2020-25414. Agency Information Collection Activities; Proposed Collection; Comment Request; Expedited Programs for Serious Conditions-Drugs and Biologics

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA or Agency) is announcing an opportunity for public comment on the proposed collection of certain information by the Agency. Under the Paperwork Reduction Act of 1995 (PRA), Federal Agencies are required to publish notice in the Federal Register concerning each proposed collection of information, including each proposed revision of an existing collection of information, and to allow 60 days for public comment in response to the notice. This notice solicits comments on information collection pertaining to “Expedited Programs for Serious Conditions—Drugs and Biologics.”

DATES:

Submit either electronic or written comments on the collection of information by January 19, 2021.

ADDRESSES:

You may submit comments as follows. Please note that late, untimely filed comments will not be considered. Electronic comments must be submitted on or before January 19, 2021. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of January 19, 2021. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are postmarked or the delivery service acceptance receipt is on or before that date.

Electronic Submissions

Submit electronic comments in the following way:

• Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else's Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov.
• If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

Written/Paper Submissions

Submit written/paper submissions as follows:

• Mail/Hand Delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
• For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.”

Instructions: All submissions received must include the Docket No. FDA-2013-D-0575 for “Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry on Expedited Programs for Serious Conditions—Drugs and Biologics.” Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.

• Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states “THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.” The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as “confidential.” Any information marked as “confidential” will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA's posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.govinfo.gov/​content/​pkg/​FR-2015-09-18/​pdf/​2015-23389.pdf.

Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the “Search” box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION CONTACT:

Domini Bean, Office of Operations, Food and Drug Administration, Three White Flint North, 10A-12M, 11601 Landsdown St., North Bethesda, MD 20852, 301-796-5733, [e-mail].

SUPPLEMENTARY INFORMATION:

Under the PRA (44 U.S.C. 3501-3521), Federal Agencies must obtain approval from the Office of Management and Budget (OMB) for each collection of information they conduct or sponsor. “Collection of information” is defined in 44 U.S.C. 3502(3) and 5 CFR 1320.3(c) and includes Agency requests or requirements that members of the public submit reports, keep records, or provide information to a third party. Section 3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A)) requires Federal Agencies to provide a 60-day notice in the Federal Register concerning each proposed collection of information, including each proposed revision of an existing collection of information, before submitting the collection to OMB for approval. To comply with this requirement, FDA is publishing notice of the proposed collection of information set forth in this document.

With respect to the following collection of information, FDA invites comments on these topics: (1) Whether the proposed collection of information is necessary for the proper performance of FDA's functions, including whether the information will have practical utility; (2) the accuracy of FDA's estimate of the burden of the proposed collection of information, including the validity of the methodology and assumptions used; (3) ways to enhance the quality, utility, and clarity of the information to be collected; and (4) ways to minimize the burden of the collection of information on respondents, including through the use of automated collection techniques, when appropriate, and other forms of information technology.

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 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 (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 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.

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

Table 1—Estimated Annual Reporting Burden ¹

Activity	Number of respondents	Number of responses per respondent	Total annual responses	Average burden per response	Total hours
Priority Review Designation Requests	70	1.44	101	30	3,030
Breakthrough Therapy Designation Requests	119	1.31	156	70	10,920
Fast Track Designation Requests	205	1.273	261	60	15,660
RMAT Designation Requests	33	1.15	38	60	2,280
Fast Track Premeeting Packages	224	1.75	392	100	39,200
Total			948		71,090
1 There are no capital costs or operating and maintenance costs associated with this collection of information.

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.

Dated: November 12, 2020.

Lauren K. Roth,

Acting Principal Associate Commissioner for Policy.