对I/II期临床试验的新型模型辅助设计进行比较审查
Haolun Shi1, Ruitao Lin2, Xiaolei Lin3
1Department of Statistics and Actuarial Science, Simon Fraser University, Burnaby, British Columbia, Canada.
Biometrical journal. Biometrische Zeitschrift
|May 13, 2024
概括
在I/II期试验中,模型辅助设计比基于模型的设计更简单,更实用. 这项研究比较了八个模型辅助设计,为临床试验中最佳剂量选择提供了建议.
科学领域:
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
- 药理学 药理学是指药理学的学科.
背景情况:
- 免疫疗法和向药物的I/II期试验需要有效的剂量确定方法.
- 基于模型的设计是计算密集的,限制了实时应用.
- 模型辅助设计通过预先计算的决策表提供了简单性和透明度.
研究的目的:
- 系统地评估和比较最近的模型辅助I/II阶段设计的操作特性.
- 将模型辅助设计与已知的基于模型的设计 (EffTox) 进行比较.
- 为各种试验场景提供基于性能指标的设计建议.
主要方法:
- 对八个模型辅助设计 (TEPI,PRINTE,Joint i3+3,BOIN-ET,STEIN,uTPI,BOIN12) 和EffTox进行了全面的数值模拟.
- 为每个预先确定的最佳生物剂量 (OBD) 位置生成了10,000个剂量场景.
- 评估了性能指标,包括选择百分比,患者分配到OBD,以及过量百分比.
主要成果:
- 该研究系统地比较了多个模型辅助和基于模型的设计的操作特性.
- 性能指标在各种模拟场景中被彻底评估.
- 确定了设计效率和安全概况的关键差异.
结论:
- 在I/II阶段试验中,模型辅助设计通常是首选的,因为它们具有实用性和透明度.
- 该研究提供了基于证据的建议,根据具体的试验目标,样本大小和初始剂量位置选择最合适的设计.
- 在临床试验的早期阶段,知情的设计选择可以优化剂量选择和患者安全.
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