FDA finalizes enforcement policy for FMT treatments

Regulatory NewsRegulatory News | 28 November 2022 |  By 

In a newly finalized guidance, the US Food and Drug Administration (FDA) laid out its criteria for applying enforcement discretion for new fecal microbiota treatments (FMT) being developed in investigational new drug applications (INDs) for Clostridioides difficile infections (CDI) in patients who do not respond to standard therapies.
The final guidance reaffirms the draft version in affirming that regulatory discretion will not apply to FMTs obtained from stool banks.
FDA said that “in development of this policy FDA has considered input from stakeholders, including physicians, scientists, and patients concerned about sufficient access to FMT for patients with C. difficile infection not responding to standard therapies. FDA has developed this policy to help facilitate access for such patients, while addressing and controlling the risks that centralized manufacturing in stool banks presents to individuals receiving such products.”
Over the past decade, FMT, which involves the transplantation of stools from healthy donors into the gastrointestinal tract of patients afflicted with recurring C. difficile, has emerged as a promising treatment. In the guidance, FDA writes that “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.”
FDA received 145 comments on the draft guidance and enforcement policy. (RELATED: Fecal Transplants to Treat C. difficile: FDA Seeks Comment on What IND Requirements to Waive, Regulatory Focus 29 February 2016)
FDA said it would waive IND requirements for these treatments, provided that:
  • “The licensed health care provider treating the patient obtains appropriate consent from the patient or his or her legally authorized representative for the use of the FMT product. The consent should include, at a minimum, a statement that the use of FMT products to treat C. difficile is investigational and a discussion of the product’s reasonably foreseeable risks.
  • The stool donor and stool are qualified by screening and testing performed under the direction of the licensed health care providers for the purpose of providing the FMT product to treat his or her patient.
  • The use of the FMT product does not raise reported safety concerns of potential significant safety concerns (e.g., concerns regarding inappropriate storage or handling, or concerns regarding administration of product collecting without appropriate screening or testing).”
In its final guidance, FDA overruled comments urging the agency to extend regulatory discretion to stool banks.
“Should the agency require physicians such as me using material from stool banks to operate under IND, I doubt that I will be able to do so, due to the administrative burden (for an uncompensated procedure, and a private practice setting, without research support staff); I will either revert to the prior directed donation over universal donation, or simply not be able to offer the treatment to patients at all,” asserted Neil Stollman, a community practitioner in California.
FDA’s final guidance


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you