes an advisory committee to advise the FDA on issues related to the development of therapies to treat rare diseases.

[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 670 Introduced in Senate (IS)]

<DOC>






117th CONGRESS
  1st Session
                                 S. 670

    To amend the Federal Food, Drug, and Cosmetic Act to accelerate 
   development of therapies across the spectrum of rare diseases and 
  conditions and facilitate patient access to such therapies, and for 
                            other purposes.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                             March 10, 2021

  Ms. Klobuchar (for herself and Mr. Wicker) introduced the following 
  bill; which was read twice and referred to the Committee on Health, 
                     Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL


 
    To amend the Federal Food, Drug, and Cosmetic Act to accelerate 
   development of therapies across the spectrum of rare diseases and 
  conditions and facilitate patient access to such therapies, 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 ``Speeding Therapy Access Today Act of 
2021''.

SEC. 2. TABLE OF CONTENTS.

    The table of contents of this Act is as follows:

Sec. 1. Short title.
Sec. 2. Table of contents.
Sec. 3. Intercenter Institute on Rare Diseases and Conditions.
Sec. 4. Rare Disease and Condition Drug Advisory Committee.
Sec. 5. Grants and contracts for development of drugs for rare diseases 
                            and conditions.

SEC. 3. INTERCENTER INSTITUTE ON RARE DISEASES AND CONDITIONS.

    (a) Establishment Required.--The first sentence of section 1014(a) 
of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 399g(a)) is 
amended by inserting ``, at least one of which shall be focused on rare 
diseases and conditions'' before the period at the end of the sentence.
    (b) Timing of Establishment.--Subsection (c) of section 1014 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 399g) is amended to 
read as follows:
    ``(c) Timing.--Not later than the date that is 1 year after the 
date of enactment of the Speeding Therapy Access Today Act of 2021, the 
Secretary shall establish, in accordance with this section and section 
529B, an Institute under subsection (a) focused on rare diseases and 
conditions, to be known as the Intercenter Institute on Rare Diseases 
and Conditions.''.
    (c) Responsibilities.--Subchapter B of chapter V of the Federal 
Food, Drug, and Cosmetic Act (relating to drugs for rare diseases or 
conditions) is amended by inserting after section 529A of such Act (21 
U.S.C. 360ff-1) the following new section:

``SEC. 529B. INTERCENTER INSTITUTE ON RARE DISEASES AND CONDITIONS.

    ``(a) Responsibilities.--In addition to carrying out activities 
listed in section 1014(a), the Intercenter Institute on Rare Diseases 
and Conditions shall--
            ``(1) serve as the Food and Drug Administration's 
        coordinating office for engagement with rare disease and 
        condition stakeholders, complementing but not supplanting 
        engagement activities between stakeholders and the review 
        divisions;
            ``(2) build, within the Food and Drug Administration, 
        knowledge and understanding associated with the review of 
        medical products to treat rare diseases and conditions, 
        including advancements in trial design, statistical analysis, 
        regulatory science, product manufacturing, and other topics as 
        determined by the Secretary;
            ``(3) implement cross-center rare disease and condition-
        focused meetings and policy development;
            ``(4) coordinate rare disease and condition-specific 
        regulatory science initiatives;
            ``(5) facilitate stakeholder engagement to the external 
        community and international regulatory agencies on rare disease 
        and condition product development;
            ``(6) establish and implement the Accelerating Lifesavings 
        Therapies in Treating Ultra-rare Disease Entities Program under 
        subsection (b); and
            ``(7) establish and carry out the rare disease and 
        condition third-party payor program under subsection (d).
    ``(b) ALTITUDE Program.--
            ``(1) In general.--The Intercenter Institute shall 
        establish and implement a program, to be known as the 
        Accelerating Lifesavings Therapies in Treating Ultra-rare 
        Disease Entities Program, to identify and make recommendations 
        to address current and emerging regulatory science and public 
        policy challenges associated with developing medical products 
        to treat rare diseases or conditions in an individual or very 
        small populations.
            ``(2) Issues.--The program under paragraph (1) shall focus 
        on issues including--
                    ``(A) manufacturing standards for therapies 
                described in such paragraph, including in non-industry 
                settings;
                    ``(B) trial designs and metrics;
                    ``(C) regulatory flexibilities for abbreviated 
                toxicology studies, overlapping animal studies, and 
                patient dosing;
                    ``(D) regulatory science, chemistry, manufacturing, 
                and other needs associated with developing such 
                therapies; and
                    ``(E) other issues as determined by the Secretary.
    ``(c) Proposals for Amending Labels.--
            ``(1) Stakeholder group.--Not later than 180 days after the 
        date of enactment of this section, the Intercenter Institute 
        shall convene a meeting of stakeholders from the rare disease 
        community, including patients, caregivers, product 
        manufacturers, third-party payors, and others, to consider 
        potential amendments to labels for medical products to treat 
        rare diseases or conditions approved pursuant to a pathway 
        under section 506.
            ``(2) Guidance.--Not later than 90 days after the date of 
        the meeting under paragraph (1), the Secretary shall issue 
        guidance to propose changes to how the labels of medical 
        products to treat rare diseases or conditions demonstrate 
        clinical benefits and reflect relevant scientific data 
        including surrogate endpoints.
    ``(d) Rare Disease and Condition Third-Party Payor Program.--
            ``(1) In general.--The Intercenter Institute shall 
        establish and carry out a voluntary rare disease and condition 
        early third-party payor feedback program--
                    ``(A) to inform coverage policies for rare disease 
                therapies; and
                    ``(B) to inform clinical trial design, patient 
                engagement, and other data collections.
            ``(2) Program requirements.--The program under paragraph 
        (1) shall--
                    ``(A) facilitate voluntary communication between 
                sponsors of medical products to treat rare diseases and 
                conditions and third-party payors; and
                    ``(B) require participation of the Centers for 
                Medicare & Medicaid Services with representation from--
                            ``(i) the Center for Medicare; and
                            ``(ii) the Center for Medicaid and CHIP 
                        Services.
            ``(3) Annual report.--The Intercenter Institute shall--
                    ``(A) on an annual basis, submit a report to that 
                Congress on--
                            ``(i) the participation within the program 
                        under paragraph (1); and
                            ``(ii) the impacts of the program under 
                        paragraph (1); and
                    ``(B) post each such report on the public website 
                of the Intercenter Institute.
            ``(4) Bulletin to medicaid directors.--Following the 
        approval, clearance, or authorization by the Food and Drug 
        Administration of a medical product to treat a rare disease or 
        condition, the Secretary shall issue a bulletin to State 
        Medicaid directors containing information to help inform 
        coverage decisions on the product by State Medicaid and 
        Children's Health Insurance programs.
    ``(e) Definition.--In this section, the terms `Intercenter 
Institute on Rare Diseases and Conditions' and `Intercenter Institute' 
refer to the Intercenter Institute on Rare Diseases and Conditions 
established pursuant to section 1014.''.

SEC. 4. RARE DISEASE AND CONDITION DRUG ADVISORY COMMITTEE.

    Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic 
Act is further amended by inserting after section 529B of such Act, as 
inserted by section 3, the following new section:

``SEC. 529C. RARE DISEASE AND CONDITION DRUG ADVISORY COMMITTEE.

    ``(a) In General.--The Secretary shall establish and maintain a 
committee, to be known as the Rare Disease and Condition Drug Advisory 
Committee (in this section referred to as the `Advisory Committee').
    ``(b) Duty of Committee.--The Advisory Committee shall advise the 
Secretary on issues associated with development of therapies to treat 
rare diseases or conditions.
    ``(c) Specific Issues.--In advising the Secretary, the Advisory 
Committee may address issues including--
            ``(1) modified or new regulatory pathways to support review 
        of therapies;
            ``(2) clinical trial design needs, including development of 
        innovative approaches to clinical trials;
            ``(3) qualifications of biomarkers or other drug 
        development tools for use in reviews;
            ``(4) modified or new standards to support the review of 
        already marketed drugs being evaluated for repurposing to treat 
        a rare disease or condition; and
            ``(5) issues--
                    ``(A) that pertain to an application for approval 
                of a therapy to treat a rare disease or condition; and
                    ``(B) with respect to which a review division has 
                requested that the Advisory Committee provide advice.
    ``(d) Membership.--
            ``(1) In general.--The Advisory Committee shall consist 
        of--
                    ``(A) not more than 15 members appointed by the 
                Secretary in accordance with paragraph (2); and
                    ``(B) the nonvoting ex officio members under 
                paragraph (3).
            ``(2) Appointed members.--
                    ``(A) Special government employees.--Members of the 
                Advisory Committee appointed pursuant to paragraph 
                (1)(A) shall serve as special Government employees (as 
                defined in section 202(a) of title 18, United States 
                Code).
                    ``(B) Eligibility.--To be eligible for appointment 
                pursuant to paragraph (1)(A), an individual shall--
                            ``(i) be eligible to serve as special 
                        Government employee (as defined in section 
                        202(a) of title 18, United States Code); and
                            ``(ii) have expertise in the fields of 
                        public policy, law, regulatory policy, 
                        economics, patient-focused product development, 
                        or patient advocacy.
                    ``(C) Composition.--Of the members of the Advisory 
                Committee appointed pursuant to paragraph (1)(A)--
                            ``(i) up to 10 shall be selected from among 
                        experts in the disciplines relevant to the 
                        activities of the Intercenter Institute on Rare 
                        Diseases and Conditions, to include at least 
                        one expert in each of--
                                    ``(I) rare disease product 
                                development;
                                    ``(II) conducting clinical trials 
                                with respect to rare diseases and 
                                conditions, including with respect to 
                                very small patient populations;
                                    ``(III) rare disease and condition 
                                natural history and related studies;
                                    ``(IV) health economics pertaining 
                                to the development of medical products 
                                for rare diseases or conditions;
                                    ``(V) manufacturing and related 
                                needs associated with medical products 
                                for rare diseases or conditions; and
                                    ``(VI) patient experience data 
                                collection; and
                            ``(ii) up to 5 shall be selected from the 
                        public, to include--
                                    ``(I) at least 4 individuals who 
                                are representatives of the rare disease 
                                patient community;
                                    ``(II) at least one individual who 
                                is directly impacted by a rare disease 
                                or condition; and
                                    ``(III) at least one person who 
                                serves as a family caregiver to a 
                                person diagnosed with a rare disease or 
                                condition.
            ``(3) Nonvoting ex officio members.--The nonvoting ex 
        officio members of the Advisory Committee under paragraph 
        (1)(B) shall consist of the following:
                    ``(A) The Secretary (or the Secretary's designee).
                    ``(B) The Director of the Intercenter Institute on 
                Rare Diseases and Conditions.
                    ``(C) The Director of the Center for Biologics 
                Evaluation and Research (or the Director's designee).
                    ``(D) The Director of the Center for Drug 
                Evaluation and Research (or the Director's designee).
                    ``(E) The Director of the Center for Devices and 
                Radiological Health (or the Director's designee).
                    ``(F) The Director of the National Center for the 
                Advancing Translational Sciences of the National 
                Institutes of Health (or the Director's designee).
                    ``(G) The Administrator of the Centers for Medicare 
                & Medicaid Services (or the Administrator's designee).
                    ``(H) Any additional officers or employees of the 
                Department of Health and Human Services as the 
                Secretary determines necessary for the Advisory 
                Committee to effectively carry out its functions.
            ``(4) Chair.--The Chair of the Advisory Committee shall be 
        the Director of the Intercenter Institute for Rare Diseases and 
        Conditions.
            ``(5) Terms.--
                    ``(A) Members.--
                            ``(i) In general.--The term of a member of 
                        the Advisory Committee appointed pursuant to 
                        paragraph (1)(A) shall be 4 years, except that 
                        any member appointed to fill a vacancy in an 
                        unexpired term shall be appointed for the 
                        remainder of that term.
                            ``(ii) Continued service.--A member 
                        appointed pursuant to paragraph (1)(A) may 
                        continue serving as a member of the Advisory 
                        Committee for up to 180 days after the 
                        expiration of that member's term if a successor 
                        has not been appointed.
                    ``(B) Reappointment.--A member of the Advisory 
                Committee who has been appointed pursuant to paragraph 
                (1)(A) for a term of 4 years may not be reappointed to 
                serve as a member of the Advisory Committee before the 
                date that is 2 years after the date of expiration of 
                that member's term.
    ``(e) Quorum.--A majority of the appointed members of the Advisory 
Committee shall constitute a quorum for the conduct of business.''.

SEC. 5. GRANTS AND CONTRACTS FOR DEVELOPMENT OF DRUGS FOR RARE DISEASES 
              AND CONDITIONS.

    (a) Authority of Secretary.--Section 5(a) of the Orphan Drug Act 
(21 U.S.C. 360ee(a)) is amended--
            (1) in paragraph (2), by striking ``and'' at the end; and
            (2) by inserting before the period at the end ``, and (4) 
        developing practices pertaining to the chemistry, 
        manufacturing, regulatory approval of, and controls of 
        individualized therapies or therapies to treat very small 
        populations''.
    (b) ALTITUDE Program.--In supporting grants and contracts under 
section 5(a)(4) of the Orphan Drug Act, as added by subsection (a), the 
Secretary of Health and Human Services shall consult with the Director 
of the Intercenter Institute on Rare Diseases and Conditions regarding 
the Accelerating Lifesavings Therapies in Treating Ultra-rare Disease 
Entities Program established under section 529B(b) of the Federal Food, 
Drug, and Cosmetic Act, as added by section 3(c) of this Act, to--
            (1) identify the regulatory science and related challenges 
        and needs associated with developing individualized therapies 
        or therapies to treat very small patient populations; and
            (2) support research to address such challenges.
                                 <all>