Biomarker Discovery: Study Design and Execution

Abstract A disease causes specific changes in the profiles (chemical and biochemical) of biological fluids and tissues before the development of clinical symptoms. Proteomic and metabolomic analysis of biological samples can reveal changes in abundance levels of proteins and metabolites, with these changes potentially functioning as useful diagnostic and prognostic clinical tests. To become a clinically approved test, a potential biomarker needs to be confirmed and validated . Confirmation and validation apply to the analytical methodology as well as the candidate biomarker. A search of the scientific and medical literature indicates that many studies reported the discovery of potential biomarkers without proper validation. In this chapter, the discussion centers on biomarker study design and execution and details the steps needed for a successful biomarker discovery.