[Federal Register Volume 79, Number 38 (Wednesday, February 26, 2014)]
[Notices]
[Pages 10814-10815]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-04095]
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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the availability of a draft guidance for industry entitled
``Enforcement Policy Regarding Investigational New Drug Requirements
for Use of Fecal Microbiota for Transplantation to Treat Clostridium
difficile Infection Not Responsive to Standard Therapies,'' dated March
2014. This draft guidance informs members of the medical and scientific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,
provided the licensed health care provider treating the patient obtains
adequate informed consent from the patient or his or her legally
authorized representative for use of the FMT products, the stool is
obtained from a donor known to either the patient or the licensed
health care provider treating the patient, and the donor and stool are
qualified by screening and testing performed under the direction of the
licensed health care provider for the purpose of providing the FMT
product to treat his or her patient. This draft guidance, when
finalized, is intended to supersede the guidance document entitled
``Enforcement Policy Regarding Investigational New Drug Requirements
for Use of Fecal Microbiota for Transplantation to Treat Clostridium
difficile Infection Not Responsive to Standard Therapies,'' dated July
2013 (July 2013 Guidance).
DATES: Although you can comment on any guidance at any time (see 21 CFR
10.115(g)(5)), to ensure that the Agency considers your comment on this
draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by March 28, 2014.
ADDRESSES: Submit written requests for single copies of the draft
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 the office in
processing your requests. The draft guidance may also be obtained by
mail by calling CBER at 1-800-835-4709 or 301-827-1800. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
guidance document.
Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written
[[Page 10815]]
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 draft document entitled
``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,'' dated March 2014.
Fecal microbiota collected from healthy individuals are being
investigated for use in the treatment of C. difficile infection that is
not responsive to standard therapies. 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 has not
yet been fully evaluated in controlled clinical trials.
In the Federal Register of July 18, 2013 (78 FR 42965), FDA
announced the availability of the July 2013 Guidance. At that time, FDA
informed members of the medical and scientific community, and other
interested persons that it intended to exercise enforcement discretion
regarding these requirements provided that the physician treating the
patient obtains adequate informed consent from the patient or his or
her legally authorized representative for the use of FMT products. FDA
received several comments on the guidance, all of which supported
enforcement discretion with regard to the continued use of FMT products
to treat C. difficile infection not responsive to standard therapies.
Since publishing the July 2013 Guidance, FDA has reviewed and
intends to modify its enforcement discretion policy. In this draft
guidance, FDA explains that it 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: (1) The licensed health care provider treating the patient
obtains adequate informed consent from the patient or his or her
legally authorized representative for the use of FMT products; (2) the
FMT product is obtained from a donor known to either the patient or the
licensed health care provider treating the patient; and (3) the stool
donor and stool are qualified by screening and testing performed under
the direction of the licensed health care provider for the purpose of
providing the FMT product to treat his or her patient. This policy does
not extend to the use of an FMT product when the FMT product is
manufactured from the stool of a donor who is not known by the patient
and/or the licensed health care provider treating the patient, or the
donor and donor stool are not qualified under the direction of the
licensed health care provider. Furthermore, 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.
FDA intends to exercise this discretion on an interim basis while
the Agency further considers the matter, and continues to evaluate its
enforcement policy. The draft guidance is being issued consistent with
FDA's good guidance practices regulation (21 CFR 10.115). The draft
guidance, when finalized, will represent FDA'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 requirement of the applicable
statutes and regulations.
II. Comments
The draft guidance is being distributed for comment purposes only
and is not intended for implementation at this time. 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 draft guidance
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.
Dated: February 20, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-04095 Filed 2-25-14; 8:45 am]
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