如何在正在进行的篮子试验中添加篮子,并借用信息借款
Libby Daniells1, Pavel Mozgunov2, Helen Barnett3
1STOR-i Centre for Doctoral Training, Department of Mathematics and Statistics, Lancaster University, Lancaster, UK.
Statistical methods in medical research
|March 20, 2025
概括
在适应性临床试验中添加新组患者可以提高功率,但可能会增加错误率的风险. 一种新的校准方法提高了这些复杂的篮子试验设计中的决策稳定性.
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:
- 篮子试验在一个协议下,在多个患者组中评估单一的治疗方法.
- 允许新篮子结合的适应性设计是可取的,但面临的挑战是小样本大小和功率.
- 对于小样本的现有贝叶斯方法没有解决添加新篮子的影响.
研究的目的:
- 通过信息借贷,研究将新篮子纳入正在进行的适应性篮子试验的方法.
- 开发和评估一种新的校准方法,用于有效性标准,以确保可靠的错误控制.
- 评估这些方法在功率和I型错误率方面的性能.
主要方法:
- 探索信息借用技术,为正在进行的试验添加篮子.
- 为有效性标准制定一种新的校准策略,考虑除了零之外的替代场景.
- 模拟研究以评估I型错误控制和新篮子合并功率.
主要成果:
- 添加新的篮子可以显著提高检测治疗效应的统计能力.
- 使用标准方法观察到错误率的潜在通货膨胀,当将新篮子纳入时.
- 拟议的校准程序证明了错误率通货膨胀的减少.
结论:
- 开发的方法在适应性试验中添加篮子时提供了更好的功率.
- 一种新的校准方法提高了疗效评估的可靠性,并控制了决策错误.
- 这项研究为适应性篮子试验设计和分析提供了更强大的框架.
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