SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a guidance for industry entitled “Expedited Programs for Serious Conditions—Drugs and Biologics.” This guidance provides a single resource for information on FDA's policies and procedures related to the following expedited programs for serious conditions: (1) Fast track designation, (2) breakthrough therapy designation, (3) accelerated approval, and (4) priority review designation. The guidance describes threshold criteria generally applicable to expedited programs, including what is meant by serious condition, unmet medical need, and available therapy. This guidance also discusses considerations for expedited development and review such as manufacturing and product quality, nonclinical studies, and clinical inspections. In addition, this guidance aligns CDER's criteria for priority review designation with CBER's criteria. Only products intended to treat a serious condition are eligible for priority review (unless otherwise eligible under specific statutory provisions).

For over 30 years, expediting the availability of promising therapies to patients with serious conditions has been a priority for FDA. With the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) (Public Law 112-122), FDA is expanding its efforts to expedite development and review of therapies intended to treat patients with serious conditions. This guidance is intended to satisfy the statutory requirements of sections 901(c)(2) and 902(b)(1)(A) of FDASIA.

Section 901(c)(2) of FDASIA requires FDA to issue a final guidance document to implement amendments to the Federal Food, Drug, and Cosmetic Act (the FD&C Act) made by section 901 of ( printed page 31118) FDASIA (Enhancement of Accelerated Approval Access to New Medical Treatments) within 1 year of the date the draft guidance issues. The discussions of accelerated approval, and other more broadly applicable provisions in this guidance, are intended to meet this requirement.

Section 902(b)(1)(A) of FDASIA requires FDA to issue a guidance document to implement requirements of section 902 (Breakthrough Therapies) within 1 year of the date the comment period closes for the draft guidance. The breakthrough therapy discussion and other more broadly applicable provisions in this guidance are intended to meet this requirement.

In the Federal Register of June 26, 2013 (78 FR 38349), FDA announced the availability of the draft guidance entitled “Expedited Programs for Serious Conditions—Drugs and Biologics.” The notice gave the public an opportunity to comment by August 26, 2013. FDA carefully considered all comments received in developing the final guidance. This guidance addresses the applicability of expedited programs to rare diseases, clarification on available therapy, and additional detail on possible flexibility in manufacturing and product quality. The guidance also includes clarification on the qualifying criteria for breakthrough therapy designation and examples of surrogate endpoints and intermediate clinical endpoints used to support accelerated approval. This guidance finalizes the draft guidance issued in June 2013.

The provisions of this guidance relating to fast track development and other issues such as serious condition and unmet medical need, replace the guidance for industry entitled “Fast Track Drug Development Programs—Designation, Development, and Application Review.” The provisions of this guidance pertaining to available therapy replace the guidance for industry entitled “Available Therapy.”

This guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents the Agency's current thinking on expedited programs for serious conditions—drugs and biologics. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

This guidance contains information collection provisions that are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The collections of information in 21 CFR 202.1, certain parts of 21 CFR part 314, 21 CFR part 601, and sections 506(b)(1), 735, and 736 of the FD&C Act (21 U.S.C. 356(b)(1), 379g, and 379h) have been approved under OMB control numbers 0910-0686, 0910-0001, 0910-0338, 0910-0389, 0910-0297, and 0910-0765.

III. Comments

Interested persons may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets Management (see ADDRESSES ). It is only necessary to send one set of comments. Identify comments with the docket number found in brackets in the heading of this document. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

Persons with access to the Internet may obtain the document at either http://www.fda.gov/​Drugs/​GuidanceComplianceRegulatoryInformation/​Guidances/​default.htm, http://www.fda.gov/​BiologicsBloodVaccines/​GuidanceComplianceRegulatoryInformation/​default.htm, or http://www.regulations.gov.