A thread on what people with ME/CFS need in the way of service

[Jonathan Edwards]

There is perhaps the argument that case studies on new drugs won't happen in these 'no off-label prescribing clinics', to build up the evidence supporting a trial.

It's a thought but the reality is that individual case studies do not 'build up the evidence supporting a trial' unless there is some reliable controlling factor supporting the observations. When we started using rituximab we did 'case studies' in a sense, but we made sure that we were gathering data that could be compared to some sort of control information. In some cases that was detailed pharmacodynamic analysis of objective changes. In others it was historical control data that was good enough to provide high probability that changes were meaningful.

For ME/CFS the problem is that we have nothing much to use of this sort. There are any number of anecdotal stories of benefits of midodrine or fludrocortisone or modafinil but strangely no physician is, deep down, sufficiently convinced of 'building up evidence' to want to do a trial.

A trial with a positive result from any drug would transform our understanding of ME/CFS in a way never seen before - way beyond DecodeME. It would be guaranteed a knighthood in the UK.

 

[Hutan]

Perhaps it's just a matter of tweaking the wording to clarify what we are asking for, or at least being able to explain the detail when asked?

We want any new off-label prescribing to be part of a focussed effort to gather good-quality, useful data about efficacy. The expectation is not necessarily that a Phase 3 triple blinded trial with placebos necessarily springs from the service fully formed. But years of prescribing a drug followed by a token study of subjective reports from the people who are dedicated users is definitely not ok.

 

[Jonathan Edwards]

Perhaps it's just a matter of tweaking the wording to clarify what we are asking for, or at least being able to explain the detail when asked?

Maybe it should tweaked but I think we have had this discussion already and my point was that the wording is actually exactly what it should be. It says nothing about blinding or placebos, just carefully designed controls in a trial - and a case study is a trial. If people do not appreciate the significance then they are probably not the people to be experimenting so would do well to stick to licensed drugs!! I may be being a bit unfair but in EndME's terms the message lands where it should do.

 

[Hutan]

Yes, it's just that the target audience isn't just good researchers; we don't want to needlessly put anyone off it because they misunderstand something.

People who are sure that a drug they takes helps them could still be able to get access to it under this model. There is flexibility in what responsible evidence gathering looks like.

To be clear, I'm very happy with what we have and with the communications there have been about it. I just want us to make sure that no one gets the wrong idea about what is proposed. And that opponents have no chance to drum up negative feelings about it.

 

[Amw66]

There have been attempts at trials. LDN has had attempts to get a trial for around 10 years
Funding has been the issue .

The accepted bps medical narrative has probably worked against trials .

 

[Robert 1973]

Yes, it's just that the target audience isn't just good researchers; we don't want to needlessly put anyone off it because they misunderstand something.

People who are sure that a drug they takes helps them could still be able to get access to it under this model. There is flexibility in what responsible evidence gathering looks like.

To be clear, I'm very happy with what we have and with the communications there have been about it. I just want us to make sure that no one gets the wrong idea about what is proposed. And that opponents have no chance to drum up negative feelings about it.

I strongly agree with this. It may be unnecessary to make it clearer, but there is nothing to lose from and potentially a lot to gain. On that basis I think we/you should definitely add some extra explanation along the lines Hutan suggests above.