Oncology Clinical Trial Design Planning Based on a Multistate Model That Jointly Models Progression-Free and Overall Survival Endpoints.

ABSTRACT: When planning an oncology clinical trial, the usual approach is to assume proportional hazards and even an exponential distribution for time-to-event endpoints. Often, besides the gold-standard endpoint overall survival (OS), progression-free survival (PFS) is considered as a second confirmatory endpoint. We use a survival multistate model to jointly model these two endpoints and find that neither exponential distribution nor proportional hazards will typically hold for both endpoints simultaneously. The multistate model provides a stochastic process approach to model the dependency of such endpoints neither requiring latent failure times nor explicit dependency modeling such as copulae. We use the multistate model framework to simulate clinical trials with endpoints OS and PFS and show ho

SUBMITTER: Erdmann A 

PROVIDER: S-EPMC11650109 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature