[Federal Register Volume 89, Number 223 (Tuesday, November 19, 2024)]
[Notices]
[Pages 91404-91405]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-26918]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-D-4311]
Frequently Asked Questions--Developing Potential 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Gene Therapy Products; Draft Guidance for Industry; Availability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 or Agency) is announcing
the availability of a draft document entitled ``Frequently asked
Questions--Developing Potential Cellular and Gene Therapy Products.''
The draft guidance document provides industry with answers to
frequently asked questions (FAQs) and commonly faced issues that arise
during the development of cellular and gene therapy (CGT) products. The
FAQs represent common questions directed to the Agency and span
multiple disciplines, including regulatory review; chemistry,
manufacturing, and controls (CMC); pharmacology/toxicology; clinical;
and clinical pharmacology.
DATES: Submit either electronic or written comments on the draft
guidance by February 18, 2025 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final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.
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-2024-D-4311 for ``Frequently asked Questions--Developing Potential
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.
[[Page 91405]]
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. 3128, 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 1-800-835-4709 or 240-402-8010. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
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
``Frequently asked Questions--Developing Potential Cellular and Gene
Therapy Products.'' The draft guidance document provides industry with
answers to FAQs and commonly faced issues that arise during the
development of CGT products. The FAQs represent common questions
directed to the Agency and span multiple disciplines, including
regulatory review; CMC; pharmacology/toxicology; clinical; and clinical
pharmacology.
The guidance was created as part of FDA's response to the PDUFA VII
commitment to increase efficiency and to support development of CGT
products by providing a repository of common questions posed to the
Office of Therapeutic Products by sponsors and other key stakeholders.
The Agency compiled FAQs received from a variety of sources, including
FDA interactions with sponsors in development programs.
The guidance covers relevant, current, and timely topics related to
the development of CGT products, which may be updated to include
additional FAQs as appropriate. Sponsors are encouraged to visit the
Cellular and Gene Therapy Guidances web page on the FDA website for a
full list of finalized as well as draft guidances relevant to the
development of CGT products.
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 ``Frequently
asked Questions--Developing Potential 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
Action of 1995 (44 U.S.C. 3501-3521). The collections of information in
21 CFR part 312 pertaining to investigational new drug applications,
clinical trials and clinical trial design, meetings with FDA, and Form
FDA 1571, have been approved under OMB control number 0910-0014. The
collections of information in section 402(j)(5)(B) of the Public Health
Service Act (42 U.S.C. 282(j)(5)(B)), which requires certification that
all applicable requirements of section 402(j) have been met on Form FDA
3674, and the collections of information in 21 CFR part 601 pertaining
to the submission of biologics license applications and Form FDA 356h
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, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or
https://www.regulations.gov.
Dated: November 7, 2024.
Kimberlee Trzeciak,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2024-26918 Filed 11-18-24; 8:45 am]
BILLING CODE 4164-01-P