Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Guidance for Industry; Availability, 67046-67047 [2022-24112]

Download as PDF 67046 Federal Register / Vol. 87, No. 214 / Monday, November 7, 2022 / Notices under the PRA because it entails no burden other than that necessary to identify the respondent, the date, the respondent’s address, and the nature of the instrument (see 5 CFR 1320.3(h)(1)). TABLE 2—ESTIMATED ANNUAL RECORDKEEPING BURDEN 1 Number of recordkeepers 21 CFR part/activity Number of records per recordkeeper Total hours § 822.31; Manufacturer records ........................................... § 822.32; Investigator records .............................................. 5 15 1 1 5 15 20 5 100 75 Total .............................................................................. ........................ ........................ ........................ ........................ 175 1 There are no capital costs or operating and maintenance costs associated with this collection of information. Explanation of Recordkeeping Burden Estimate: FDA expects that at least some of the manufacturers will be able to satisfy the PS requirement using information or data they already have. For purposes of calculating burden, however, FDA has assumed that each PS order can only be satisfied by a 3-year clinically based surveillance plan, using three investigators. These estimates are based on FDA’s knowledge and experience with PS. We have adjusted our burden estimate, which has resulted in a decrease to the currently approved burden. Our estimated burden for the information collection reflects an overall decrease of 4,780 hours and a corresponding decrease of 145 responses. We believe these adjustments more accurately reflect the current number of requests associated with postmarket surveillance of medical devices. Dated: November 1, 2022. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2022–24232 Filed 11–4–22; 8:45 am] BILLING CODE 4164–01–P document provides recommendations to sponsors interested in studying multiple versions of a cellular or gene therapy product in an early phase clinical trial for a single disease. Sponsors have expressed interest in gathering preliminary evidence of safety and activity using multiple versions of a cellular or gene therapy product in a single clinical trial, where each version of the product is distinct and is generally submitted to FDA in a separate investigational new drug application (IND). The guidance provides recommendations for conducting such studies, including how to organize and structure the INDs, submit new information, and report adverse events. The guidance announced in this notice finalizes the draft guidance of the same title dated September 2021. DATES: The announcement of the guidance is published in the Federal Register on November 7, 2022. ADDRESSES: You may submit either electronic or written comments on Agency guidances at any time as follows: Electronic Submissions DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2021–D–0776] Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. khammond on DSKJM1Z7X2PROD with NOTICES Average burden per recordkeeping Total annual records ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Guidance for Industry.’’ The guidance SUMMARY: VerDate Sep<11>2014 16:45 Nov 04, 2022 Jkt 259001 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 PO 00000 Frm 00040 Fmt 4703 Sfmt 4703 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–0776 for ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; 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 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 E:\FR\FM\07NON1.SGM 07NON1 Federal Register / Vol. 87, No. 214 / Monday, November 7, 2022 / Notices 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: khammond on DSKJM1Z7X2PROD with NOTICES Jessica Gillum, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993–0002, 240– 402–7911. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a document entitled ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Guidance for Industry.’’ The guidance document provides recommendations to sponsors interested in studying multiple VerDate Sep<11>2014 16:45 Nov 04, 2022 Jkt 259001 versions of a cellular or gene therapy product in an early phase clinical trial for a single disease. Sponsors have expressed interest in gathering preliminary evidence of safety and activity using multiple versions of a cellular or gene therapy product in a single clinical trial, where each version of the product is distinct and is generally submitted to FDA in a separate IND. The objective of these early phase clinical studies is to guide which version(s) of the product to pursue for further development in later phase studies. Thus, these studies are not intended to provide primary evidence of effectiveness to support a marketing application and generally are not adequately powered to demonstrate a statistically significant difference in efficacy between the study arms. The guidance provides recommendations for conducting such studies, including how to organize and structure the INDs, submit new information, and report adverse events. In the Federal Register of September 30, 2021 (86 FR 54207), FDA announced the availability of the draft guidance entitled ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early Phase Clinical Trial.’’ FDA received several comments on the draft guidance and those comments were considered as the guidance was finalized. Changes to the guidance include clarifying how to continue the umbrella trial after a study arm has been closed and adding examples of changes that result in multiple versions of a cellular or gene therapy product. In addition, editorial changes were made to improve clarity. The guidance announced in this notice finalizes the draft guidance of the same title dated September 2021. This guidance is being issued consistent with FDA’s good guidance practices regulation (21 CFR 10.115). The guidance represents the current thinking of FDA on ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial.’’ 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. 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. PO 00000 Frm 00041 Fmt 4703 Sfmt 4703 67047 The previously approved collections of information are subject to review by OMB under the PRA. The collections of information in 21 CFR part 312 and Form FDA 1572 have been approved under OMB control number 0910–0014. III. Electronic Access Persons with access to the internet may obtain the 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: November 1, 2022. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2022–24112 Filed 11–4–22; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2022–N–2671] Drug Supply Chain Security Act Implementation and Readiness Efforts for 2023; Public Meeting; Request for Comments AGENCY: Food and Drug Administration, HHS. Notice of public meeting; request for comments. ACTION: The Food and Drug Administration (FDA, the Agency, or we) is announcing the following virtual public meeting entitled ‘‘Drug Supply Chain Security Act Implementation and Readiness Efforts for 2023’’ to allow supply chain stakeholders an opportunity to share their perspectives. The topics to be discussed are stakeholder experiences with implementation and overall readiness regarding implementation of enhanced drug distribution security requirements that will go into effect on November 27, 2023, standards for the interoperable data exchange of product tracing information, requests for product tracing information or verification from FDA for the purpose of investigating suspect or illegitimate products or for recalls, steps taken to build capacity for package-level tracing, pharmaceutical distribution supply chain best practices, and, in general, the impact that the Drug Supply Chain Security Act (DSCSA) requirements would have on public health, including patient safety and access to prescription drugs, and on SUMMARY: E:\FR\FM\07NON1.SGM 07NON1

Agencies

[Federal Register Volume 87, Number 214 (Monday, November 7, 2022)]
[Notices]
[Pages 67046-67047]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-24112]

-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-0776]

Studying Multiple Versions of a Cellular or Gene Therapy Product
in an Early-Phase Clinical Trial; 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 final guidance entitled ``Studying Multiple
Versions of a Cellular or Gene Therapy Product in an Early-Phase
Clinical Trial; Guidance for Industry.'' The guidance document provides
recommendations to sponsors interested in studying multiple versions of
a cellular or gene therapy product in an early phase clinical trial for
a single disease. Sponsors have expressed interest in gathering
preliminary evidence of safety and activity using multiple versions of
a cellular or gene therapy product in a single clinical trial, where
each version of the product is distinct and is generally submitted to
FDA in a separate investigational new drug application (IND). The
guidance provides recommendations for conducting such studies,
including how to organize and structure the INDs, submit new
information, and report adverse events. The guidance announced in this
notice finalizes the draft guidance of the same title dated September
2021.

DATES: The announcement of the guidance is published in the Federal
Register on November 7, 2022.

ADDRESSES: You may submit either electronic or written comments on
Agency guidances at any time as follows:

Electronic Submissions

Submit electronic comments in the following way:
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-0776 for ``Studying Multiple Versions of a Cellular or Gene
Therapy Product in an Early-Phase Clinical Trial; 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 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

[[Page 67047]]

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: Jessica Gillum, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a document entitled
``Studying Multiple Versions of a Cellular or Gene Therapy Product in
an Early-Phase Clinical Trial; Guidance for Industry.'' The guidance
document provides recommendations to sponsors interested in studying
multiple versions of a cellular or gene therapy product in an early
phase clinical trial for a single disease. Sponsors have expressed
interest in gathering preliminary evidence of safety and activity using
multiple versions of a cellular or gene therapy product in a single
clinical trial, where each version of the product is distinct and is
generally submitted to FDA in a separate IND. The objective of these
early phase clinical studies is to guide which version(s) of the
product to pursue for further development in later phase studies. Thus,
these studies are not intended to provide primary evidence of
effectiveness to support a marketing application and generally are not
adequately powered to demonstrate a statistically significant
difference in efficacy between the study arms. The guidance provides
recommendations for conducting such studies, including how to organize
and structure the INDs, submit new information, and report adverse
events.
In the Federal Register of September 30, 2021 (86 FR 54207), FDA
announced the availability of the draft guidance entitled ``Studying
Multiple Versions of a Cellular or Gene Therapy Product in an Early
Phase Clinical Trial.'' FDA received several comments on the draft
guidance and those comments were considered as the guidance was
finalized. Changes to the guidance include clarifying how to continue
the umbrella trial after a study arm has been closed and adding
examples of changes that result in multiple versions of a cellular or
gene therapy product. In addition, editorial changes were made to
improve clarity. The guidance announced in this notice finalizes the
draft guidance of the same title dated September 2021.
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Studying Multiple Versions of a Cellular
or Gene Therapy Product in an Early-Phase Clinical Trial.'' 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. 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 312 and Form FDA 1572 have been approved
under OMB control number 0910-0014.

III. Electronic Access

Persons with access to the internet may obtain the 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: November 1, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-24112 Filed 11-4-22; 8:45 am]
BILLING CODE 4164-01-P

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