使用现实世界的数据来预测健康结果-预测设计:应用和样本大小规划
Stella Erdmann1, Dominic Edelmann2, Meinhard Kieser1
1Institute of Medical Biometry, University of Heidelberg, Heidelberg, Germany.
Biometrical journal. Biometrische Zeitschrift
|July 26, 2023
概括
这项研究引入了一种用于临床试验的新型预测设计,利用现有的对照数据有效评估新疗法. 这种方法为传统的随机对照试验 (RCT) 提供了成本高效和更快的替代方案.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 卫生经济学 卫生经济学
背景情况:
- 随机对照试验 (RCT) 是治疗疗效的标准,但资源密集.
- 从RCT和真实世界数据 (RWD) 中获得的大量现有的患者控制数据未得到充分利用.
- 需要更高效,更实用的研究设计.
研究的目的:
- 调查用于评估新治疗效果的预测设计的可行性.
- 确定如何利用外部控制数据来告知治疗效果估计.
- 在这个新设计中开发偏差评估和样本大小计算的方法.
主要方法:
- 开发一个预测模型来估计未来患者在对照条件下的结果.
- 对新疗法进行单臂试验,并将结果与预测对照进行比较.
- 调查线性预测模型的假设,以准确预测反事实结果.
- 为预测设计开发统计测试和样本大小公式.
主要成果:
- 预测设计可以通过利用外部数据源提供有关治疗效果的有价值信息.
- 线性预测模型可以在特定假设下准确预测患者的反事实.
- 建立了一个评估人口中平均治疗效果的框架.
- 开发了一个R Shiny应用程序,以促进预测设计的实际应用.
结论:
- 预测设计为传统的RCT提供了一个更有效和更具成本效益的替代方案.
- 这种创新方法利用现有数据,减少试验持续时间和参与者的负担.
- 开发的方法和工具支持在临床研究中实施预测设计.
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