Notice2024-09287
Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry; Availability
Primary source
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Published
April 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of a draft document entitled "Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry." Allogeneic cells of human origin may be expanded in culture to manufacture medical products consisting of live cells, inactivated cells, cell lysates, or other cell-based materials such as cell-derived particles. The draft guidance document provides sponsors of allogeneic cell-based medical products recommendations for determining the appropriate cell safety testing to support an investigational new drug application (IND) or a biologics license application (BLA). Cell safety testing should be based on a risk analysis that considers the expansion potential of the cells, the reagents that are used to expand the cells in culture, and the number of individuals the cell-based medical product is capable of treating.
Full Text
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<title>Federal Register, Volume 89 Issue 84 (Tuesday, April 30, 2024)</title>
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[Federal Register Volume 89, Number 84 (Tuesday, April 30, 2024)]
[Notices]
[Pages 34251-34253]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-09287]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-D-1243]
Safety Testing of Human Allogeneic Cells Expanded for Use in
Cell-Based Medical Products; Draft Guidance for Industry; Availability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 ``Safety Testing of Human
Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft
Guidance for Industry.'' Allogeneic cells of human origin may be
expanded in culture to manufacture medical products consisting of live
cells, inactivated cells, cell lysates, or other cell-based materials
such as cell-derived particles. The draft guidance document provides
sponsors of allogeneic cell-based medical products recommendations for
determining the appropriate cell safety testing to support an
investigational new drug application (IND) or a biologics license
application (BLA). Cell safety testing should be based on a risk
analysis that considers the expansion potential of the cells, the
reagents that are used to expand the cells in culture, and the number
of individuals the cell-based medical product is capable of treating.
DATES: Submit either electronic or written comments on the draft
guidance by July 29, 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:
<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-2024-D-1243 for ``Safety Testing of Human Allogeneic Cells Expanded
for Use in Cell-Based Medical Products; Draft 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.
[[Page 34252]]
<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 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: 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 draft document entitled
``Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-
Based Medical Products; Draft Guidance for Industry.'' Allogeneic cells
of human origin may be expanded in culture to manufacture medical
products consisting of live cells, inactivated cells, cell lysates, or
other cell-based materials such as cell-derived particles. The draft
guidance document provides sponsors of allogeneic cell-based medical
products recommendations for determining the appropriate cell safety
testing to support an IND or a BLA. Cell safety testing should be based
on a risk analysis that considers the expansion potential of the cells,
the reagents that are used to expand the cells in culture, and the
number of individuals the cell-based medical product is capable of
treating. This guidance does not address the measurement or analysis of
cell characteristics that may be relevant to biological activity.
Viral and microbial contamination is a potential risk for all cell-
based medical products, especially when the cells are cultured
extensively during manufacturing. Contamination may be present in the
source cells, or the cells may become contaminated with adventitious
agents during manufacturing. In addition, genomic changes that result
in tumorigenic cells can occur during extensive culture.
The purpose of this draft guidance is to provide guidance on safety
testing to assist manufacturers in addressing the requirements of 21
CFR 610.18(c)(1) and 312.23(a)(7), and other relevant regulations, as
applicable, with respect to human allogeneic cells expanded for use in
cell-based medical products. FDA's recommendations for cell safety
testing reflect a risk-based approach that takes into consideration
both the specific characteristics of the cells and their proposed use.
The recommendations in this draft guidance apply to cultured
allogeneic cells, including cell banks, that are sources of the
intended constituents of the final drug product, as well as combination
products that contain an allogeneic cell or cell-based biologic
constituent part in combination with a drug and/or device. The
recommendations in this draft guidance also apply to genetically
modified allogeneic cells that have been transduced with viral and/or
plasmid vectors, and cells that have undergone genome editing. This
guidance does not apply to cell substrates that are used during
manufacturing of non-cell-based products such as viruses, gene therapy
vectors, or recombinant proteins.
The draft guidance, when finalized, is intended to supplement the
following two final guidances: ``Chemistry, Manufacturing, and Control
(CMC) Information for Human Gene Therapy Investigational New Drug
Applications (INDs); Guidance for Industry'' dated January 2020, and
``Guidance for FDA Reviewers and Sponsors: Content and Review of
Chemistry, Manufacturing, and Control (CMC) Information for Human
Somatic Cell Therapy Investigational New Drug Applications (INDs)''
dated April 2008.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of another human gene therapy final guidance document
entitled ``Considerations for the Use of Human-and Animal-Derived
Materials and Components in the Manufacture of Cell and Gene Therapy
and Tissue-Engineered Medical Products; Draft Guidance for Industry.''
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 ``Safety
Testing of Human Allogeneic Cells Expanded for Use in Cell-Based
Medical 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 draft 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 9 CFR 113.47 and 113.53 have been approved under OMB control number
0579-0013; 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; the collections of information in 21 CFR part 610
have been approved under OMB control number 0910-0139; and the
collections of information in 21 CFR part 1271 have
[[Page 34253]]
been approved under OMB control number 0910-0543.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances,https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances,https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or
<a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Dated: April 25, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-09287 Filed 4-29-24; 8:45 am]
BILLING CODE 4164-01-P
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