Tuesday, 13 January 2015

ClinicSpeak: evidence-based vs. eminence-based medicine vs. quackery

The current evidence-base does not support any treatments for progressive MS. #ClinicSpeak #MSBlog #MSResearch

"We have had numerous discussions on this blog about evidence-based medicine (EBM) in relation to the treatment and management of MS. The Pharma industry equates EBM with getting a license and being able to market their treatment for a particular indication. Clinicians equate EBM with irrefutable evidence that a particular treatment works and is better than placebo or another treatment; this does not mean it has to be a licensed therapy. Licenses are really about money. What we clinicians use to make a decision is a weighing-up of the evidence; we usually require support from our peer-group before accepting the evidence as being good enough. In other words it is hard to be an outlier when it comes to practicing EBM. The latter is at odds with innovators; i.e. those clinicians who try something based on a scientific rationale or some other reason. We don't want to stop innovation as that is how a large number of advances have happened in medicine. You can usually identify innovators from quacks; their background and motivations for trying something are very different."

"For people with a chronic disease EBM can be frustrating as patients often want treatments that only promise a benefit; they are prepared to take a chance. The medical community is usually resistant to prescribing treatments that only promise a benefit because the scientific rationale is poor, the evidence-base is non-existent, the treatment may be risky or often the people offering the therapy are charlatans who are simply in it for the money."

"One of the main issues I have to counteract in MS is the off-label prescribing of DMTs in progressive MS. An example is alemtuzumab or bone marrow transplantation. The hype around these treatments has led to unrealistic expectations; even in MSers with progressive disease who are aware that the evidence base supporting these treatments in advanced MS is poor. I am continually having to explain to people why these treatments are inappropriate to use without a sound evidence-base in progressive MS. The other reason for push back is the risks associated with these treatments, balanced by undefined benefits. Saying no to my patients is increasingly making me unpopular. What we really need is better trials in progressive MS based on new insights, for example the use of combination therapy strategies, i.e. anti-inflammatory therapies in combination with neuroprotectives and new drugs that may promote remyelination. The problem is the regulators and pharma industry have yet to develop an appropriate and rational development plan for testing combination therapies in progressive MS. I hope the progressive MS alliance will address this issue."

"I read the editorial below in last week's BMJ (British Medical Journal) about EBM in orthopaedics and sports medicine; it made me smile as so much of it is relevant to neurology and the treatment of multiple sclerosis. I have provided some excerpts of you to read and ponder over."



Lohmander & Roos. The evidence base for orthopaedics and sports medicine. Cite this as: BMJ 2015;350:g7835

Excerpts

..... Medicine rests on an uneven evidence base. Some interventions are supported by large multicentre randomised controlled trials that have a low risk of bias and are powered for hard endpoints—a high level of evidence. Others depend on retrospective observational data that provide a lower level of evidence. Yet others were theorised and considered biologically or mechanistically plausible and are heirlooms of “eminence based medicine.”......

..... Some interventions are just plain wrong and have real costs and harms, without countervailing benefits. Medical reversals may occur when well done clinical trials, systematic reviews, and meta-analyses of trials find current practice to be no better than a lesser treatment or placebo. .....

...... Clinical impressions can be deceiving. Where high level evidence speaks against abundant clinical experience and ingrained and unquestioned routine, cognitive dissonance results. Defenders of questioned treatments focus on potential scientific flaws in the published trials to invalidate trial results and thereby to decrease their level of cognitive dissonance, while ignoring the inherent biases of clinical experience and the phenomenon of the physician as a placebo reactor. It was, for example, suggested that participants in sham controlled surgical trials “may not be of entirely sound mind” and research performed on such people “not generalisable to mentally healthy patients.”.....

.... Confirmation bias reigns and we ignore, or do not want to be exposed to, information or opinions that challenge what we already believe, while wanting to hear information and beliefs that confirm what we already believe. This human trait contributes to overconfidence in personal beliefs and maintains and strengthens beliefs in the face of contrary evidence. The effects are stronger for emotionally charged issues and deeply entrenched views. As a result, proponents of questioned interventions fight hard for their interventions and specialties and often delay change, when the appropriate and ethical action would be to abandon ship.....

CoI: multiple

6 comments:

  1. If you had a disease like PPMS and there was no approved medicine, would you want an untested treatment? If someone told you there was a ninety plus percent chance the treatment was a fraud, would you still want to take the chance even if only to prove the fraud?

    This may be an unfair question, but when science has thus far failed to provide a treatment which passes evidence based trials, should doctors prescribe an untested approach knowing it may be little more than a placebo? As a patient, I tend towards the "whatever works for me" approach even if the pills are only "ragus" pills (sugar backwards). Is there harm in allowing the patients mind to feign health when it appears hope is lost? Sadly, I am the type who immediately goes and looks up all new meds for the evidence, but I would not begrudge the patient who chooses ignorance when current research shows little near term cause for hope.

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    1. As long as the "whatever works for me" approach doesn't have the unfortunate side-effect of severe anorexia of the wallet ;-)

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  2. for PPMS I have read where there are sub-types with one being inflammation/immune-based; however I hear little of PPMS sub-types being discussed - perhaps because some of this sub-type research is fairly recent. Hence, when a study shows only a certain percentage to respond, and the overall impact is low I wonder if that is the treatment and/or not sub-typing the PPMS?

    also, that there is a wide variation in the progression rate of PPMSers would seem to indicate something is different. seems the way its done is to try DMT's and if a person doesn't respond then its PPMS. actually, I am now confused as I have read where some PPMSers have responded to DMT's; though then some are switched to a diagnosis of RRMS despite no major attack. then there are doctors who think PPMSers have low-level attacks.

    lastly, for bone marrow transplant there are anecdotal reports from PPMSers who went to Russia and state they are better post-treatment. to tie this into the above, I would like to know what was their PPMS sub-type (or if they were RRMS and not PPMS). seriously, why haven't any researchers followed up with these individuals? it's just a few at the moment, and so the cost to dig deeper would not seem that much. from what I can tell from patient blogs is that patients are speaking to each other; though I haven't read where any researchers have contacted these individuals.

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    1. Relapsing PPMSers and PPMSers, the former have gadolinium lesions (maybe you don't see the relapses) and respond to some of the current MS drugs.

      The labelling has issues when it comes to treatments as label someone with progressive MS and then you say there are no treatments but there are progressive MSers who will benefit from anti-inflammatories and these tend to be the people with the active lesions on MRI.

      People early in their disease may respond better than people later in the disease because they have brain reserve.

      Why haven't researchers followed up there is an issue of (a) collecting the data and (b) patient confidentiality If people are in registers like the MS register there disease course could be follwoed and if enough of people are honest and say what they are dong then something may come from it

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    2. Thanks for explaining more about the differences. I will ask me doctor at the next visit about gadolinium lesions.

      I will reach out to the PPMSers who have returned from Russia and reported positive results, and ask if they have given any thought to working with the neuros on a case study write-up. I agree that something more formal would help out.

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  3. The sad truth I list you don't know whether these treatments will work on progressive ms in a individual! So of course the tendency will be to give them a try! I feel the best hope at the minute for progressive ms is genbac1 of geneuro.

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