Abstract
Personalized medicine has reached the mainstream, accounting for more new drug approvals and a promising pipeline of candidate therapeutics. Recent advances in genomics, computational biology, medical imaging, diagnostic technologies, and translational medicine are creating the possibility for scientists to develop diagnostic tools and new treatments for cancer, genetic disorders, and infectious diseases that may be particularly effective in biomarker-defined subpopulations. Drug development under this model creates new challenges that will require the need for increased regulatory flexibility, novel clinical trial designs, and translational science development. In this review, the authors highlight key developmental and regulatory challenges in the advancement of personalized medicines and their associated companion diagnostics with the need for innovative clinical trial designs to support drug/diagnostic development and registration. Further, the clinical complexities of implementing new technologies are considered, such as high-throughput next-generation sequencing in personalized medicine, and offer a glimpse of the regulatory and policy considerations shaping this methodology in multimarker diagnostic development.