The FDA has recently put
the medical community on notice that the use of fecal transplants for the
treatment of Clostridium difficile diarrhea constitutes use of an unapproved
biologic. This is another fascinating
and discouraging FDA intrusion into medical practice that threatens to set back
the use of a clearly beneficial and safe therapy for years. What is wrong with
these people?
First, there are a number of studies published on the use of
fecal transplants in the treatment of this deadly infection. C. difficile diarrhea is estimated to kill
14,000 Americans every year and the numbers of fatalities has steadily risen
over the last 20 years or so. Although initial therapy is 90% or more
efficacious, the relapse rate is as high as 25%. For those patients who have multiple bouts or
whose initial infection is especially severe, fecal transplants offer superior
efficacy in every study performed so far. The best review of this topic can be
found via this link.
In one controlled
study (albeit small), the efficacy of fecal transplant in recurrent C diff
diarrhea was 81% and that for standard therapy was 31% - a very statistically
significant difference. In the studies reported so far – hundreds of patients
treated – there have been no important safety signals whatsoever. Therefore,
the risk benefit ratio appears to be all benefit and no risk.
In addition, the procedure is not so pleasant. One obtains stool from a volunteer or
volunteers, suspends it in saline and milk, filters it through gauze and
administers it to the patient via a nasogastric tube inserted through the
nose going to the duodenum. The patient
has to have a bowel cleansing with massive laxative therapy the day before the
transplant and has to take a pill to reduce stomach acid prior to receiving the
stool transplant. Sounds like fun for
both physician and patient, right? This means that not everyone is going to
want to do this and only those with a significant problem will do so.
So – what is the FDA’s problem? First, they are not in
control. Beyond that, I suppose they interpret this intervention as doing their
job to protect Americans from unsafe and ineffective therapies. But other than the studies done so far, what
do they want? If they want larger,
controlled studies, who will pay for these studies? How long will they take? Will we have to have
a manufacturer that is approved provide a volunteer stool sample within 6 hours
so it can be used? How does that work?
Also – which FDA are we speaking to? The letter comes from CBER. But I understood that the use of biologics
for therapy would be regulated through the antiinfectives division in CDER . .
. ??
This entire approach is reminiscent of the FDA’s disastrous
attempt to make antibiotic trials more stringent by making them impossible to
perform or impossibly expensive to perform or both.
My recommendation here is that the FDA get together with
Gastroenterologists and ID specialists and define a clinical guideline similar
to the Cleveland Clinic approach such that physicians are aware of a
recommended standard approach to this procedure. But the FDA should not be regulating
this. This is yet another bridge too
far.
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