Furthermore, limited knowledge, insufficient research funding, and a lack of diagnostic tools diminish a clinician’s ability to provide optimal care. P2P Report Press Release
Funded by the NIH’S Pathways to Prevention (P2P) program to identify research gaps and methodological and scientific weaknesses in “topics that have incomplete or underdeveloped research”, the report did just that. In order to meet the criteria for having a report done a disorder must meet:
- Have a primary or secondary disease prevention focus.
- Have broad public health importance—key considerations are the severity of the problem and the feasibility of interventions.
- Have limited published data or incomplete or underdeveloped research.
- Have difficulty completing a systematic review and producing a report synthesizing published literature due to lack of randomized controlled trials.
- Have two or more NIH ICs or Offices committed to addressing the topic by participating in workshop activities (i.e., sponsor, steering committee, post-workshop meetings).
The second criteria means that the NIH recognizes chronic fatigue syndrome is a real problem; i.e. that it has “broad public health importance”.
No researchers, advocates or ME/CFS experts were involved. The Pathways to Prevention “Advancing the Research on Myalgic Encephalomyelitis/Chronic Fatigue Syndrome” was done entirely from outside the field. The five-member panel with its experts in the fields of biostatistics, epidemiology, clinical care, and clinical trial research came to ME/CFS freshly – they looked at it with open eyes – and they came down strongly on the need for more research.
The Institute of Medicine (IOM) report got much more press, but this report should, in some ways, cheer the ME/CFS community more. It suggested, as the IOM report did – but more so – that given a good look at the data, outsiders are going to roll up on the side of the ME/CFS community.
The report wasn’t perfect – some real opportunities were missed – but in its entirety the report was a major step forward for the chronic fatigue syndrome community. Powerful statements in the introductory section underscored the fact that the authors recognized the need present.
- “ME/CFS is an unmet public health need with an economic burden estimated to be between $2 billion and $7 billion in the United States.”
- “ME/CFS results in major disability for a large proportion of the people affected.”
- “Limited knowledge and research funding creates an additional burden for patients and health care providers.”
- “ME/CFS is an area where the research and health care community has frustrated its constituents, by failing to appropriately assess and treat the disease and by allowing patients to be stigmatized.”
The P2P panel started off the body of the document in similar fashion to the IOM by hammering home the key message that ME/CFS exists! It’s a real disease.
If ME/CFS is a waste-basket disorder – if it’s a bunch of disconnected disorders thrown together – then it’s easier to justify not spending much money on it. The idea that chronic fatigue syndrome is not real, may, in fact, be one of the key issues that’s stopping the NIH from funding it properly.
If ME/CFS, on the other hand, is a real disease which costs the U.S. around $5 billion a year, and results in major disability, etc. then the NIH has been mostly ignoring a real problem for many years.
If it’s true that ME/CFS has “a tremendous impact at the individual, family, and societal level” but that “clinicians have a “poor understanding of the condition” and “patients are typically underserved” then the NIH needs to step up.
Basic Needs Unmet
“Innovative biomedical research is urgently needed to develop a clear definition and diagnostic tools that will serve as a launch for bench to beside program that produce treatments.”
The report provided a long laundry list of unmet needs. It asserted that the most basic questions – what ME/CFS is and how it should be diagnosed – prevent the field from moving forward and patients are bearing the brunt of that.
It reported patients often have to make “extraordinary efforts at extreme personal and physical costs” simply to find a physician”. It stated that the scientific community has a “responsibility” to address the issues that ME/CFS bring, but that “limited” public and private research dollars have been available.
Treatments are unproven and have not been produced. Patients are often inappropriately labeled as having a primary psychological disorder. Work is” urgently needed” to develop a clear case definition and validated diagnostic tools and endpoints. The small clinical trials done have been fraught with methodological problems, lack power and are unable to produce insights into the disorder. Psychological therapies (which have dominated clinical trials) are not the way out. Long-term studies are needed. Few doctors understand appropriate management strategies (let alone treatment strategies). Little understanding exists of how the disease starts or manifests itself on a molecular level.
How to fix these problems? Much larger rigorously designed studies employing validated endpoints and an acceptable definition are needed. (Simply fixing the methodological problems cited and then produce studies of the size and the scope needed to properly research this disease, would require a major influx of dollars.)
Besides the methodological shortcomings the field is also simply dogged by a lack of research. The panel stated that research into many of the biological factors associated with ME/CFS has been neglected. Overall “minimal progress over the past twenty years to improve the state of science regarding ME/CFS” has been made.
“Strong evidence” nevertheless exists, of “immunologic and inflammatory pathologies, neurotransmitter signaling disruption, micorobiome perturbation, and metabolic or mitochondrial abnormalities exists”. The panel highlighted the need to better understand the role herpesviruses and other pathogens, infectious mononucleosis and genetics play.
The panel proposed the following actions take place:
Creating the Foundation
- A team of stakeholders (e.g., patients, clinicians, researchers, federal agencies) be assembled to reach consensus on the definition and parameters of ME/CFS. Among others diagnostic biomakers and clearly defined end points need to be produced.
- A national and international research network should be developed to clarify the case definition and to advance the field.
- A network of collaborative centers, focused on producing biomarkers and a variety of other factors should be established.
- A Working Group should be established to produce valid methodological approaches to ME/CFS.
- Genetic, epigenomic and protein expression, neuroimaging and metobolomics studies should be used to identify biomarkers. Epidemiological studies are needed to identify people at risk and understand prognosis and co-morbidity. Diagnostic and prognostic algorithms should be developed to identify who will develop ME/CFS following infection or other triggers. Drug repurposing efforts should be made to identify new ways of treating ME/CFS. Immunologic mechanisms of ME/CFS and pathways associated with disease progression must be defined and characterized. Identical twin studies should be done.
- More investigator-initiated studies, a career development pathway and small grant mechanisms, to assist minority entry into the field are needed.
- Partnerships across institutions to advance the research and develop new scientists should occur.
- Longitudinal studies (e.g., the Health and Retirement Study, the Nurses’ Health Study) should be leveraged.
- A national repository and central archive of de-identified data and tissue samples should be created.
- NIH Institutes and Centers not presently represented in the Trans-NIH ME/CFS Working Group should collaborate to capitalize on the tremendous opportunities to learn from other disciplines and diseases (e.g., Gulf War Syndrome, Lyme disease, fibromyalgia, multiple sclerosis, and Parkinson’s disease).
- The Oxford Definition should be retired.
- A curriculum should be produced to facilitate ME/CFS knowledge acquisition in physicians.
- The Health Resources and Services Administration (HRSA) should facilitate practitioner training.
- The NIH Clinical Center for clinical trials should be used and fast-track testing of new therapies be explored.
- A meeting on the state of ME/CFS treatment should be convened by the FDA and NIH.
- Telemedicine or home visits should be used to assist the severely ill and those unable to reach practitioners.
- Demonstration projects of patient-centered medical homes for people with ME/CFS should be developed.
- How people recover from ME/CFS should be identified.
- Another ME/CFS Expert Panel should be convened in five years to monitor progress.
Some Missed Opportunities
The report could have been stronger. Despite calls from several quarters the report never requested specific dollar amounts. While the body of the document clearly indicated ME/CFS was not receiving the funding necessary an explicit statement that ME/CFS receive funding commensurate with its size and needs would have been helpful. (On the other hand, the panel was asked to identify gaps in research not recommend dollar amounts.)
The panelists appeared to pull their punches linguistically at times, in the recommendations half of the document. After stating that biomedical research is urgently needed they “recommended” investing in it. Instead of stating that creating opportunities for new researchers are “needed” to energize the field they stated new researchers “could” energize the field. Their characterization of the number of researchers in the field as “relatively” small was inaccurate. With ME/CFS receiving ten to forty times less funding that other chronic disorders the number of researchers working in the field is positively tiny.
Time To Step Forward
Anyone reading the report could only draw one conclusion: the chronic fatigue syndrome field has many fundamental needs that need to be taken care of for the field to move forward. Centers of Excellence need to be produced. Research needs to be expanded. Studies need to be larger and more rigorously composed. New investigators need to be encouraged. Curriculums need to be developed. Physicians need to be trained.
The report offers a roadmap which if followed would change how this disorder is funded, researched and treated, profoundly. It provides, as Jenny Spotila, noted in a recent blog, a report card that can be used to assess the governments response.
The federal government has funded two independent reports – the IOM and P2P reports – that say essentially the same thing: it’s time for the NIH to finally get off its duff and engage with this disease like it’s a real disease. It’s time provide some real funding. The federal government commissioned these reports. Will it follow their recommendations? They haven’t yet….
- Check out Is ME/CFS Stuck for more on a doctor’s take of the federal governments lack of response so far – and what the patient community must do to move forward.
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