[Federal Register Volume 90, Number 184 (Thursday, September 25, 2025)]
[Notices]
[Pages 46223-46225]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2025-18650]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2025-D-3049]
Postapproval Methods To Capture Safety and Efficacy Data for Cell
and Gene Therapy Products; Draft Guidance for Industry; Availability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 document entitled ``Postapproval
Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy
Products; Draft Guidance for Industry.'' The draft guidance document
discusses methods and approaches for capturing postapproval safety and
efficacy data for cell and gene therapy (CGT) products. Given the
potential for long-lasting effects of CGT products, and the generally
limited number of participants treated in clinical trials, the
collection of postapproval study data for CGT products is important for
gathering data on product safety and effectiveness over time.
DATES: Submit either electronic or written comments on the draft
guidance by December 24, 2025 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final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.
ADDRESSES: You may submit comments on any guidance at any time as
follows:
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.
[[Page 46224]]
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.
FOR FURTHER INFORMATION CONTACT: Tami Belouin, Center for Biologics
Evaluation and Research, Food and Drug Administration, 240-402-7911.
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
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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.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2025-18650 Filed 9-24-25; 8:45 am]
BILLING CODE 4164-01-P