83 FR 32311 - Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 134 (July 12, 2018)

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ``Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry.'' The draft guidance provides sponsors, who are developing a human gene therapy (GT) product, recommendations regarding the design of long term follow-up (LTFU) observational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. The draft guidance, when finalized, is intended to supersede the document entitled ``Guidance for Industry: Gene Therapy Clinical Trials--Observing Participants for Delayed Adverse Events'' dated November 2006. This draft guidance, when finalized, is also intended to supplement the guidance entitled ``Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Draft Guidance for Industry.''

[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32311-32312]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-14867]


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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Products; Draft Guidance for Industry; Availability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 draft document entitled ``Long Term Follow-Up 
After Administration of Human Gene Therapy Products; Draft Guidance for 
Industry.'' The draft guidance provides sponsors, who are developing a 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. The draft guidance, when finalized, is intended to supersede 
the document entitled ``Guidance for Industry: Gene Therapy Clinical 
Trials--Observing Participants for Delayed Adverse Events'' dated 
November 2006. This draft guidance, when finalized, is also intended to 
supplement 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.''

DATES: Submit either electronic or written comments on the draft 
guidance by October 10, 2018 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-2018-D-2173 for ``Long Term Follow-Up After Administration of Human 
Gene Therapy Products; 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.
     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.thefederalregister.org/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 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

[[Page 32312]]

the SUPPLEMENTARY INFORMATION section for electronic access to the 
draft guidance document.

FOR FURTHER INFORMATION CONTACT: Jonathan McKnight, 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 
``Long Term Follow-Up After Administration of Human Gene Therapy 
Products; Draft Guidance for Industry.'' The draft 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. The draft 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 in the draft guidance are GT product-specific clinical 
considerations for monitoring subjects under a LTFU protocol and 
recommendations on patient monitoring for licensed GT products. The 
draft guidance, when finalized, is intended to supersede the guidance 
entitled ``Guidance for Industry: Gene Therapy Clinical Trials--
Observing Participants for Delayed Adverse Events'' dated November 
2006. The draft guidance, when finalized, is also intended to 
supplement 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,'' published elsewhere in this issue of the Federal Register. 
Also, elsewhere in this issue of the Federal Register, FDA is 
announcing the availability of another draft guidance entitled 
``Chemistry, Manufacturing, and Control Information for Human Gene 
Therapy Investigational New Drug Applications; 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 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. This guidance 
is not subject to Executive Order 12866.

II. Paperwork Reduction Act of 1995

    This draft 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. 3501-3520). 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 draft guidance 
at either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: July 5, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-14867 Filed 7-11-18; 8:45 am]
BILLING CODE 4164-01-P