View all text of Part A [§ 351 - § 360n-2]
§ 356. Expedited approval of drugs for serious or life-threatening diseases or conditions
(a) Designation of a drug as a breakthrough therapy
(1) In general
(2) Request for designation
(3) Designation
(A) In general
(B) ActionsThe actions to expedite the development and review of an application under subparagraph (A) may include, as appropriate—
(i) holding meetings with the sponsor and the review team throughout the development of the drug;
(ii) providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the nonclinical and clinical data necessary for approval is as efficient as practicable;
(iii) involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review;
(iv) assigning a cross-disciplinary project lead for the Food and Drug Administration review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the review team and the sponsor; and
(v) taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment.
(b) Designation of drug as fast track product
(1) In general
(2) Request for designation
(3) Designation
(c) Accelerated approval of a drug for a serious or life-threatening disease or condition, including a fast track product
(1) In general
(A) Accelerated approval
(B) Evidence
(2) Limitation
(A) In generalApproval of a product under this subsection may be subject to 1 or both of the following requirements:
(i) That the sponsor conduct an appropriate postapproval study or studies to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit.
(ii) 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.
(B) Studies not required
(C) Postapproval study conditions
(D) Studies begun before approval
(3) Expedited withdrawal of approval
(A) In generalThe Secretary may withdraw approval of a product approved under accelerated approval using expedited procedures described in subparagraph (B) if—
(i) 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);
(ii) 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;
(iii) other evidence demonstrates that the product is not shown to be safe or effective under the conditions of use; or
(iv) the sponsor disseminates false or misleading promotional materials with respect to the product.
(B) Expedited procedures describedExpedited 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 proposed 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 proposal to withdraw 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.
(d) Review of incomplete applications for approval of a fast track product
(1) In generalIf the Secretary determines, after preliminary evaluation of clinical data submitted by the sponsor, that a fast track product may be effective, the Secretary shall evaluate for filing, and may commence review of portions of, an application for the approval of the product before the sponsor submits a complete application. The Secretary shall commence such review only if the applicant—
(A) provides a schedule for submission of information necessary to make the application complete; and
(B) pays any fee that may be required under section 379h of this title.
(2) Exception
(e) Construction
(1) Purpose
(2) Construction
(f) Awareness effortsThe Secretary shall—
(1) develop and disseminate to physicians, patient organizations, pharmaceutical and biotechnology companies, and other appropriate persons a description of the provisions of this section applicable to breakthrough therapies, accelerated approval, and and 1
1 So in original.
fast track products; and(2) establish a program to encourage the development of surrogate and clinical endpoints, including biomarkers, and other scientific methods and tools that can assist the Secretary in determining whether the evidence submitted in an application is reasonably likely to predict clinical benefit for serious or life-threatening conditions for which significant unmet medical needs exist.
(g) Regenerative advanced therapy
(1) In general
(2) CriteriaA drug is eligible for designation as a regenerative advanced therapy under this subsection if—
(A) the drug is a regenerative medicine therapy (as defined in paragraph (8));
(B) the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
(C) preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition.
(3) Request for designation
(4) Designation
(5) Actions
(6) Access to expedited approval pathwaysAn application for a regenerative advanced therapy under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] may be—
(A) eligible for priority review, as described in the Manual of Policies and Procedures of the Food and Drug Administration and goals identified in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012; and
(B) eligible for accelerated approval under subsection (c), as agreed upon pursuant to subsection (a)(3)(B), through, as appropriate—
(i) surrogate or intermediate endpoints reasonably likely to predict long-term clinical benefit; or
(ii) reliance upon data obtained from a meaningful number of sites, including through expansion to additional sites, as appropriate.
(7) Postapproval requirementsThe sponsor of a regenerative advanced therapy that is granted accelerated approval and is subject to the postapproval requirements under subsection (c) may, as appropriate, fulfill such requirements, as the Secretary may require, through—
(A) the submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such as electronic health records;
(B) the collection of larger confirmatory data sets, as agreed upon pursuant to subsection (a)(3)(B); or
(C) postapproval monitoring of all patients treated with such therapy prior to approval of the therapy.
(8) Definition
(h) Limited population pathway for antibacterial and antifungal drugs
(1) In generalThe Secretary may approve an antibacterial or antifungal drug, alone or in combination with one or more other drugs, as a limited population drug pursuant to this subsection only if—
(A) the drug is intended to treat a serious or life-threatening infection in a limited population of patients with unmet needs;
(B) the standards for approval under section 355(c) and (d) of this title, or the standards for licensure under section 351 of the Public Health Service Act [42 U.S.C. 262], as applicable, are met; and
(C) the Secretary receives a written request from the sponsor to approve the drug as a limited population drug pursuant to this subsection.
(2) Benefit-risk consideration
(3) Additional requirementsA drug approved under this subsection shall be subject to the following requirements, in addition to any other applicable requirements of this chapter:
(A) LabelingTo indicate that the safety and effectiveness of a drug approved under this subsection has been demonstrated only with respect to a limited population—
(i) all labeling and advertising of an antibacterial or antifungal drug approved under this subsection shall contain the statement “Limited Population” in a prominent manner and adjacent to, and not more prominent than—(I) the proprietary name of such drug, if any; or(II) if there is no proprietary name, the established name of the drug, if any, as defined in section 353(e)(3) of this title, or, in the case of a drug that is a biological product, the proper name, as defined by regulation; and
(ii) the prescribing information for the drug required by section 201.57 of title 21, Code of Federal Regulations (or any successor regulation) shall also include the following statement: “This drug is indicated for use in a limited and specific population of patients.”.
(B) Promotional material
(4) Other programs
(5) Guidance
(6) Advice
(7) Termination of limitations
(8) Rules of construction
(9) Reporting and accountability
(A) Biennial reporting
(B) GAO report
(June 25, 1938, ch. 675, § 506, as added Pub. L. 105–115, title I, § 112(a), Nov. 21, 1997, 111 Stat. 2309; amended Pub. L. 112–144, title VIII, § 803, title IX, §§ 901(b), 902(a), July 9, 2012, 126 Stat. 1079, 1083, 1086; Pub. L. 114–255, div. A, title III, §§ 3033(a), (c), 3042, Dec. 13, 2016, 130 Stat. 1101, 1103, 1112; Pub. L. 117–328, div. FF, title III, § 3210(a), Dec. 29, 2022, 136 Stat. 5822.)