On Nov. 4, the U.S. Food and Drug Administration (FDA) convened a public hearing entitled, “Use of Fecal Microbiota for Transplantation (FMT) to Treat Clostridium difficile Infection Not Responsive to Standard Therapies.” The purpose of the public hearing was for FDA to obtain stakeholder input on FMT to treat C. difficile infection not responsive to standard therapies, including clinical evidence of effectiveness, evaluation of safety, impact of FDA’s current enforcement policy on FMT product development, and the future and path forward for FMT.
The agenda included public testimony from industry, researchers, clinicians and patients. AGA’s comments were presented by Colleen Kelly, MD, co-principal investigator of the AGA FMT National Registry. Her presentation included unpublished data from the AGA registry in addition to guiding principles on FMT developed by the AGA Center for Gut Microbiome Research and Education scientific advisory board, which state that:
- AGA supports continued patient access to FMT for treatment of C. difficile infection that fails standard therapies, where the evidence supports its safety and efficacy.
- For other indications with a weaker evidence base, AGA supports the use of FMT only in the context of a formal clinical study or single patient compassionate use with appropriate scientific, ethical and regulatory review.
- AGA does not support the use of FMT outside the supervision of a licensed health care professional.
- AGA recognizes a continued need for systematic data collection on short- and long-term outcomes of FMT for all indications, including treatment of C. difficile infection.
- AGA supports continued innovation in the development of microbiota-based therapies that are standardized, manufactured products and have demonstrated safety and efficacy in appropriate clinical trials.
Other AGA members who gave public testimony included Jessica R. Allegretti, MD, Paul Feuerstadt, MD, AGAF, Norman B. Javitt, MD, PhD, Stacy Kahn, MD, Sahil Khanna, MBBS, and Eugene F. Yen, MD. Diane Hoffmann, JD, MS, member of the AGA Center for Gut Microbiome Research and Education scientific advisory board, also spoke at the public hearing.
The use of FMT to treat multiply recurrent or refractory C. difficile infection is currently permitted by the FDA under an enforcement policy that was first announced in 2013. The latest version of the enforcement policy was published in 2016 as a draft guidance from the FDA. The FDA has not yet announced a timeline to finalize the policy.
The AGA FMT National Registry is a critical effort to track short- and long-term patient outcomes and potential risks associated with FMT. The registry’s goal is to track 4,000 patients for 10 years. If you perform FMT, please contribute to this important initiative. Learn more at www.gastro.org/FMTRegistry.