The end of placeboes is in sight

[rvallee]

Interesting article about overturning the use of placebo in clinical trials by relying on real-world data instead, to essentially use the patient population as control. It sadly sidesteps some facts about how the placebo-controlled part is sometimes omitted already without properly acknowledging it. PACE is typically referred to as a RCT despite being open label and having multiple treatment arms that muddy interpretation.

It also misses how some segments of medicine are currently specifically based on placebo and as such are impossible to properly control, their very aim is to change perception, not affect physiology. It seems that much of discussion about the rigor of clinical trials and medical research in general is based on aspirations about what they could be, rather than how they actually are.

There is some merit to this idea, to rely on real-world data, but what seems to be forgotten is the issue of patient populations where data is not properly recorded. In our case this would be devastating as most of the information is wrong except for patient-reported outcomes, which are rarely recorded at all and when they do get recorded are very distorted, even on official records.

So there remains the question of whether the comparisons would be fair or based on aspirational things like "50% of IAPT users recover" despite being 50% of a % of a % and the definition of "recovery" being meaningless despite being used in real-world practice. Meanwhile Australia still relies on misleading guidelines that claim 97% recovery rates for "CFS". Those serve as meaningless comparisons as they are completely made-up and further distorted.

“The primary purpose of a clinical trial is not to treat the individual who participates, it is to treat future patients,” says Charlotte Blease, a Keane Scholar at research initiative OpenNotes, which seeks to make healthcare more transparent. “Most patients are not debriefed after clinical trials and nobody even bothers to tell them afterwards what happened to the drug or what they were allocated, and that's really a terrible way to treat clinical participants.”

Fortunately, a potential solution to the ethical quandary raised by placebo groups is on the horizon. The digitisation of health records means real world evidence such as doctors’ notes and pathology reports could be used to create a synthetic control arm to replace clinical trials’ existing control arms.

This means rather than having patients taking a placebo in a clinical trial, the same results can be achieved by identifying patients in the real world at the same stage of illness and within a similar demographic. The same methodology that would usually be applied to analyse a control group can be applied to real world data to generate results that can be compared against the new treatment. For instance, this could involve analysing the efficacy of the real-world treatments being received by patients with a certain stage of cancer.

https://www.wired.co.uk/article/end-of-placebo-trials

 

[alktipping]

considering all the stories I have heard about peoples medical notes histories being very poorly recorded or with the wrong information .even patients notes being mixed in from other patients who have the same first and last names . this is pretty much guaranteed to fail .

 

[Snow Leopard]

I don't think this is justified except when the outcome is mortality - and in those cases, I don't think placebo controlled trials are the norm anyway.

 

[Peter T]

Not at all relevant here, but I have someone sifting for me some fifteen plus years of unsorted filing, papers, some junk mail and even unopened post. It included two previous attempts at new filing systems only very partially implemented. The proportion of unopened post varies from cardboard box to cardboard box depending on how well or otherwise I was at the time that I gave up on that heap and boxed it. Several months into the process averaging two or three hours per week sorting, the end of next week should see us get back to my original filing system that contains ordered information predating the first significant ME relapse that forced me to stop work completely.

My helper now knows the route to the nearest recycling point very well, and the company managing the site has probably noted a spike in their paper through put. Much of the process felt like losing a burden, particularly at first when we were addressing the last four years of almost totally unopened post, but interestingly now getting further back I am reliving previous relapses, which feels less positive.

What made me think about it in this context is that today we opened the envelope of a long term follow up request relating to the FINE study. It went into the shredding pile unexamined as it so obviously not worth expending any mental energy on now. However it did bring home how problematic follow up results must be in studies like this. What percentage of the drop out in such long term follow up is because the subject was not even well enough to deal with their post at that point?

 

[Adrian]

Interesting article about overturning the use of placebo in clinical trials by relying on real-world data instead, to essentially use the patient population as control.

Whilst it is good to look at real world data about treatments which may not be as effective as in trials (or more) it is important that strong methodologies would apply.

But it is concerning that they would suggest it as a replacement for trials. I would have thought this would mean releasing untried treatments into the general population and basically saying have a go and in a few years we will see what worked. Rather than having a carefully monitored (and ethically approved trial) which includes the ability to stop the trial if harm is happening.

 

[Jonathan Edwards]

Placebobabble

 

[rvallee]

considering all the stories I have heard about peoples medical notes histories being very poorly recorded or with the wrong information .even patients notes being mixed in from other patients who have the same first and last names . this is pretty much guaranteed to fail .

Most importantly in that outcomes are not actually part of those files. Or at least not consistently. They are all dependent on asking the right questions and that almost never happens. There's the additional fallacy of "patient didn't come back to see me therefore the issue is likely resolved".

It makes a lot of sense with diligent and accurate record-keeping. That is not even close to be the case. Some day, just not yet.