Seminar: Early Phase Design Considerations for Oncology Drug Development in the Era of Immunotherapy and Targeted Agents

Uncertainty about optimality of doses of anti-cancer agents approved in recent years, including PD-L1 blockade agents and targeted agents, has led to a movement in the oncology drug development field to reconsider the traditional approach for drug development. Following in the path of approvals for nivolumab and pembrolizumab, trial designs for dose finding trials have ballooned from sample sizes in the range of 12-50 to the hundreds or more.  In addition, post-marketing trials have demonstrated that lower doses of certain drugs may be as efficacious as approved doses.  The has contributed to a renewed focus on ‘dose optimization,’ causing statisticians, researchers, patient advocates and regulators to realize the profound inadequacies of traditional phase I dose finding designs. Trial approaches need to incorporate the adaptive nature of a seamless toxicity-efficacy paradigm, while maintaining practical aspects of trial implementation, statistical properties, endpoint measurement and safety and well-being of patients. This talk will discuss these aspects and provide recent context of drug approvals in oncology. This seminar is part of the Frontiers in Biostatistics seminar series.

Elizabeth Garrett-Mayer, PhD, FSCT,  joined ASCO in 2017 as CENTRA’s division director for biostatistics and research data governance and became CENTRA’s first vice president in 2022. CENTRA leads ASCO’s research efforts, including the TAPUR Study, ASCO’s COVID-19 Registry, and research projects aimed at increasing minority enrollment and expanding eligibility criteria in clinical trials.