
[Federal Register Volume 88, Number 248 (Thursday, December 28, 2023)]
[Notices]
[Pages 89707-89709]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-28596]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-D-4299]


Potency Assurance for Cellular and Gene Therapy Products; Draft 
Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA, Agency, or we) is 
announcing the availability of a draft guidance entitled ``Potency 
Assurance for Cellular and Gene Therapy Products.'' FDA is issuing this 
draft guidance to provide recommendations to help assure the potency of 
human cellular therapy or gene therapy (CGT) products at all stages of 
the product lifecycle. FDA is recommending a comprehensive approach to 
potency assurance of CGT products that is grounded in quality risk 
management. For investigational products, we describe how to 
progressively implement a strategy for potency assurance during product 
development and provide additional considerations to help assure the 
potency of products that are undergoing rapid clinical development. For 
licensed products, we describe requirements for potency assurance, 
including testing required for lot release.

DATES: Submit either electronic or written comments on the draft 
guidance by March 27, 2024 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:

[[Page 89708]]

     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-2023-D-4299 for ``Potency Assurance for Cellular and Gene Therapy 
Products.'' Received comments 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.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of the guidance to the 
Office of Communication, Outreach and Development, Center for Biologics 
Evaluation and Research (CBER), Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send 
one self-addressed adhesive label to assist the office in processing 
your requests. The guidance may also be obtained by mail by calling 
CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the guidance document.

FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft document entitled 
``Potency Assurance for Cellular and Gene Therapy Products.'' FDA is 
issuing this draft guidance to provide recommendations to help assure 
the potency of human CGT products that are regulated as biological 
products under section 351 of the Public Health Service Act (42 U.S.C. 
262).
    In this draft guidance, we provide recommendations for developing a 
science- and risk-based strategy to help assure the potency of human 
CGT products. A potency assurance strategy is a multifaceted approach 
that reduces risks to the potency of a product through: (1) 
manufacturing process design, (2) manufacturing process control, (3) 
material control, (4) in-process testing, and (5) potency lot release 
assays. The goal of a potency assurance strategy is to ensure that 
every lot of a product released will have the specific ability or 
capacity to achieve the intended therapeutic effect.
    In this draft guidance, we emphasize that potency assays and their 
corresponding acceptance criteria should be designed to make meaningful 
contributions to potency assurance by reducing risks to product 
potency. We provide illustrative examples of approaches to potency 
assay development that are grounded in quality risk management. Due to 
the diversity of CGT products and the product-specific nature of 
potency assays, the recommendations in this draft guidance regarding 
the selection and design of potency assays are necessarily general.
    This draft guidance, when finalized, is intended to supersede the 
document entitled ``Guidance for Industry: Potency Tests for Cellular 
and Gene Therapy Products,'' dated January 2011.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the current thinking of FDA on potency 
assurance for cellular and gene therapy products. It does not establish 
any rights for any person and is not binding on FDA or the public. You 
can use an alternative approach if it satisfies the requirements of the 
applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. The 
previously approved collections of information are subject to review by 
the Office of Management and Budget (OMB) under the Paperwork Reduction 
Act of 1995 (PRA) (44 U.S.C. 3501-3521). The collections of information 
in 21 CFR part 211 have been approved under OMB control number 0910-
0139; the collections of information in 21 CFR 312.23 have been

[[Page 89709]]

approved under OMB control number 0910-0014; the collections of 
information in 21 CFR 600.14 have been approved under OMB control 
number 0910-0458; and the collections of information in 21 CFR part 601 
have been approved under OMB control number 0910-0338.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, http://www.fda.gov/regulatory-information/search-fda-guidance-documents, or 
https://www.regulations.gov.

    Dated: December 21, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-28596 Filed 12-27-23; 8:45 am]
BILLING CODE 4164-01-P


