[Federal Register Volume 82, Number 221 (Friday, November 17, 2017)]
[Notices]
[Pages 54385-54386]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-24837]


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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Serious Conditions; Draft Guidance for Industry; Availability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 draft document entitled ``Expedited Programs for 
Regenerative Medicine Therapies for Serious Conditions; Draft Guidance 
for Industry.'' The draft guidance, when finalized, will provide 
stakeholders engaged in the development of regenerative medicine 
therapies with FDA's current thinking on the expedited development and 
review of these products. The draft guidance describes the expedited 
programs available to sponsors of regenerative medicine therapies for 
serious or life-threatening diseases or conditions (referred to in the 
draft guidance as serious conditions), including those products 
designated as regenerative advanced therapies (which FDA refers to as 
``regenerative medicine advanced therapy'' (RMAT) designation); 
describes how the Center for Biologics Evaluation and Research (CBER) 
will work with sponsors and encourage flexibility in clinical trial 
design to facilitate the development of data to demonstrate the safety 
and effectiveness of regenerative medicine therapies being developed to 
address unmet medical needs in patients with serious or life-
threatening diseases or conditions; and describes the opportunities for 
sponsors of regenerative medicine therapies to interact with CBER 
review staff.

DATES: Submit either electronic or written comments on the draft 
guidance by February 15, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to http://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 http://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; Draft Guidance for Industry.'' 
Received comments will be placed in the docket and, except for those 
submitted as ``Confidential Submissions,'' publicly viewable at http://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 http://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

[[Page 54386]]

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 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 draft 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 draft 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 draft 
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 draft document entitled 
``Expedited Programs for Regenerative Medicine Therapies for Serious 
Conditions; Draft Guidance for Industry.'' The draft guidance document 
describes the expedited programs available to sponsors of regenerative 
medicine therapies for serious or life-threatening diseases or 
conditions (referred to in the draft 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.
    The draft guidance document addresses regenerative medicine 
therapies, which are defined in section 506(g)(8) of the Federal Food, 
Drug, and Cosmetic Act (the FD&C Act), as amended by the Cures Act, as 
including cell therapies, therapeutic tissue engineering products, 
human cell and tissue products, and combination products using any such 
therapies or products, except for those regulated solely under section 
361 of the Public Health Service Act (PHS Act) (42 U.S.C. 264) and 21 
CFR part 1271. Gene therapies, including genetically modified cells, 
that lead to a durable modification of cells or tissues can meet the 
definition of a regenerative medicine therapy. Under section 506(g) of 
the FD&C Act, RMAT designation is available for a drug (i.e., a human 
drug, including a drug that is a biological product) that meets the 
definition of regenerative medicine therapy; is intended to treat, 
modify, reverse, or cure a serious or life-threatening disease or 
condition; and with respect to which preliminary clinical evidence 
indicates the potential to address unmet medical needs for such disease 
or condition. A combination product (biologic-device, biologic-drug, or 
biologic-device-drug) can be eligible for RMAT designation when the 
biological product provides the greatest contribution to the overall 
intended therapeutic effects of the combination product (i.e., the 
primary mode of action in the combination product is conveyed by the 
biological product component).
    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; Draft Guidance for 
Industry.''
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the current thinking of FDA on ``Expedited 
Programs for Regenerative Medicine Therapies for Serious Conditions; 
Draft Guidance for Industry.'' 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 draft 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 in ``Guidance for 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 draft guidance 
at either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: November 13, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-24837 Filed 11-16-17; 8:45 am]
 BILLING CODE 4164-01-P