Guidance: Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products

Postapproval Methods To Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry; Availability

DATES:

Submit either electronic or written comments on the draft guidance by December 24, 2025 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.

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No. FDA-2025-D-3049 for "Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry." 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 draft 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. 3103, Silver Spring, MD 20993-0002. Send one self-addressed adhesive label to assist the office in processing your requests. The draft guidance may also be obtained by mail by calling CBER at 800-835-4709 or 240-402-8010 or emailing [email protected]. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a draft document entitled "Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry." The draft guidance discusses methods and approaches for capturing postapproval safety and efficacy data for CGT products. Given the potential for long-lasting effects of CGT products, and the generally limited number of participants treated in clinical trials, postapproval monitoring is important for gathering data on product safety and effectiveness over time. Additional postapproval efficacy and safety data is essential to better understand the long-term safety and effectiveness of CGT products and address any concerns that emerge. The guidance does not address data collected for the purpose of expanding clinical indications.

The guidance was created as part of FDA's response to the Prescription Drug User Fee Act (PDUFA VII) commitment to increase efficiency and to support development of CGT products. The Agency held a public meeting on April 27, 2023, entitled "Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products" and solicited comments from the public to Docket No. FDA-2023-N-0398. FDA published a report of the meeting on October 18, 2023. Stakeholder input presented during the meeting and comments received to the docket were used to inform the content of this guidance.

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 "Postapproval Methods to Capture Safety and Efficacy Data for Cell 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.

As we develop final guidance on this topic, FDA will consider comments on costs or cost savings the guidance may generate, relevant for Executive Order 14192.

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 11 pertaining to electronic records and signatures have been approved under OMB control number 0910-0303. The collections of information in 21 CFR parts 50 and 56 pertaining to the protection of human subjects, informed consent, and institutional review boards have been approved under OMB control number 0910-0130. The collections of information in 21 CFR part 600 for general records and adverse experience reporting pertaining to biological products have been approved under OMB control number 0910-0308.

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, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.