Efficacy paradox and proportional contextual effect (PCE)

The “efficacy paradox” is when the effect of a treatment tested in an RCT, or evidence-based guideline advice, differs markedly from treatment benefits observed in clinical practice. This arises because in RCT reporting and guideline development treatment efficacy is judged by the separation of the treatment group from the placebo group (the specific treatment effect) whereas in clinical practice it is the overall treatment effect, which includes both specific and contextual responses, that patients experience. This paradox causes a disconnect between guidelines and clinical practice and ignores the importance of contextual response in clinical care. This article fully explains and discusses these issues and presents a possible way to reduce the paradox through an alteration in RCT reporting that shifts the focus to overall treatment benefit and the proportion (“proportional contextual effect”) that is explained by placebo and contextual effects.