Responder Analyses and the Assessment of a Clinically Relevant Treatment Effect
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*Steven Snapinn, Amgen 

Keywords: Clinical endpoint, Dichotomization, Minimum clinically important difference, Power

Ideally, a clinical trial should be able to demonstrate not only a statistically significant improvement in the primary efficacy endpoint, but also that the magnitude of the effect is clinically relevant. One proposed approach to address this question is a responder analysis, in which a continuous primary efficacy measure is dichotomized into “responders” and “non-responders.” In this presentation I will discuss various weaknesses with this approach, including a potentially large cost in statistical efficiency, as well as its failure to achieve its main goal. I will propose an alternative approach in which the assessments of statistical significance and clinical relevance are separated.