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

Area of Science:

• Microbiology
• Gastroenterology
• Regulatory Science

Background:

• Fecal microbiota transplantation (FMT) shows promise for various conditions, notably recurrent Clostridioides difficile infection.
• However, the existing evidence base for FMT's safety and efficacy is largely considered low-quality.
• This limits its widespread adoption and regulatory clarity.

Purpose of the Study:

• To critique the current quality of evidence supporting fecal microbiota transplants.
• To propose a novel governance framework for generating high-quality clinical evidence.
• To outline necessary adjustments to regulatory approaches, specifically the FDA's enforcement strategy.

Main Methods:

• Analysis of the existing literature on fecal microbiota transplant safety and efficacy data.
• Development of a governance proposal focused on incentivizing rigorous research.
• Examination of current Food and Drug Administration (FDA) regulations and enforcement policies.

Main Results:

• Current data supporting fecal microbiota transplants (FMT) for any indication, including recurrent Clostridioides difficile infection, is of low quality.
• A governance proposal is presented to stimulate the creation of high-quality evidence.
• The proposal advocates for incentivizing well-designed randomized controlled trials (RCTs) for stool and stool-derived microbial products.

Conclusions:

• The current evidence landscape for FMT is insufficient to support its broad clinical application.
• A shift towards incentivizing high-quality RCTs is crucial for advancing the field.
• The proposed governance model necessitates a modification of the FDA's current enforcement approach without altering existing statutes or regulations.