[Federal Register Volume 78, Number 138 (Thursday, July 18, 2013)]
[Notices]
[Pages 42965-42966]
From the Federal Register Online via the Government Publishing 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]
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