SUPPLEMENTARY INFORMATION:

In compliance with 44 U.S.C. 3507, FDA has submitted the following proposed collection of information to OMB for review and clearance.

Expedited Programs for Serious Conditions—Drugs and Biologics

OMB Control Number 0910-0765—Extension

This information collection supports Agency regulations and associated guidance pertaining to expedited programs for serious conditions. The purpose of our regulations in 21 CFR part 312, subpart E is to establish procedures designed to expedite the development, evaluation, and marketing of new therapies intended to treat persons with life-threatening and severely debilitating illnesses, especially where no satisfactory alternative therapy exists. While the statutory standards of safety and effectiveness apply to all drugs, the many kinds of drugs that are subject to them, and the wide range of uses for those drugs, demand flexibility in applying the standards.

We have developed the guidance for industry entitled “Expedited Programs for Serious Conditions—Drugs and Biologics” as a single resource for information on FDA's policies and procedures related to the following ( printed page 10096) 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. The 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. It also clarifies the qualifying criteria for breakthrough therapy designation and provides examples of surrogate endpoints and intermediate clinical endpoints used to support accelerated approval.

A sponsor or applicant who seeks fast track designation is required to submit to us a request showing that the drug product: (1) Is intended for a serious or life-threatening condition and (2) has the potential to address an unmet medical need. We expect that most information to support a designation request will have been gathered under existing requirements for preparing an investigational new drug application (IND), new drug application (NDA), or biologics license application (BLA). If such information has already been submitted to us, the information may be summarized in the fast track designation request. A designation request should include, where applicable, additional information not specified elsewhere by statute or regulation. For example, additional information may be needed to show that a product has the potential to address an unmet medical need where an approved therapy exists for the serious or life-threatening condition to be treated. Such information may include clinical data, published reports, summaries of data and reports, and a list of references. The amount of information and discussion in a designation request need not be voluminous, but it should be sufficient to permit a reviewer to assess whether the criteria for fast track designation have been met.

After we make a fast track designation, a sponsor or applicant may submit a premeeting package that may include additional information supporting a request to participate in certain fast track programs. The premeeting package serves as background information for the meeting and should support the intended objectives of the meeting. As with the request for fast track designation, we expect that most sponsors or applicants will have gathered such information to meet existing requirements for preparing an IND, an NDA, or a BLA. These may include descriptions of clinical safety and efficacy trials not conducted under an IND ( e.g., foreign studies) and information to support a request for accelerated approval. If such information has already been submitted to us, the information may be summarized in the premeeting package.

We also developed the guidance document entitled “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.” The guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions with FDA's recommendations on the expedited development and review of these therapies. The guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious or life-threatening diseases or conditions, including those products designated as regenerative advanced therapies (which FDA refers to as “regenerative medicine advanced therapy” (RMAT) designation). The guidance also describes considerations in the clinical development of regenerative medicine therapies and opportunities for sponsors of regenerative medicine therapies to interact with the Center of Biologics Evaluation and Research review staff.

The guidance documents are available on our website at https://www.fda.gov/​regulatory-information/​search-fda-guidance-documents and were issued consistent with our good guidance practice regulations in 21 CFR 10.115, which provide for public comment at any time.

In the Federal Register of November 18, 2020 (85 FR 73487), we published a 60-day notice requesting public comment on the proposed collection of information. No comments were received.

We estimate the burden of this collection of information as follows:

Based on a review of the information collection since our last request for OMB approval, we have increased our burden estimates by 389 responses and 35,325 hours. As reflected in table 1, we estimate that 70 respondents will submit 101 requests for priority review designation annually. We assume an average of 30 hours is needed to prepare such a request.

We estimate that 119 respondents will submit 156 requests for breakthrough designation annually and assume that an average of 70 hours is needed to prepare such a request.

We estimate 205 respondents will submit 261 requests for fast track designation requests annually and assume that an average of 60 hours is needed to prepare such a request.

Of the requests for fast track designation made per year, we granted approximately 224 requests from 392 respondents, and for each of these granted requests, a premeeting package was submitted. We therefore assume an average burden of 100 hours per respondent for preparing a premeeting package.

Finally, we estimate 33 respondents will submit 38 requests for RMAT designation and assume that an average of 60 hours is needed to prepare such a request.