Benchmarking progression-free survival ratio as primary endpoint in precision oncology clinical trials.

ABSTRACT: Progression Free Survival Ratio (PFSratio), as defined as the ratio between PFS on investigational treatment (PFS2) and PFS on the last prior therapy (PFS1), is a popular endpoint in precision oncology (PO) studies. In this work, five methodologies for PFSratio-based trial analysis (count-based, Kaplan Meier, Kernel-based Kaplan Meier, parametric and midrank) and two for trial design (GBVE and Weibull) are benchmarked. The Kernel-based Kaplan Meier analysis is most recommended, as it handles informative censoring and does not require PFS1/PFS2 distribution assumptions. Sample size and power calculation methods perform best when applied to settings with expected high PFS1/PFS2 correlation and median ratio. Analysis of five clinical trials (MOSCATO 01, WINTHER, MASTER, SHIVA and POG570) from

SUBMITTER: Nichetti F 

PROVIDER: S-EPMC12820104 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature