Potency Assurance for Cellular and Gene Therapy Products; Draft Guidance for Industry; Availability, 89707-89709 [2023-28596]
Download as PDF
Federal Register / Vol. 88, No. 248 / Thursday, December 28, 2023 / Notices
FOR FURTHER INFORMATION CONTACT:
khammond on DSKJM1Z7X2PROD with NOTICES
Ranjani Prabhakara, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 75, Rm. 6648,
Silver Spring, MD 20993–0002, 240–
402–4652.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a revised draft guidance for industry
entitled ‘‘Quality Considerations for
Topical Ophthalmic Drug Products.’’
This revised draft guidance provides
information regarding quality
considerations for ophthalmic drug
products consistent with the
requirements outlined in section
501(a)(2)(B) of the Federal Food, Drug,
and Cosmetic Act (FD&C Act) (21 U.S.C.
351(a)(2)(B)) and 21 CFR parts 210 and
211 for all drug products, 21 CFR part
601 for biological products, 21 CFR part
4 for combination products, and, for
ophthalmic drug products with a U.S.
Pharmacopeia (USP) monograph, the
applicable criteria from the USP. The
revised draft guidance also provides
recommendations to industry on the
documentation that should be submitted
in the chemistry, manufacturing, and
controls (CMC) section of NDAs,
ANDAs, and BLAs for certain CMC
attributes for ophthalmic drug products.
This revised draft guidance revises
the guidance of the same name
published on October 13, 2023 (88 FR
70997). FDA is revising this draft
guidance to address microbiological
considerations related to product
sterility for all ophthalmic drug
products subject to current good
manufacturing practice (CGMP)
requirements and the prevention of
contamination of ophthalmic drug
products packaged in multidose
containers, given several recent recalls
of ophthalmic drug products and
instances of consumer injury and death
from microbiologically contaminated
ophthalmic drug products.
FDA is also revising the draft
guidance to clarify its stated scope. As
originally published, the scope
explicitly included NDA, ANDA, and
BLA products regulated by the Center
for Drug Evaluation and Research; OTC
monograph drugs marketed under
section 505G of the FD&C Act (21 U.S.C.
355h); and combination products. It was
not FDA’s intention to specifically
exclude products that are not marketed
under an approved application or under
section 505G of the FD&C Act; however,
the draft guidance may have been
interpreted that way. Therefore, FDA is
clarifying that the guidance also applies
to other drugs that, while also subject to
VerDate Sep<11>2014
20:14 Dec 27, 2023
Jkt 262001
CGMP requirements, are not marketed
under a drug application, including
drugs compounded by outsourcing
facilities pursuant to section 503B of the
FD&C Act (21 U.S.C. 353b).
This revised draft guidance is being
issued consistent with FDA’s good
guidance practices regulation (21 CFR
10.115). The revised draft guidance,
when finalized, will represent the
current thinking of FDA on ‘‘Quality
Considerations for Topical Ophthalmic
Drug 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. Previous Submission of Comments
In commenting on this revised draft
guidance, you do not need to reiterate
comments that you previously
submitted regarding the draft guidance
issued on October 13, 2023. Your
previously submitted comments will
still be considered. You may instead
submit updates to previously submitted
comments, as needed, and comments
related to the new section on
microbiological considerations and the
clarified scope of this revised draft
guidance.
III. 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 314 for
NDAs and ANDAs have been approved
under OMB control number 0910–0001.
The collections of information in 21
CFR part 601 for BLAs have been
approved under OMB control number
0910–0338. The collections of
information in 21 CFR parts 210 and
211 pertaining to CGMP have been
approved under OMB control number
0910–0139. The collections of
information in 21 CFR 201.56 and
201.57 relating to certain prescription
product labeling requirements have
been approved under OMB control
number 0910–0572. The collections of
information for section 351(k)
submission of the Public Health Service
Act (42 U.S.C. 262(k)) have been
approved under OMB control number
0910–0718. The collections of
information pertaining to human drug
compounding under section 503B of the
FD&C Act have been approved under
OMB control number 0910–0858.
PO 00000
Frm 00054
Fmt 4703
Sfmt 4703
89707
IV. Electronic Access
Persons with access to the internet
may obtain the revised draft guidance at
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
Dated: December 21, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–28595 Filed 12–27–23; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2023–D–4299]
Potency Assurance for Cellular and
Gene Therapy Products; Draft
Guidance for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA, Agency, or we) is
announcing the availability of a draft
guidance entitled ‘‘Potency Assurance
for Cellular and Gene Therapy
Products.’’ FDA is issuing this draft
guidance to provide recommendations
to help assure the potency of human
cellular therapy or gene therapy (CGT)
products at all stages of the product
lifecycle. FDA is recommending a
comprehensive approach to potency
assurance of CGT products that is
grounded in quality risk management.
For investigational products, we
describe how to progressively
implement a strategy for potency
assurance during product development
and provide additional considerations
to help assure the potency of products
that are undergoing rapid clinical
development. For licensed products, we
describe requirements for potency
assurance, including testing required for
lot release.
DATES: Submit either electronic or
written comments on the draft guidance
by March 27, 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:
SUMMARY:
Electronic Submissions
Submit electronic comments in the
following way:
E:\FR\FM\28DEN1.SGM
28DEN1
89708
Federal Register / Vol. 88, No. 248 / Thursday, December 28, 2023 / Notices
khammond on DSKJM1Z7X2PROD with NOTICES
• 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–
2023–D–4299 for ‘‘Potency Assurance
for 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
VerDate Sep<11>2014
20:14 Dec 27, 2023
Jkt 262001
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 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:
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 ‘‘Potency
Assurance for Cellular and Gene
Therapy Products.’’ FDA is issuing this
draft guidance to provide
PO 00000
Frm 00055
Fmt 4703
Sfmt 4703
recommendations to help assure the
potency of human CGT products that
are regulated as biological products
under section 351 of the Public Health
Service Act (42 U.S.C. 262).
In this draft guidance, we provide
recommendations for developing a
science- and risk-based strategy to help
assure the potency of human CGT
products. A potency assurance strategy
is a multifaceted approach that reduces
risks to the potency of a product
through: (1) manufacturing process
design, (2) manufacturing process
control, (3) material control, (4) inprocess testing, and (5) potency lot
release assays. The goal of a potency
assurance strategy is to ensure that
every lot of a product released will have
the specific ability or capacity to
achieve the intended therapeutic effect.
In this draft guidance, we emphasize
that potency assays and their
corresponding acceptance criteria
should be designed to make meaningful
contributions to potency assurance by
reducing risks to product potency. We
provide illustrative examples of
approaches to potency assay
development that are grounded in
quality risk management. Due to the
diversity of CGT products and the
product-specific nature of potency
assays, the recommendations in this
draft guidance regarding the selection
and design of potency assays are
necessarily general.
This draft guidance, when finalized,
is intended to supersede the document
entitled ‘‘Guidance for Industry:
Potency Tests for Cellular and Gene
Therapy Products,’’ dated January 2011.
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 potency assurance for 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 Act of 1995 (PRA) (44 U.S.C.
3501–3521). 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 312.23 have been
E:\FR\FM\28DEN1.SGM
28DEN1
89709
Federal Register / Vol. 88, No. 248 / Thursday, December 28, 2023 / Notices
approved under OMB control number
0910–0014; the collections of
information in 21 CFR 600.14 have been
approved under OMB control number
0910–0458; and the collections of
information in 21 CFR part 601 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-regulatoryinformation-biologics/biologicsguidances, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
Dated: December 21, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–28596 Filed 12–27–23; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Resources and Services
Administration
Agency Information Collection
Activities: Proposed Collection: Public
Comment Request; Information
Collection Request Title: Advanced
Nursing Education Program Specific
Form OMB No. 0915–0375—Revision
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services.
ACTION: Notice.
AGENCY:
In compliance with the
requirement for opportunity for public
comment on proposed data collection
projects of the Paperwork Reduction Act
of 1995, HRSA announces plans to
submit an Information Collection
Request (ICR), described below, to the
Office of Management and Budget
(OMB). Prior to submitting the ICR to
OMB, HRSA seeks comments from the
public regarding the burden estimate,
below, or any other aspect of the ICR.
DATES: Comments on this ICR should be
received no later than February 26,
2024.
khammond on DSKJM1Z7X2PROD with NOTICES
SUMMARY:
Submit your comments to
paperwork@hrsa.gov or mail the HRSA
Information Collection Clearance
Officer, Room 14N39, 5600 Fishers
Lane, Rockville, Maryland 20857.
FOR FURTHER INFORMATION CONTACT: To
request more information on the
proposed project or to obtain a copy of
the data collection plans and draft
instruments, email paperwork@hrsa.gov
or call Joella Roland, the HRSA
Information Collection Clearance
Officer, at (301) 443–3983.
SUPPLEMENTARY INFORMATION: When
submitting comments or requesting
information, please include the ICR title
for reference.
Information Collection Request Title:
Advanced Nursing Education (ANE)
Program Specific Form OMB No. 0915–
0375—Revision
Abstract: HRSA provides advanced
nursing education grants to educational
institutions to increase the supply,
distribution, quality of, and access to
advanced education nurses through the
ANE Programs. The ANE Programs are
authorized by section 811 of the Public
Health Service Act (42 U.S.C. 296j), as
amended. This clearance request is for
continued approval of the information
collection OMB No. 0915–0375 with
revisions. This revision request seeks to
add the Advanced Nursing EducationNurse Practitioner Residency and
Fellowship (ANE–NPRF) Program and
the Maternity Care Nursing Workforce
Expansion Program to the ANE Program
Specific Form, and to remove programs
that have closed, which include the
Advanced Nursing Education-Nurse
Practitioner Residency (ANE–NPR)
Program and the Advanced Nursing
Education-Nurse Practitioner Residency
Integration Program. The activities
previously supported under the ANE–
NPR and the Advanced Nursing
Education-Nurse Practitioner Residency
Integration Program are now supported
under the ANE–NPRF Program.
Need and Proposed Use of the
Information: Section 811 of the Public
Health Service Act provides the
Secretary of Health and Human Services
with the authority to award grants to
and enter into contracts with eligible
entities to meet the costs of: (1) projects
that support the enhancement of
advanced nursing education and
ADDRESSES:
practice; and (2) traineeships for
individuals in advanced nursing
education programs. Under this section,
HRSA makes awards to entities who
train and support nurses characterized
as ‘‘advanced education nurses.’’ In
awarding such grants, funding
preference is given to applicants with
projects that will substantially benefit
rural or underserved populations or
help meet public health nursing needs
in state or local health departments;
special consideration is given to an
eligible entity that agrees to extend the
award to train advanced education
nurses who will practice in designated
Health Professional Shortage Areas.
The ANE Program Specific Form
allows HRSA to effectively target
funding and measure the impact of the
ANE Programs in meeting the legislative
intent and program goals of supporting
the enhancement of advanced nursing
education and creating opportunities for
individuals in advanced nursing
education programs to increase the
number of advanced practice nurses,
especially in rural and underserved
areas. Additionally, collecting this data
assists HRSA in carrying out the most
impactful program and ensuring
resources are used responsibly. The
proposed updates to this information
collection are to accurately list the
current ANE Programs.
Likely Respondents: Likely
respondents will be current ANE
Programs awardees and new applicants
to ANE Programs.
Burden Statement: Burden in this
context means the time expended by
persons to generate, maintain, retain,
disclose, or provide the information
requested. This includes the time
needed to review instructions; to
develop, acquire, install, and utilize
technology and systems for the purpose
of collecting, validating, and verifying
information, processing and
maintaining information, and disclosing
and providing information; to train
personnel and to be able to respond to
a collection of information; to search
data sources; to complete and review
the collection of information; and to
transmit or otherwise disclose the
information. The total annual burden
hours estimated for this ICR are
summarized in the table below.
TOTAL ESTIMATED ANNUALIZED BURDEN HOURS
Form name
(includes the ANE program specific tables and attachments)
Number of
respondents
Advanced Nursing Education Workforce .............................
Nurse Anesthetist Traineeship .............................................
VerDate Sep<11>2014
20:14 Dec 27, 2023
Jkt 262001
PO 00000
Frm 00056
Number of
responses per
respondent
156
64
Fmt 4703
Sfmt 4703
Total
responses
1
1
E:\FR\FM\28DEN1.SGM
156
64
28DEN1
Average
burden per
response
(in hours)
Total burden
hours
7
7
1,092
448
Agencies
[Federal Register Volume 88, Number 248 (Thursday, December 28, 2023)]
[Notices]
[Pages 89707-89709]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-28596]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-D-4299]
Potency Assurance for Cellular and Gene Therapy Products; Draft
Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA, Agency, or we) is
announcing the availability of a draft guidance entitled ``Potency
Assurance for Cellular and Gene Therapy Products.'' FDA is issuing this
draft guidance to provide recommendations to help assure the potency of
human cellular therapy or gene therapy (CGT) products at all stages of
the product lifecycle. FDA is recommending a comprehensive approach to
potency assurance of CGT products that is grounded in quality risk
management. For investigational products, we describe how to
progressively implement a strategy for potency assurance during product
development and provide additional considerations to help assure the
potency of products that are undergoing rapid clinical development. For
licensed products, we describe requirements for potency assurance,
including testing required for lot release.
DATES: Submit either electronic or written comments on the draft
guidance by March 27, 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:
[[Page 89708]]
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-2023-D-4299 for ``Potency Assurance for 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.
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 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: 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
``Potency Assurance for Cellular and Gene Therapy Products.'' FDA is
issuing this draft guidance to provide recommendations to help assure
the potency of human CGT products that are regulated as biological
products under section 351 of the Public Health Service Act (42 U.S.C.
262).
In this draft guidance, we provide recommendations for developing a
science- and risk-based strategy to help assure the potency of human
CGT products. A potency assurance strategy is a multifaceted approach
that reduces risks to the potency of a product through: (1)
manufacturing process design, (2) manufacturing process control, (3)
material control, (4) in-process testing, and (5) potency lot release
assays. The goal of a potency assurance strategy is to ensure that
every lot of a product released will have the specific ability or
capacity to achieve the intended therapeutic effect.
In this draft guidance, we emphasize that potency assays and their
corresponding acceptance criteria should be designed to make meaningful
contributions to potency assurance by reducing risks to product
potency. We provide illustrative examples of approaches to potency
assay development that are grounded in quality risk management. Due to
the diversity of CGT products and the product-specific nature of
potency assays, the recommendations in this draft guidance regarding
the selection and design of potency assays are necessarily general.
This draft guidance, when finalized, is intended to supersede the
document entitled ``Guidance for Industry: Potency Tests for Cellular
and Gene Therapy Products,'' dated January 2011.
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 potency
assurance for 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
Act of 1995 (PRA) (44 U.S.C. 3501-3521). 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 312.23 have been
[[Page 89709]]
approved under OMB control number 0910-0014; the collections of
information in 21 CFR 600.14 have been approved under OMB control
number 0910-0458; and the collections of information in 21 CFR part 601
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: December 21, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-28596 Filed 12-27-23; 8:45 am]
BILLING CODE 4164-01-P