May 12th, 2013

FDA: Fecal Transplants Need Investigational New Drug Application

Gastroenterologists, ID doctors, C diff-sufferers, and microbiome-obsessed humanoids everywhere were treated to this surprising news recently — a decision by the FDA about fecal microbiota transplantation (FMT). From an email sent by the IDSA:

Because fecal microbiota transplantation (FMT) is not approved for any therapeutic purposes, an investigational new drug (IND) application is needed for the use of FMT to treat any disease including C. difficile infection.

More information is on the American Gastroenterological Association site here. This FDA decision was confirmed at a public workshop May 2-3 — “sold out” I’ve been told by one attendee — a meeting which included many interested clinicians and researchers. The FDA plans to hold internal discussions to further refine the regulatory issues surrounding FMT.

I certainly understand the perspective of the FDA, which must consider patient safety as its top priority. But the immediate practical effect of this decision is that getting FMT to the patients who need it — those with recurrent C diff — will now be much more difficult.

And the FDA decision underscores a profound irony in regulatory protocols in our country. A person struggling with C diff can walk into any pharmacy, Whole Foods, Trader Joe’s, GNC, etc, and spend a small fortune on various probiotics, the efficacy of which is marginal at best.

Yet to get FMT, a procedure that has demonstrated quite remarkable efficacy in multiple studies — including this prospective randomized controlled trial — that same patient will need his or her clinician to file an “emergency use” IND application, which is no easy task.

Let’s hope these FDA “internal discussions” proceed expediently. Otherwise, maybe clinicians doing FMT should just start calling it a “supplement.”

11 Responses to “FDA: Fecal Transplants Need Investigational New Drug Application”

  1. Bill Ray says:

    This may cause our neighbor to DIE! Sceduled for next week with non-rufundable reservations, etc.

    Pretty stupid FDA.

  2. robert barker says:

    I can only hope that therapeutic use of feces doesn’t suffer the same fate as colchicine.

  3. Rob Menak says:

    If only the FDA required this rigorous nonsense for other “feces” they’ve readily approved. e.g. donepezil 23 mg

  4. Philip MD says:

    Actually it’s great idea to re-frame FMT as supplement and or offer self treatment kit!

  5. Sue says:

    Thank you for explaining this. I wonder if the FDA’s decision will lead to more at-home transplants. We’ve come so far with C. diff. I pray the FDA proceeds quickly and wisely.

  6. Whit Johnson says:

    I would hope someone would file suit agst FDA seeking an injunction agst reg which addresses neither food nor drug.
    Oh wait, it IS food.
    Or was.

  7. Joyce says:

    Time to barrage the FDA with e-mails and letters that they are preventing us from practicing evidence-based medicine!

  8. Nice article. Actually, I am most concerned that the IND requirement will preclude attempts at treating acutely ill patients with life-threatening C.diff infections with FMT. To my mind, this is the most critical need for urgent study.
    I find the FDA’s decision very regrettable.

  9. anonymous says:

    This is heartbreaking. I am a 19 year old otherwise healthy college student with recurring C diff. (failed vanco.) I had an appointment scheduled for FMT for NEXT WEEK and was really counting on it to take care of C diff. before I study abroad for a year…

  10. Paul Sax says:

    Note that there are at least two sites in Boston (MGH and Lahey) that have an IND approved for FMT.

  11. Maggie says:

    A work around is to just perform a simple home enema with saline mixed with the stool of your healthy partner. If desired you can have them tested for disease but I view that as unnecessary since it is my own family and we are certainly aware of our disease states. This is done throughout Europe. The United States is just “ick” conscious. Worked great for us.

HIV Information: Author Paul Sax, M.D.

Paul E. Sax, MD

Contributing Editor

NEJM Journal Watch
Infectious Diseases

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