Biomarkers are becoming an indispensable addition to clinical development. Modern biotechnologies such as omics have brought unprecedented opportunities for biomarker discovery and applying the knowledge of biomarkers in clinical trials. However, significant challenges remain in many areas including statistical modeling.
In this presentation, the concept of biomarkers and their roles in drug discovery will be briefly reviewed. The presentation will focus on statistical issues and advancement on the utility of the biomarkers and their impact on early clinical trials such as proof of mechanism (POM). Specifically, biomarker strategies in early clinical trial decision making using omics data, e.g. quantification of pharmacological effect, correlative analysis between biomarker and clinical endpoints and relevant statistical challenges, will be discussed. Case studies will be presented.