Last week’s issue of the New Yorker featured a terrific article about fecal microbiota transplantation, or FMT. Much of the article focused on OpenBiome, a nonprofit stool bank spun off from MIT that screens donors, processes samples, and ships them to hospitals around the country. For those who are unfamiliar with FMT, it is a startlingly effective treatment for recurrent C. difficile infection. C. diff infections have become among the most common hospital-acquired infections in the United States, causing more than 300,000 hospitalizations and 14,000 deaths annually. And unfortunately, many of these infections are resistant to antibiotics, with resistance rates rising rapidly. But FMT may provide a way forward: a recent randomized trial (antibiotics versus antibiotics plus FMT) was stopped early, when 94% of patients in the FMT group were cured, as compared to roughly 30% of those in the antibiotics groups.
Coincidentally, I’ve been working with OpenBiome over the past few months on an interesting question that the New Yorker article touched on only briefly: how should the FDA regulate FMT to best ensure its safety and efficacy? At present, the FDA is proposing to regulate FMT as a biologic drug. However, many (including OpenBiome’s co-founder, Mark Smith) have argued that it ought to be regulated like human tissue, which from a scientific standpoint it resembles more closely than it does a small molecule drug, given the challenge of characterizing stool’s active ingredients and providing consistency across batches. OpenBiome’s Policy Director, Carolyn Edelstein, and I are currently working on a paper examining the pluses and minuses of the FDA’s current approach. I want to briefly summarize a few key points of our paper here, but essentially we argue that classifying FMT as a drug is simultaneously underregulatory and overregulatory. Our primary goal is to ensure that patients have access to safe, effective treatments – and that means the FDA should be more involved in regulating some aspects of FMT, and less involved in others.