Balance between “evidence” and “trials”

I was alerted to this in my twitter stream today- what is the importance of the randomised trials versus the “real world evidence”. The authors have mentioned about the high cost of “randomised trials”- which of course, are necessary in order to define compliance with the regulations.

In fact, I have always felt that the drug trials are “too good to be true”; and overwhelmingly “positive”; especially if they are biologics. Ipso facto, you can’t argue with the logic of blocking pathways. How efficacious are these is left to open judgement and by vanity metrics like “progression free survival”. Albeit, the cost to define the overall survival is very expensive, no doubt.

There’s a new kid on the block- Real World Evidence (RWE). From the quoted write up:

Although the definition of RWE is evolving, most associate RWE with data derived from medical practice among heterogeneous sets of patients in real-life settings, such as insurance claims data and clinical data from electronic health records.

My only contention- how do they address the heterogeneity in “real world data”? How will they weed out any erroneous assumptions? How will they actually separate the manifestations of other competing co-morbidities (if any) with the presumed actual effect of the drug in isolation.

RWE provides important insights into patterns of care, limitations to market uptake, health care use costs, and discovery of toxicities otherwise masked in highly selected patients inherently enrolled in RCTs.

This point is perfectly valid. The selection of patients in trials is subject to a lot of bias at the outset. Indeed, its a foregone conclusion that recurrent/advanced/progressive patients are unlikely to have “improved” outcomes- save the firepower for the “preserved” patients. But that skews the results towards a more favourable subset.

I am more interested to know how the actual molecular profiling will help in patient selection; especially for definitive treatments like radiation therapy. “Precision Oncology” is after all personalisation of treatments. More importantly, it should help in pre-selection of patients for dose escalation without the increased incidence of side effects. That would be the holy grail of treatment schedules.

A great read and food for thought.

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