2023/02/13 by Masahiro Kojima, Kojima, Masahiro
Decision Sciences · Mathematics · #FOS: Computer and information sciences #Methodology (stat.ME) #Optimal Experimental Design Methods #Statistical Methods and Inference #Statistical Methods in Clinical Trials
paper · pdf · doi:10.48550/arxiv.2302.06088
openalex publication_date 2023/02/13 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/28
Recently, the strategy for dose optimization in oncology has shifted to conduct Phase 2 randomized controlled trials with multiple doses. Optimal biologic dose selection from Phase 1 trial data to determine candidate doses for Phase 2 trials has been gaining attention. This study proposes a novel adaptive cohort size determination method for optimal biologic dose-finding to accelerate trials. The cohort size expansion is determined adaptively depending on the toxicity and efficacy data of the ongoing trial. In a simulation, the proposed method shortened the trial duration and maintained accuracy. The trial duration was reduced by an average of approximately 20% compared with the non-adaptive cohort size determination design. The cohort size expansion is demonstrated using a simple example.