Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry; Availability, 34251-34253 [2024-09287]
Download as PDF
<![CDATA[
34251
Federal Register / Vol. 89, No. 84 / Tuesday, April 30, 2024 / Notices
ANNUAL BURDEN—CURRENTLY APPROVED INFORMATION COLLECTIONS—Continued
Total
number of
respondents
Instrument
CCWIS Self-Assessment: Design Requirements ................................
CCWIS Self-Assessment: Financial ....................................................
CCWIS Self-Assessment: ....................................................................
Reporting ..............................................................................................
CCWIS Self-Assessment: Security ......................................................
CCWIS Self-Assessment: Title IV–E Foster Care Maintenance Eligibility ..................................................................................................
CCWIS Self-Assessment: User Experience ........................................
Total number
of responses
per
respondent
(3 years)
Average
burden
hours per
response
Total
burden
hours
Annual
burden
hours
55
55
1
1
24
10
1320
550
440
183
55
55
1
1
10
10
550
550
183
183
55
55
1
1
10
10
550
550
183
183
9020
3,002
Total Annual Burden for Currently Approved Generics
ANNUAL BURDEN—POTENTIAL ADDITIONAL INFORMATION COLLECTION REQUESTS
Instrument
Total
number of
respondents
Total number
of responses
per
respondent
(3 years)
Average
burden
hours per
response
Total
burden
hours
Annual
burden
hours
Future Tools to be developed ..............................................................
55
1
10
550
183
Authority: 5 U.S.C. 301; 42 U.S.C.
470, 620 et seq., 622(b), 629b(a), 652(b),
654A, 670 et seq., 671(a), 1302, and
1396a(a).
Mary C. Jones,
ACF/OPRE Certifying Officer.
[FR Doc. 2024–09226 Filed 4–29–24; 8:45 am]
BILLING CODE 4184–25–P
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Medical Products; Draft Guidance for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.
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Draft Guidance for Industry.’’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 cellderived particles. The draft guidance
document provides sponsors of
allogeneic cell-based medical products
lotter on DSK11XQN23PROD with NOTICES1
SUMMARY:
VerDate Sep<11>2014
17:27 Apr 29, 2024
Jkt 262001
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:
• 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
PO 00000
Frm 00055
Fmt 4703
Sfmt 4703
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–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 https://www.regulations.gov
or at the Dockets Management Staff
between 9 a.m. and 4 p.m., Monday
through Friday, 240–402–7500.
E:\FR\FM\30APN1.SGM
30APN1
lotter on DSK11XQN23PROD with NOTICES1
34252
Federal Register / Vol. 89, No. 84 / Tuesday, April 30, 2024 / Notices
• 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-201509-18/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. 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
VerDate Sep<11>2014
17:27 Apr 29, 2024
Jkt 262001
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 cellderived 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
PO 00000
Frm 00056
Fmt 4703
Sfmt 4703
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 TissueEngineered 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
E:\FR\FM\30APN1.SGM
30APN1
Federal Register / Vol. 89, No. 84 / Tuesday, April 30, 2024 / Notices
been approved under OMB control
number 0910–0543.
ADDRESSES:
You may submit comments
on any guidance at any time as follows:
III. Electronic Access
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’’).
Persons with access to the internet
may obtain the draft guidance at https://
www.fda.gov/vaccines-blood-biologics/
guidance-compliance-regulatoryinformation-biologics/biologicsguidances,https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2024–D–1244]
Considerations for the Use of Humanand Animal-Derived Materials and
Components in the Manufacture of Cell
and Gene Therapy and TissueEngineered Medical Products; Draft
Guidance for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
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.’’
The draft guidance document provides
manufacturers of cellular and gene
therapy (CGT) and tissue-engineered
medical products (TEMPs) with
recommendations regarding assuring the
safety, quality, and identity of materials
of human and animal origin used in the
manufacture of these products. In
addition, recommendations are
provided regarding the chemistry,
manufacturing, and control (CMC)
information submitted in an
investigational new drug application
(IND) relating to the use of human- and
animal-derived materials.
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.
lotter on DSK11XQN23PROD with NOTICES1
SUMMARY:
VerDate Sep<11>2014
17:27 Apr 29, 2024
Jkt 262001
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–1244 for ‘‘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.’’
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
PO 00000
Frm 00057
Fmt 4703
Sfmt 4703
34253
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-201509-18/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. 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,
E:\FR\FM\30APN1.SGM
30APN1
]]>Agencies
[Federal Register Volume 89, Number 84 (Tuesday, April 30, 2024)]
[Notices]
[Pages 34251-34253]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-09287]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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:
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-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 https://www.regulations.gov or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday, 240-402-7500.
[[Page 34252]]
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. 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 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: 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
