Read this letter. It spells out all the issues and connects the dots. The author has granted permission to share it. I hope it inspires you as much as it has me.
Dear Secretary Sibelius, Dr. Koh, Dr. Maier, Dr. Unger, Dr. Lee, Dr. Fineberg, and Dr. Behney,
As a physician, health services researcher, and person affected by myalgic encephalomyelitis/ chronic fatigue syndrome (ME/CFS), I am writing to you today to request that you consider strongly the letter expert ME/CFS scientists and physicians sent September 23, 2013 and cancel the Department of Health and Human Services’ contract with the Institute of Medicine (IOM) to construct a clinical case definition for ME/CFS. While I am appreciative of DHHS’ continued interest in ME/CFS and recognize the important and influential role the IOM plays in the health of the nation, I believe that the money and resources spent on such a contract might not only be duplicative and better spent on other areas of ME/CFS research but may end up being harmful to patients in the short-term, by subjecting them to inappropriate treatments, and in the long-term, by obstructing and obscuring research progress. Patients, patient advocates, clinicians, and researchers understand these issues and thus, rather than greeting the contract with joy and enthusiasm expected, are instead contacting you with their concerns.
Duplicative efforts will waste time, resources, and money
1) Over the decades, a number of different clinical case definitions from different countries have been proposed for ME/CFS. Expert clinicians and researchers have reviewed all of them, found many to be unsatisfactory in describing patients, and, thus, came up with two different consensus-based definitions in the last decade, the Canadian Consensus Criteria (CCC, 2003) and the Myalgic Encephalomyelitis - International Consensus Criteria (ME-ICC, 2011). The experts recognize that both the CCC and ME-ICC need further validation and refinement but agree that these definitions are adequate enough to be used NOW both for clinical and research purposes. Indeed, use of the CCC has already yielded a possible treatment, rituximab, for some patients via a successful small trial in Norway.
2) DHHS’ own CFS Advisory Committee (CFSAC), recommended in October 2012 that “at least one stakeholders’ (Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS) experts, patients, advocates) workshop in consultation with CFSAC members [be convened] to reach a consensus for a case definition useful for research, diagnosis and treatment of ME/CFS beginning with the 2003 Canadian Consensus Definition for discussion purposes.” CFSAC did not ask for separate meetings to construct separate clinical and research definitions but for meetings to construct a case definition useful for multiple purposes.
3) Several studies, including the Centers for Disease Control and Prevention’s Multi-Site Clinical Assessment Study, and a meeting, an Evidence-based Methodology Workshop, are already being planned by the National Institutes of Health to address the issue of case definitions. It is unclear how IOM involvement would add value to the processes already underway. Instead too many cooks may spoil the broth.
4) Whenever patients, clinicians, researchers, advocates, or CFSAC have asked for increased funds for ME/CFS research and care, like for a Request for Applications (RFA) or for a Center of Excellence, they are told that there is no money. Yet, DHHS has money now to spend on a meeting rather than these repeated worthwhile requests?
Separate research/ clinical case definitions are harmful to patients, obstructive/ destructive to research efforts
The last 3 decades have demonstrated that the separation of clinical care and research has resulted in suboptimal, even harmful care, of patients and little progress in our understanding of the cause(s) of and treatment for ME/CFS.
As shown recently by the US Food and Drug Administration’s excellent final report from their ME/CFS Drug Development Workshop (The Voice of the Patient), post-exertional malaise (PEM), exacerbation of all ME/CFS symptoms (including pain, exhaustion, sore throat, insomnia, cognitive problems, etc.) with mild physical/ cognitive activity, is a key feature and disabling symptom of ME/CFS. PEM, not chronic fatigue, is why patients are bedridden, homebound, unemployed, and unable to walk a block. Clinicians from around the globe who see and take care of thousands of ME/CFS patients regularly are well aware of this symptom and thus chose it as a required symptom when constructing both the CCC and ME-ICC. The management of PEM is also different from chronic fatigue; rather than push patients to ignore PEM and to continue to engage in mental or physical activity, which could result in not only temporary but prolonged disability, experienced clinicians tell patients to balance their activity with rest to decrease the onset or severity of PEM.
In contrast, the emphasis on fatigue by the 1994 Fukuda and other case definitions promote the image of ME/CFS as a benign illness that can be overcome merely by a positive attitude, increased exercise, healthy diet, and enough sleep. This is reinforced by European-based clinical trials of cognitive behavioral therapy (CBT) and graded exercise therapy (GET) that claim to substantially improve the health of or even cure ME/CFS patients. These are double-blind randomized placebo-controlled clinical trials so they must be the best and last word in care, right? This treatment information is distributed widely in usually trusted resources such as the online medical database UpToDate. Yet a careful reading of those trials shows that frequently, subjects were selected primarily because of chronic fatigue and that a common primary outcome measure was fatigue reduction. PEM was neither required for subject selection nor measured as an outcome. (Aside from the fact that none of the trials report objective increases in activity, for example, via actigraphy.)
Consequently, when the results of those trials are applied in practice to patients with symptoms beyond only chronic fatigue, over 50% of thousands of patients surveyed over the last decade have stated that those treatments made them worse, not better. Patients who follow their physicians’ directions faithfully have ended up bedridden, some for days, others for years. The most recent IOM contract announcement mentions the 2007 NICE Guidelines for CFS/ME from the United Kingdom, where CBT and GET are mainstays of treatment. The NICE guidelines were not deemed to be “nice” but rather “unfit for purpose” by the ME Association and UK patients, who asked for a judicial review of that document. When the majority of people receiving a treatment are not getting better or even getting worse, we should ask WHY, not cling to the results of trials and doubt the words and experiences of patients.
Because most physicians are not educated about PEM and the limits of GET/ CBT trials, patients who do not improve substantially with or defer CBT or GET are either blamed for non-compliance or viewed as depressed, malingerers, or hypochondriacs. In 2011, the Centers for Disease Control and Prevention reported that 85% of clinicians still viewed ME/CFS as a wholly (14%) or partially psychiatric disorder (71%). A quarter of clinicians recommended referral to a psychologist as an initial treatment. This perception, coupled with lack of knowledge, is why hundreds of thousands of patients all over the United States cannot find a single knowledgeable and sympathetic physician to take care of them. It doesn’t matter if the patient visits Dr. “Average” at a rural private practice clinic or Dr. “Expert” at a metropolitan internationally respected university medical center. The attitude displayed and advice given is rarely different; when choosing “experts”, even those selected for their methodological/ analytic rather than clinical/ basic science skills, will DHHS or IOM consider screening for knowledge about or attitudes towards ME/CFS? Will those who view it as a primarily psychological or psychiatric illness be screened out? I understand that the current IOM Gulf War Illness panel is currently facing criticism from Gulf War veterans and even from the chairman of the GWI advisory committee, Jim Binns, that the panel includes members who don't think GWI is a physical illness. Will any ME/CFS IOM committee have the same problem?
This history is largely why I and other patients, now joined by expert clinicians and researchers, experience a collective shudder of fear and horror when they hear DHHS plans to a) construct a clinical case definition employing professionals unfamiliar with ME/CFS, b) separate from a research case definition, c) at several separate meetings no less. ME/CFS’s past is filled with examples of ineffective and harmful ideas and treatments visited upon patients without listening to their stories nor to those of the clinicians taking care of them. Confusion and harm has already been incurred by applying research based on one definition (e.g. Oxford-based PACE trials) to patients diagnosed with another definition (Fukuda) and by employing a research case definition (Fukuda), without a solid clinical grounding, that focuses on the wrong symptom. Why make that same mistake again?
We now have two case definitions, CCC and ME-ICC, vetted by experienced clinicians that are already being used in both practice and research. I see no need to waste further time, money, or energy on another consensus-based meeting when those resources could be better used to validate/ refine these definitions or find biomarkers, diagnostic tests, or treatments. Patients’ lives are passing by each minute, never to be regained; don’t make us to wait another 3 decades!