The FDA Stakeholder’s Meeting for Chronic Fatigue Syndrome (ME/CFS) is set for April 25th/26th in Bethesda, Maryland.
The FDA Stakeholder Team began an effort in 2011 to accelerate drug development and discovery at the FDA for ME/CFS. With Chronic Fatigue Syndrome (ME/CFS) the only major disorder without have an FDA approved drug, our first goal was to get the FDA to create a “Stakeholders Meeting’ to clear up impediments to drug approval for this disorder. Patients, advocates and legislators provided support for this effort.
In July 2012, following a teleconference with members of the group, FDA announced it would host a “cross-agency coordinated multidisciplinary scientific workshop to address drug and biomarker development issues in ME/CFS”, making this the first Stakeholder’s Meeting at the FDA for a disease since the HIV/AIDS epidemic. On Sept 13, 2012 the FDA held an ME/CFS Advocacy Teleconference with patients and patient advocates.
Hundreds of patients and advocates have sent 1,000’s of testimonials to the FDA over the past couple of months and recently, Bob Miller’s hunger strike resulted in the inclusion of NIH personnel in the meeting.
Now the FDA has set the date (April 25th/26th, 2013) for a 1 1/2 day Stakeholders Meeting in Bethesda, Maryland. In response to requests from the FDA Stakeholder Team the FDA stated:
I am responding to your February 15 inquiry about the status of planning for the FDA ME/CFS Stakeholder meeting. The public workshop will be held on the afternoon of April 25 and all day, April 26, at the Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, MD 20814, Phone 301-897-9400.
The public workshop will be held on the afternoon of April 25 and all day, April 26, at the Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, MD 20814, Phone 301-897-9400.
Beginning at 1:00 pm on April 25, FDA will gather patients’ perspectives on CFS and ME through a facilitated group discussion focusing on two main topics: (1) disease symptoms and daily impacts that matter most to patients; and (2) patients’ perspectives on current approaches to treating CFS and ME.
The second day will include a scientific discussion with patients, clinicians, researchers and government experts focusing on how to identify quantitative outcome measures to determine if disease symptoms improve with specific drug interventions.
Additional information will be posted soon (see link below), including the Federal Register Notice that will describe the meeting format and provide details about how to register to attend the meeting.
Much remains to be done to determine the participants and agenda. Thanks for making this happen and thanks for your support.
We’ll be in touch.
The FDA Stakeholder Team
Much of the difficulty may be determining an objective means of proving drugs work for a very specific population well characterized population.
Ideally, a trial should have a single endpoint based on just one outcome measure. However, many larger (read more expensive) trials have a primary (composite) endpoint consisting of multiple outcome measures. Trial endpoints can be a quantitative measurement of a biochemical biomarker for example or a socioeconomic parameter such as quality-of-life.
Choosing the most important endpoints may also influence what drugs are tested. Big Pharma doesn’t really have much for improving specific cognitive issues such as reduced processing speed for example, but would a drug like Rituximab or Ampligen make a difference on multiple endpoints – in subgroups?
Lots of questions to be asked before April.
FDA
F-orever
D-elayed
A-mpligen
Thanks for posting Cort. Bob had asked the ME/CFS community to begin thinking of ideas for this meeting. Do you have any idea how he plans to gather this input, or how we can effectively put our heads together beforehand?
I think the whole community of ME / CFS patients have to push the FDA and other regulatory authorities for conditional approval of Ampligen, the only drug currently useful for us!
The rest is just idle chatter!
The next meeting in April we have to put on the agenda the use of Ampligen!
We want the approval of Ampligen!
Carla
Is this Stakeholder announcement information available in a concise form that would fit on a postcard-size mailing that I could print up and send to my doctors, present and past? Karen Hart
Karen,
This link has the most updated info about the meeting and FDA is supposed to keep it updated.
http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm
You can take the important data and place it on a postcard.
Bob
Dear Cort and friends,
Any officials from the CDC attending/invited? What do people involved with this conference feel the other outcomes to be addressed, outside of Ampligen approval? I am hoping to see Ampligen available ASAP, from a compassionate care platform, as well as more education and differentiation of M.E., as I am observing many saying they have M.E./CFS, when they are not aware of the true seriousness of M.E.. I noticed my blog was pulled, can only assume it was the death discussion…But insist that this fact must be emphasized, and a topic despite the negativity of it. And, financially, the earlier these patients are recognized and treated, the less impact on governmental disability funds. What a rewarding exchange! I loved the ‘Lazarus Effect’ comment one friend made, and I am one wh!o had a wonderful response to the Ampligen.Why can’t the patients be taught and tested for diagnostic markers? Let’s lessen the confusion for the patients, and address education for physicians. I am very optimistic and feel that our local governmental representatives become involved. Optimism rules.
To better health, Mary Silvey, RN