Sunday, September 30, 2012

Really? The FDA said that? FDA considers CFS to be a serious or life-threatening condition.

FDA considers CFS to be a serious or life-threatening condition.

I was skimming various blogs and found this blog post. I'll post one paragraph (I broke it up for easier reading but kept the bullet point markers) she wrote but the entire post is worth reading. It looks like an excellent blog:

  • FDA considers CFS to be a serious or life-threatening condition. This means that drug applications can qualify for the Accelerated Approval Process. Accelerated Approval brings drugs to market after shorter clinical trials, and additional studies are done after that approval. This is how many HIV drugs have been approved, as well as drugs for cancer and other condition.

  • FDA will offer a webinar on “Excellence in Advocacy” in November 2012. The purpose of the webinar is to show advocates how they can speed the development and approval of treatments through engagement of researchers and pharmaceutical companies. I find it a little ironic that FDA will be telling us how to be better advocates, but I think this will be an important session. FDA has been through this process with HIV and other serious conditions; they must have a perspective on what works and what does not.

  • Ampligen will be considered for approval at a public meeting on December 20, 2012.This meeting will feature an FDA panel, as well as public comments from clinicians, patients and others.

  • FDA will hold the 1.5 to 2 day stakeholder meeting in the spring of 2013. This meeting will bring together other federal agencies, researchers, clinicians, pharmaceutical companies and patients. It emerged during the call that a major goal of the meeting will be identifying consensus endpoints. In this context, endpoints are the reliable quantitative measures that will be used in clinical trials to evaluate whether a treatment is working.

  • FDA takes no position on the name issue of ME v. CFS. What they focus on is whether a treatment improves patient condition. They are using the term ME/CFS as a framework for that process but take no position on lumping vs. splitting in defining the illness.

  • There are currently 8 open applications for new drugs to treat CFS. Dr. Teresa Michele stated that many of those applications are for nutritional supplements, with small early trials. FDA expects that agreement on measurable endpoints will speed and increase research into other treatments.

So I went to the FDA's website:

Drug Development for Myalgic Encephalomyelitis and Chronic Fatigue Syndrome (ME and CFS)

FDA will be planning a series of activities focused on drug development to treat the symptoms of Myalgic Encephalomyelitis and Chronic Fatigue Syndrome (ME and CFS) in order to explore:
  • The burden of disease that impacts the quality of life for ME and CFS patients;
  • The quantitative outcome measures or endpoints that determine if disease symptoms improve with intervention; and
  • How drug efficacy should be clinically tested based on these endpoints or measurements.
Please send correspondence regarding ME and CFS to
Please Note: The list of activities will be updated as each activity is announced.
Teleconference between FDA and Patients/Patient Advocates – September 13, 2012
Additional Resources:

Yesterday a friend posted this article from prohealth on facebook (which is what started me off on my quest for more info). An excerpt from the article:

She [Sandra Kweder MD] quickly demolished any concerns about whether the FDA believes chronic fatigue syndrome is a ‘serious and life-threatening disorder’ - stating that the agency considers ME/CFS on a par with cancer, diabetes, epilepsy, heart failure and other serious diseases.

This was an important statement, given that ‘serious and life-threatening’ is a key criterion in the FDA’s decisions on drugs selected for expedited review.

Let’s take a brief look at the options this designation may open up in future consideration of drugs for chronic fatigue syndrome:
Fast track – Drugs on Fast track status are accorded more frequent meetings with the FDA and are eligible for 'accelerated approval'; and the application can be submitted in pieces rather than all at once.

Accelerated Approval - The FDA can conditionally approve a drug before it has final confirmation of its effectiveness, thereby allowing the drug company to get the drug out to the patients more quickly and giving it the opportunity to raise funds for the final studies. This is particularly important for small drug companies with limited financial resources, such as Ampligen maker Hemispherx.

Priority Review - The FDA will aim to review the drug in six months instead of 10 months.
The FDA's designation of ME/CFS as a ‘serious and life-threatening disorder’ should make it more difficult for the NIH and CDC to justify spending such small sums on ME/CFS, and it gives ME/CFS advocates another plank they can use to promote their cause in the federal arena.