S12 Nonclinical Biodistribution Considerations for Gene Therapy Products; International Council for Harmonisation; Draft Guidance for Industry; Availability, 50536-50538 [2021-19410]
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BILLING CODE 6730–02–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2021–D–0875]
S12 Nonclinical Biodistribution
Considerations for Gene Therapy
Products; International Council for
Harmonisation; Draft Guidance for
Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
BILLING CODE 6715–01–P
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘S12
Nonclinical Biodistribution
Considerations for Gene Therapy
Products.’’ The draft guidance was
prepared under the auspices of the
International Council for Harmonisation
(ICH), formerly the International
Conference on Harmonisation. The draft
guidance provides harmonized
recommendations for the conduct and
overall design of nonclinical
biodistribution (BD) studies for gene
therapy (GT) products. Considerations
for interpretation and application of the
BD data to support a nonclinical
development program and inform the
design of clinical trials are also
provided. The recommendations in the
guidance endeavour to facilitate the
development of investigational GT
products, while avoiding unnecessary
use of animals, in accordance with the
3Rs (reduce/refine/replace) principles.
The draft guidance is intended to
promote harmonization of
recommendations for BD studies for
investigational GT products and
facilitate a more efficient and timely
nonclinical development program.
DATES: Submit either electronic or
written comments on the draft guidance
by November 8, 2021 to ensure that the
Agency considers your comment on this
SUMMARY:
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draft guidance before it begins work on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–
2021–D–0875 for ‘‘S12 Nonclinical
Biodistribution Considerations for Gene
Therapy Products.’’ Received comments
will be placed in the docket and, except
for those submitted as ‘‘Confidential
Submissions,’’ are 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
E:\FR\FM\09SEN1.SGM
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Federal Register / Vol. 86, No. 172 / Thursday, September 9, 2021 / Notices
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 this 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, or to the
Division of Drug Information, Center for
Drug Evaluation and Research, Food
and Drug Administration, 10001 New
Hampshire Ave., Hillandale Building,
4th Floor, Silver Spring, MD 20993–
0002. Send one self-addressed adhesive
label to assist that 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 guidance document.
FOR FURTHER INFORMATION CONTACT:
Regarding the guidance: Stephen
Ripley, Center for Biologics Evaluation
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19:38 Sep 08, 2021
Jkt 253001
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 71, Rm. 7301, Silver Spring,
MD 20993–0002, 240–402–7911.
Regarding the ICH: Jill Adleberg,
Center for Drug Evaluation and
Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 51, Rm. 6364, Silver Spring,
MD 20993–0002, 301–796–5259,
Jill.Adleberg@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft guidance for industry entitled
‘‘S12 Nonclinical Biodistribution
Considerations for Gene Therapy
Products.’’ The draft guidance was
prepared under the auspices of ICH. ICH
has the mission of achieving greater
regulatory harmonization worldwide to
ensure that safe, effective, high-quality
medicines are developed, registered,
and maintained in the most resourceefficient manner.
By harmonizing the regulatory
requirements in regions around the
world, ICH guidelines have
substantially reduced duplicative
clinical studies, prevented unnecessary
animal studies, standardized the
reporting of important safety
information, standardized marketing
application submissions, and made
many other improvements in the quality
of global drug development and
manufacturing and the products
available to patients.
The six Founding Members of the ICH
are FDA; the Pharmaceutical Research
and Manufacturers of America; the
European Commission; the European
Federation of Pharmaceutical Industries
Associations; the Japanese Ministry of
Health, Labour, and Welfare; and the
Japanese Pharmaceutical Manufacturers
Association. The Standing Members of
the ICH Association include Health
Canada and Swissmedic. Additionally,
the Membership of ICH has expanded to
include other regulatory authorities and
industry associations from around the
world (refer to https://www.ich.org/).
ICH works by involving technical
experts from both regulators and
industry parties in detailed technical
harmonization work and the application
of a science-based approach to
harmonization through a consensusdriven process that results in the
development of ICH guidelines. The
regulators around the world are
committed to consistently adopting
these consensus-based guidelines,
realizing the benefits for patients and for
industry.
As a Founding Regulatory Member of
ICH, FDA plays a major role in the
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development of each of the ICH
guidelines, which FDA then adopts and
issues as guidance for industry. FDA’s
guidance documents do not establish
legally enforceable responsibilities.
Instead, they describe the Agency’s
current thinking on a topic and should
be viewed only as recommendations,
unless specific regulatory or statutory
requirements are cited.
In June 2021, the ICH Assembly
endorsed the draft guideline entitled
‘‘S12 Nonclinical Biodistribution
Considerations for Gene Therapy
Products’’ and agreed that the guidance
should be made available for public
comment. The draft guidance is the
product of the Safety Expert Working
Group of the ICH. Comments about this
draft guidance will be considered by
FDA and the Safety Expert Working
Group.
FDA is thus announcing the
availability of a draft guidance for
industry entitled ‘‘S12 Nonclinical
Biodistribution Considerations for Gene
Therapy Products.’’ The draft guidance
discusses the definition of BD and gives
examples of GT products that are within
the scope of the document. The
guidance also provides
recommendations for the design of
nonclinical BD studies, such as the test
article and dose level(s) administered,
the animal species tested, route of
administration, biofluid and tissue
collection procedure, and other design
elements. In addition, specific
considerations, such as BD assay
methods, GT expression product levels,
and product immunogenicity are
provided. The recommendations in the
draft guidance are intended to promote
harmonization in the development of a
nonclinical development program for
GT products to support clinical trial
design, and reduce the use of animals,
in accordance with the 3Rs (reduce/
refine/replace) principles.
This draft guidance has been left in
the original ICH format. The final
guidance will be reformatted and edited
to conform with FDA’s good guidance
practices regulation (21 CFR 10.115) and
style before publication. The draft
guidance, when finalized, will represent
the current thinking of FDA on ‘‘S12
Nonclinical Biodistribution
Considerations for 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
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Federal Register / Vol. 86, No. 172 / Thursday, September 9, 2021 / Notices
previously approved FDA collections of
information. Therefore, clearance by the
Office of Management and Budget
(OMB) under the Paperwork Reduction
Act of 1995 (PRA) (44 U.S.C. 3501–
3521) is not required for this guidance.
The previously approved collections of
information are subject to review by
OMB under the PRA. The collections of
information in 21 CFR part 314 have
been approved under OMB control
number 0910–0001 and in 21 CFR part
601 under OMB control number 0910–
0338.
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at https://
www.regulations.gov, https://
www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.fda.gov/vaccines-blood-biologics/
guidance-compliance-regulatoryinformation-biologics/biologicsguidances.
Dated: September 2, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for
Policy.
[FR Doc. 2021–19410 Filed 9–8–21; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2021–N–0352]
Intent To Prepare an Environmental
Impact Statement for Certain
Sunscreen Drug Products for Over-theCounter Use; Reopening of the
Comment Period
AGENCY:
Food and Drug Administration,
HHS.
Notice of intent; reopening of
the comment period.
ACTION:
The Food and Drug
Administration (FDA or Agency) is
reopening the comment period for
public scoping on the environmental
impact statement (EIS) described in the
notice entitled ‘‘Intent To Prepare an
Environmental Impact Statement for
Certain Sunscreen Drug Products for
Over-the-Counter Use’’ that appeared in
the Federal Register of May 13, 2021.
The Agency is taking this action to
allow interested persons additional time
to submit comments.
DATES: FDA is reopening the comment
period for public scoping on the EIS
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SUMMARY:
VerDate Sep<11>2014
19:38 Sep 08, 2021
Jkt 253001
identified in the notice published May
13, 2021 (86 FR 26224). To ensure the
Agency considers your comments on the
draft EIS, submit either electronic or
written comments on the scoping
process discussed in the notice by
September 23, 2021.
ADDRESSES: You may submit comments
as follows. Please note that late,
untimely filed comments will not be
considered. Electronic comments must
be submitted on or before September 23,
2021. The https://www.regulations.gov
electronic filing system will accept
comments until 11:59 p.m. Eastern Time
at the end of September 23, 2021.
Comments received by mail/hand
delivery/courier (for written/paper
submissions) will be considered timely
if they are postmarked or the delivery
service acceptance receipt is on or
before that date.
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
PO 00000
Frm 00015
Fmt 4703
Sfmt 4703
identified, as confidential, if submitted
as detailed in ‘‘Instructions.’’
Instructions: All submissions received
must include the Docket No. FDA–
2021–N–0352 for ‘‘Intent To Prepare an
Environmental Impact Statement for
Certain Sunscreen Drug Products for
Over-the-Counter Use.’’ Received
comments, those filed in a timely
manner (see ADDRESSES), 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-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.
FOR FURTHER INFORMATION CONTACT:
Trang Q. Tran, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
E:\FR\FM\09SEN1.SGM
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]]>Agencies
[Federal Register Volume 86, Number 172 (Thursday, September 9, 2021)]
[Notices]
[Pages 50536-50538]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-19410]
=======================================================================
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-0875]
S12 Nonclinical Biodistribution Considerations for Gene Therapy
Products; International Council for Harmonisation; 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 guidance for industry entitled ``S12
Nonclinical Biodistribution Considerations for Gene Therapy Products.''
The draft guidance was prepared under the auspices of the International
Council for Harmonisation (ICH), formerly the International Conference
on Harmonisation. The draft guidance provides harmonized
recommendations for the conduct and overall design of nonclinical
biodistribution (BD) studies for gene therapy (GT) products.
Considerations for interpretation and application of the BD data to
support a nonclinical development program and inform the design of
clinical trials are also provided. The recommendations in the guidance
endeavour to facilitate the development of investigational GT products,
while avoiding unnecessary use of animals, in accordance with the 3Rs
(reduce/refine/replace) principles. The draft guidance is intended to
promote harmonization of recommendations for BD studies for
investigational GT products and facilitate a more efficient and timely
nonclinical development program.
DATES: Submit either electronic or written comments on the draft
guidance by November 8, 2021 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-2021-D-0875 for ``S12 Nonclinical Biodistribution Considerations
for Gene Therapy Products.'' Received comments will be placed in the
docket and, except for those submitted as ``Confidential Submissions,''
are 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
[[Page 50537]]
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 this 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, or to the Division of Drug Information, Center for Drug
Evaluation and Research, Food and Drug Administration, 10001 New
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD
20993-0002. Send one self-addressed adhesive label to assist that
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
guidance document.
FOR FURTHER INFORMATION CONTACT:
Regarding the guidance: Stephen Ripley, 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.
Regarding the ICH: Jill Adleberg, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
51, Rm. 6364, Silver Spring, MD 20993-0002, 301-796-5259,
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``S12 Nonclinical Biodistribution Considerations for Gene
Therapy Products.'' The draft guidance was prepared under the auspices
of ICH. ICH has the mission of achieving greater regulatory
harmonization worldwide to ensure that safe, effective, high-quality
medicines are developed, registered, and maintained in the most
resource-efficient manner.
By harmonizing the regulatory requirements in regions around the
world, ICH guidelines have substantially reduced duplicative clinical
studies, prevented unnecessary animal studies, standardized the
reporting of important safety information, standardized marketing
application submissions, and made many other improvements in the
quality of global drug development and manufacturing and the products
available to patients.
The six Founding Members of the ICH are FDA; the Pharmaceutical
Research and Manufacturers of America; the European Commission; the
European Federation of Pharmaceutical Industries Associations; the
Japanese Ministry of Health, Labour, and Welfare; and the Japanese
Pharmaceutical Manufacturers Association. The Standing Members of the
ICH Association include Health Canada and Swissmedic. Additionally, the
Membership of ICH has expanded to include other regulatory authorities
and industry associations from around the world (refer to https://www.ich.org/).
ICH works by involving technical experts from both regulators and
industry parties in detailed technical harmonization work and the
application of a science-based approach to harmonization through a
consensus-driven process that results in the development of ICH
guidelines. The regulators around the world are committed to
consistently adopting these consensus-based guidelines, realizing the
benefits for patients and for industry.
As a Founding Regulatory Member of ICH, FDA plays a major role in
the development of each of the ICH guidelines, which FDA then adopts
and issues as guidance for industry. FDA's guidance documents do not
establish legally enforceable responsibilities. Instead, they describe
the Agency's current thinking on a topic and should be viewed only as
recommendations, unless specific regulatory or statutory requirements
are cited.
In June 2021, the ICH Assembly endorsed the draft guideline
entitled ``S12 Nonclinical Biodistribution Considerations for Gene
Therapy Products'' and agreed that the guidance should be made
available for public comment. The draft guidance is the product of the
Safety Expert Working Group of the ICH. Comments about this draft
guidance will be considered by FDA and the Safety Expert Working Group.
FDA is thus announcing the availability of a draft guidance for
industry entitled ``S12 Nonclinical Biodistribution Considerations for
Gene Therapy Products.'' The draft guidance discusses the definition of
BD and gives examples of GT products that are within the scope of the
document. The guidance also provides recommendations for the design of
nonclinical BD studies, such as the test article and dose level(s)
administered, the animal species tested, route of administration,
biofluid and tissue collection procedure, and other design elements. In
addition, specific considerations, such as BD assay methods, GT
expression product levels, and product immunogenicity are provided. The
recommendations in the draft guidance are intended to promote
harmonization in the development of a nonclinical development program
for GT products to support clinical trial design, and reduce the use of
animals, in accordance with the 3Rs (reduce/refine/replace) principles.
This draft guidance has been left in the original ICH format. The
final guidance will be reformatted and edited to conform with FDA's
good guidance practices regulation (21 CFR 10.115) and style before
publication. The draft guidance, when finalized, will represent the
current thinking of FDA on ``S12 Nonclinical Biodistribution
Considerations for 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
[[Page 50538]]
previously approved FDA collections of information. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. The previously approved collections of
information are subject to review by OMB under the PRA. The collections
of information in 21 CFR part 314 have been approved under OMB control
number 0910-0001 and in 21 CFR part 601 under OMB control number 0910-
0338.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at https://www.regulations.gov, https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances.
Dated: September 2, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-19410 Filed 9-8-21; 8:45 am]
BILLING CODE 4164-01-P
