The Food and Drug Administration has relaxed certain requirements for using fecal microbiota therapy in Clostridium difficile infections, a decision that may encourage payers to consider coverage options for the treatment.
In April, the FDA said it would require providers who want to offer fecal transplants to apply for investigational new drug status -- a process several physicians and researchers told the agency was too onerous.
Now, with C. diff. infection rates at historic highs, according to the Centers for Disease Control and Prevention, the FDA is backing off the requirement for investigational new drug status applications and will let providers use fecal microbiota therapy in patients not responding to standard therapies as long as treating physicians acquire informed consent.
"Informed consent," the FDA said, "should include at a minimum a statement that the use of FMT products to treat C. difficile is investigational and a discussion of its potential risks."
Like other infections often acquired in a hospital setting, antibiotic resistant C. difficile has become increasingly difficult to treat with existing antibiotics, and also life-threatening.
The CDC has estimated that about 14,000 Americans died from a C. difficile infection in 2011 -- out of about 500,000 infections -- although a USA Today investigation of Agency for Healthcare Research and Quality billing data pegged the number at around 30,000, which is on a par with annual motor vehicle deaths.The costs for treatment have been hard to pin down -- although they're certainly significant.
The CDC estimates that C. difficile infections cost the U.S. healthcare system an extra $1 billion annually.
One 2010 review of C. difficile studies estimated that average per case costs range from $2,870 to $4,846 for primary infections, from $13,655 to $18,067 for recurrent infections and as much as $90,000 for special-needs patients or those who end up in an intensive care unit. A more recent study presented at a meeting of the American Society of Health System Pharmacists estimated that the per case cost is about $35,000.
The standard treatment for C. difficile, meanwhile, has been facing challenges of its own. Metronidazole and vancomycin, for more severe cases, are the usual antibiotic treatments, but according to the Mayo Clinic, as many as 30 percent of patients fail the first-line treatment, while about 40 percent of those with a recurrence experience a further relapse.
And the treatments themselves are only going so far in the arms race of evolution, with C. difficile and other "nightmare bacteria," in the words of CDC Director Thomas Frieden, MD, growing increasingly resistant to modern antibiotics.
Enter fecal microbiota transplants, an old school therapy (possibly dating back to 4th century China and officially investigated by Western doctors in 1958) that by some estimates can have a 90 percent success rate, although some clinical researchers are skeptical of those and other positive findings recently published in the New England Journal of Medicine.
The process essentially shares the healthy gut bacteria of one person with an infected patient by way of a donor's feces, administered orally, nasally or directly into the colon. Fecal microbiota therapy does come with its own risks and costs, namely testing to ensure that a donor's bacteria profile doesn't include dangerous species, although Johan Bakken, MD, a Duluth, Minnesota, gastroenterologist, told the Canadian Medical Association Journal that the risk of transmitting contagious agents through fecal transplants is largely "theoretical" and that there are no reported cases of such transmissions.
In the U.S. and Canada, there's been growing interest in the therapy, and the Mayo Clinic has been one provider increasingly offering it. The Mayo Clinic in Arizona first performed a colonoscopic fecal transplant in 2011 for a patient with severe refractory C. difficile pseudomembranous colitis, with donated stool from the patient's brother.
"Unbelievably, the patient left the hospital 24 hours after the procedure, after having been bedridden for weeks," Mayo Clinic osteopathic doctor Robert Orenstein said in a media release.
Physicians may or may not be able to get reimbursed for the treatment -- most can bill for the colonoscopic portions of the transplant, if that's the method used -- although there may be a path ahead for it. The Centers for Medicare & Medicaid Services recently created a new code for fecal transplants, HCPCS Code G0455, covering the preparation and installation of fecal microbiota by any mechanism.
If fecal microbiota therapy does show efficacy -- there's currently an NIH-funded trial in progress -- it may end up helping reduce Medicare patient readmissions, because C. difficile is particularly dangerous in elderly patients living in nursing facilities.
The interest in the treatment also hints at a slow paradigm shift in medicine's approach to bacteria.
"Patients with (C. difficile) are missing certain gut flora, usually as a result of antibiotic use," the Mayo Clinic's Orenstein said. "I suspect some kind of signaling takes place between healthy bacteria and the mucosa of the gut, and without that signaling, C. difficile can take over. Restoring the missing flora seems to be the key."