[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5452-5454]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01710]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2173]


Long Term Follow-Up After Administration of Human Gene Therapy 
Products; 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 ``Long Term Follow-Up 
After Administration of Human Gene Therapy Products.'' The guidance 
document provides sponsors, who are developing a human gene therapy 
(GT) product, recommendations regarding the design of long term follow-
up (LTFU) observational studies for the collection of data on delayed 
adverse events following administration of a GT product. This guidance 
finalizes the draft guidance of the same title dated July 2018 and 
supersedes the document entitled ``Guidance for Industry: Gene Therapy 
Clinical Trials--Observing Subjects for Delayed Adverse Events'' dated 
November 2006. This guidance also supplements the guidance entitled 
``Testing of Retroviral Vector-Based Human Gene Therapy Products for 
Replication Competent Retrovirus during Product Manufacture and Patient 
Follow-up; Guidance for Industry.''

DATES: The announcement of the guidance is published in the Federal 
Register on January 30, 2020.

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

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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-2018-D-2173 for ``Long Term Follow-Up After Administration of Human 
Gene Therapy Products.'' 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.
     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.gpo.gov/fdsys/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.
    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 Walker Udechukwu, 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 ``Long 
Term Follow-Up After Administration of Human Gene Therapy Products; 
Guidance for Industry.'' This guidance provides a brief introduction of 
the product characteristics, patient-related factors, and the 
preclinical and clinical data that should be considered when assessing 
the need for LTFU observations for your GT product. This guidance also 
describes the Agency's current recommendations for the conduct of LTFU 
studies, specifically the information/data to support a sponsor's 
rationale for the duration and design of a LTFU protocol when clinical 
trials are initiated. Also included this guidance are GT product-
specific clinical considerations for monitoring subjects under a LTFU 
protocol and recommendations on patient monitoring for licensed GT 
products. This guidance is intended to supersede the guidance entitled 
``Guidance for Industry: Gene Therapy Clinical Trials--Observing 
Participants for Delayed Adverse Events'' dated November 2006. This 
guidance supplements the guidance entitled ``Testing of Retroviral 
Vector-Based Human Gene Therapy Products for Replication Competent 
Retrovirus during Product Manufacture and Patient Follow-up; Draft 
Guidance for Industry''.
    In the Federal Register of July 12, 2018 (83 FR 32311), FDA 
announced the availability of the draft guidance of the same title 
dated July 2018. FDA received several comments on the draft guidance 
and those comments were considered as the guidance was finalized. The 
guidance announced in this notice finalizes the draft guidance dated 
July 2018.
    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 ``Long Term Follow-Up After Administration 
of Human 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

    This guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521). The 
collections of information in 21 CFR parts 50 and 56 have been approved 
under OMB control number 0910-0755; the collections of information in 
21 CFR part 58 have been approved under OMB control number 0910-0119; 
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-blood-biologics/guidance-
compliance-regulatory-information-biologics/

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biologics-guidances or https://www.regulations.gov.

    Dated: January 27, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01710 Filed 1-29-20; 8:45 am]
 BILLING CODE 4164-01-P


