PS17:Navigating ICH E20: Opportunities and Challenges in Future Oncology Trials with Adaptive Designs

In September 2025, the FDA announced the availability of a draft guidance for industry prepared under ICH E20, Adaptive Design for Clinical Trials. This draft guidance provides comprehensive recommendations for confirmatory clinical trials that employ adaptive designs to evaluate treatment effects of investigational drugs within the context of their overall development programs. It offers a structured framework for adaptive designs, emphasizing their potential to enhance trial efficiency while addressing the risks associated with inadequate planning, analysis, or governance. The introduction of ICH E20 has a significant impact on oncology clinical trials. Under the FDA's Project Optimus, oncology trials are required to make critical decisions on dose and target populations early in development, often relying on short-term endpoints such as tumor response. To improve efficiency, sponsors are increasingly adopting adaptive designs, such as seamless phase 2/3 trials, in oncology. However, it is unclear if there are gaps between current oncology adaptive design practices and the principles outlined in ICH E20. Key questions include the justification of adaptation rules, the definition of estimands, and the development of simulation plans in the context of regulatory decision-making. This transdisciplinary session will connect statistical methods with cross-functional drug development efforts and provide an in-depth exploration of the challenges and opportunities involved in aligning oncology trial practices with the principles outlined in ICH E20. Speakers from regulatory affairs and biostatistics in industry, the FDA, and academia will discuss recent advancements in oncology-focused adaptive designs and present case studies featuring adaptations for dose optimization, population enrichment, and more. Key design elements will be analyzed, including adaptation rules informed by short-term endpoints, methods for integrating data across trial phases, and the timing of interim and final analyses in event-driven settings. This session will also provide a comparative assessment of current practices versus the recommendations in ICH E20.