@Jonathan Edwards
I really agree with you that I wish more ME docs would publish on their research. Many of them have their own treatment plans which they tout as useful. They may be - or they may not be. If they are, one must wonder why they don't publish something. Some of them do - Dr. Nancy Klimas is well known for publishing a great deal on her research while she treats patients, and continuing to seek funding for some therapies she would like to try (e.g. anakinra - she hasn't gotten the funding yet to do a trial). I can think of one very well known ME specialist who has promoted a wide variety of therapies over the years, with accompanying theories as to causation, yet to my knowledge has never published anything. He charges his patients quite a bit, and he doesn't share knowledge with the medical community. I think some of his ideas are intriguing, but they should be scientifically validated.
At the same time, I don't think it's unreasonable for doctors to do what they can to treat patients and to alleviate symptoms. One must be somewhat cautious when dealing with highly risky therapies of questionable benefit. However, I think there is sufficient justification right now for a doctor to treat a well-informed patient with rituximab based on the phase 2 double blind trials of the Norwegians. Sure, phase 3 evidence would be great to have, but in the meantime, patients are suffering, and doing nothing is not always the right choice. It's one thing to expect an ME specialist to treat patients and also put the data into a trial, but your average GP who is just trying to help his patients probably doesn't have enough patients, time, or specialized knowledge to do a trial in ME. You can argue that patients should then see a specialist in ME, and in theory, I agree - but practically, there are nowhere near enough ME specialists to treat the patients out there. Because the medical community has failed to really deal with this issue in a coordinated, effective way, compassionate doctors are left to their own to try to help their patients. It is an unenviable position for both doctor and patient, and one that can only really be solved by the medical community as a whole addressing the ME problem with resources proportional to its impact on patients.
Dr. Edwards, you are not part of the problem here - you have recognized a need to bring science and reason, as well as an open mind, to a difficult medical problem. In that sense, most of your peers are not so forward thinking.
I have, with the help of various physicians, the vast majority of whom have not been ME specialists, tried therapies that seemed at least plausible and where the risk did not seem excessive. Some have failed, but a few have been helpful. I would prefer to do it as part of a clinical trial, but given that my choice is often between doing nothing while waiting for a trial and doing something to try to help with symptoms or even get at the cause, I think you can see why I'm not willing to wait.
Obviously PCP's aren't going to all start trying rituximab on their patients, but I don't think it would unreasonable for a rheumatologist or even a hematologist with relevant experience to try it on a patient. I don't think it's at all unreasonable for PCP's to try drugs that may be effective, and to go only on the patient's subjective opinion of efficacy for now, provided the drug isn't overly risky.
I suspect that in your career as a rheumatologist, you had a few patients with very rare or unexplained illnesses that lacked known, effective therapies that you had to get creative with, and who did not number enough to be included in a trial. In these cases, one cannot just rely on evidence, but must use scientifically plausible, if unproven, treatments. Many docs will limit this to severe, life threatening illnesses where the risk of doing nothing is exceedingly high. I would argue that it's reasonable to apply a similar standard in treating patients who have unexplained illnesses with severe quality of life implications. Patients should be advised as to the risks, but ultimately if a patient wishes to take a more aggressive approach, a physician should respect this decision. It is, after all, the patient's life and health that is at risk.
That doesn't change the fact that ME specialists who continue to offer treatments to a wide array of patients should do so in the context of a clinical trial. I suspect many believe the treatments they offer are ineffective.
FDA approval shouldn't be the standard for insurance paying for treatments. They are too slow and bureaucratic an organization, and they do not seem to take ME seriously at all. I believe that a physician believing a treatment is medically justified and plausible is sufficient grounds for insurance to pay for a treatment. I'm sure some physicians would abuse this, but on the whole, I think that's a lot better than patients suffering because bureaucrats and pencil pushers get in between highly educated medical professionals and patients who need their help.
It's such a catch-22 right now for patients. We feel like we are in a no-win situation. Many, like me, never thought it possible they could end up in a situation where they were seriously ill and for decades the medical establishment, government, and insurance companies basically ignored them. That said, science has not failed us, but rather its practitioners. It unfortunately drives desperate patients to unscientific options, which I understand is exactly what you are trying to avoid. No one who is sick should be forced to fight the battle alone, with the medical profession having turned their collective backs on the patient - yet this is exactly what happens every day in the ME world. I don't think doctors understand how much patients are hurt by the lack of support and validation, the questioning of one's sanity and/or honesty, and the implication that having ME is a character flaw rather than an illness.
My younger sister has a very serious medical illness which is generally considered terminal - but one which is well understood and whose reality and severity is unquestioned. I don't envy her diagnosis. However, our experiences with the medical profession have been polar opposites. She has been treated by some of the world's finest physicians, using FDA approved drugs costing hundreds of thousands of dollars per year in a major, world-renowned university medical center in the USA. She has great trust in her doctors. I live in a very different world. When I meet a new doctor, I must worry about how he/she will judge me - and in fact often do not disclose that I have ME, for fear that it will lead the doctor to not take me seriously or want to help me. That is a sad state of affairs.
Again, I am not venting at you. You just happen to be the one who is here and actually bothers to listen to this underserved group of patients.
