A Proposition on the Test Concerning the Existence of Responders

Abstract

Evidence-based medicine, nowadays, has become an indispensable rationale for clinical decision making and is based on a statistical analysis of the double-blind placebo controlled trial. For the analysis, in recent years, the Cox proportional hazards model has brought more precise interpretation of the differences in heterogeneity between participants of the trial. Nevertheless, when a statistically significant efficacy is gained, usually the intervention is widely carried out in practice for the similar patient group to the participants.

However, there remains the possibility that the significance is derived from the efficacy only to a part of the participants, that is, the existence of responders. Moreover, as heterogeneity is based on predictable risk factors and when the existence of responders is derived from an unknown confounder, the existence of the confounder is not elucidated by subgroup analysis.

In this issue, the way of estimating the presence or absence of the responders is proposed by means of Akaike information criterion (AIC), provided that a little ingenuity is needed to apply the AIC to the statistics composed of both the intervention and the placebo group data, as a pair.