Rachel Sachs and Carolyn Edelstein have a fascinating new paper about the difficulties that FDA will confront in regulating a promising new therapy involving … poop.
Fecal microbiota transplantation—the medical euphemism for taking poop from a healthy person, blending it up, and putting it in a sick person’s intestines—has shown extraordinary promise in treating C. difficile infections. There’s also some suggestion it could help treat other painful chronic conditions, including ulcerative colitis and Crohn’s disease.
In 2013, the FDA announced that it would have to regulate the transplanted fecal microbiome as a drug. When stool is used to treat disease, the agency explained, it falls within the broad statutory definition of a “drug.” But Sachs and Edelstein have mixed feelings about FDA regulation.
Broadly speaking, there is a clear need for oversight of the use of fecal microbiota for transplantation, given the risks posed by stool-borne transmissible illnesses. The uniformity and expertise that the FDA can offer the industry are important institutional advantages both for stool providers navigating their legal responsibilities and for physicians seeking guidance on how to best help their patients.
However, the FDA’s decision has proven problematic for scientific as well as legal and economic reasons. Not only does stool defy the typical scientific characterization that the FDA has long applied to small-molecule and biologic drugs, but the potential do-it-yourself nature of the treatment poses particular concerns in the context of a regime involving periods of regulatory exclusivity. The goals of any regulation of [fecal microbiota transplantation] for the treatment of recurrent C. difficile infection should be to optimize access to the therapy while maintaining strict screening and reporting standards and oversight.
Among other things, Sachs and Edelstein worry that laws drafted to regulate very different kinds of drugs may require FDA to grant a single company an exclusive license, for an extended period, to sell stool for medical purposes. The company would presumably use that exclusive license to jack up the prices for stool.
High prices could have the perverse effect of shifting stool into an unregulated gray market. As Sachs and Edelstein note, “although there is little chance that patients will manufacture traditional small molecule therapies in their bathrooms, processing stool for transplantation at a basic level requires very little training or equipment.”
The paper is full of fascinating stuff like this, including Sachs’s and Edelstein’s bottom-line recommendation that FDA ought to treat stool more like cord blood and other tissues than as a conventional drug. Go read it!