[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]


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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.

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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notice finalizes the draft guidance of the same title dated September 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


