Clin Trials: September 2, 2013

Background The development of personalised (stratified) medicine is intrinsically dependent on an understanding of treatment-effect mechanisms (effects on therapeutic targets that mediate the effect of the treatment on clinical outcomes). There is a need for clinical trial data for the joint evaluation of treatment efficacy, the utility of predictive markers as indicators of treatment efficacy, and the mediational mechanisms proposed as the explanation of these effects.

Purpose (1) To review the problem of confounding (common causes) for the drawing of valid inferences concerning treatment-effect mechanisms, even when the data have been generated using a randomised controlled trial, and (2) to suggest and illustrate solutions to this problem of confounding. Read More