Expectancy Optimized Trials (EXOT) – Offene Verumbedingungen in klinischen Studien

Abstract

Clinical trials to evaluate the therapeutic effect of pharmacological interventions are mostly conducted as double-blind comparative studies. The study drug must prove its superiority over an active established treatment (comparator) and/or a placebo. This traditional study design assumes an additive relationship between pharmacological and placebo effects. Recent research demonstrates that this model is probably not apt to establish the therapeutic efficiency in clinical practice. The real therapeutic benefit of a new drug becomes visible only through modifications and adaptations of the study design that simulate the clinical treatment context. Such a modification is seen in the introduction of an open verum treatment arm, in which participants are certain to receive the study drug. This treatment realizes expectancy effects that are inherent to clinical practice. The expectancy effects are unspecified treatment effects and represent the placebo effect. The aim of the open verum condition and the inherent optimized placebo effect is to maximize the participants¡¯ therapeutic benefit through the improved drug effect. Additionally, this procedure mimics clinical practice more realistically and enables a more effective estimate of the therapeutic efficiency of the drug under investigation. The drug's therapeutic efficiency in clinical practice, including the expectancy effects, represents a new but relevant criterion and objective for pharmacological research.