Statistical Test for Composite Hypothesis in Clinical Research |Biomedgrid

In clinical evaluation of the safety and efficacy of a test treatment under investigation, a typical approach is to test for the null hypothesis of no treatment difference in efficacy in randomized clinical trials (RCT). The investigator would reject the null hypothesis of no treatment difference and then conclude the alternative hypothesis that the treatment is efficacious. In practice, however, this typical approach based on test for efficacy alone may not be appropriate for a full assessment of both efficacy and safety of the test treatment under study. Alternatively, [1] suggested testing composite hypothesis by taking both safety and efficacy into consideration. In this article, appropriate statistical test for a composite hypothesis of non-inferiority in efficacy and superiority in safety is derived. The impact on power calculation for sample size requirement when switching from a single hypothesis (e.g., for efficacy) to a composite hypothesis (e.g., for both safety and efficacy) is also examined.