[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5451-5452]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01704]


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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; 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 final guidance entitled ``Human Gene Therapy for 
Rare Diseases; Guidance for Industry.'' The final 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. The guidance announced in this 
notice finalizes the draft guidance of the same title dated July 2018.

DATES: The announcement of the guidance is published in the Federal 
Register on January 30, 2020.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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-2258 for ``Human Gene Therapy for Rare Diseases; 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.govinfo.gov/content/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 guidance to the 
Office of Communication, Outreach and Development, Center for Biologics

[[Page 5452]]

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 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 guidance document.

FOR FURTHER INFORMATION CONTACT: Shruti Modi, 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; Guidance for Industry.'' This guidance 
provides recommendations to stakeholders developing human 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.
    In the Federal Register of July 12, 2018 (83 FR 32303), FDA 
announced the availability of the draft guidance of the same title. FDA 
received several comments on the draft guidance and those comments were 
considered as the guidance was finalized. The guidance announced in 
this notice finalizes the draft guidance dated July 2018.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of two other human gene therapy guidance documents 
entitled ``Human Gene Therapy for Hemophilia; Guidance for Industry'' 
and ``Human Gene Therapy for Retinal Disorders; Guidance for 
Industry.''
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on recommendations for stakeholders developing 
human GT products for retinal disorders affecting adult and pediatric 
patients. 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.

II. Paperwork Reduction Act of 1995

    This 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. 3501-3521). 
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 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 guidance at 
either https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances or 
https://www.regulations.gov.

    Dated: January 27, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01704 Filed 1-29-20; 8:45 am]
 BILLING CODE 4164-01-P