Dose Selection of Targeted Oncology Drugs in Early Development: Time for Change?

Abstract Adequate dose selection for targeted oncology drugs can result in better efficacy and minimize the risk for toxicities that lead to high rates of treatment discontinuation and dose reductions. Less than optimal dose selection, in many cases, is likely due to tolerability-driven dose selection that was developed for cytotoxic chemotherapy drugs as far back as the early 1940s. This chapter addresses the following topics: (1) discusses the importance of optimal dose selection from the perspectives of different stakeholders, (2) highlights current dose selection approaches for targeted oncology drugs, and (3) provides proposed approaches for optimal dose selection. A few case studies are provided at the end of the chapter to highlight the different issues discussed in the main text.