Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability, 14897-14898 [2022-05538]

Download as PDF Federal Register / Vol. 87, No. 51 / Wednesday, March 16, 2022 / Notices adverse event reporting, we assume it takes respondents 0.6 hour to submit a voluntary adverse event report via the SRP, 1 hour to submit a mandatory adverse event report via the SRP (except CTP, which estimates 0.6 hour), and 0.6 hour to submit a mandatory AER via the ESG (gateway-to-gateway transmission). CTP used two data sources to estimate the reporting burden for tobacco product AEs. CTP researched the number of voluntary AE reports submitted to the center since the launch of the first tobacco questionnaire in the SRP in 2014. Our records indicated a total of 1,426 initial reports over the last 7 full calendar years. We used the total number of reports to average the number of yearly reports to 204. As referenced above, the premarket tobacco product application rule requires firms to submit adverse experience reports for tobacco products with marketing orders. The burden for these mandatory reports has been approved under OMB control number 0910–0879. For this collection, we have included 1 hour to acknowledge the inclusion under this collection. Therefore, the estimate for CTP voluntary and mandatory reports is expected to be 123 hours. The submission of mandatory reports associated with drug products and biological drug products is accounted for and approved under OMB control number 0910–0230; the submission of mandatory reports associated with the Vaccine Adverse Event Reporting System is accounted for and approved under OMB control number 0910–0308; medical device report submissions are accounted for and approved under OMB control number 0910–0437; and the submission of mandatory reports associated with animal drug products is accounted for and approved under OMB control number 0910–0284. Dated: March 7, 2022. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2022–05514 Filed 3–15–22; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration jspears on DSK121TN23PROD with NOTICES1 [Docket No. FDA–2021–D–0398] Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. VerDate Sep<11>2014 17:16 Mar 15, 2022 Jkt 256001 The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ‘‘Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry.’’ The draft guidance document provides recommendations to sponsors developing human gene therapy products incorporating genome editing (GE) of human somatic cells. Specifically, the guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application in order to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing, product testing, preclinical safety assessment, and clinical trial design. DATES: Submit either electronic or written comments on the draft guidance by June 14, 2022 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 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 PO 00000 Frm 00078 Fmt 4703 Sfmt 4703 14897 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–0398 for ‘‘Human Gene Therapy Products Incorporating Human Genome Editing; 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 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. E:\FR\FM\16MRN1.SGM 16MRN1 14898 Federal Register / Vol. 87, No. 51 / Wednesday, March 16, 2022 / Notices 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: jspears on DSK121TN23PROD with NOTICES1 I. Background FDA is announcing the availability of a draft guidance document entitled ‘‘Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry.’’ The draft guidance document provides recommendations to sponsors developing human gene therapy products incorporating GE of human somatic cells. Specifically, the guidance provides recommendations regarding information that should be provided in an IND application in order to assess the safety and quality of the investigational GE product as required in 21 CFR 312.23. This includes information on product design, product manufacturing, product testing, preclinical safety assessment and clinical trial design. Elsewhere in this issue of the Federal Register, FDA is announcing the availability of another human gene therapy draft guidance document entitled ‘‘Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products; Draft Guidance for Industry.’’ 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 ‘‘Human Gene Therapy Products Incorporating Human Genome Editing.’’ 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 VerDate Sep<11>2014 17:16 Mar 15, 2022 Jkt 256001 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 have been approved under OMB control number 0910–0014; the collections of information in 21 CFR parts 210 and 211 have been approved under OMB control number 0910–0073; the collections of information in 21 CFR part 1271 have been approved under OMB control number 0910–0543. 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: March 10, 2022. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2022–05538 Filed 3–15–22; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting Pursuant to section 10(d) of the Federal Advisory Committee Act, as amended, notice is hereby given of the following meeting. The meeting will be closed to the public in accordance with the provisions set forth in sections 552b(c)(4) and 552b(c)(6), Title 5 U.S.C., as amended. The grant applications and the discussions could disclose confidential trade secrets or commercial property such as patentable material, and personal information concerning individuals associated with the grant applications, the disclosure of which would constitute a clearly unwarranted invasion of personal privacy. PO 00000 Frm 00079 Fmt 4703 Sfmt 4703 Name of Committee: National Institute of Diabetes and Digestive and Kidney Diseases Special Emphasis Panel; PAR19–319: NIDDK Central Repositories Non-renewable Sample Access (X01). Date: May 6, 2022. Time: 2:00 p.m. to 4:00 p.m. Agenda: To review and evaluate contract proposals. Place: National Institutes of Health, Democracy II, 6707 Democracy Blvd., Bethesda, MD 20892 (Telephone Conference Call). Contact Person: Najma S. Begum, Ph.D. Scientific Review Officer, Review Branch, DEA, NIDDK, National Institutes of Health Room, 7349, 6707 Democracy Boulevard, Bethesda, MD 20892–5452, (301) 594–8894, begumn@niddk.nih.gov. (Catalogue of Federal Domestic Assistance Program Nos. 93.847, Diabetes, Endocrinology and Metabolic Research; 93.848, Digestive Diseases and Nutrition Research; 93.849, Kidney Diseases, Urology and Hematology Research, National Institutes of Health, HHS) Dated: March 11, 2022. Miguelina Perez, Program Analyst, Office of Federal Advisory Committee Policy. [FR Doc. 2022–05562 Filed 3–15–22; 8:45 am] BILLING CODE 4140–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting Pursuant to section 10(d) of the Federal Advisory Committee Act, as amended, notice is hereby given of the following meeting. The meeting will be closed to the public in accordance with the provisions set forth in sections 552b(c)(4) and 552b(c)(6), Title 5 U.S.C., as amended. The grant applications and the discussions could disclose confidential trade secrets or commercial property such as patentable material, and personal information concerning individuals associated with the grant applications, the disclosure of which would constitute a clearly unwarranted invasion of personal privacy. Name of Committee: National Institute of Diabetes and Digestive and Kidney Diseases Special Emphasis Panel; Characterization of Islet-derived Extracellular Vesicles for Improved Detection, Monitoring, Classification, and Treatment of Type 1 Diabetes Special Emphasis Panel. Date: April 6, 2022. Time: 12:00 p.m. to 6:00 p.m. Agenda: To review and evaluate grant applications. E:\FR\FM\16MRN1.SGM 16MRN1

Agencies

[Federal Register Volume 87, Number 51 (Wednesday, March 16, 2022)]
[Notices]
[Pages 14897-14898]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-05538]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-0398]


Human Gene Therapy Products Incorporating Human Genome Editing; 
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 ``Human Gene Therapy 
Products Incorporating Human Genome Editing; Draft Guidance for 
Industry.'' The draft guidance document provides recommendations to 
sponsors developing human gene therapy products incorporating genome 
editing (GE) of human somatic cells. Specifically, the guidance 
provides recommendations regarding information that should be provided 
in an investigational new drug (IND) application in order to assess the 
safety and quality of the investigational GE product, including 
information on product design, product manufacturing, product testing, 
preclinical safety assessment, and clinical trial design.

DATES: Submit either electronic or written comments on the draft 
guidance by June 14, 2022 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-0398 for ``Human Gene Therapy Products Incorporating Human 
Genome Editing; 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 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.

[[Page 14898]]

    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 guidance document 
entitled ``Human Gene Therapy Products Incorporating Human Genome 
Editing; Draft Guidance for Industry.'' The draft guidance document 
provides recommendations to sponsors developing human gene therapy 
products incorporating GE of human somatic cells. Specifically, the 
guidance provides recommendations regarding information that should be 
provided in an IND application in order to assess the safety and 
quality of the investigational GE product as required in 21 CFR 312.23. 
This includes information on product design, product manufacturing, 
product testing, preclinical safety assessment and clinical trial 
design.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of another human gene therapy draft guidance document 
entitled ``Considerations for the Development of Chimeric Antigen 
Receptor (CAR) T Cell Products; Draft Guidance for Industry.''
    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 ``Human Gene 
Therapy Products Incorporating Human Genome Editing.'' 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 have been approved under OMB control 
number 0910-0014; the collections of information in 21 CFR parts 210 
and 211 have been approved under OMB control number 0910-0073; the 
collections of information in 21 CFR part 1271 have been approved under 
OMB control number 0910-0543.

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: March 10, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-05538 Filed 3-15-22; 8:45 am]
BILLING CODE 4164-01-P
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