Sec. 804. Postapproval studies and program integrity for accelerated approval drugs
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Section 506(c) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 356(c) ) is amended— by striking paragraph
(2)and inserting the following: Approval of a product under this subsection may be subject to 1 or both of the following requirements: That the sponsor conduct an appropriate postapproval study or studies (which may be augmented or supported by real world evidence) to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit. That the sponsor submit copies of all promotional materials related to the product during the preapproval review period and, following approval and for such period thereafter as the Secretary determines to be appropriate, at least 30 days prior to dissemination of the materials. 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. 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 . The Secretary may require such study or studies to be underway prior to approval. ; and by striking paragraph
(3)and inserting the following: The Secretary may withdraw approval of a product approved under accelerated approval using expedited procedures described in subparagraph (B), if— the sponsor fails to conduct any required postapproval study of the product with due diligence, including with respect to conditions specified by the Secretary under paragraph (2)(C); a study required to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit of the product fails to verify and describe such effect or benefit; other evidence demonstrates that the product is not shown to be safe or effective under the conditions of use; or the sponsor disseminates false or misleading promotional materials with respect to the product. Expedited procedures described in this subparagraph shall consist of, prior to the withdrawal of accelerated approval— providing the sponsor with— due notice; an explanation for the proposed withdrawal; an opportunity for a meeting with the Commissioner of Food and Drugs or the Commissioner’s designee; and an opportunity for written appeal to— the Commissioner of Food and Drugs; or a designee of the Commissioner who has not participated in the proposed withdrawal of approval (other than a meeting pursuant to subclause (III)) and is not a subordinate of an individual (other than the Commissioner) who participated in such proposed withdrawal; providing an opportunity for public comment on the notice proposing to withdraw approval; 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 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. Subject to subparagraph (B), the labeling for a product approved under accelerated approval shall include— a statement indicating that the product was approved under accelerated approval; a statement indicating that continued approval of the product is subject to postmarketing studies to verify clinical benefit; identification of the surrogate or intermediate endpoint or endpoints that supported approval and any known limitations of such surrogate or intermediate endpoint or endpoints in determining clinical benefit; and a succinct description of the product and any uncertainty about anticipated clinical benefit and a discussion of available evidence with respect to such clinical benefit. The labeling requirements of subparagraph
(A)shall apply only to products approved under accelerated approval for which the predicted effect on irreversible morbidity or mortality or other clinical benefit has not been verified. With respect to any application pending before the Secretary on the date of enactment of the Food and Drug Amendments of 2022, the Secretary shall allow any applicable changes to the product labeling required to comply with subparagraph
(A)to be made by supplement after the approval of such application. Not later than September 30, 2025, 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 circumstances in which the Secretary considered real world evidence submitted to support postapproval studies required under this subsection that were completed after the date of enactment of the Food and Drug Amendments of 2022 . . Section 506B(a) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 356b(a) ) is amended— by redesignating paragraph
(2)as paragraph (3); and by inserting after paragraph
(1)the following: 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 of Health and Human Services shall issue guidance describing— 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; the use of novel clinical trial designs that may be used to conduct appropriate postapproval studies as may be required under section 506(c)(2)(A) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 356(c)(2)(A) ), as amended by subsection (a); and the expedited procedures described in section 506(c)(3)(B) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 356(c)(3)(B) ). The Secretary shall issue— draft guidance under paragraph
(1)not later than 18 months after the date of enactment of this Act; and final guidance not later than 1 year after the close of the public comment period on such draft guidance. 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— determining eligibility of participants for such a program; and developing and implementing a process for applying to, and participating in, such a program. 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— novel endpoints developed through the pilot program established under this subsection; and as appropriate, the use of real world evidence and real world data to support the validation of efficacy endpoints, including surrogate and intermediate endpoints, for rare diseases. 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. 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. 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.
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Sec. 804
Postapproval studies and program integrity for accelerated approval drugs
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