By Rachel Sachs
Yesterday, the FDA announced a new draft guidance regarding its exercise of its enforcement discretion around the investigational new drug (IND) requirements as they apply to fecal microbiota transplantation, or FMT. For almost three years now, the FDA has exercised its enforcement discretion for FMT under a rather permissive set of guidelines, enabling patients to access FMT while companies shepherd a set of products through the clinical trial process. I recently co-authored an article about the FDA’s regulation of FMT, and I’m concerned about this new guidance, in terms of safety, access, and regulatory clarity. This is one of the wonkiest posts I’ve written in some time (and that’s saying something), so I’ll endeavor to be as clear as possible.
What is FMT and why is it important? To make a long story short, it’s a poop transplant. Filtered stool from a healthy donor is transplanted into the gastrointestinal tract of a sick patient. Although scientists are continuing to explore the use of FMT for a range of indications, we already know that FMT is a startlingly effective treatment for recurrent C. difficile infection. C. difficile infections have become among the most common hospital-acquired infections in the United States, with more than 450,000 total incident infections annually. Unfortunately, many of these infections are resistant to antibiotics: with those 450,000 infections came 80,000 recurrences and 29,000 deaths. But FMT may provide a way forward. A recent randomized trial of patients with recurrent C. difficile infections was stopped early, when 94% of patients in the FMT group were cured, as compared to roughly 30% of those getting only antibiotics.
How is the FDA involved? In May 2013, the FDA announced that fecal microbiota would be regulated as a drug. All uses of FMT would therefore need to be part of an IND application, and patients who wanted to be treated with FMT for recurrent C. difficile would need to participate in a clinical trial to do so. Physicians and scientists responded with concern, arguing that available evidence supporting FMT’s effectiveness as a therapy for recurrent C. difficile infection was too compelling for regulators to restrict its availability to clinical trials. As such, in July 2013, the FDA announced that it would exercise enforcement discretion when FMT was used to treat patients “with C. difficile infection not responding to standard therapies,” so long as the treating physician obtained informed consent. Continue reading