October 20, 2017

FDA restrictions on fecal transplants relaxed

The FDA recently formally announced that it would exercise “enforcement discretion” for its rule that an Investigational New Drug (IND) permit be approved before fecal transplants could be used to treat Live-threatening infections with Clostridium Difficile bacteria.  While not changing written policy guidelines the FDA’s existing policies, the announcement gives doctors an opportunity to treat patients who have granted their consent after being informed about the possible risks of the procedure. This decision effectively reverses the decision that was made before which decided to more tightly regulate any use of fecal microbiota transplants without first applying for an IND, a process which would effectively make use of this treatment very difficult for most doctors to use.

Fecal transplants are widely regarded as the only effective treatment option for recurrent Clostridium difficile infection which comes back in spite of treatment with antibiotics. Making it easier for doctors to use this treatment approach can possibly save many lives with low estimates for deaths related to c. diff of at least 14,000 a year in the United States. Before this decision, patients and their families faced an illness with no approved treatment while doctors hands were tied when it came to performing this procedure.

While the FDA’s guidance is addressed specifically toward the use of fecal transplants to treat Clostiridium Difficile, and does not cover its use for other illnesses it may also encourage more exploration as a treatment option for Inflammatory Bowel Diseases such as Ulcerative Colitis and Crohn’s Disease where there have been encouraging individual stories and there is also ongoing research.  In fact Clostridium Difficile infections are sometimes found in patients who already have Ulcerative Colitis and / or Crohn’s Disease.

Here is the text of the decision from the Federal Register listing which is also available at this link. http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Vaccines/ucm361379.htm?source=govdelivery

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

 

I.  INTRODUCTION
We, FDA, are informing 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 Clostridium difficile (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.  FDA intends to exercise this discretion on an interim basis while the agency develops appropriate policies for the study and use of FMT products under IND.
FDA’s guidance documents, including this guidance, do not establish legally enforceable responsibilities. Instead, guidances describe the FDA’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in FDA’s guidances means that something is suggested or recommended, but not required.

II.  DISCUSSION
Fecal microbiota collected from healthy individuals are being investigated for use in the treatment of C. difficileinfection. 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 profiles 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.” The purpose of this 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.
The workshop to discuss the regulatory and scientific issues associated with FMT was held on May 2-3, 2013. FDA noted that use of FMT and clinical studies to evaluate its safety and effectiveness are subject to regulation by FDA, and that the complex nature of FMT products presents specific scientific and regulatory challenges. 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.  In the weeks since the workshop, FDA has received numerous inquiries about the application of the IND regulations to the administration of FMT products, and many expressed concern about the use of these products under IND.
FDA acknowledges these concerns and intends to exercise enforcement discretion regarding the IND requirements for the use of 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.
FDA intends to exercise this discretion while we further consider the matter. During this period of enforcement discretion, FDA will continue to work with any sponsors who wish to submit INDs for this use of FMT.
This enforcement discretion 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.