Keywords: clinical trials; composite endpoints; primary endpoints
One key component of a successful clinical trial is a correct selection of the primary endpoint. However, in some diseases, especially in rare diseases, patients are heterogeneous and may have different symptoms. Therefore, it is challenging to identify one single primary endpoint which can efficiently measure all patients' disease manifestation. Instead, a group of multiple endpoints need to be considered in order to assess the totality of the evidence. In these scenarios, non-standard multiple testing adjustment approaches should be implemented to control the overall study type I error rate. In this presentation, we will discuss issues related to the use of multiple endpoints for making regulatory decisions. We will also discuss the good use of the composite endpoints in clinical trials when the evaluation of drug's efficacy needs to rely on assessing totality of the evidence. Real case examples will be shared for illustrating issues and solutions.