Title: Modernizing the Accelerated Approval Pathway Act of 2022
Policy area: Health

Summary:
Modernizing the Accelerated Approval Pathway Act of 2022
This bill modifies provisions relating to the accelerated approval of drugs for serious or life-threatening diseases or conditions, including by requiring the Food and Drug Administration (FDA) to publish its reasons when it determines a postapproval study to be unnecessary. The bill also establishes a pilot program and an FDA council to support the development of drugs for rare diseases and ensure the appropriate use of the accelerated approval pathway, respectively.

[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 4446 Introduced in Senate (IS)]

<DOC>






117th CONGRESS
  2d Session
                                S. 4446

   To modernize the process of accelerated approval of a drug for a 
           serious or life-threatening disease or condition.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                             June 22, 2022

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

_______________________________________________________________________

                                 A BILL


 
   To modernize the process of accelerated approval of a drug for a 
           serious or life-threatening disease or condition.

    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 ``Modernizing the Accelerated Approval 
Pathway Act of 2022''.

SEC. 2. MODERNIZING ACCELERATED APPROVAL.

    (a) In General.--Section 506(c) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 356(c)) is amended--
            (1) in paragraph (2)--
                    (A) by redesignating subparagraphs (A) and (B) as 
                clauses (i) and (ii), respectively, and adjusting the 
                margins accordingly;
                    (B) by striking ``Approval of a product'' and 
                inserting the following:
                    ``(A) In general.--Approval of a product'';
                    (C) in clause (i) of such subparagraph (A), as so 
                redesignated, by striking ``appropriate postapproval 
                studies'' and inserting ``an appropriate postapproval 
                study or studies (which may be augmented or supported 
                by real world evidence)''; and
                    (D) by adding at the end the following:
                    ``(B) Studies not required.--If the Secretary does 
                not require that the sponsor of a product approved 
                under accelerated approval conduct a postapproval study 
                under this paragraph, the Secretary shall publish on 
                the website of the Food and Drug Administration the 
                rationale for why such study is not appropriate or 
                necessary.
                    ``(C) Postapproval study conditions.--Not later 
                than the time of approval of a product under 
                accelerated approval, the Secretary shall specify the 
                conditions for a postapproval study or studies required 
                to be conducted under this paragraph with respect to 
                such product, which may include enrollment targets, the 
                study protocol, and milestones, including the target 
                date of study completion.
                    ``(D) Studies begun before approval.--The Secretary 
                may require such study or studies to be underway prior 
                to approval.''; and
            (2) in paragraph (3)--
                    (A) by redesignating subparagraphs (A) through (D) 
                as clauses (i) through (iv), respectively, and 
                adjusting the margins accordingly;
                    (B) by striking ``The Secretary may'' and inserting 
                the following:
                    ``(A) In general.--The Secretary may'';
                    (C) in clause (i) of such subparagraph (A), as so 
                redesignated, by striking ``drug with due diligence'' 
                and inserting ``product with due diligence, including 
                with respect to conditions specified by the Secretary 
                under paragraph (2)(C)'';
                    (D) in clause (iii) of such subparagraph (A), as so 
                redesignated, by inserting ``shown to be'' after 
                ``product is not''; and
                    (E) by adding at the end the following:
                    ``(B) Expedited procedures described.--Expedited 
                procedures described in this subparagraph shall consist 
                of, prior to the withdrawal of accelerated approval--
                            ``(i) providing the sponsor with--
                                    ``(I) due notice;
                                    ``(II) an explanation for the 
                                proposed withdrawal;
                                    ``(III) an opportunity for a 
                                meeting with the Commissioner or the 
                                Commissioner's designee; and
                                    ``(IV) an opportunity for written 
                                appeal to--
                                            ``(aa) the Commissioner; or
                                            ``(bb) a designee of the 
                                        Commissioner who has not 
                                        participated in the proposal 
                                        withdrawal of approval (other 
                                        than a meeting pursuant to 
                                        subclause (III)) and is not 
                                        subordinate of an individual 
                                        (other than the Commissioner) 
                                        who participated in such 
                                        proposed withdrawal;
                            ``(ii) providing an opportunity for public 
                        comment on the proposing to withdrawal 
                        approval;
                            ``(iii) the publication of a summary of the 
                        public comments received, and the Secretary's 
                        response to such comments, on the website of 
                        the Food and Drug Administration; and
                            ``(iv) convening and consulting an advisory 
                        committee on issues related to the proposed 
                        withdrawal, if requested by the sponsor and if 
                        no such advisory committee has previously 
                        advised the Secretary on such issues with 
                        respect to the withdrawal of the product prior 
                        to the sponsor's request.''.
    (b) Reports of Postmarketing Studies.--Section 506B(a) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356b(a)) is amended--
            (1) by redesignating paragraph (2) as paragraph (3); and
            (2) by inserting after paragraph (1) the following:
            ``(2) Accelerated approval.--Notwithstanding paragraph (1), 
        a sponsor of a drug approved under accelerated approval shall 
        submit to the Secretary a report of the progress of any study 
        required under section 506(c), including progress toward 
        enrollment targets, milestones, and other information as 
        required by the Secretary, not later than 180 days after the 
        approval of such drug and not less frequently than every 180 
        days thereafter, until the study is completed or terminated. 
        The Secretary shall promptly publish on the website of the Food 
        and Drug Administration the information so reported, in an 
        easily searchable format.''.
    (c) Enforcement.--Section 301 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 331), is amended by adding at the end the 
following:
    ``(fff) The failure of a sponsor of a product approved under 
accelerated approval pursuant to section 506(c)--
            ``(1) to conduct with due diligence any postapproval study 
        required under section 506(c) with respect to such product; or
            ``(2) to submit timely reports with respect to such product 
        in accordance with section 506B(a)(2).''.
    (d) Guidance.--
            (1) In general.--The Secretary of Health and Human Services 
        shall issue guidance describing--
                    (A) how sponsor questions related to the 
                identification of novel surrogate or intermediate 
                clinical endpoints may be addressed in early-stage 
                development meetings with the Food and Drug 
                Administration;
                    (B) the use of novel clinical trial designs that 
                may be used to conduct appropriate post-approval 
                studies as may be required under section 506(c)(2)(A) 
                of the Federal Food, Drug, and Cosmetic Act, as amended 
                by subsection (a);
                    (C) the expedited procedures described in section 
                506(c)(3)(B) of the Federal Food, Drug, and Cosmetic 
                Act; and
                    (D) considerations related to the use of surrogate 
                or intermediate clinical endpoints that may support the 
                accelerated approval of an application under 
                506(c)(1)(A), including considerations in evaluating 
                the evidence related to any such endpoints.
            (2) Final guidance.--The Secretary shall issue--
                    (A) draft guidance under paragraph (1) not later 
                than 18 months after the date of enactment of this Act; 
                and
                    (B) final guidance not later than 1 year after the 
                close of the public comment period on such draft 
                guidance.
    (e) Rare Disease Endpoint Advancement Pilot.--
            (1) In general.--The Secretary of Health and Human Services 
        shall establish a pilot program under which the Secretary will 
        establish procedures to provide increased interaction with 
        sponsors of rare disease drug development programs for purposes 
        of advancing the development of efficacy endpoints, including 
        surrogate and intermediate endpoints, for drugs intended to 
        treat rare diseases, including through--
                    (A) determining eligibility of participants for 
                such program; and
                    (B) developing and implementing a process for 
                applying to, and participating in, such a program.
            (2) Public workshops.--The Secretary shall conduct up to 3 
        public workshops, which shall be completed not later than 
        September 30, 2026, to discuss topics relevant to the 
        development of endpoints for rare diseases, which may include 
        discussions about--
                    (A) novel endpoints developed through the pilot 
                program established under this subsection; and
                    (B) as appropriate, the use of real world evidence 
                and real work data to support the validation of 
                efficacy endpoints, including surrogate and 
                intermediate endpoints, for rare diseases.
            (3) Report.--Not later than September 30, 2027, the 
        Secretary 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 
        describing the outcomes of the pilot program established under 
        this subsection.
            (4) Guidance.--Not later than September 30, 2027, the 
        Secretary shall issue guidance describing best practices and 
        strategies for development of efficacy endpoints, including 
        surrogate and intermediate endpoints, for rare diseases.
            (5) Sunset.--The Secretary may not accept any new 
        application or request to participate in the program 
        established by this subsection on or after October 1, 2027.
    (f) Accelerated Approval Council.--
            (1) General.--Not later than 180 days after the date of 
        enactment of this Act, the Secretary of Health and Human 
        Services shall establish an intra-agency coordinating council 
        within the Food and Drug Administration to ensure the 
        consistent and appropriate use of accelerated approval across 
        the Food and Drug Administration, pursuant to section 506(c) of 
        the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356(c)).
            (2) Membership.--The members of the Council shall consist 
        of the following senior officials, or a designee of such 
        official, from the Food and Drug Administration and relevant 
        Centers:
                    (A) The Director of the Center for Drug Evaluation 
                and Research.
                    (B) The Director of the Center for Biologics 
                Evaluation and Research.
                    (C) The Director of the Oncology Center of 
                Excellence.
                    (D) The Director of the Office of New Drugs.
                    (E) The Director of the Office of Orphan Products 
                Development.
                    (F) The Director of the Office of Tissues and 
                Advanced Therapies.
                    (G) The Director of the Office of Medical Policy.
                    (H) At least 3 directors of review division 
                overseeing products approved under accelerated 
                approval, including at least one director of a review 
                division within the Office of Neuroscience.
            (3) Duties of the council.--
                    (A) Meetings.--The Council shall convene not fewer 
                than 3 times per calendar year to discuss issues 
                related to accelerated approval, including any relevant 
                cross-disciplinary approaches related to product review 
                with respect to accelerated approval.
                    (B) Policy development.--The Council shall directly 
                engage with product review teams to support the 
                consistent and appropriate use of accelerated approval 
                across the Food and Drug Administration. Such 
                activities may include--
                            (i) developing guidance for Food and Drug 
                        Administration staff and best practices for, 
                        and across, product review teams, including 
                        with respect to communication between sponsors 
                        and the Food and Drug Administration and the 
                        review of products under accelerated approval;
                            (ii) providing training for product review 
                        teams; and
                            (iii) advising review divisions on product-
                        specific development, review, and withdrawal of 
                        products under accelerated approval.
            (4) Publication of a report.--Not later than 1 year after 
        the date of enactment of this Act, and annually thereafter, the 
        council shall publish on the public website of the Food and 
        Drug Administration a report on the activities of the council.
    (g) Rule of Construction.--Nothing in this section (including the 
amendments made by this section) shall be construed to affect products 
approved pursuant to 506(c) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 356(c)) prior to the date of enactment of this Act.
                                 <all>