All Times EDT
Keywords: multi-component endpoints, rare disease
Heterogeneous rare diseases which affect multiple organ systems may be better accommodated by endpoints that assess treatment effect across multiple endpoints within the same patient. Appropriate utilization of multi-component endpoints as primary endpoints in rare disease study design can be more clinically meaningful and can also increase the effect size.
In this discussion, we will summarize and discuss the existing approaches for derivation of a clinically meaningful composite endpoints, as well as the existing statistical approach for simultaneously testing for multi-component endpoints. Specifically, the varieties of parametric and non-parametric global testing approaches such as O'Brien Rank Sum, O'Brien OLS and GLS test, etc. will be presented and discussed. And combination of global testing approach and multiplicity adjustment approach for efficient testing of global hypothesis and individual hypothesis for each individual component will also be summarized and discussed.