[Federal Register Volume 84, Number 33 (Tuesday, February 19, 2019)]
[Notices]
[Pages 4825-4826]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-02691]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2017-D-6159]
Expedited Programs for Regenerative Medicine Therapies for
Serious Conditions; 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 ``Expedited Programs for
Regenerative Medicine Therapies for Serious Conditions; Guidance for
Industry.'' The guidance document provides sponsors engaged in the
development of regenerative medicine therapies for serious or life-
threatening diseases or conditions with FDA's recommendations on the
expedited development and review of these therapies. The guidance
describes the expedited programs available to sponsors of regenerative
medicine therapies for serious or life-threatening diseases or
conditions, including those products designated as regenerative
advanced therapies (which FDA refers to as ``regenerative medicine
advanced therapy'' (RMAT) designation). The guidance also describes
considerations in the clinical development of regenerative medicine
therapies and opportunities for sponsors of regenerative medicine
therapies to interact with the Center of Biologics Evaluation and
Research (CBER) review staff.
The guidance announced in this notice finalizes the draft guidance
of the same title dated November 2017.
DATES: The announcement of the guidance is published in the Federal
Register on February 19, 2019.
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-2017-D-6159 for ``Expedited Programs for Regenerative Medicine
Therapies for Serious Conditions; 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.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
Docket: For access to the docket to read background documents or
the
[[Page 4826]]
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
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: Tami Belouin, 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
``Expedited Programs for Regenerative Medicine Therapies for Serious
Conditions; Guidance for Industry.'' The guidance describes the
expedited programs available to sponsors of regenerative medicine
therapies for serious or life-threatening diseases or conditions
(referred to in the guidance as serious conditions), including those
products designated as RMATs; provides information about the provisions
in the 21st Century Cures Act (Cures Act) (Pub. L. 114-225) regarding
the use of the accelerated approval pathway for regenerative medicine
therapies that have been granted designation as an RMAT; describes how
CBER will encourage flexibility in clinical trial design to facilitate
the development of data to demonstrate the safety and effectiveness of
regenerative medicine therapies that are being developed to address
unmet needs in patients with serious conditions; and describes the
opportunities for sponsors of regenerative medicine therapies to
interact with CBER review staff.
In the Federal Register of November 17, 2017 (82 FR 54385), FDA
announced the availability of the draft guidance of the same title
dated November 2017. FDA received several comments on the draft
guidance and those comments were considered as the guidance was
finalized. In addition, editorial changes were made to improve clarity.
The guidance announced in this notice finalizes the draft guidance
dated November 2017.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of a document entitled ``Evaluation of Devices Used
with Regenerative Medicine Advanced Therapies; 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 ``Expedited Programs for Regenerative
Medicine Therapies for Serious Conditions.'' 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 guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are 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 312 have been approved under
OMB control number 0910-0014; the collections of information regarding
formal meetings described in the draft guidance, ''Formal Meetings
Between the FDA and Sponsors or Applicants of PDUFA Products,'' have
been approved under OMB control number 0910-0429; the collections of
information in 21 CFR part 601 have been approved under OMB control
number 0910-0338; the collections of information for expedited programs
in ''Guidance for Industry: Expedited Programs for Serious Conditions--
Drugs and Biologics,'' have been approved under OMB control number
0910-0765; the collections of information in 21 CFR part 314 have been
approved under OMB control number 0910-0001.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or
https://www.regulations.gov.
Dated: February 13, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-02691 Filed 2-15-19; 8:45 am]
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