Notice2024-01789
Considerations for the Development of Chimeric Antigen Receptor T Cell Products; Guidance for Industry; Availability
Primary source
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Published
January 30, 2024
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled "Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products; Guidance for Industry." The guidance is intended to assist sponsors, including industry and academic sponsors, developing ex vivo- manufactured CAR T cell products. The guidance provides CAR T cell specific recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and design of clinical studies for oncology indications (including hematologic malignancies and solid tumors). The guidance announced in this notice finalizes the draft guidance of the same title dated March 2022.
Full Text
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<title>Federal Register, Volume 89 Issue 20 (Tuesday, January 30, 2024)</title>
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[Federal Register Volume 89, Number 20 (Tuesday, January 30, 2024)]
[Notices]
[Pages 5910-5911]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-01789]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-0404]
Considerations for the Development of Chimeric Antigen Receptor T
Cell Products; 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 final guidance entitled ``Considerations for the
Development of Chimeric Antigen Receptor (CAR) T Cell Products;
Guidance for Industry.'' The guidance is intended to assist sponsors,
including industry and academic sponsors, developing ex vivo-
manufactured CAR T cell products. The guidance provides CAR T cell
specific recommendations regarding chemistry, manufacturing, and
control (CMC), pharmacology and toxicology, and design of clinical
studies for oncology indications (including hematologic malignancies
and solid tumors). The guidance announced in this notice finalizes the
draft guidance of the same title dated March 2022.
DATES: The announcement of the guidance is published in the Federal
Register on January 30, 2024.
ADDRESSES: You may submit either electronic or written comments on
Agency guidances at any time as follows:
Electronic Submissions
Submit electronic comments in the following way:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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-2021-D-0404 for ``Considerations for the Development of Chimeric
Antigen Receptor (CAR) T Cell Products; Guidance for Industry.''
Received comments will be placed in the docket and, except for those
submitted as ``Confidential Submissions,'' publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday, 240-402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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: Tami Belouin, 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 document entitled
``Considerations for the Development of Chimeric Antigen Receptor (CAR)
T Cell Products; Guidance for Industry.'' The guidance is intended to
assist sponsors, including industry and academic sponsors, developing
ex vivo-manufactured CAR T cell products. The guidance provides CAR T
cell specific recommendations regarding CMC, pharmacology and
toxicology, and design of clinical studies for oncology indications
(including hematologic malignancies and solid tumors). Recommendations
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specific to autologous or allogeneic CAR T cell products are noted in
the guidance. The guidance also provides recommendations for analytical
comparability studies for CAR T cell products. While the guidance
specifically focuses on CAR T cell products, some of the information
and recommendations provided may also be applicable to other
genetically modified lymphocyte products, such as CAR Natural Killer
cells or T cell receptor modified T cells.
In the Federal Register of March 16, 2022 (87 FR 14893), FDA
announced the availability of the draft guidance of the same title
dated March 2022. FDA received numerous comments on the draft guidance
and those comments were considered as the guidance was finalized.
Changes to the guidance include clarifying the scope, focusing on
treatment for oncology indications, and the recommendations for CAR T
cells manufactured using cellular starting material from patients who
have received CAR T cells previously, potency for CAR T cells that
express multiple transgene elements, stability studies, and clinical
monitoring. In addition, editorial changes were made to improve
clarity. The guidance announced in this notice finalizes the draft
guidance dated March 2022.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of another human gene therapy final guidance entitled
``Human Gene Therapy Products Incorporating Human Genome Editing;
Guidance for Industry.''
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Considerations for the Development of
Chimeric Antigen Receptor (CAR) T Cell 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 (44 U.S.C. 3501-3521). The collections of information in 21
CFR part 50 have been approved under OMB control number 0910-0130; 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
part 312 have been approved under OMB control number 0910-0014; the
collections of information in 21 CFR part 601 have been approved under
OMB control number 0910-0338; and the collections of information in 21
CFR part 1271 have been approved under OMB control number 0910-0543.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
<a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or
<a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Date: January 24, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-01789 Filed 1-29-24; 8:45 am]
BILLING CODE 4164-01-P
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