A proposal for ME Action: a commitment to evidence-based medicine

[JaneL]

But there is a difference that comes from culture and that difference is what is causing this misconception: the percentage split between

1. patients whose treatments are defined by scientific consensus and
2. patients making a good elective scientific bet that is not yet consensus

Maybe the split is 90:10 in the US and 99:1 in the UK, who knows?

Which treatments for ME should be classed under the second category? What makes a treatment a “good elective scientific bet”?

 

[InfiniteRubix]

Those are are questions that are answered by best practice research governance and project management, from which useful categories of status could be defined.

It doesn't matter if some categories are totally empty, which could be the case for nearly everything for us because of research neglect. We have to get those principles right first though, and take the world as we actually find it. Which scientific findings go where is a second order question.

 

[Guest 2176]

You mention Ampligen, which, as you say, does not have sufficient research evidence to support its use. As a hypothetical question, do you think it would be right for MEAction to advocate for individuals to be allowed access to Ampligen treatment on compassionate grounds?

I do! For one, from what I've read, the side effects were low, probably much lower than Rituximab, so from a cost benefit standpoint it makes a lot of sense.

Was there not thr usual standard of evidence supporting it having an effect? Correct. But not no effect at all. And one has to ethically weigh that against what not doing anything treatment wise in ME/CFS does , to morale and mortality.

Interestingly I think i may agree on a few points with @Jonathan Edwards , although we disagree on the topic that shall remain nameless (until December 1st). We agree on the evidence for efficacy of several treatments used in me/cfs--ivig for example, and probably ampligen--but we disagree on the cost benefit analysis and ethics.

I think that if there is weak evidence, but not no evidence, and there is a small sample size, things are worth trying in this illness and getting covered by insurance. "First do no harm" means also that if physicians offer no treatments they are harming the patients by neglect , because the suicide rates are astronomical.

Saline is a good example. It increases blood volume and produces profound but short lasting relief in many patients. Now, as @Jonathan Edwards is pointed out, the effect is/should not be lasting. Well--so what? The effect of feeling okay for three hours in this illness is HUGE from a psychological benefit. Palliative care is important in this illness for quality of life and also patients should have more access to experimental treatments even when the evidence base is poor. Its not the main thing that should be focused on (the main thing is an aggressive protest movement that improves funding, and allows for the evidence base to grow and become higher quality --"quantity has a quality all its own") but it should be done.

 

[Trish]

I do! For one, from what I've read, the side effects were low, probably much lower than Rituximab, so from a cost benefit standpoint it makes a lot of sense.

I would agree with you with one proviso - that the makers of Ampligen supply it on the basis of a clinical trial, so outcomes are properly recorded and studied and in the longer term either it will be withdrawn as ineffective for ME (as with Rituximab) or provided to many more people if it turns out to be effective.

 

[Jaybee00]

I agree with @debored13 here. I disagree with the approach of this exercise’s “cautionary principle”. Cautionary Principle might be OK for knee arthritis, but not good for a serious disease where you get worse over time. I believe aggressive approaches to treatment— with informed consent—are needed.

I support those who want to wait for multi-million dollar, multi-center RCT studies, but that’s not for me— not interested in waiting 25 years for this.

Cautionary principle: ME Action has a commitment to stick to established facts. We strive for high accuracy and prefer to err on the side of caution.

 

[Guest 2176]

I would agree with you with one proviso - that the makers of Ampligen supply it on the basis of a clinical trial, so outcomes are properly recorded and studied and in the longer term either it will be withdrawn as ineffective for ME (as with Rituximab) or provided to many more people if it turns out to be effective.

The makers of ampligen cant fund this. But the fda could subsidize it. Maybe we can build one less f35 and spend the savings on some kind of compassionate Care program

 

[Trish]

I disagree with the approach of this exercise’s “cautionary principle”.

I think the point is not what individual's decide to do. If patients want to experiment with unproven treatments that is their choice, but as an ME organisation, all information it produces about those treatments should clarify the evidence base when listing them so patients can make informed decisions.

It is about MEAction ensuring that the actions it takes and the materials it publishes are based on sound science. So for example, it would not be appropriate to publish a document that says some ME doctors recommend drug X for ME, without also explaining clearly whether there is clinical trial evidence supporting the efficacy of drug X in treating ME, and the strength of that clinical trial evidence - whether, for example, there has been a large multi centre double blind placebo controlled trial, and what it means if there has not.

 

[Wilhelmina Jenkins]

While I support those who are currently on Ampligen being allowed to continue, I do not think that ME Action should support the expansion of its use until the company that makes it answers the questions that were raised when FDA decided not to move it forward for approval. I would like to see an independent trial, preferably done by NIH.

 

[InfiniteRubix]

Can I offer a suggestion to short circuit this?

Otherwise, I think this ethical and philosophical mishmash for pwME has at least another 10 years left in it , leaving unnecessary space for confusion, misinterpretation, bias and very bad behaviour IMHO (especially as matters shoot off into the sunset irretrievably over time). I believe that this thread, the thread closed until 1st December and the MEpedia thread all circle the same point, and is also why @Michiel Tack 's letter was important. Community discussions risk being disproportionately driven by political considerations, because if it's not driven by the science, what else is it driven by?

I know that I'm going to be very direct, so please tell me if I've got this wrong and why.

I don't see how any structured, dependable, referable, authoritative or credible discussions can come from this site or MEpedia without settling the primacy of the first principle below, definitively. I write that with affection and live concern, because that first principle will be eroded otherwise.

So….:

The focus should be to settle the below structure of principles as indisputable - improving and fleshing them out. Anything else is a dangerous misstructure of time in our specific pwME context. I know that I am still labouring the same point boringly from my previous posts, but that is because I believe that it hasn't sunk in:

1. Firstly, accept that scientific findings and associated treatments exist on a spectrum of status/quality/consensus/etc, and that this spectrum is critically important and determinable
   1. Scientific consensus, vs some/partial consensus, vs mere ideas (can be properly defined and via previous example starting points I gave)
      
   2. There may be sub-criteria that detail some treatments e.g. duration of effect
2. Secondly, that this spectrum should be normalised as a frame of reference, ideally systemised as referable structured knowledge
   1. For example, as drop down options in MEpedia, options whose availability are defined by wiki admins/moderators, rather than as.hoc
3. Thirdly, only then are treatments systemised
   1. Tagging for status etc.
      
   2. Of course MEpedia articles already exist, but it's the logical chain of assumptions that I'm referring to
4. Fourthly, agreement that advocacy organisations should reflect that ethos/status in communications and strategy
   1. Agreeing that where they do not, there will be an explicit statement of exception with reasoning
      
   2. There maybe be different approaches or disagreements about the status of specific treatments, but brushing over the entire concept is dangerous

That list is serially dependent - you shouldn't be doing one without getting the previous points correct, either explicitly or implicitly, systematically or culturally. Otherwise it's a menu of whether process, probity or ethics are undermined.

All this seems so painfully obvious that I think some people may believe that I'm pointing out the inane, going back to first principles… It doesn't normally matter, if everyone is implicitly in agreement. But that's the point - we haven't even got everyone on the same page about point 1(!). That first point should not be in dispute and it apparently is - some recent threads could not have happened otherwise. That elephant in the room must be put to bed and can be, now.

To highlight where we appear to be, imagine that you are establishing a database that is tracking the status of scientific findings, you structure the database appropriately first. You then populate the database with treatments. You apply the structure of tags or other variables to each treatment. But to repeat, point one on the list above is an open issue, up in the air, going round in circles (implicitly or explicitly in many posts and several threads). So we couldn't even create such a database if we wanted to, wiki or otherwise, because you cannot structure anything if that basic first principle is in dispute, explicitly or implicitly.

As a result, there are a lot of "cart before the horse" discussions about which medicines are where and their specific benefits. These are a dangerous distraction from those first principles, because not everyone is on the same page. The principles might appear obvious, to the point of boring. And yet here we are, with this post going in circles to make the same point that I made before.

So, assuming I've not wildly misunderstood, the only thing that matters at the moment is settling the primacy of the first point, asking

• whether anyone disagrees with the first point and why? The issue is skirted around too often.
  
  
• then maybe address with a poll, if need be. Such a basic premise cannot be left open on a science focused platform

I'd love to be put in my place and reassured. But, as things stand, the long-term lifeblood of this site arguably depends on this. The S4ME committee are clearly on-board IMHO - the implicit must be made explicit to allow for acceptance as a norm by users.

Credibility is like an egg. You cannot unscramble it easily once scrambled.

 

[InfiniteRubix]

I maybe pasted into the wrong thread. But the MEpedia, the 1st Dec thread and this are all the same topic at root, at least in large part, IMHO.

 

[InfiniteRubix]

@Michiel Tack 's letter dealt with this all in different ways. My live worry is that, even with that letter, we are left here on S4ME with point 1 above put in question. The idea that a finding/treatment has a status as consensus/good bet/mere idea is determinable, important and not to be brushed over.

 

[Jonathan Edwards]

Cautionary Principle might be OK for knee arthritis, but not good for a serious disease where you get worse over time.

I don't know where the 'knee arthritis' came from but I can guess. Would you not consider this a serious disease that gets worse with time (and shortens life expectancy very considerably):

The cautionary principle that I have recommended over the years on these forums comes from working with people who without treatment might not be able to walk, feed themselves, clean themselves or anything else much. I have just as much respect for PWME and spent today contributing to a meeting aimed at providing ME research. But arguments that somehow the case is special for PWME leave me pretty cold.

 

[Trish]

But arguments that somehow the case is special for PWME leave me pretty cold.

I agree we have to be careful not to suggest ME is worse or more disabling or more deserving of research than any other condition. Having had a close friend die of motor neurone disease recently and another now going rapidly downhill physically and cognitively with Parkinsons' disease I think noone should argue ME is the only disease remaining with no effective treatment.

 

[InfiniteRubix]

arguments that somehow the case is special for PWME leave me pretty cold.

This is on the money. Being especially unfortunate, relative to many illnesses, mustn't make us subjective to the point of being blind or narcissistic. We must remain objective.

 

[Jonathan Edwards]

"First do no harm" means also that if physicians offer no treatments they are harming the patients by neglect , because the suicide rates are astronomical.

Saline is a good example. It increases blood volume and produces profound but short lasting relief in many patients. Now, as @Jonathan Edwards is pointed out, the effect is/should not be lasting. Well

I don't think I buy the assumption in the first argument. I strongly suspect that a lot of patients spend a lot of money on treatments offered (that should not be offered) because they are desperate and then find themselves not just with a chronic illness but with no money and in some cases no home as a result. I am sceptical that being honest with people and saving them expense is more likely to lead to suicide.

Saline increases blood volume, but so does drinking a pint of lager and eating some potato chips. The effect with the lager can probably be achieved quicker and is exactly the same. My objection to saline is not that its effect is short lasting but that you can get the same effect by normal ingestion. Some people would like to claim otherwise but I have looked into it in some detail in the past and I can see nothing to support that.

What seems such a crying shame is that millions and millions of dollars are spent on useless treatments for PWME that could be used for research to find something that works. As long as patients and physicians keep each other happy by sidestepping meaningful research nothing much will be learnt.

 

[Barry]

We must remain objective

Which is a major challenge for anyone who is severely ill, but is is exactly right nonetheless.

 

[InfiniteRubix]

Which is a major challenge for anyone who is severely ill, but is is exactly right nonetheless.

As someone who just ranted in my own long post, I couldn't agree more

 

[InfiniteRubix]

eating some potato chips

Cautionary tangential observation... I literally have been eating crisps for that reason - a vice with a purpose if I want a treat... And now my cholesterol has raised... Absurd, I know. But a cautionary tale of the nonsense we subject ourselves to.

What seems such a crying shame is that millions and millions of dollars are spent on useless treatments for PWME that could be used for research to find something that works

This

 

[Wilhelmina Jenkins]

I am sceptical that being honest with people and saving them expense is more likely to lead to suicide.

I can only speak from my own experience, but I agree with this. The majority of those that I have known personally, as well as those from whom I am separated by one degree, are those who have traveled from doctor to doctor, searching for a cure that doesn’t exist. Despair sets in.

I remember back in the ‘90s, there was a panic in the community when one of the top “experts” said that 12 of his patients had died in one year. I kept asking “Died of what?” until I found out that 6 were suicides and 6 were treatment-related.

I believe that most of us have thought about suicide because this is such a nightmare disease. But I think that we do better when we try to hold each other up for the long haul rather than perusing cures that have not yet been found.

 

[Hoopoe]

I've definitely made myself worse with trying to cure myself with dubious treatments. Better to accept that it's incurable (for now) and try to maintain quality of life.

Also a lot of money wasted that could have gone into research.

I think one reason this happens is that nobody tells us the harsh truth. That it's an incurable disease and that medicine doesn't have the answer. Instead there is often a lot of indifference or psychologization or downplaying or comments like "you're in perfect health" that create the impression that the problem is more a lack of good will on the part of the healthcare provider or poor patient/doctor relationship or bad guidelines. That logically then leads to the search for the "right doctor".

That's not the only factor of course, it's also incredibly difficult to just accept that there is no treatment. We patients also like to tell each other that it's curable.