Document ID: FDA-2019-D-5392-0019
Agency: fda
Document Type: Notice
Title: Interpreting Sameness of Gene
Therapy Products Under the Orphan
Drug Regulations; Guidance for
Industry; Availability
Posted Date: 2021-09-30T04:00Z

[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54206-54207]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-21324]

[[Page 54206]]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-5392]

Interpreting Sameness of Gene Therapy Products Under the Orphan 
Drug Regulations; 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 ``Interpreting Sameness 
of Gene Therapy Products Under the Orphan Drug Regulations.'' The 
guidance document provides FDA's current thinking on certain criteria 
that help determine sameness of human gene therapy products for the 
purpose of orphan-drug designation and orphan-drug exclusivity. The 
guidance is intended to assist stakeholders, including industry and 
academic sponsors who seek orphan-drug designation and orphan-drug 
exclusivity, in the development of gene therapies for rare diseases. 
The guidance announced in this notice finalizes the draft guidance of 
the same title dated January 2020.

DATES: The announcement of the guidance is published in the Federal 
Register on September 30, 2021.

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-2019-D-5392 for ``Interpreting Sameness of Gene Therapy Products 
Under the Orphan Drug Regulations.'' 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, 240-402-7500.
     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, 240-402-7500.
    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 
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: Sana Hussain, 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 document entitled 
``Interpreting Sameness of Gene Therapy Products Under the Orphan Drug 
Regulations.'' The guidance provides FDA's current thinking on certain 
criteria that help determine sameness of human gene therapy products 
for the purpose of orphan-drug designation and orphan-drug exclusivity. 
The guidance is intended to assist stakeholders, including industry and 
academic sponsors who seek orphan-drug designation and orphan-drug 
exclusivity, in the development of gene therapies for rare diseases. 
The guidance focuses specifically on factors FDA intends to consider 
when determining sameness for gene therapy products and does not 
address sameness determinations for other types of products.
    In the Federal Register of January 30, 2020 (85 FR 5445), FDA 
announced the availability of the draft guidance of the

[[Page 54207]]

same title dated January 2020. Additionally, in the Federal Register of 
April 23, 2020 (85 FR 22740), FDA announced that it is extending the 
comment period on the notice published January 30, 2020. FDA received 
several comments on the draft guidance; the comments generally 
supported the approach described in the guidance and requested 
additional clarification. Those comments were considered as the 
guidance was finalized, and changes to the guidance include adding 
clarification and examples, as feasible. In addition, editorial changes 
were made to improve clarity. The guidance announced in this notice 
finalizes the draft guidance dated January 2020.
    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 ``Interpreting Sameness of Gene Therapy 
Products Under the Orphan Drug Regulations.'' 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

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. Therefore, 
clearance by the Office of Management and Budget (OMB) under the 
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not 
required for this guidance. The previously approved collections of 
information are subject to review by OMB under the PRA. The collections 
of information in 21 CFR part 316 have been approved under OMB control 
number 0910-0167 and the collections of information in 21 CFR part 312 
have been approved under OMB control number 0910-0014.

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, 
https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: September 24, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21324 Filed 9-29-21; 8:45 am]
BILLING CODE 4164-01-P