As the 16 January, 2015 deadline for responding to the controversial P2P draft report draws near, and in the interests of balance and representing the whole community, Phoenix Rising presents two differing views on how to react. Today, Clark Ellis flags up important content to critique and to praise in the report. In her article, Gabby Klein makes the case for protesting the P2P process and not responding to its content.
P2P, or not P2P, that is the question.
So what’s the answer? When you boil it down there are only really two options.
1. The P2P process is flawed and invalid and we should either fight it, or ignore it, rather than participate.
2. Flawed or not, it is happening and we should feed into the process.
If you want to feed into the process, even while remaining opposed perhaps, then this article will hopefully let you know what you can do without you having to analyse the nine-page draft report, or try to digest the lengthy discussion on the forum.
The draft was posted a few days before Christmas and is not as bad as many feared, but nevertheless it does have a number of really bad bits that we should challenge.
If you want to give feedback on the P2P report content then you have until January 16th. THIS COMING FRIDAY! After which a final report will be produced and published.
Simple instructions on how to provide feedback on the report and where to send them too (email or mail) can be found here.
The P2P site states, ”The information in this report is intended to help health care decision-makers—patients and clinicians, health system leaders, and policymakers, among others—make well-informed decisions and thereby improve the quality of health care services.”
Although the statement is somewhat ambiguous, it seems to point to any new disease definition being for clinical use, not for research. Clinical definitions should be looser than research definitions as not everyone with the same disease has the same symptoms.
It is an important distinction and even the International Consensus Criteria caters to the variable symptomology in patients, whereas a definition as used for research purposes would need to be stricter to ensure participants had a solid diagnosis. Though even the clinical definition that we are talking about in this the case of the P2P, should not be so loose that it encompasses many different fatiguing diseases.
My hope is that we end up with something between CCC and ICC, but I rarely get what I want. Does anybody, though?
Something we often forget is that us patients and advocates are largely on the same side. We all just want this nightmare to end, we want to see meaningful action from government agencies to get us there.
People might disagree with someone else’s assessment and whatever their call to arms might be, and there is nothing wrong with that. That doesn’t make them the enemy. We’re in this together, doing the best we can with limited personal resources.
I am completely behind advocates who state that the P2P process is worse than a joke from a Christmas cracker, and I think there is some evidence that the opposition to the process so far has been worthwhile. But, I personally think that at this stage, we have to be pragmatic and accept that P2P is happening regardless.
The money has been spent, the draft is here and the final version will follow soon. It is likely to impact us directly—positive or negative—whether we want it to or not.
After that, HHS will do something with it and this may make no difference, it might make things worse, or it might make things better. Whatever your prediction, I believe we have a better chance of a favourable outcome if we provide feedback.
Change occurs incrementally. And it is slow. This report and any outcomes from it may or may not represent change. We’ll have to see. But I think the draft report is much better than it could have been, and some parts should have our support. Other parts of the report are dangerous, ludicrous, or pointless, and should receive our strong opposition.
As @Sean states in the discussion thread for the draft report, “We mustn’t let the perfect be the enemy of the good.”
At this stage, I don’t think I will choose to spend any more energy actively opposing the process as I’m not sure that energy would be well spent. Despite its inadequacies, this report could still take us forward—though that is by no means certain, and has a better chance if we provide feedback.
I will be giving feedback on the content of the draft report—both positive and negative.
So what are the highlights?
Before we get to that, I would absolutely recommend reading the nine-page draft report if you can. For those who can’t, I have tried to come up with a list of five things that are probably most worth commenting on and which most people in the discussion thread seem to share a somewhat common view on. A suggested response is given in each case.
You may copy all or part of this, or write your own if you prefer. After the five bad things there are five good things listed about the report which are worth praising.
A special thanks to @Sasha for drafting a number of these points on the forum which I have unremorsefully stolen. Some I have adapted, so they may not represent her views accurately.
First, the bad.
1. Economic burden misstated as only $1 billion.
Line 6: ME/CFS is an unmet public health need with an economic burden estimated to be greater than $1 billion.
Suggested response:
This appears to be based on an incorrect and unsubstantiated statement made by the workshop facilitator, Dr. Carmen Green, during her introductory statement at the P2P workshop on 9 December (“As you have heard, ME/CFS is a major health issue for over one million adults, has an economic burden associated with $1 billion”). Given the number of patients and the severity of the disease, this figure clearly cannot be right.
In fact, the latest research on economic burden is a 2008 paper by Jason et al. (The economic impact of ME/CFS: individual and societal costs. Jason LA, Benton MC, Valentine L, Johnson A, Torres-Harding S. Dynamic Medicine 2008, 7:6, available at https://www.dynamic-med.com/content/7/1/6/ )Using a community-based sample of patients, he estimated economic burden at $18.7 billion. Using a tertiary sample, the estimate was $24.0 billion.
2. Education needed for patients and health care providers:
Line 79: Educational efforts are needed to help patients and their health care providers better understand this disease and scientific processes.
Line 87: Overall, limited patient and professional education has impaired progress in managing ME/CFS.
Suggested Response:
There is certainly an education problem which would benefit from an up-to-date message and appropriate resources to address the issue, but the need is predominantly within the medical community, and that of the broader general public, not in the patient.
In an environment where support has been so fundamentally lacking, patients in this community have had to become experts in their own disease and its management, well above the usual level of education found within other patient communities that have benefited from doctor-led support.
ME/CFS patients invariably find their doctors, the social services professions and the wider public are misinformed, hold out-of-date and disproven beliefs and often lack even a basic evidence-based understanding of the disease. Efforts to improve education should be weighted accordingly.
3. Research funding:
Lines 5-9: An estimated one million people, mostly women, are affected. ME/CFS is an unmet public health need with an economic burden estimated to be greater than $1 billion. 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.
Lines 213-221: Investing in bench-to-bedside to policy research for ME/CFS is recommended […] The NIH Institutes and Centers […] and other U.S. Department of Health and Human Services (HHS) agencies should coordinate research efforts to promote efficiency and effectiveness, while also using public/private partnerships to leverage and catalyze the use of existing NIH infrastructure and dollars.
Lines 390-391: There is a role for new and ongoing policies to spark innovation and fund new research. For instance, new avenues are needed to fund research, such as the Prescription Drug User Fee Act.
Suggested Response:
The report (lines 5-9) acknowledges that an estimated one million people in the US have ME/CFS and that it results in major disability for a large proportion of those affected. According to the UK Department of Health, “Estimates suggest that up to 25% of people with CFS/ME are so seriously affected that they are unable to perform most basic personal tasks and are confined to bed or spend the majority of the day in bed.”
Despite this disease burden upon patients and economic burden on society, the NIH spends an average of $5 million a year, and ME/CFS is 229th out of 237 ailments on NIH’s grant list, in terms of funds allocated. https://report.nih.gov/categorical_spending.aspx/
MS has a similar disability profile to ME/CFS but affects only 4 people for every 10 with ME/CFS. Nevertheless, it typically receives about $115 million in annual NIH research funding: so in proportion, MS receives roughly 60 times as much funding as ME/CFS.
The report must call upon ME/CFS to have approximate pro rata research parity with comparable diseases like MS, which would be an annual budget of $287 million.
Although the NIH’s budget has been cut in recent years, this is irrelevant. Other diseases must forego a tiny fraction of their research income so that ME/CFS can have its decades-overdue fair cut of the total NIH pot. Immediate fair redistribution is essential to allow progress.
The report as it stands, is lacking as it does not address the shortcomings of the NIH in direct funding of disease research into ME/CFS (lines 213-221 and 390-391).
4. Alternative medicine treatments are needed
Lines 272-276: Patients often choose clinical trials or complementary and alternative medicine because effective treatment is not available and because traditional health care is not meeting their needs. Studies investigating homeopathy, non-pharmacologic, complementary, and alternative medicine treatments are needed. Studies addressing biopsychosocial parameters (including the mind-body connection), function, and QOL should be encouraged.
Whereas some patients do choose alternative medicines due to the lack of mainstream evidence-based treatments, there is no strong evidence that these alternative treatments are worth pursuing. Despite hundreds of these alternative treatments being available already, patients remain sick.
Patients want mainstream pharmacologic treatments for a serious mainstream disease, based on appropriate trials and evidence, not alternatives such as homeopathy and biopsychosocial therapies that have a history of failure and lack evidence to support their appropriateness, effectiveness and safety.
5. Psychological therapies recommended as a standard part of multimodal treatment
Lines 92-93: Although psychological repercussions (e.g., depression) often follow ME/CFS, this is not a psychological disease in etiology.
Lines 113-116: Existing treatment studies (cognitive behavioral therapy [CBT] and graded exercise therapy [GET]) demonstrate measurable improvement, but this has not translated to improvements in quality of life (QOL). Thus, they are not a primary treatment strategy and should be used as a component of multimodal therapy.
Lines 282-284: Studies addressing biopsychosocial parameters (including the mind-body connection), function, and QOL should be encouraged.
Lines 362-364: The modest benefit from CBT should be studied as adjunct to other modalities of treatment such as self-management. Future treatment studies should evaluate multimodal therapies.
Lines 384-385: We believe there is a specific role for multimodal therapy.
The report (lines 92-93) acknowledges that ME/CFS does not have a psychological etiology. However, the report recommends ‘multimodal therapies’ with CBT as a component (lines 113-116, 362-364, and 384-385), and that studies addressing biopsychosocial parameters and function, the mind-body connection, biopsychosocial function, and QOL should be encouraged (lines 282-284).
There is nothing wrong, psychologically, with patients with ME/CFS that is not part of having a chronic disease. If patients are struggling psychologically to cope and to adjust to the distress of disease, then they should be able to access appropriate support to help them to cope, which might include some psychological therapies. If they are depressed, they should be offered treatment for their depression.
However, there should be a strict limit where psychological treatment is concerned so that patients are not given psychological interventions that are not supported by strong evidence of benefit. If patients do not require help to cope psychologically with having such a debilitating disease, they should be left alone, as is the case for other serious diseases such as multiple sclerosis, Parkinson’s disease or cancer.
There are no grounds for studying ME/CFS as a special psychological case, when it isn’t one, or imposing psychological treatments on patients who neither want nor need them. The current wording of the proposal would only serve to undermine progress. The overriding need is for biomedical research into the disease and appropriate recognition as an organic disease.
Now the good.
Some of the statements in the report are helpful and should be singled out for positive feedback. There may be some people out there who will try to get these statements removed or watered down, and if you don’t want them removed or edited then have your say.
The first positive is unquestionably positive, but it does highlight an inconsistency in the report that you may wish to comment on.
1. Oxford Definition should be retired:
Line 38: The Oxford criteria (published in the Journal of the Royal Society of Medicine in February 1991) are flawed and include people with other conditions, confounding the ability to interpret the science.
Line 365: Thus, for needed progress to occur we recommend (1) that the Oxford definition be retired.
Although this point is unquestionably good, we would still recommend specific feedback regarding this point, as follows:
While the report’s recommendation that the Oxford definition of ME/CFS be retired, is welcomed, it makes no sense for the report to reject the Oxford definition so strongly and yet include Oxford-definition trials, including the PACE trial, when assessing the efficacy of cognitive behavioural therapy (CBT) and graded exercise therapy (GET).
The report states (lines 108 and 113-114) that “Patients with ME/CFS are hopeful that research will lead to a cure […] Existing treatment studies (cognitive behavioural therapy [CBT] and graded exercise therapy [GET]) demonstrate measurable improvement”. This is seriously misleading: it gives the impression that the CBT/GET studies assessed were studies on ME/CFS patients, conflicting with the acknowledgement in lines 38-39 that the criteria are flawed and include people with other conditions.
Meta-analyses of ‘ME/CFS’ CBT and GET studies are presented in the recent AHRQ report (pp. 44, 59, and 60, with the diagnostic inclusion criteria listed on the pages that follow): https://www.effectivehealthcare.ahrq.gov/ehc/products/586/2004/chronic-fatigue-report-141209.pdf/
When the Oxford-definition studies are removed, the remaining CBT studies are a failure and the single remaining GET study only barely reaches statistical significance. Given that it included only 25 patients and, by its nature, could not be blinded, its results are extremely weak.
Please note that although data were collected in relation to Fukuda symptoms, it is not the case that a subset of Fukuda-criteria patients can be identified. Patients were asked about Fukuda criteria only in relation to the past week, not the required six months (p. 156 in this document: https://www.meactionuk.org.uk/FULL-Protocol-SEARCHABLE-version.pdf/ and in the ‘CFS case definition: the International (Centers for Disease Control and Prevention, CDC) criteria’ section of this document: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3776285/
It is important to make it clear in lines 113-114 that CBT and GET studies on these loosely defined ME/CFS patients, do not translate to measurable improvement in patients with ME/CFS as defined by stricter definition criteria.
2. Recognises the urgent need for innovative biomedical research:
Line 186: Innovative biomedical research is urgently needed to identify risk and therapeutic targets, and for translation efforts.
3. Spells out the biomedical research that is needed, from the development of biomarkers to the collection of biobank materials to ‘omics’-based drug repurposing:
Line 221-276: This is a long section of text—See draft report.
4. Emphasises the need to address issues that are meaningful to patients:
Line 80: The scientific community also has a responsibility to address issues that are meaningful to patients.
5. Not just fatigue:
Line 106: We noted a consistent constellation of symptoms: fatigue, post-exertional malaise, neurocognitive deficit, and pain.
Lines 82-86: There is reproducible evidence of neurocognitive dysfunction with abnormalities in functional magnetic resonance imaging (fMRI) and positron emission tomography (PET) studies. Strong evidence indicates immunologic and inflammatory pathologies, neurotransmitter signaling disruption, microbiome perturbation, and metabolic or mitochondrial abnormalities in ME/CFS, potentially important for defining and treating ME/CFS.
Provide your feedback here and remember to quote the line numbers from the report that your comments relate to.