Biomarker and translational research play a vital role in drug development to assess target engagement, understand mechanism of action, provide insight on drug safety and efficacy, and expedite the discovery and implementation of new diagnostic tools. Among various technologies used for translational research, ligand binding assays and mass spectrometry have been used widely to measure endogenous proteins and metabolites as biomarkers in circulation and tissues to provide meaningful information to guide drug development. Obtaining robust translational data in clinical development is very challenging due to the complexity of diseases and limitations of the current technologies. In addition, the evolving regulatory landscape related to the application of biomarkers in clinical trials poses significant challenges for anyone who works in the field. There is a gap in knowledge on biomarker specific issues and approaches. To fill the gap, this short course will step through the fundamentals of biomarkers assays and their applications in clinical trials. The short course will focus primarily on the discussion of technologies, key issues in assay development and some regulatory considerations to ensure continuity of experience and best practices, sharing institutional knowledge and making literature more accessible.

Learning Objectives:

• Gain a high-level overview for the role of Biomarkers in clinical development
• Apply fit for purpose/ Context of Use in their own assay development and validation
• Understand LBA and LC/MS unique aspects of biomarker assay validation/qualification
• Learn the unique aspects and considerations that apply to biomarker assays
• Broaden their knowledge beyond LBA and LCMS technology to help integrate with other technologies
• Identify regulatory framework for biomarkers in drug development