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适应性试验设计的信心区间II:案例研究和实际指导
David S Robertson1, Thomas Burnett2, Babak Choodari-Oskooei3
1MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Statistics in medicine
|August 9, 2025
概括
适应性临床试验中的置信区间 (CI) 可能不可靠. 本研究评估了二阶段组序列设计的CI,提供了选择和报告试验后的指导方针.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 制药统计 制药统计
背景情况:
- 传统的置信区间 (CI) 在适应性临床试验中表现出覆盖不足和不一致.
- 监管指南强调在解释自适应性试验的CI时要谨慎.
- 在现有的CI方法中,对于适应性设计缺乏明确的偏好.
研究的目的:
- 探索和评估适应性临床试验的置信区间 (CI).
- 介绍CIs的扩展案例研究,用于两个阶段的组序列设计.
- 为在适应性试验环境中选择和报告CI提供准则.
主要方法:
- 对适应性设计的CI施工方法的方法审查 (第一部分).
- 一个双阶段组序列试验的扩展案例研究.
- 针对案例研究设置的拟议CI的全面模拟研究.
主要成果:
- 置信区间 (CI) 在适应性试验中表现出明显不同的特性.
- 该研究促进了对有效的CI程序在适应性试验后的扩大理解.
- 模拟结果突出了不同CI方法之间的性能差异.
结论:
- 研究人员需要明确的指导来选择和报告适应性试验的CI.
- 选择CI显著影响治疗效果的解释.
- 拟议的指导方针旨在提高CI在自适应设计中的可靠性和一致性.
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