Assessment of clinical relevance by considering point estimates and associated confidence intervals

For the proof of efficacy of a new drug in a placebo-controlled clinical trial it is not sufficient merely to demonstrate a statistically significant treatment difference. In recent years, regulatory authorities have strongly recommended assessing additionally whether the observed effect size is also of clinical relevance. This opinion is reflected in various guidelines which are of the utmost importance for the successful approval of a new drug. Clinical relevance can be investigated by responder analyses or by considering the point estimates on the original scale together with the associated confidence intervals. In this paper, we focus on the latter approach and discuss the suitability of different criteria which are commonly applied in medical research.