83 FR 32303 - Human Gene Therapy for Rare Diseases; Draft Guidance for Industry; Availability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(FDA or Agency) is announcing the availability of a draft document entitled ``Human Gene Therapy for Rare Diseases; Draft Guidance for Industry.'' The draft guidance document provides recommendations to stakeholders developing a human gene therapy (GT) product intended to treat a rare disease in adult and/or pediatric patients regarding the manufacturing, preclinical, and clinical trial design issues for all phases of the clinical development program. Such information is intended to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the GT product itself.

[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32303-32305]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-14871]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2258]


Human Gene Therapy for Rare Diseases; Draft Guidance for 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 
Rare Diseases; Draft Guidance for Industry.'' The draft guidance 
document provides recommendations to stakeholders developing a human 
gene therapy (GT) product intended to treat a rare disease in adult 
and/or pediatric patients regarding the manufacturing, preclinical, and 
clinical trial design issues for all phases of the clinical development 
program. Such information is intended to assist sponsors in designing 
clinical development programs for such products, where there may be 
limited study population size and potential feasibility and safety 
issues as well as issues relating to the interpretability of 
bioactivity/efficacy outcomes that may be unique to rare diseases or to 
the nature of the GT product itself.

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

[[Page 32304]]

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-2258 for ``Human Gene Therapy for Rare Diseases; 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 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

    The Orphan Drug Act of 1983 (Pub. L. 97-414) defines a rare disease 
as a disease or condition that affects fewer than 200,000 persons in 
the United States. Since most rare diseases have no approved therapies, 
there is a significant unmet need for effective treatments. However, 
developing safe and effective products to treat rare diseases can be 
challenging. For example, it may be more difficult to find and recruit 
such patients into clinical trials, and many rare diseases exhibit a 
number of variations or subtypes. Consequently, patients may have 
highly diverse clinical manifestations and rates of disease progression 
with unpredictable clinical courses. Despite these challenges, GT-
related research and development continue to grow at a rapid rate, with 
several products advancing in clinical development.
    FDA is announcing the availability of a document entitled ``Human 
Gene Therapy for Rare Diseases; Draft Guidance for Industry.'' The 
draft guidance provides recommendations to stakeholders developing a GT 
product intended to treat a rare disease in adult and/or pediatric 
patients regarding the manufacturing, preclinical, and clinical trial 
design issues for all phases of the clinical development program. Such 
information is intended to assist sponsors in designing clinical 
development programs for such products, where there may be limited 
study population size and potential feasibility and safety issues as 
well as issues relating to the interpretability of bioactivity/efficacy 
outcomes that may be unique to rare diseases or to the nature of the GT 
product itself.
    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 Retinal Disorders; 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 Rare Diseases.'' 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

[[Page 32305]]

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 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. 2018-14871 Filed 7-11-18; 8:45 am]
BILLING CODE 4164-01-P