拡大コホートを伴う抗がん剤用量探索試験の計画、実装、意思決定の実例

抄録

For phase 1 dose-finding studies, a variety of model-based approaches and study designs with expansion cohorts have been proposed in place of the conventional 3＋3 design, and the benefits of these designs have been reported. However, there are limited reports on the practical details of such statistical work using actual clinical trial data. We present practical procedures for planning, conducting, and decision making in a model-based approach with both dose-escalation and expansion parts using actual clinical trial data, focusing on statistical considerations.