All Times EDT
Keywords: enrichment designs
For rare diseases, patients are often heterogeneous. It may be beneficial for clinical trials to use composite endpoints by incorporating multiple components (either all or some components) or winning on multiple endpoints (e.g., 3 out of 5) (Mielke et al., 2020). Efficient and effective statistical methods are needed to handle these types of endpoints to increase the chance of detecting the treatment effect.
Statistical methods such as the win ratio approach (Redfors et al., 2020), the Finkelstein-Schoenfeld method (Finkelstein and Schoenfeld, 2019), and O’Brien rank sum test (O’Brien, 1984) have been used to increase the chance of detecting drug’s efficacy for important endpoints.
Aside from statistical methods for analyzing the efficacy endpoints, innovative designs including enrichment designs and multiple stage designs such as cross-over designs, sequential parallel comparison designs (Fava et al. 2003), and sequential enriched design (Chen et al. 2011) are instrumental in increasing trial efficiency.
In this presentation, I will discuss issues and challenges in utilizing two-stage enrichment designs and offer potential solutions for small-sized clinical trials, including pediatric trials.