[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the availability of a draft guidance for industry entitled 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 This draft guidance informs members of the medical and scientific 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 This draft guidance, when 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draft guidance before it begins work on the final version of the 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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