Document ID: FDA-2017-D-6159-0016
Agency: fda
Document Type: Notice
Title: Expedited Programs for Regenerative Medicine Therapies for Serious
Conditions; Guidance for Industry; Availability
Posted Date: 2019-02-19T05:00Z

[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    The guidance announced in this notice finalizes the draft guidance 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]
 BILLING CODE 4164-01-P