[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32306-32307]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-14875]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA 2018-D-2238]


Human Gene Therapy for Hemophilia; Draft Guidance for Industry; 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 
Hemophilia; Draft Guidance for Industry.'' The draft guidance document 
provides recommendations to stakeholders developing human gene therapy 
(GT) products for the treatment of hemophilia. The draft guidance 
provides recommendations on the clinical trial design and related 
development of coagulation factor VIII (hemophilia A) and IX 
(hemophilia B) activity assays, including how to address discrepancies 
in factor VIII and factor IX activity assays. The draft guidance also 
includes recommendations regarding preclinical considerations to 
support development of GT products for the treatment of hemophilia.

DATES: Submit either electronic or written comments on the draft 
guidance by October 10, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2238 for ``Human Gene Therapy for Hemophilia; 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.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-

[[Page 32307]]

800-835-4709 or 240-402-8010. See the SUPPLEMENTARY INFORMATION section 
for electronic access to the draft 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 draft document entitled 
``Human Gene Therapy for Hemophilia; Draft Guidance for Industry.'' The 
draft guidance document provides recommendations to stakeholders 
developing GT products for the treatment of hemophilia. The draft 
guidance provides recommendations on the clinical trial design and 
related development of coagulation factor VIII (hemophilia A) and IX 
(hemophilia B) activity assays, including how to address discrepancies 
in factor VIII and factor IX activity assays. The draft guidance also 
includes recommendations regarding preclinical considerations to 
support development of GT products for the treatment of hemophilia. 
Hemophilia therapy in the United States has progressed from replacement 
therapies for on-demand treatment of bleeding to prophylaxis to reduce 
the frequency of bleeding. GT products for hemophilia are being 
developed as single-dose treatments that may provide long-term 
expression of the missing or abnormal coagulation factor in the patient 
at steady levels to reduce or eliminate the need for exogenous factor 
replacement.
    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 Retinal Disorders; 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 Hemophilia.'' 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 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. 2018-14875 Filed 7-11-18; 8:45 am]
 BILLING CODE 4164-01-P