ABSTRACT
References (for Appendix). . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67 3.6 Some Further Statistical Considerations . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68 3.7 Model Selection and Validation. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69 3.8 Tests for Translational Process. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69
3.9 Animal Model versus Human Model . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 71 3.10 Translation among Study Endpoints . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 72 3.11 Trial Simulation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 References . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74
3.1 Introduction A general understanding of the molecular principles by which diverse types of
biomarker assays function can be an important facilitator for optimizing the statistical analyses of biomarker data, generated in translational medicine (TM) studies. The purpose of this chapter is to provide a general overview of various types of biomarker assays, including those analyzing a spectrum of macromolecules ranging from nucleic acids (DNA and RNA) and small-molecule metabolites to larger molecular-weight proteins as analytes.
