Notice2021-21324
Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Guidance for Industry; Availability
Primary source
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Published
September 30, 2021
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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 The guidance announced in this notice finalizes the draft guidance of the same title dated January 2020.
Full Text
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<title>Federal Register, Volume 86 Issue 187 (Thursday, September 30, 2021)</title>
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[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54206-54207]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<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-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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at
the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through
Friday, 240-402-7500.
<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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>,
<a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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