"6 choose 4": A framework to understand and facilitate discussion of strategies for overall survival safety monitoring
Godwin Yung, Kaspar Rufibach, Marcel Wolbers, Mark Yan, Jue Wang
公開日: 2024/10/5
Abstract
Advances in anticancer therapies have significantly contributed to declining death rates in certain disease and clinical settings. However, they have also made it difficult to power a clinical trial in these settings with overall survival (OS) as the primary efficacy endpoint. Therefore, two approaches have been recently proposed for the pre-specified analysis of OS as a safety endpoint (Fleming et al., 2024; Rodriguez et al., 2024). In this paper, we provide a simple, unifying framework that includes the aforementioned approaches (and a couple others) as special cases. By highlighting each approach's focus, priority, tolerance for risk, and strengths or challenges for practical implementation, this framework can help to facilitate discussions between stakeholders on "fit-for-purpose OS data collection and assessment of harm" (American Association for Cancer Research, 2024). We apply this framework to a real clinical trial in large B-cell lymphoma to illustrate its application and value. Several recommendations and open questions are also raised.