PS16: Optimizing Dose Selection Across Clinical, Operational, and Regulatory Dimensions

Standard statistical methods for dose finding trials seek to learn a dose response profile to determine the minimum dose required to attain near maximal efficacy. However, efficacy-centric dose selection is frequently insufficient to support decisions that optimize downstream clinical development. Robust dose selection must balance multiple competing objectives, including safety and tolerability, pharmaceutical feasibility, operational complexity, and long-term financial viability. High-performing development programs increasingly integrate these cross-functional considerations into a unified decision-making framework, in which modern statistical methods play a central enabling role. Substantial gains in development efficiency and decision quality can be achieved through judicious application of common statistical tools, including tailored utility functions, adaptive trial design, and probabilistic projections of program value (e.g., net present value). Although incorporating these approaches increases operational complexity, the incremental costs are small relative to potential benefits: more informed dose selection, reduced downstream risk, and improved likelihood of success in confirmatory development. This session brings together perspectives from industry statisticians, clinical program leadership, and regulatory science to explore how broader decision-analytic objectives can be rigorously and transparently incorporated into dose-finding studies within current regulatory expectations. This session will comprise three 18-20 minute presentations, each followed by 5 minutes of audience Q&A.