Case studies from industry

Two complex innovative trial designs using a hierarchy of endpoints will be presented. The first is an adaptive platform trial for the treatment of ALS. The second is a confirmatory study in heart failure that borrows information from a previous trial. In both ALS and heart failure the standard primary endpoint is an integration of survival and/or hospitalization events with function. Traditional analyses of these endpoints are non-parametric joint rank analyses. However, in innovative trial designs such as platform trials or trials with historical borrowing, a parametric analysis is often needed to accommodate complexities within the setting. In both examples, sharing of information from multiple data sources is utilized and analysis models must account for potential differences across these sources. The Bayesian paradigm lends a natural framework for synthesis of these data through hierarchical modeling.

Hierarchical composite endpoints

Complex and innovation trial design

Case studies

Bayesian analysis