AGA, FDA met to discuss fecal transplant regulation: What you need to know

Gary Wu

Several members of the American Gastroenterological Association recently met with representatives from the FDA’s Center for Biologics Evaluation and Research to offer clinical and research perspectives on fecal microbiota transplantation and to gain a better understanding of CBER’s thoughts on the regulation of FMT for Clostridioides difficile infection.

Healio Gastroenterology and Liver Disease spoke with two members of the Scientific Advisory Board for the AGA Center for Gut Microbiome Research and Education about the meeting: Gary D. Wu, MD, from the University of Pennsylvania, as well as Alexander Khoruts, MD, from the University of Minnesota Microbiota Therapeutics Program.

Here are three things you need to know about how the meeting came about, and what was discussed.

Healio: What was behind putting this meeting together with the FDA?

Wu: There has been much ongoing discussion about the impact of stool banks on recruitment into clinical trials designed to determine the efficacy of products for the treatment of Clostridioides difficile infection. Together with the need to protect the safety of patients receiving FMT, FDA has issued a number of draft guidance’s pertaining to the practice of FMT and regulation of stool banks. In the absence of a final guidance, an enforcement discretion policy allows FMT to be performed without an [investigational new drug application] for the purposes of treating recurrent CDI.

The AGA represents a broad constituency of clinicians and researchers who, along with patients, have many questions pertaining to the future of stool banks, how this will affect the practice of FMT, and the prospects for new therapeutics. AGA met with the FDA for three reasons:

To learn the FDA’s current thoughts about the uncertainties described above in an attempt to bring some clarity to AGA’s constituencies;

To provide a variety of viewpoints representative of the broad constituency of AGA about the regulatory issues pertaining to FMT; and

To express AGA’s desire to provide assistance to the FDA during this current period of uncertainty in the field.

Healio: What are some of the needs and concerns of clinical and research communities regarding FMT?

Khoruts: As a clinician and researcher, my concerns were:

Continued access for patients who can benefit from FMT to this treatment;

Safety of FMT; and

Continued innovation in this rapidly developing field.

Termination of enforcement discretion carries distinct risks. For example, patients may increasingly turn to do-it-yourself protocols if FMT becomes less accessible. That could negatively impact their safety.

Current placebo-controlled trials are able to capture only a very narrow slice of the patient population with recurrent CDI. Many of these patients are immunosuppressed, have underlying bowel problems such as IBS or IBD, and have a multitude of other medical problems. Most of these patients are excluded from trials. Therefore, it will be difficult to extrapolate the results of these trials to populations that are actually seen by physicians in practice.

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Healio: What were some things the AGA came into the meeting with the FDA hoping to accomplish?

Khoruts: The AGA did not arrive with a specific position on regulation of FMT. The association wanted to make sure the FDA heard the voices of different stakeholders, including patients, clinicians and researchers, in addition to the views from the industry.

AGA expressed a need for innovative trials that would capture the complexity of the real-world patient population and offered the expertise of its members in advising the FDA on such trial design. One of the AGA initiatives discussed in the meeting was the AGA FMT National Registry, which tracks actual physician practice and long-term outcomes.