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.