[Federal Register Volume 78, Number 138 (Thursday, July 18, 2013)]
[Notices]
[Pages 42965-42966]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-17223]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-0811]


Guidance for Industry: Enforcement Policy Regarding 
Investigational New Drug Requirements for Use of Fecal Microbiota for 
Transplantation To Treat Clostridium difficile Infection Not Responsive 
to Standard Therapies; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a guidance for industry entitled ``Enforcement 
Policy Regarding IND Requirements for Use of Fecal Microbiota for 
Transplantation to Treat Clostridium difficile Infection Not Responsive 
to Standard Therapies,'' dated July 2013. This guidance informs members 
of the medical and scientific community and other interested persons 
that we intend to exercise enforcement discretion regarding the 
investigational new drug (IND) requirements for the use of fecal 
microbiota for transplantation (FMT) to treat C. difficile infection 
not responding to standard therapies. FDA intends to exercise this 
discretion provided that the treating physician obtains adequate 
informed consent from the patient or his or her legally authorized 
representative for the use of FMT products. Informed consent should 
include, at a minimum, a statement that the use of FMT products to 
treat C. difficile is investigational and a discussion of its potential 
risks. This policy does not extend to other uses of FMT. FDA intends to 
exercise this discretion on an interim basis while we further consider 
the matter. This guidance has an immediate implementation date because 
FDA has

[[Page 42966]]

determined that prior public participation is not feasible or 
appropriate.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of the guidance to 
the Office of Communication, Outreach and Development (HFM-40), Center 
for Biologics Evaluation and Research (CBER), Food and Drug 
Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-
1448. Send one self-addressed adhesive label to assist that office in 
processing your requests. See the SUPPLEMENTARY INFORMATION section for 
electronic access to the guidance document.
    Submit electronic comments on the guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Paul E. Levine, Jr., Center for 
Biologics Evaluation and Research (HFM-17), Food and Drug 
Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-
1448, 301-827-6210.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Enforcement Policy Regarding IND Requirements for Use of 
Fecal Microbiota for Transplantation to Treat Clostridium difficile 
Infection Not Responsive to Standard Therapies,'' dated July 2013. This 
guidance is being issued consistent with FDA's good guidance practices 
(GGP) regulation Sec.  10.115 (21 CFR 10.115). This guidance is being 
implemented without prior public comment because the Agency has 
determined that prior public participation is not feasible or 
appropriate (Sec.  10.115(g)(2)). The Agency made this determination 
because the guidance requires immediate implementation for public 
health reasons. This guidance deals with an urgent issue affecting 
patients with life-threatening infections with C. difficile. Although 
this guidance document is immediately in effect, it remains subject to 
comment in accordance with the Agency's GGPs regulation.
    Fecal microbiota collected from healthy individuals are being 
investigated for use in the treatment of C. difficile infection. 
Published data suggest that the use of fecal microbiota to restore 
intestinal flora may be an effective therapy in the management of 
refractory C. difficile infection. However, the efficacy and safety 
profile of this intervention have not yet been fully evaluated in 
controlled clinical trials.
    In the Federal Register of February 25, 2013 (78 FR 12763), FDA 
announced a public workshop, entitled ``Fecal Microbiota for 
Transplantation,'' which was held on May 2 and 3, 2013. The purpose of 
the workshop was to provide a forum for the exchange of information, 
knowledge, and experience among the medical and scientific community 
about the regulatory and scientific issues associated with FMT. During 
that workshop, and in subsequent communications, physicians and 
scientists expressed concern to FDA that FMT is not appropriate for 
study under the Agency's IND regulations (21 CFR part 312). Some health 
care providers stated that applying IND requirements will make FMT 
unavailable and suggested that an alternative regulatory approach is 
needed to ensure the widespread availability of FMT for individuals 
with C. difficile infection unresponsive to standard therapies.
    FDA acknowledges these concerns. The Agency intends to exercise 
enforcement discretion regarding the IND requirements for the use of 
FMT to treat C. difficile infection not responding to standard 
therapies, provided that the treating physician obtains adequate 
informed consent from the patient or his or her legally authorized 
representative for the use of FMT products. Informed consent should 
include, at a minimum, a statement that the use of FMT products to 
treat C. difficile is investigational and a discussion of its potential 
risks. FDA intends to exercise this discretion on an interim basis 
while the Agency further considers the matter.
    This policy does not extend to other uses of FMT. Data related to 
the use and study of FMT to treat diseases or conditions other than C. 
difficile infection are limited, and study of FMT for these other uses 
is not included in this enforcement policy.
    This guidance represents the Agency's current thinking on this 
topic. It does not create or confer any rights for or on any person and 
does not operate to bind FDA or the public. An alternative approach may 
be used if such approach satisfies the requirements of the applicable 
statutes and regulations.

II. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

III. Electronic Access

    Persons with access to the Internet may obtain the guidance at 
either http://www.fda.gov/RegulatoryInformation/Guidances/default.htm 
or http://www.regulations.gov.

    Dated: July 12, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-17223 Filed 7-17-13; 8:45 am]
BILLING CODE 4160-01-P