"6 choose 4": A framework to understand and facilitate discussion of strategies for overall survival safety monitoring

• DOI: 10.48550/arxiv.2410.04020
• Publication Date: 2024-10-04
• Authors: Godwin Yung; Kaspar Rufibach; Marcel Wolbers; Mark Yan; Jue Wang
• Keywords: Methodology (stat.ME); FOS: Computer and information sciences; Applications (stat.AP); Statistics - Applications; Statistics - Methodology

Advances in anticancer therapies have significantly contributed to declining death rates certain disease and clinical settings. However, they also made it difficult power a trial these settings with overall survival (OS) as the primary efficacy endpoint. A number of statistical approaches therefore been recently proposed for pre-specified analysis OS safety endpoint (Shan, 2023; Fleming et al., 2024; Rodriguez 2024). In this paper, we provide simple, unifying framework that includes aforementioned (and couple others) special cases. By highlighting each approach's focus, priority, tolerance risk, strengths or challenges practical implementation, can help facilitate discussions between stakeholders on "fit-for-purpose data collection assessment harm" (American Association Cancer Research, We apply real large B-cell lymphoma illustrate its application value. Several recommendations open questions are raised.