[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32302-32303]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-14870]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2236]


Human Gene Therapy for Retinal Disorders; Draft Guidance for 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 ``Human Gene Therapy for 
Retinal Disorders; Draft Guidance for Industry.'' The draft guidance 
provides recommendations to stakeholders developing human gene therapy 
(GT) products for retinal disorders affecting adult and pediatric 
patients. The draft guidance focuses on issues specific to GT products 
for retinal disorders and provides recommendations related to product 
development, preclinical testing, and clinical trial design for such GT 
products.

DATES: Submit either electronic or written comments on the draft 
guidance by October 10, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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:
    <bullet> 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.
    <bullet> 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:
    <bullet> 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.
    <bullet> 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-2236 for ``Human Gene Therapy for Retinal Disorders; 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.
    <bullet> 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

[[Page 32303]]

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 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: Angela Moy, 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 
``Human Gene Therapy for Retinal Disorders; Draft Guidance for 
Industry.'' The draft guidance provides recommendations to stakeholders 
developing GT products for retinal disorders affecting adult and 
pediatric patients. These disorders vary in etiology, prevalence, 
diagnosis, and management, and include genetic as well as age-related 
diseases. These disorders manifest with central or peripheral visual 
impairment and often with progressive visual loss. The draft guidance 
focuses on issues specific to GT products for retinal disorders and 
provides recommendations related to product development, preclinical 
testing, and clinical trial design for such GT products.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of two other human gene therapy draft guidance 
documents entitled ``Human Gene Therapy for Hemophilia; Draft Guidance 
for Industry'' and ``Human Gene Therapy for Rare Diseases; 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 for Retinal Disorders.'' 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 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 part 50 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; 
the collections of information in 21 CFR part 211 have been approved 
under OMB control number 0910-0139; 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 part 601 have been approved 
under OMB control number 0910-0338; the collections of information in 
the guidance entitled ``Expedited Programs for Serious Conditions--
Drugs and Biologics'' have been approved under OMB control number 0910-
0765; and the collections of information in the guidance entitled 
``Formal Meetings Between the FDA and Sponsors or Applicants'' have 
been approved under OMB control number 0910-0429.

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. 2018-14870 Filed 7-11-18; 8:45 am]
 BILLING CODE 4164-01-P