Hutan
Senior Member
- Messages
- 1,099
- Location
- New Zealand
The problem is, and I feel ME clinicians know this, is the likelihood of multiple ME subsets, misdiagnosed ME patients, and other unknown confounding factors, that without some knowledge of pathophysiology in at least one subset then drug trials for drugs that really could work for a subset are doomed.
I currently think we need to understand more than near zero about ME and have more accurate diagnostic capabilities before RCTs are really worth spending the resources to do. So in that sense I disagree with many from S4ME and understand much more why patients and doctors experiment with off-label treatments.
But, by using a placebo treatment arm and blinding, even if quite a small percentage of participants have a significant response to the drug, then that will probably show up in the treatment averages. If researchers include a scattergram when reporting on a blinded trial, and relevant outcomes are chosen, then it's likely that good responses in any significant subset will be obvious. And that can lead to a closer look at what it is about those responders that is different to others, and to larger trials by other researchers, to see if there continues to be a subset of responders.
The cost of an RCT is not that much compared to the many costs of not doing RCTs, including
* many patients travelling to try a treatment that doesn't work;
* many patients losing credibility with their doctors and family and friends when they try to try an unproven treatment;
* many patients needlessly suffering side effects;
* clinicians damaging their reputations by promoting unproven treatments;
* the understanding of ME/CFS being delayed, as dead ends aren't quickly discarded and promising leads aren't followed up by a range of researchers;
* successful treatments not being accorded the credibility that they should have, with the result that any benefit only accrues to a select few able to access clinicians willing to prescribe the drug.