ISSUE: The use of surrogate endpoints for overall survival has been advocated as a way to accelerate drug development, approval, and reimbursement decisions, particularly for treatments for smaller populations or diseases where death rates are lower and take longer to occur. Thus, their use could facilitate addressing unmet medical needs in a timely manner. Mitigating the impact of recent regulatory requirements to change Phase 3 trial designs in oncology, such as crossover, has also been given as a rationale for using surrogate endpoints. For NSCLC, hyper-progression on immune-oncology treatments and crossover have been identified as potential factors leading to different results in the surrogacy validation process. Yet, a comprehensive framework and methods to understanding and validating surrogacy in oncology are currently lacking. Recent scientific and public debates show that surrogate endpoints are under attack, and a more rigorous and consistent evaluation is desired to provide greater confidence in their use and pave the way for more effective clinical development and care decisions.

OVERVIEW: Silvia Paddock moderates the session and sets the scene by presenting the challenges of surrogacy validation in oncology and summarizing its particularities in NSCLC. Dalia Dawoud provides an update on recent requirements for surrogacy validation at NICE and the implications for future HTAs. Advanced statistical methods can reduce the uncertainty around predictions from the surrogate to the final outcome. Billy Amzal illustrates this for NSCLC and shows that progression-free survival can serve as potential surrogate for overall survival. He also discusses options to transfer the findings into a real-world setting. Jeff Allen provides an overview of the potential benefits of using surrogate endpoints for people living with cancer and recent initiatives on surrogacy validation in NSCLC at Friends of Cancer Research. The audience and panelists reflect on the presentations in 15 minutes.