Rethinking pragmatic randomised controlled trials: introducing the “cohort multiple randomised controlled trial” design

The idea of multiple, patient-centered RCTs in a large cohort is a
breath of fresh air in the current regulation-dominated area of clinical
trials. I think its value to the community will be enhanced if attention
is also focused on the outcomes, usually of a nominal or categorical
nature, which are meaningful to and sought by the patients, their
families, and the community. We often measure what we can or what can
distinguish one treatment from another, but do not ask ourselves as often
whether that is what makes a difference to the patient, and that is what
s/he desires from the treatment. While using many measurements in clinical
trials, I have felt that there is a cut-off point on each scale which is
the threshold for what a patient or his physician would call an acceptable
or satisfactory response. Estimating the proportion of patients who
achieve this threshold simulates the real life situation far more
faithfully than the precise measurements on scales such as the VAS. Hence
my plea to consider this in the design of cmRCTs.

Competing interests:
None declared