“CFS and ME is a serious disease or set of diseases for which there are currently no FDA approved therapies” ‘Voice of the Patient’ Document

The FDA’s Patient Focused Drug Development Initiative marks the first time the FDA is implicitly

listening people

The Chronic Fatigue Syndrome community spoke and the FDA really listened…

including patients narratives and needs into their drug development process. Designed to more “systematically gather patients perspectives on their conditions and available therapies,” the Initiative was one response to Congress’s concern that chronic illnesses were being left out.

How the Initiative will ultimately affect drug development remains to be seen, but, in as misunderstood a disorder as ME/CFS, simply having the FDA listen to ME/CFS patients and then accurately report on what they said is a big step forward.

The good news is that the FDA really listened. The ‘Voice of the Patient’ document they produced emphasizes the costs (severity) of this illness, and cites the same two major symptoms (post-exertional malaise, cognitive problems) as the Canadian Consensus Criteria (CCC). If the IOM contract goes forward, this ‘Voice of the Patient’ document provides validation by an important branch of the government of key aspects of the CCC.

The FDA’s frequent statements, both in this document and elsewhere, acknowledging that they consider chronic fatigue syndrome to be a serious disorder on the order of heart disease and kidney failure, make it more and more difficult to continue to push the severity of this tragically misnamed disorder under the rug any more.

No one who sits through patient testimonies at CFSAC can help but be staggered by the problems many people with ME/CFS face, or the emotional and financial costs this illness produces. At CFSAC patients are, for the most part, preaching to the converted. The FDA Stakeholder’s meeting gave the chronic fatigue syndrome community a chance to talk to an entirely new audience – an audience desperately in need of hearing the real facts. This document suggests that audience heard them.

Key Themes

“The patient input … strengthens our understanding of the burden of CFS and ME on patients …”‘Voice of the Patient’ Document

That the key themes the FDA laid out are in fact the key themes of this illness suggests the FDA listened well.


The FDA highlighted the knock-out punch that felled so many people with ME/CFS so quickly

Acute Onset – The first theme the FDA mentioned is one of the most striking aspects of this illness – the fact that many people can remember the day they got ill. This sudden onset is just not just interesting, but scientifically relevant as well. The fact that the FDA put this theme first – a theme that can only be intellectually intriguing to the medically community –  was encouraging.

Huge Loss of Functionality – The FDA noted that many people with ME/CFS “were formerly highly successful and productive professionals or students … who now struggle with even the simplest aspects of day to day living.”

Key Symptoms Outlined – “Of over 50 symptoms identified, the most frequently mentioned included severe fatigue or exhaustion, impairments in cognitive functioning (e.g., concentrating or processing information), chronic pain, sleep difficulties, blood pressure drops and dizziness, sensitivity to light, sound and temperature, and susceptibility to infection.”

The Key Symptom in Chronic Fatigue Syndrome Highlighted – The FDA highlighting of PEM, again suggests that they heard and picked up on a key aspect of ME/CFS: ”Post-exertional malaise refers to crashes that exacerbate symptoms that are often caused by even minimal exertion. They can last for days, weeks or even months.”

Treatments Desperately Needed – People with ME/CFS have tried many treatments, often without success. “Patients are desperate for research and development of treatments that can: (a) better relieve their most significant symptoms and (b) address the underlying cause(s) of their disease.”

Devastating Effects on All Aspects of Life – “CFS and ME takes a devastating toll on the lives of many patients and their families, including loss of careers, decreased quality of family life, social isolation, and feelings of hopelessness.

Panel Discussion

In the overview of the panel discussion, the FDA emphasized that patients reported ME/CFS being much more than about fatigue, again highlighted the cognitive and post-exertional problems, reported that crashes can be triggered by even minimal exertion, and that abrupt onset often occurred after some sort of systemic infection.


The FDA highlighted three categories of symptoms: cognitive symptoms, physical symptoms, and post-exertional malaise/crashes.

brain image

The FDA suggested that cognitive and neurological symptoms were the most distressing found in ME/CFS

Cognitive – The FDA gave first place to cognitive and neurological symptoms which they suggested, based on a show of hands, were the most debilitating. (Personally, I would disagree with that . Fatigue/PEM is much more debilitating for me, but then again I’m a lot healthier than most.)

After listing the many different types of cognitive symptoms found, the FDA noted that some formerly very successful people reported an inability to conduct basic activities such as reading or writing, that concentration periods could be very short, decision-making was ‘very stressful’, and communicating could be very difficult.

Physical – Severe fatigue and exhaustion and weakness lead a long list of physical symptoms, with “many participants” reporting they were unable to stand for long or walk “even a few blocks” or sustain activities for long. The FDA categorized symptoms according to how frequently they were perceived to have occurred.

  • Many participants” reported problem with sleep (insomnia, unrefreshing sleep, etc.), chronic pain (headaches, migraines, nerve pain, stomach pain), sore throats, hypersensitivity to light, sound, and other stimuli.
  • Some people reported orthostatic intolerance and similar symptoms (dizziness, spatial disorientation, and gastrointestinal symptoms.
  • A few reported problems with depression, fear and anxiety – “not as a symptom, but as a consequence of the devastating impact of the disease.” (Note that depression, fear and anxiety – all common outcomes of chronic illnesses – were at the bottom of the list.)

Post-Exertional Malaise

Some patients expressed the expectation and “legitimate fear of what happens when we overexert ourselves” knowing that “you will pay for it.”’Voice of the Patient’ document

The FDA – listening closely – gave the most attention to post-exertional malaise and the problem of ‘crashes’. Recognizing that PEM, more than anything else, defines the chronic fatigue syndrome experience, they dug more deeply into this symptom than any other.

It was the ‘collective experience’, the FDA said, of ‘acute, debilitating PEM’ that the audience gave the most attention to.

person lying down

The FDA considered post-exertional malaise to be the key symptom in ME/CFS…

It was gratifying to hear this for two reasons: One, the CCC, ICC and Pediatric Definition of ME/CFS agree. Two, the degree of post-exertional malaise found in ME/CFS is different from other disorders.

The fact that this term, which was coined by the ME/CFS community, has entered the lexicon of the medical community attests to that fact. (The first record of it in Pubmed is a 1994 paper by Dr. Komaroff.) Fibromyalgia has fibro-fog, cancer fatigue has ‘chemo-fog’, ME/CFS has ‘brain-fog’ – three similar terms to describe the similar cognitive issues found, but only ME/CFS has PEM or post-exertional malaise, and only in ME/CFS do ‘crashes’ make up such a major part of the illness.

Eskimos have different words for the many different types of snow; people with fibromyalgia have wonderfully descriptive terms for pain, and the chronic fatigue syndrome community’s ‘snow’ is ‘crashing’ – a wonderfully descriptive term in itself. ME/CFS patients described complete exhaustion, collapse, intense physical pain, incoherency, blacking out, memory loss, flu-like symptoms, being like a ‘brick’, feeling like their heads were ‘going to explode’, and the need for total darkness.

The fear caused by the unpredictability of crashes, the surprisingly mild exertion needed to provoke a crash, how the ‘postness’ of PEM can vary from minutes to days, were all noted by the FDA. It is interesting that the section on triggering crashes did not mention exercise, but poor sleep, stress, infection, and weather changes were mentioned. While crashes are a core part of ME/CFS, the FDA acknowledged that their duration and degree of severity ranges widely.


Impact is an important topic, as disorders with greater impact (and more unmet needs) are given some breaks in the FDA drug review process. A drug to treat lupus, for instance, which did not meet its endpoints at years end was nevertheless approved because of the dearth of treatment options available for that disorder.

FDA clearly understood the impact ME/CFS causes: they cited the ‘severe limitations’, the loss of careers, the need to take less fulfilling and less responsible jobs, the disability, the physical and social isolation, the inability to make plans given the illness’s variability, the ‘harsh financial’ losses, and, finally and fittingly, the feelings of ‘hopelessness, emptiness and despair’ that that these impacts can produce.


The FDA began by noting how ‘striking’ the many different treatments people with ME/CFS used were, and the widely varying degrees of efficacy achieved.

Despite the harsh treatment Hemispherx and Ampligen received from the FDA, the ‘Voice of the Patient’ document reported dramatic reports of improvement, including one person for whom Ampligen was a ‘miracle drug’. Stating that many patients went through a ‘complex process of trial and error’, the FDA acknowledged that patients were working hard to find relief.

Physical therapies such as stretching worked for some, but not for others.  Patients reported clever ways they’d adapted to their reduced energy levels, as well as a large variety of diagnostic tools and biomarkers (yes, the FDA said biomakers) clinicians used to monitor their progress.


Confusion and frustration marked ME/CFS patients search for good treatments

The FDA noted – much to the patients credit – that they felt that even small increments in improvement, could yield substantial improvements in quality of life.  That’s the mark of a ‘mature’ group of people who, while not happy with their situation, have adjusted well to it.

One of the most interesting aspects raised was the reduction in the effectiveness of treatments over time. For some reason, drugs that work tend to lose effectiveness in some people (many people?) – and then need to modulated or switched out – with something that does work, which then loses its effectiveness, which then needs to be switched out, etc.. This is an example of a system that Dr. Cheney said tended to pull back patients after he’d pushed in a healthier direction.

Frustration at the difficulty in  finding knowledgeable practitioners was  voiced, and then finally, and importantly, the FDA reported the willingness of some patients to undergo significant risks in the search for effective treatments.


It was a good first step for the FDA, but it was only a first step.  A guidance document remains to be produced, but more importantly the issues that have left a large community without the benefit of drugs or even drug company interest need

drugs for CFS

The FDA has a lot of work to do before drugs start flowing to ME/CFS patients

It was a good first step for the FDA, but it was only a first step.  A guidance document remains to be produced, but more importantly the issues that have left a large community without the benefit of drugs or even drug company interest need to be addressed – and it’s not clear that the FDA, at this point, is interested in taking that on.

Tomorrow the FDA will be the subject of a congressional hearing that will address how well it is implementing  the FDASIA bill that authorized the patient initiative, and directed the FDA to get more drugs more quickly to people with chronic illnesses.  The four key parts of FDASIA include

With regards to ME/CFS we expect questions to be asked about the FDA’s outreach (or lack of it) to drug companies for the Stakeholder Meeting.


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