FMT and Microbial Medical Products: Generating High-Quality Evidence through Good Governance

Abstract

This article argues that current data for the safety and efficacy of fecal microbiota transplants as a treatment for any indication, including recurrent Clostridioides difficile infection, is low-quality. It develops a governance proposal that encourages production of high-quality evidence by incentivizing well-designed RCTs of stool and stoolderived microbial products. The proposal would require that FDA change its current enforcement approach, but it would not require any change in statutes or regulations.

Donors' experiences and attitudes of fecal microbiota transplantation: An empirical bioethics study from China

“…This may be due to the development of human microbiome research and media reports that have attracted public attention in recent years. The commercial prospects of stool-derived products can be affected by the way in which stool is regulated (or not regulated) [30]. With increasing interest in human microbiome and microbiome-based therapies, the related ownership issues that arise alongside therefore need to be revisited.…”

Where Stool is a Drug: International Approaches to Regulating the use of Fecal Microbiota for Transplantation

“…53 As of April 2019, there are 28 active or enrolling studies in the U.S. for potential therapeutic applications of FMT outside of rCDI. 54 Still, FMT's investigational status and the release of multiple draft guidances have led to some confusion in the medical community as to under what conditions it may be offered to rCDI patients. 55…”

The Impact of Regulatory Policies on the Future of Fecal Microbiota Transplantation

“…Some experts in the field believe that FMT could simply be a transitional treatment available only until a microbiota-based drug is formally approved by the FDA. 6 Others do not think that FMT will or should be abandoned as an approach for restoring full functionality of intestinal microbiota and treating serious and sometimes fatal diseases, such as recurrent CDI (rCDI) that cannot be cured with antibiotics alone. There are at least two possible reasons why FMT might only be available for a temporary time period.…”