FDA Guidelines for FMT use
FMT Regulations
In the United States, FMT is not approved as a therapy. It can be prescribed under what is called discretionary enforcement for individuals suffering from C. diff not responding to standard therapies, or it can be used in a clinical trial under an Investigational New Drug designation, which limits access to trials being run.
In 2013, the Food and Drug Administration (FDA) created a policy regarding the use of FMT. In order to monitor its use, FMT was designated as an Investigational New Drug (IND), meaning it can be used in clinical trials. However, because FMT has the ability to be life saving for Clostridium difficile (C. diff) infections, the FDA also allows it to be used for recurrent C. diff infections not responding to standard therapies. This is called special guidance. In 2022, the FDA issued additional guidance that required FMT to be administered by the provider who is choosing the donor, screening the stool, and curating the FMT for delivery. This is the guidance Biome Basics abides by.
FMT Summary
The FDA guidance allows physicians to treat patients with FMT who have C. diff not responding to standard therapy as long as they obtain informed consent.
The FDA 2016 draft guidance was never enacted so the current regulations do not require donors to be “known” to either the patient or physician.
The FDA does not specify the delivery route of FMT or a standardized process for preparation of FMT.
Treatment for anything other than C. diff not responsive to standard therapy must be done as part of an IND application through the FDA approval process.
Autologous FMT (FMT made from your own stool, for your own use) is still considered an IND and is regulated as such.
Excerpt from the 2013 FDA Draft Guidance:
“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.”
Other leaders in FMT
OpenBiome is historically the non-profit leader in delivering FMT to physicians in the United States. We have modeled many of our practices after theirs. They provide very limited FMT for emergency cases of C. diff at this time.
However, their website still provides great information about FMT. They have created an excellent summary of the current status of FMT regulation. A portion of that summary is shared below. Flora Medicine encourages all physicians and patients to utilize the OpenBiome website to learn about FMT and its use. They have provided tens of thousands of FMT to patients suffering from C. diff infection (CDI) and provide a wide variety of resources on their website for both physicians and patients.
Can physicians perform FMT without an IND application (outside of a clinical trial)?
Yes, to treat patients with C. difficile infections not responsive to standard therapy. When the FDA announced in May 2013 that it would regulate fecal microbiota as an investigational drug, medical professional societies, physicians, and patients reacted with concern over how the policy would limit access to a therapy with 90% efficacy rates for this illness and no known short-term safety risks (Kassam et al., 2013).
Responding to these concerns, FDA issued guidance in July 2013 stating that it would exercise “enforcement discretion” – that is, it would allow doctors to provide FMT (for patients with C. difficile infections not responding to standard therapies) without filing an IND application."
What are the conditions for performing FMT without an IND application?
Physicians may only use FMT without an IND application to treat patients with C. difficile not responsive to standard therapy. Physicians must also obtain adequate informed consent from the patient or a legal representative, that includes, at minimum, a statement that the use of FMT products to treat CDI is investigational, and a discussion of the therapy’s potential risks and alternative options.
There are no FDA requirements about how to obtain stool for FMT. Physicians may use material from a donor identified by the patient or physician, or from a universal stool bank. There are also no requirements dictating the route of administration for FMT (e.g. colonoscopy, naso-enteric delivery, oral capsule)."
I have seen a draft FDA guidance stating that physicians using stool from a stool bank must do so under IND. Is this the new policy?
In March 2016, FDA released its second draft guidance to propose that enforcement discretion be narrowed so that physicians who obtain material from stool banks to treat CDI that is not responsive to standard therapy do so under an IND. This document is a draft for discussion purposes, and does not alter current enforcement discretion policy.
The prevailing FDA guidance, published in July 2013, states that physicians may perform FMT outside of an Investigational New Drug (IND) application to treat CDI that is not responsive to standard therapy, so long as they obtain informed consent." However, in November of 2022, after the approval of Rebyota’s fecal-derived drug, the FDA guidelines became more strict. Healthcare providers curating FMT for patient use were no longer considered stool banks. Flora Medicine was no longer considered a stool bank. After rigors in testing increased and staffing shortages ensued, Flora Medicine made the difficult decision to stop making FMT for our patients. With Rebyota on the market and Vowst, we feel we’ve bridged the gap to ensuring patients still have options available to them.