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After indicating that it would seek to strengthen the regulation of fecal microbiota for transplantation (FMT) for Clostridium difficile infection, the FDA has reversed course and announced that it will leave such treatment up to the discretion of physicians.

The FDA’s initial stance was to require that physicians get agency approval before using FMT to treat the potentially life-threatening gastrointestinal infection. That position was prompted, the agency stated, by its view that FMT is a biological product and drug. Thus, an Investigational New Drug application (IND) would have to be submitted prior to use, “to assure that subjects are not exposed to unreasonable risks,” noted FDA spokesperson Curtis Allen.

The original policy did make exceptions for clinical emergencies. In such cases, “the request to use [FMT] may be made via telephone or other rapid means of communication, and authorization may be given by the FDA official over the telephone,” Mr. Allen pointed out. “In these situations … treatment may begin prior to FDA’s receipt of the written IND submission that is to follow the initial emergency request.”

Criticism of the initial FDA decision was swift and forceful. According to Mr. Allen, some participants at an FDA public workshop in early May “expressed concerns about the administrative aspects of submitting an IND” and “expressed the concern that FDA involvement in the process would deprive ill patients of curative or palliative therapy.”

In an interview, Lawrence J. Brandt, MD, a professor of medicine and surgery at Albert Einstein College of Medicine, in New York City, said, “I have the greatest respect for the FDA and for the responsibility it has to protect the population against injury from inappropriate and unsafe products. I do think, however, that the restrictions placed on FMT in the treatment of recurrent Clostridium difficile infection are unduly restrictive.”

Dr. Brandt, who is also the emeritus chief of the Division of Gastroenterology at Montefiore Medical Center, in New York City, noted that thousands of FMT cases have been presented in various formats, with “an acknowledged serious adverse event frequency of close to zero.” The main point of a study presented at Digestive Disease Week in May (abstract 998), of which he was an author, “was that in a patient who has severe or complicated Clostridium difficile infection not responding to antibiotics, FMT represents a potentially valuable therapy with a cure rate of 92%.”

Against such findings, he suggested, concerns about long-term risks have to be put into perspective. “About 25,000 patients per year die of Clostridium difficile infection in the United States. If you went over to each of them and said, ‘You can die from the Clostridium difficile or you might die 20 years from now from a disease you might contract from the fecal transplant,’ what would most of them answer?”

In June, the FDA responded to these concerns by stating that it will “exercise enforcement discretion” and no longer require prior approval of FMT for C. difficile treatment.


Dr. Brandt and Mr. Allen reported no relevant financial conflicts of interest.