The job of Patient Advocate came upon me uninvited. I did not apply for this job, nor did I have any qualifications for it. I am a sculptor, not a doctor or a researcher. My daughter became sick with a mysterious fatigue illness and I was the obvious person to fill the job. Learning the job of a PA unfolds over time and there is no instruction manual. Certain ideas and thoughts can be transferred from former jobs and former lives, but much has to be learned from scratch. It is helpful in doing the PA job if you have a lot of time and a lot of money, as the solution to this disease takes a great deal of both. It would also be helpful to have an education in bio-chemistry, of which I have none. The most important qualification that a Patient Advocate needs is persistence and discipline. A PA also needs to remain objective and detached, even under the most extreme conditions. Every Patient Advocate has a patient. My patient is my daughter. The objective of this particular Patient Advocate is to make his daughter better. How to set about it is another matter, and turns out to be a complex and sustained set of illusive problems. While most doctors look at a broad and confusing set of symptoms and try to attach treatments to an entire cohort of partially differentiated patients, the PA’s problem is slightly different. The Patient Advocate, by job definition, is obliged to help one person - in this case, his daughter - his patient. Thus the PA is looking at one narrow and confusing set of symptoms, which makes his problem slightly easier.
I am the patient advocate of my 40-year-old daughter. She is housebound in St. Paul MN with CFS/ME. This blog presents reports from several lectures or conferences. It also attempts to define, in my own way, the role of a Patient Advocate. The premise is simple. I make my observations in hopes that they might be beneficial to others, in the same spirit of generosity that so many others' comments have been useful to me. These entries are presented for information only purposes. In no way should they be taken as medical advice. I am not a doctor and I do not want to be one.
CFSAC testimony - Dr. Joan Grobstein on Responsibility
The CFSAC committee met again on October 3-4, 2012. Public testimony can be seen in the above youtube clip and an accompanying one. As usual the testimonies of patients and advocates were varied, heartfelt and stirring. Over the years these presentation have done a great deal to publicly give a face to ME/CFS and to elaborate, in the most personal and often harrowing terms, the toll that it takes on patients. Collectively these testimonies are the strongest evidence of the case against the government for their weak response to this medical emergency. This year there is some slight evidence that the CFSAC committee and the federal government itself, principally the FDA, is waking up from their long slumber and beginning to take ME/CFS seriously.
Many years ago I came to realize that the testimonies of Dr. Joan Grobstein are particularly articulate and insightful. Dr. Grobstein has the capacity to distill various problems down to their essential matter - and to cut out the crap. Dr. Grobstein has repeatedly clarified the central issues and constructed a path that will lead us out of the quagmire impeding research and treatment into this illness. It has always been my opinion that this woman should be given a position where she could "make things happen". Certainly her background as a physician gives her a unique and important perspective. When concerned people are gathered to design a plan of how to attack this illness, Dr. Grobstein's name should be first on the list.
Dr. Joan Grobstein's oral presentation at the October CFSAC meeting begins at 38:50 on the youtube video . Previous testimonies of Dr. Grobstein are available elsewhere on this blog or on youtube. Each of them is noteworthy.
In no way should this focus on Dr. Grobstein be seen as diminishing the myriad fine efforts of all the participants in the public testimony. Their testimonies do us all a very great service and I thank these individuals for the special time and effort that it takes to give testimony.
Two important items of Dr. Grobstein follow: The first is a transcript of Dr. Grobstein's oral statement delivered at the CFSAC meeting. The second is Dr. Grobsteins' five-page written submission.
"Hello. I’m Dr. Joan
Grobstein. I’m a physician.
My topic is responsibility.
Recently we’ve seen accomplished scientists honestly and
courageously admit that their original findings about XMRV were mistaken. They took responsibility for an
error. At its best, this is how
science works: form a hypothesis,
test the hypothesis, revise the hypothesis. We would like to see the same honesty and courage from
scientists at CDC about the empiric definition. The hypothesis that the empiric definition defines the same
disease as either Fukuda or the Canadian definitions has been tested and found
to be wrong. Scientists at CDC
must take responsibility for this error.
The original paper on XMRV in ME/CFS patients has been retracted. CDC
should retract all papers using the empiric definition or, at least, rename
them to indicate that they are studies of Idiopathic Chronic Fatigue and major
depression, not ME or CFS.
NIH must also take responsibility: fund studies that will clarify the definition controversy,
as it did with XMRV. Because we
saw money appear quickly to study XMRV, we know that funds are available. It’s clear that CDC’s new study about
the definition is unlikely to be impartial. They are vigorously defending their position. During a recent phone contact with
patients Dr. Unger said CDC could be “lumpers one day and splitters the
next”. This is a remarkably
unscientific statement. The
scientific method demands that we rigorously define the conditions of our
understanding this, how can Dr. Unger be trusted to undo the harm to ME and CFS
patients that the definitional conflict has caused? Leaders at CDC must take responsibility and ensure that
science done at CDC is impeccable.
There are biomarkers for Canadian-defined ME/CFS
patients. Does CDC wish to conceal
that these biomarkers are not found in empiric-defined CFS patients?
Turning now to the responsibilities of the voting members of
CFSAC, its staff, and its ex officio members, we’re all aware that very few
recommendations made by this Committee have been implemented.
For example, CFSAC made three specific revenue-neutral
recommendations which have been
ignored. The first, from 2009,
condemned the use of the empiric definition. Despite this recommendation, CDC continues to publish
studies using the empiric. The
second recommended removing the Toolkit from the CDC website. The third recommended making
IACFS/ME’s Primer widely available.
These last two recommendation were made 4 months ago. Today, the Toolkit remains on the CDC
website. Meanwhile, CFSAC’s own
website has a new link to the Toolkit and no link to the Primer.
Who’s responsible for the lack of action? Committee members have fulfilled their
responsibilities. It says on the
website they “asked” that these three things be done. On the contrary, they are experts, and, as such, they made
recommendations to the Secretary.
Yet neither the ex officio members nor the staff have apparently worked
on implementation. Dr. Koh’s
comments gave no indication that he is aware of the importance of these
recommendations. In a letter to
advocates explaining his refusal to meet, Dr. Koh said, “CFSAC provides a
mechanism to ensure stakeholders are engaged and have the
offer input.” Instead, it appears that
CFSAC provides a mechanism to ensure stakeholders are contained and have
opportunities to offer input that will be ignored.So
what is the point of CFSAC? If it
cannot accomplish these small, high impact tasks how can it accomplish what we
really need: significantly
increased funding for research, better education and improved clinical
care? CFSAC costs $235,000
annually. That sum could fund a
study of natural killer cell function in Canadian-defined patients, or create a
mechanism to identify an undiagnosed low-income patient, or educate a few more
physicians. I’m a taxpayer. I’m concerned that we’re spending
almost a quarter of a million dollars each year on a committee whose
recommendations are routinely ignored by DHHS.
Here’s a suggestion.
Ask ex officio members to take responsibility. Ask staff members to assign each recommendation to the
agency or agencies with responsibility for implementing that
recommendation. Ask each ex
officio member to report on activities to implement each recommendation at
every meeting. If nothing is
happening ask for explanations from higher levels within the agencies.
We need to see progress.
[Please read my written testimony which contains other
suggestions for progress.]
If you fund it they will come.
Progress in our understanding of ME and CFS has been hampered by a lack of funding for good research. We have been told by the NIH that funding levels are low because very few high quality applications are received addressing these diseases. Yet we are told by researchers who have had repeated success in obtaining NIH funding for research in other areas that their applications for funding for projects concerning CFS have been repeatedly rejected. Funding for clinical trials has been rare. This has resulted in a vicious cycle: few people enter the field out of fear that they will not be able to obtain funding, and thus there is a smaller pool of high quality applicants and applications. It is time to break that cycle with dedicated funding for ME and CFS research.
Following the model that resulted in Requests for Applications issued on September 21, 2012, by the National Cancer Institute, I suggest that several Institutes at the NIH develop a list of provocative questions (PQs) about ME and CFS that then lead to Funding Opportunity Announcements, using RO1 and R21 mechanisms. The total funding available in the NCI announcements was $64-84 million. In addition, $21 million was made available for a Clinical Trials Network, something that is also desperately needed to further our knowledge of ME and CFS. The relevant institutes that should be involved in developing the PQs are: National Cancer Institute (increased incidence of early malignancy), National Eye Institute (multiple ophthalmologic problems), National Heart, Lung, and Blood Institute (orthostasis, post-exertional malaise and altered blood volume), National Human Genome Research Institute (increased incidence in family members), National Institute of Allergy and Infectious Diseases (multiple co-infections, possible infectious etiology, immunological abnormalities), National Institute of Arthritis and Musculoskeletal and Skin Diseases (joint and muscle involvement), National Institute of Child Health and Human Development (pediatric ME and CFS), National Institute on Deafness and Other Communication Disorders (hyperacusis), National Institute of Diabetes and Digestive and Kidney Disorders (associated irritable bowel syndrome), Nation Institute of Environmental Science (associated chemical sensitivity), National Institute of General Medical Science (basic processes of cellular energy allocation), National Institute of Mental Health (adjustment of patient, family and community to chronic disease), National Institute on Minority Health and Health Disparities (no access to diagnosis or treatment for minorities and low-income individuals), National Institute of Neurology Diseases and Stroke (cognitive dysfunction, sleep disorders, autonomic nervous system dysfunction, possible disorders of glymphatic system), National Institute of Nursing Research (need for nursing care for homebound and bedbound patients, quality of life in a chronic illness), National Center for Complementary and Alternative Medicine (patients forced to use alternative medicine because no mainstream treatments available), NIH Clinical Center (clinical studies of bedbound patients), and National Center for Advancing Translational Sciences (need for rapid implementation of bench science in these diseases).
Many of these divisions are already represented on the Trans-NIH ME/CFS Research Working Group. This group should be tasked with formulating the Provocative Questions with the input of both expert clinicians, who have been working with affected patients for years, and also expert patients. Because patients have been largely abandoned by the scientific and medical communities, many have developed considerable expertise in pathophysiology and available therapeutics. We know which Questions are the most Provocative, because we live with these questions every day.
The Working Group is currently reported to be meeting monthly. An accelerated schedule will be necessary to develop and prioritize the Provocative Questions, so that RFAs can be issued as soon as possible. A high priority should be given to studies of potential therapeutic agents, because there are no therapeutic agents currently approved for the treatment of ME or CFS. Similarly, there are no validated biomarkers to use for diagnosis or to follow responses to therapies. This is also a very high priority. Of note, expert clinicians are using therapies that they believe to be effective and also use biomarkers to evaluate those therapies.
Unlike cancer, which has been well-funded and extensively studied for decades, ME and CFS have largely been ignored. Although there are over 5000 published studies, most are small and many have not used rigorous criteria for inclusion of subjects. The required funding for this first initiative is therefore larger than in the cancer initiative described above. It is, of course, impossible to know how much funding is needed to advance our knowledge of ME and CFS to the point where patients’ lives will be measurably improved. However, it is possible to make an educated guess based on current NIH funding for Multiple Sclerosis, a disease which causes a similar level of disability and may well be a related or overlapping condition. MS is estimated to affect approximately 400,000 Americans and receives $121 million per year from NIH for research. ME and CFS are estimated to affect at least 1,000,000 Americans. It therefore seems reasonable to allocate $302 million annually to ME and CFS research.
A Barrier to Productive Research Besides Funding
The biggest problem for ME and CFS research has been a somewhat esoteric one: there have been multiple definitions of the disease, some of which have been overly broad, have not focussed on the most important and unique features of the diseases, and have been shown to include large numbers of patients with other disorders as their primary diagnosis. This has resulted in wildly varying results and has made it appear that there are no consistent biomarkers or responses to therapeutic agents. It has also given an erroneous picture of the diseases. For example, because large numbers of patients with major depressive disorder were included in cohorts defined by a definition developed by the CDC (the empiric definition) and probably also by another definition developed by physicians in the UK (the Oxford criteria), it has appeared that ME and CFS could be psychological disorders which may respond to cognitive behavioral therapy and a program of increasing exercise intensity (called graded exercise therapy or GET). However, even in this diluted population the benefits of these therapies are quite minimal. There is evidence that GET is actually harmful to patients with ME and CFS defined by more stringent criteria preferred by expert clinicians. It is therefore extremely important that research funded by the NIH use an appropriate definition during patient selection.
The International Association for Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (IACFS/ME), an organizations of clinical and research experts, has recommended using the Canadian Consensus Criteria (CCC) to define ME/CFS in its CFS/ME Primer for Clinical Practitioners. Patient groups also support this definition. Thus, it seems reasonable to use this definition in NIH research.
Most (but not all, as noted above) research in the past has used the Fukuda definition, developed at the CDC in 1994. The problem with Fukuda is that it does not insist on post-exertional malaise, which is probably the cardinal symptom of ME. It also does not give a higher priority to the presence of other very important features of ME: orthostasis (not even mentioned in Fukuda), cognitive dysfunction and sleep disorders. Until the issues in the definition are clarified, it will be necessary for every study funded by the NIH to also classify patients using Fukuda and, in addition to standard data analysis, to separately analyze the data for patients who meet Fukuda criteria and those who meet Canadian criteria (many will meet both). Thus, over time it will get clearer if there are distinctions between groups defined by these two criteria. It seems likely that CCC defines ME and Fukuda defines a less-specific group that has been called CFS. However, only consistent data analysis will provide this clarification.
There was recently an effort among researchers, including the CDC, to establish a minimal data set for ME and CFS research in order to begin to answer some of the questions about differences between definitions and also basic information about the diseases. However, I have recently heard that this effort has stalled, for unclear reasons. This is unfortunate. This could be one of the Provocative Questions: what data should be collected in every study in order to make patient cohorts uniform (or at least clarify when they are not) and enhance our understanding of these diseases? An NIH-funded review of the 5000 studies already done to see whether the definition used correlates in any way with the results would be very helpful.
The only study that is currently being done about the definition is a study by the CDC. This is problematic, because the CDC is the originator of both the Fukuda and the empiric definitions. They have continued to publish research using the empiric definition as recently as this year, although it has been condemned by CFSAC (see below), and most researchers now agree that it is overly broad and that research using the empiric definition is biased toward demonstrating psychopathology in CFS. Although it is possible that the next CDC definition will be an improvement, this seems unlikely. In a recent question-and-answer session with patients, the head of the CDC’s CFS program, Dr. Elizabeth Unger said that she thought the CDC could be “lumpers one day and splitters another” (lumpers referring to more inclusive definitions and splitters to more precise ones). She also said that she thought that heterogeneity has been the barrier [to moving forward in our understanding of ME and CFS]. It is unnerving that she thinks that heterogeneity is the barrier, and yet she is unwilling to improve consistency by promoting a splitter-definition over a lumper. She may feel more of an allegiance to past failed efforts of the CDC to appropriately define the diseases than she does to rapidly advancing research that will help to alleviate the suffering of patients.
It is obvious that more research must be done to specifically address the problems that the multiple definitions have created. NIH should be actively promoting this effort. A possible solution is an NIH-funded symposium whose task would be to address the definition problem and the resulting methodological challenges with the goal of creating standards for research within a year.
It is important that outside groups either validate or challenge CDC’s efforts. However, this should not delay other research, especially in the areas of biomarkers and therapeutics. Patients should not have to wait for therapies because researchers are squabbling over the small details of definitions. We have two serviceable definitions: Canadian and Fukuda. For now, the best solution is probably to recruit only patients who meet both sets of criteria, but it may also be possible to use a study design that incorporates patients who meet one and/or the other. It is also possible to improve Fukuda by requiring post-exertional malaise. In any case, it must be very clear what definition is used, and it will be important to analyze data collected in such a way that it can be leveraged to clarify the definition problem. It is vital that the definition used be stated in the abstract to the article, so that clinicians and researchers can rapidly ascertain the study’s relevance to their patient population. Continuation of the effort to delineate minimal data sets is also very important.
Yet another barrier: will
To members of the patient community it does not appear that HHS is really interested in promoting research or other solutions to the problems and challenges that American citizens with ME and CFS face. We have asked to meet with high level officials in the Department and have been told that our only avenue of communication with the Department is the Chronic Fatigue Syndrome Advisory Committee (CFSAC). Patients have attended meetings of the Committee with great difficulty and at great personal cost. The Committee has made some excellent recommendations to the Secretary, but these recommendations have mostly not been implemented. Most recently, three revenue-neutral recommendations have been ignored: a condemnation of the empiric definition (2009), a recommendation to remove the CDC’s clinical guidelines from their website (2012--see Mary Dimmock’s testimony about the Toolkit), and a recommendation to post the IACFS/ME Primer instead (2012). There have also been multiple recommendations to increase funding for research and for centers of excellence for research and clinical care. NIH research funding for CFS is currently $6 million per year. This level of funding is 220th out of 232 diseases. There is no funding for ME, i.e., no funding that requires using the Canadian definition. There are no centers of excellence.
No HHS official has given an explanation as to why the three important, revenue-neutral recommendations (empiric definition, toolkit and primer) have been ignored. To date, Dr. Koh’s comments have given no indication that he is aware of those recommendations or of their importance. At this point, many patients are asking, what is the point of this committee? If it cannot accomplish small tasks that probably only require a single bureaucrat’s signature how can it ever accomplish something more substantive like significantly increased funding for research, education and clinical care? What, then, is the purpose of this committee? CFSAC costs $234,532 annually. This money could fund a study of natural killer cell function in Canadian Consensus Criteria-defined ME/CFS patients and/or a study to determine appropriate dosing of rituximab for ME. As a taxpayer, I am extremely concerned that we are spending nearly a quarter of a million dollars each year on a committee whose recommendations are routinely ignored by HHS. We have begun discussions about this problem with our legislators.
We feel that CFSAC, as it is currently functioning, is not meeting our need for an urgent, coordinated and fully funded federal response to ME and CFS. Here is a link to the reply we received from Dr. Koh, Assistant Secretary for Health, when we asked to meet with him and key deputies to discuss our concerns: http://bit.ly/TnoClG . The full text of the letter can also be read in testimony submitted to CFSAC by Denise Lopez-Majano. Of note, in response to a request for patients to have direct input on the Ad Hoc workgroup Dr. Koh stated, “While the [Ad hoc] workgroup and its charge are inherently internal to HHS, some of the members are also ex officio members of the CFSAC where they hear the stakeholder perspective. CFSAC provides a mechanism to ensure stakeholders are engaged and have the opportunities to offer input.” I would suggest that CFSAC keeps stakeholders engaged in activity that consistently leads to no results and gives the opportunity to offer input that is consistently ignored. This has to change.
If CFSAC is to become a more effective avenue for cooperation between the patient, clinical and research communities and the federal government that produces substantive action to rapidly improve the lives of citizens with ME and CFS, we need to see some changes in how it functions. We need to be able to question the ex officio members and Dr. Koh directly. We need frequent updates on the activities of the Ad hoc workgroup (we have had none to date). We need to see the promised report from the NIH the State of the Knowledge conference 18 months ago. We need to discuss many other important issues. We need to see substantive recommendations implemented. We need to see progress. If CFSAC continues to be ineffective and its recommendations are ignored, it should be disbanded. A more productive process must be implemented. We need to meet with Dr. Koh and key deputies from HHS to discuss these issues.
Thanks for post and to Dr Grobstein for her suggestions on how to move past the ghastly waste of patient's lives and treasure which the CFSAC has been. Note that those of us with ME the WHO accepted neurological disease, do not meet CFS Fukuda or CCC's agglomerated definition of ME/CFS. Patients with ME defined by Ramsey or Hyde as an acute,infectious onset neurological disease must be scientifically studied and not ignored as we have been since the CFS chimera was imposed on typical ME epidemics.ReplyDelete
Thanks for this interesting post. We are lucky to have Dr Grobstein. What an excellent analysis of what has gone wrong and what needs doing. It is extraordinary that CFSAC's recommendations continue to be ignored - even, as she says, the cost-neutral ones.ReplyDelete
Hello. I'm Erik Johnson.ReplyDelete
I'm an Incline Village survivor and participant in the 1987 Holmes et al "CFS patient study group" to define this "new syndrome", as
Dr. Cheney called it, before it had a name.
My topic is "responsibility"
When Dr Cheney asked me to "step up" and volunteer to help provide the immunological evidence that would force the CDC to listen to us about being truly ill, I refused. I told him that I wanted no part of it, and to get someone else.
There were others who were sicker than I, "Use one of them, instead." I said.
But Dr Cheney replied, "You are the only member of the original Tahoe cohort that is EBV negative. No one else can take your place.
We need your blood to prove this isn't CEBV Syndrome. You HAVE to volunteer."
So I did.
But there was something I expected in return.
For volunteering to start this new syndrome and becoming a prototype, researchers were supposed to talk to me about this strange sicknesss that happened to us at Lake Tahoe.
This was THEIR responsibility.
The first speaker, Dr Rosemary Underhill places a "request for some research" into cluster outbreaks.ReplyDelete
Since no "CFS researchers" are interested in the Lake Tahoe one, would someone please have
Dr Underhill contact me directly?