Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early Phase Clinical Trial; Draft Guidance for Industry; Availability, 54207-54208 [2021-21322]

Download as PDF Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices same title dated January 2020. Additionally, in the Federal Register of April 23, 2020 (85 FR 22740), FDA announced that it is extending the comment period on the notice published January 30, 2020. FDA received several comments on the draft guidance; the comments generally supported the approach described in the guidance and requested additional clarification. Those comments were considered as the guidance was finalized, and changes to the guidance include adding clarification and examples, as feasible. In addition, editorial changes were made to improve clarity. The guidance announced in this notice finalizes the draft guidance dated January 2020. 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 ‘‘Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.’’ 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. LOTTER on DSK11XQN23PROD with NOTICES1 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 316 have been approved under OMB control number 0910–0167 and the collections of information in 21 CFR part 312 have been approved under OMB control number 0910–0014. III. Electronic Access Persons with access to the internet may obtain the guidance at either https://www.fda.gov/vaccines-bloodbiologics/guidance-complianceregulatory-information-biologics/ biologics-guidances, https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents, or https://www.regulations.gov. Dated: September 24, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21324 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Draft Guidance for Industry.’’ The draft 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 is a distinct product that should be submitted to FDA in a separate investigational new drug application (IND). The draft guidance describes the regulatory framework for conducting such studies, including recommendations on how to organize and structure the INDs, submit new information, and report adverse events. DATES: Submit either electronic or written comments on the draft guidance by December 29, 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: SUMMARY: 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 PO 00000 Frm 00059 Fmt 4703 Sfmt 4703 54207 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; Draft Guidance for Industry.’’ Received comments will be placed in the docket and, except for those submitted as ‘‘Confidential Submissions,’’ publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240–402–7500. • Confidential Submissions—To submit a comment with confidential information that you do not wish to be 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 E:\FR\FM\30SEN1.SGM 30SEN1 54208 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https:// www.govinfo.gov/content/pkg/FR-201509-18/pdf/2015-23389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https:// www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ‘‘Search’’ box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500. You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)). Submit written requests for single copies of the draft guidance to the Office of Communication, Outreach and Development, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993–0002. Send one self-addressed adhesive label to assist the office in processing your requests. The draft guidance may also be obtained by mail by calling CBER at 1– 800–835–4709 or 240–402–8010. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. FOR FURTHER INFORMATION CONTACT: Tami Belouin, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993–0002, 240– 402–7911. SUPPLEMENTARY INFORMATION: LOTTER on DSK11XQN23PROD with NOTICES1 I. Background FDA is announcing the availability of a draft document entitled ‘‘Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Draft Guidance for Industry.’’ The draft 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 is a distinct product that should be 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 VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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 draft guidance describes the regulatory framework for conducting such studies, including recommendations on how to organize and structure the INDs, submit new information, and report adverse events. 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 ‘‘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 draft guidance at either https://www.fda.gov/vaccines-bloodbiologics/guidance-complianceregulatory-information-biologics/ biologics-guidances, https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents, or https://www.regulations.gov. Dated: September 24, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21322 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P PO 00000 Frm 00060 Fmt 4703 Sfmt 4703 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2021–D–0368] Investigator Responsibilities—Safety Reporting for Investigational Drugs and Devices; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘Investigator Responsibilities—Safety Reporting for Investigational Drugs and Devices.’’ The draft guidance provides recommendations to help clinical investigators comply with the safety reporting requirements of investigational new drug application (IND) studies and investigational device exemption (IDE) studies. The guidance is intended to help clinical investigators of drugs identify safety information that is considered an unanticipated problem involving risk to human subjects or others and that therefore requires prompt reporting to institutional review boards (IRBs) and to help clinical investigators of devices identify safety information that meets the requirements for reporting unanticipated adverse device effects (UADEs) to sponsors and to IRBs. DATES: Submit either electronic or written comments on the draft guidance by November 29, 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: SUMMARY: 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 E:\FR\FM\30SEN1.SGM 30SEN1

Agencies

[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54207-54208]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-21322]

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

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; Draft Guidance for Industry;
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft document entitled ``Studying Multiple
Versions of a Cellular or Gene Therapy Product in an Early-Phase
Clinical Trial; Draft Guidance for Industry.'' The draft 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 is a distinct product that should be
submitted to FDA in a separate investigational new drug application
(IND). The draft guidance describes the regulatory framework for
conducting such studies, including recommendations on how to organize
and structure the INDs, submit new information, and report adverse
events.

DATES: Submit either electronic or written comments on the draft
guidance by December 29, 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-0776 for ``Studying Multiple Versions of a Cellular or Gene
Therapy Product in an Early-Phase Clinical Trial; Draft Guidance for
Industry.'' Received comments will be placed in the docket and, except
for those submitted as ``Confidential Submissions,'' publicly viewable
at https://www.regulations.gov or at the Dockets Management Staff
between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.
Confidential Submissions--To submit a comment with
confidential information that you do not wish to be 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

[[Page 54208]]

except in accordance with 21 CFR 10.20 and other applicable disclosure
law. For more information about FDA's posting of comments to public
dockets, see 80 FR 56469, September 18, 2015, or access the information
at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in
the heading of this document, into the ``Search'' box and follow the
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852, 240-402-7500.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single copies of the draft guidance to
the Office of Communication, Outreach and Development, Center for
Biologics Evaluation and Research (CBER), Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-
0002. Send one self-addressed adhesive label to assist the office in
processing your requests. The draft guidance may also be obtained by
mail by calling CBER at 1-800-835-4709 or 240-402-8010. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
guidance document.

FOR FURTHER INFORMATION CONTACT: Tami Belouin, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a draft document entitled
``Studying Multiple Versions of a Cellular or Gene Therapy Product in
an Early-Phase Clinical Trial; Draft Guidance for Industry.'' The draft
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 is a distinct product
that should be 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 draft guidance
describes the regulatory framework for conducting such studies,
including recommendations on how to organize and structure the INDs,
submit new information, and report adverse events.
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 ``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 draft guidance
at either 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: September 24, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21322 Filed 9-29-21; 8:45 am]
BILLING CODE 4164-01-P

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