Biomarker-based designs of phase III clinical trials for personalized medicine

Abstract

Advances in biotechnology and genomics have accelerated development of molecularly targeted treatments and prognostic and predictive biomarkers, particularly, in oncology. This chapter provides an overview of various biomarker-based designs for phase III randomized clinical trials to evaluate clinical utility of a biomarker or biomarker-based treatment, including biomarker-strategy, enrichment, and randomize-all designs. We also provide a simulation comparison of the randomize-all designs in terms of their ability to assert treatment efficacy for the correct patient population. Complex adaptive designs with development and validation of predictive biomarkers are also discussed.