2025/06/09 by Saurabh Bhandari, Michael J. Daniels, Bhandari, Saurabh +3
Mathematics · #Advanced Causal Inference Techniques #FOS: Computer and information sciences #Mathematical Biology Tumor Growth #Methodology (stat.ME) #Statistical Methods in Clinical Trials
paper · pdf · doi:10.48550/arxiv.2506.07387
openalex publication_date 2025/06/09 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/28
In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian approach for assessing treatment effects in these trials, where data scarcity is a major concern. Our approach synthesizes tumor burden, a key biomarker for evaluating patient response to oncology treatments, and survival outcome, a widely used endpoint for assessing clinical benefits, by jointly modeling longitudinal and survival data. The proposed method, along with its novel estimand, aims to efficiently capture signals of treatment efficacy in early-phase studies and holds potential for development as an endpoint in Phase 3 confirmatory studies. We conduct simulations to investigate the frequentist characteristics of the proposed estimand in a simple setting, which demonstrate relatively controlled Type I error rates when testing the treatment effect on outcomes.