UPDATE: This post has corrections (*) from Jocelyn.
The two-day meeting in Bethesda, MD (April 25-26) that had FDA policy makers, drug makers, and patients coming together was a big step for the FDA and those with Chronic Fatigue Syndrome or Myalgic Encephalomyelitis. If nothing else, it’s a sign that the FDA is listening–but what did it take for them to listen? Three decades of complacency, deferred drugs, and a man not eating for 11 days
One of the revelations that happened over the two-day meeting that *rattled the patient community is Kim McCleary leaving as longtime CEO of CFIDS Association.
(Some have said her stepping down is of concern; other patients say it will allow someone with a more patient-focused policy as opposed to one centered on research that was also tied to the CDC).
Additionally, the absence of Dr. Paul Cheney and other *prominent clinicians worried some patients at the meeting. In general, patients are concerned that the FDA is not meeting with or listening to the best and brightest in the field who work directly with patients or treat them. But the FDA is starting to bring in new faces and is starting to drop its long-time grip to CDC definitions and assumptions.
(Clinicians are not normally expected at these types of meetings. Of more concern was Dr. Dan Peterson not being invited, and that the most ill patients were unable to attend by the very nature that transport is near impossible without sending them into dangerous levels of system shock).
As Robert Miller told the board, patients need fast-tracked drugs: FDA did it for emergency HIV scripts and are doing it for Alzheimer’s drugs.
One sign of hope at the meeting was a representative from Hemispherx Biopharma (maker of the rejected drug Ampligen) reaffirming their willingness to work with FDA despite the drug board turning down Ampligen for widespread and general use. Drug makers will continue to be discouraged if rejection comes so readily as if a rubberstamp of ‘No’ was made for the ME/CFS drug sector. Patients cannot continue to co-opt other drugs for their symptoms and expect results prescribed for other conditions.
Some positives: the FDA announced before the meeting that ME/CFS made it into the supposed priority list of diseases to research and develop treatment plans for over the next three years. The FDA told patients that NIH has funds to to develop standardized testing for patients and drugs (the hope is that FDA and other federal medical authorities recognize questionnaires and acute, short-term tests don’t capture patients’ experiences with crashes, recovery, and ongoing symptoms). *From public testimony, some notable comments were:
Dr. James N. Baraniuk: A biomarker is better than subjective criteria.
Dr. Judy Mikovits: FDA would benefit better by looking to already-gathered knowledge bases and collaborations on data as compared to discussions on symptoms.
This patient community has little political capital behind it as well. U.S. Senator Harry Reid (NV) is one of the few politicians who acknowledges the condition, but his fight for any issue anymore is questionable.
The series produced for KDKA-AM focused on retroviruses or similar as a plausible cause for the condition. This came with the admission that I don’t know what causes this condition. Lacking an across-the-board biomarker, there are many plausible causes, yet the *trigger remains the same: patients suffer symptoms after “(an) insult to their immune system, which can include viruses, immunizations, or mold-related illness, among others.”
Patients have staked out their beliefs on blogs, social media, and through their respective treatment regimens–that leaves regulators having to weigh different types of evidence and patients cannibalizing their own resources by criticizing others’ stances.
The ME/CFS patient community needs brave researchers, less-than-patient doctors, and diligent advocates that don’t have this condition. The FDA has stuck its head out the door for the first time in three decades–will it follow through and continue to engage the patient community? Or is this a dog and pony show with empty promises?