Serious Or Life-Threatening

During the stakeholder teleconference with FDA on September 13, 2012, Dr. Sandra Kweder said that FDA considers ME/CFS to be a serious or life-threatening condition. In the world of FDA regulations, this is a very important designation. Here’s the full quote from Dr. Kweder:

We consider your condition to be in the category of serious or life threatening diseases. Okay, so all of the measures to move things through rapidly, all of the tools that we have here at FDA to try and expedite reviews or expedite development and work with companies to try and encourage them along that would apply to, you know, immediately life threatening cancer, as far as we’re concerned they apply to this condition. This is a serious condition and I just want to make that clear. We consider it in the same category because there are no approved treatments for this condition and we understand how seriously and severely peoples’ lives are impacted by this disease – by this condition. (FDA Stakeholder Teleconference Transcript, pp. 13-14)

But what does this actually mean? How did FDA make this determination? Richard Klein, FDA Office of Special Health Issues, provided me with some background information.

FDA’s definitions of serious and life-threatening come from the Federal Regulations governing FDA. According to the regulations, a serious disease or condition “has substantial impact on day-to-day functioning.  . . . Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.” (21 CFR 312.300) It is patently obvious that CFS meets this definition because of its devastating impact on day-to-day functioning.

The definition of life-threatening is found in several regulations. In the section on investigational new drugs, “life-threatening” means “(1) Diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted; and (2) Diseases or conditions with potentially fatal outcomes, where the end point of clinical trial analysis is survival.” (21 CFR 312.81) Another section of the regulations says “Immediately life-threatening disease or condition means a stage of disease in which there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment.” (21 CFR 312.300)

So is FDA saying that CFS is a life-threatening condition? I know many patients believe that it is a life-threatening condition, and Dr. Lily Chu had made a strong case for why research should examine the issue. But I suspect FDA has not drawn this conclusion. Note that Dr. Kweder said “serious OR life-threatening,” according to the transcript. Several patients discussed the possibility that CFS is life-threatening, but no one from FDA said that it was. It is unclear to me whether FDA believes CFS is life-threatening.

Having said that, it is very clear that FDA believes CFS is a serious condition. This is a very important designation because serious (or life-threatening) conditions are eligible for several drug review programs intended to speed drugs to market. In my next post, I will attempt to explain what these programs are and how they might help those of us waiting for treatment.


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4 Responses to Serious Or Life-Threatening

  1. Thanks for this clarification, Jennie. It’s very helpful and I agree with your analysis.

    I think it would be difficult to make a case that ME/CFS is “life-threatening” under those criteria (despite the possibly related deaths which occur from time to time) but “serious” has to be pretty much impossible to refute for most of us on the evidence and with those criteria.

    However, there may be a distinction to be drawn between patients whose lives are severely impacted and those for whom it is less so. That may be a problem, as it seems to be the disease itself which is classified rather than the indvidual patient. So while some patients with this condition might be classified as having a “serious” disease, others who are less impacted, might not – and I’m not sure where that would leave the overall classification of the disease.

    Of course, this is the heart of the problem with ME/CFS which is that there is such a huge disparity between patients’ experiences. I think it’s clear to many of us that we are probably dealing with a range of illnesses, not just one discrete condition. And that might be an obstacle to achieving the proper classification of “serious” for ME/CFS – which is what we so desperately need.

    Thanks again for all your work. I hope the pre-emptive resting is proving useful. I’ve had to cut right back on everything for the time being as I’ve gone downhill quite badly recently. And that’s the way it goes…..

    Best wishes – as ever.

    • Jennie Spotila says:

      I am not sure if the variation in individual patient severity matters to the FDA in this instance. For example, there is a well-defined range of severity in breast cancer. What matters is recognizing CFS in the class of “serious or life threatening.” That designation triggers some specific programs for drug review that I’ll cover in my next post.

      I am so sorry to hear you are struggling, Valerie. I hope you can still enjoy the holidays.

  2. Anne says:

    Thanks for the clarification, Jennie, much appreciated.

  3. Kelly Latta says:

    @Valerie Eliot Smith
    I think the problem here may be somewhat similar to AIDS only not remotely on the same scale. AIDS in and of itself does not kill anyone despite the nearly 100% mortality rate without treatment. Patients die of AIDS-related diseases such as Karposi’s Sarcoma. Or it could be said they die from complications. I know that sounds like wordplay, but it is similar to how the FDA defines severe or life-threatening. Being precise is important.

    I think it could be said that some ME and CFS patients die from complications related to ME and CFS, but this is where longitudinal studies would help.

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