Will FDA Step Up?
The Federal Drug Administration held a conference call today to speak with CFS patients, advocacy groups and other interested parties. This is the first time in the history of this disease that FDA has communicated with the patient community in such a broad and direct way. I was fortunate enough to attend the conference call, and offer this summary of the discussion.
This call happened through the efforts of patients to secure an FDA Stakeholders Meeting. The meeting premise is modeled on a similar process between FDA, HIV patients and others in the 1980s and 1990s. Through sustained communication, FDA and HIV patients came to an understanding of the need for accelerated drug approvals balanced with FDA’s focus on safety and effectiveness. CFS patients and advocates have secured a commitment from Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER), to hold a similar meeting on ME/CFS. The goal of that Stakeholders Meeting is to discuss acceleration of treatments, access to treatments and evaluation of treatments. Understanding the burden of disease from the patient perspective and how to measure those effects are an essential part of developing new therapies.
Today’s call was led by Dr. Sandra Kweder, Deputy Director of the Office of New Drugs within CDER, and she provided some key information:
- 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 conditions.
- 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.
Two issues emerged during the discussion through the comments made by patients on the call. First was the issue of case definition. While FDA insisted that it does not take a position on definition or name, many patients pointed out that case definition shapes research results. It’s been well established that some criteria sweep in people with idiopathic chronic fatigue or depression. In order to evaluate if a drug helps people with ME/CFS, then the case population must be carefully defined. FDA seems to recognize this as an issue, but there was no clarity on what requirements they might make in study design.
The second issue was endpoint measurements: how do we know if a treatment is working? What can be measured in clinical trials to determine effectiveness? In a condition like high blood pressure, it’s clear that the desired endpoint is a lower blood pressure measurement. But in our disease, what can be reliably and quantitatively measured as endpoints? Multiple suggestions were made, including VO2max, natural killer cells, and actimetry data. Debra Waroff offered the “salad” endpoint: whether she is well enough to make a salad. Dr. Kweder seemed to like that measure, as it is a measurement of functional improvement in patients’ lives. Identifying a consensus around endpoints will be a major feature of the 2013 meeting.
Overall, I think this call was very positive. I would have liked to have heard more on FDA’s processes for orphan drugs and accelerated approvals. I also want to know more about how the 2013 meeting is being planned, and how patients can add the most value to that process. But I give FDA a lot of credit for holding today’s conference call and appearing to be so open to our input. If this is a sign of things to come, then I think we can have high expectations for the meeting in 2013.