[Federal Register Volume 83, Number 173 (Thursday, September 6, 2018)]
[Notices]
[Pages 45249-45251]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-19303]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket Nos. FDA-1999-D-0081, FDA-2008-D-0205, FDA-2018-D-2173, FDA-
2018-D-2236, FDA-2018-D-2238, and FDA-2018-D-2258]


Draft Guidances Relating to the Development of Human Gene Therapy 
Products; Availability; Extension of Comment Period

AGENCY: Food and Drug Administration, HHS.

ACTION: Notification; extension of comment period.

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SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
extending the comment period for the notices of availability for six 
draft guidance documents relating to the development of human gene 
therapy products that appeared in the Federal Register of July 12, 
2018. The Agency is taking this action in response to requests for an 
extension to allow interested persons additional time to submit 
comments and any new information.

DATES: FDA is extending the comment period on the six documents that 
published on July 12, 2018 (see SUPPLEMENTARY INFORMATION). Submit 
either electronic or written comments by December 10, 2018, to ensure 
that the Agency considers your comment on these draft guidances before 
it begins work on the final version of the guidances.

ADDRESSES: You may submit comments as follows. Please note that late, 
untimely filed comments will not be considered. Electronic comments 
must be submitted on or before December 10, 2018. The https://www.regulations.gov electronic filing system will accept comments until 
midnight Eastern Time at the end of December 10, 2018. Comments 
received by mail/hand delivery/courier (for written/paper submissions) 
will be considered timely if they are postmarked or the delivery 
service acceptance receipt is on or before that date.

[[Page 45250]]

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-1999-D-0081 for ``Testing of Retroviral Vector-Based Human Gene 
Therapy Products for Replication Competent Retrovirus During Product 
Manufacture and Patient Follow-up; Draft Guidance for Industry;'' 
Docket No. FDA-2008-D-0205 for ``Chemistry, Manufacturing, and Control 
Information for Human Gene Therapy Investigational New Drug 
Applications; Draft Guidance for Industry;'' Docket No. FDA-2018-D-2173 
for ``Long Term Follow-Up After Administration of Human Gene Therapy 
Products; Draft Guidance for Industry;'' Docket No. FDA-2018-D-2236 for 
``Human Gene Therapy for Retinal Disorders; Draft Guidance for 
Industry;'' Docket No. FDA-2018-D-2238 for ``Human Gene Therapy for 
Hemophilia; Draft Guidance for Industry;'' or Docket No. FDA-2018-D-
2258 for ``Human Gene Therapy for Rare Diseases; Draft Guidance for 
Industry.'' Received comments, those filed in a timely manner (see 
ADDRESSES), 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 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.

FOR FURTHER INFORMATION CONTACT: Jenifer Stach, 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

    In the Federal Register of July 12, 2018, FDA published notices of 
availability with a 90-day comment period for six draft guidance 
documents listed in the following table. Three of the six draft 
guidance documents provide recommendations to stakeholders developing 
gene therapies for retinal disorders, hemophilia, and rare diseases. 
The remaining three guidance documents provide recommendations to 
sponsors manufacturing gene therapies; namely, how to provide 
chemistry, manufacturing and controls information for gene therapy 
products, additional recommendations regarding the testing for 
replication competent retrovirus during the manufacture of retroviral 
vector-based gene therapy products and during the follow-up monitoring 
of patients who received retroviral vector-based gene therapy products, 
and recommendations regarding the design of long-term follow-up 
observational studies for the collection of data on delayed adverse 
events following administration of a gene therapy product. Comments 
were requested on these draft guidances by October 10, 2018.

               Six Draft Guidances Published July 12, 2018
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                                     Draft guidance
           Docket No.                document title          FR cite
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FDA-1999-D-0081................  Testing of Retroviral  83 FR 32309
                                  Vector-Based Human
                                  Gene Therapy
                                  Products for
                                  Replication
                                  Competent Retrovirus
                                  During Product
                                  Manufacture and
                                  Patient Follow-up;
                                  Draft Guidance for
                                  Industry.
FDA-2008-D-0205................  Chemistry,             83 FR 32307
                                  Manufacturing, and
                                  Control Information
                                  for Human Gene
                                  Therapy
                                  Investigational New
                                  Drug Applications;
                                  Draft Guidance for
                                  Industry.
FDA-2018-D-2173................  Long Term Follow-Up    83 FR 32311
                                  After Administration
                                  of Human Gene
                                  Therapy Products;
                                  Draft Guidance for
                                  Industry.
FDA-2018-D-2236................  Human Gene Therapy     83 FR 32302
                                  for Retinal
                                  Disorders; Draft
                                  Guidance for
                                  Industry.
FDA-2018-D-2238................  Human Gene Therapy     83 FR 32306
                                  for Hemophilia;
                                  Draft Guidance for
                                  Industry.

[[Page 45251]]

 
FDA-2018-D-2258................  Human Gene Therapy     83 FR 32303
                                  for Rare Diseases;
                                  Draft Guidance for
                                  Industry.
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    The Agency has received requests for a 60-day extension of the 
comment period for the six draft guidance documents. These requests 
conveyed concern that the current 90-day comment period does not allow 
sufficient time to develop a meaningful or thoughtful response to the 
draft guidance documents.
    FDA has considered these requests and is extending the comment 
period for the six draft guidance documents for 60 days, until December 
10, 2018. The Agency believes that a 60-day extension allows adequate 
time for interested persons to submit comments.

II. References

    The following references are on display in the Dockets Management 
Staff (see ADDRESSES) and are available for viewing by interested 
persons between 9 a.m. and 4 p.m., Monday through Friday; they are also 
available electronically at https://www.regulations.gov.

1. Letter from Robert Falb, Director, U.S. Policy and Advocacy, 
Alliance for Regenerative Medicine, to Dr. Peter Marks, Director, 
Center for Biologics Evaluation and Research, FDA (July 23, 2018).
2. Letter from Sesquile Ramon, Ph.D., Director, Science and 
Regulatory Affairs, Biotechnology Innovation Organization, to FDA 
Dockets Management Staff (August 3, 2018).

    Dated: August 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-19303 Filed 9-5-18; 8:45 am]
 BILLING CODE 4164-01-P