量身定制的指南,用于将Estimand框架应用于队列内试验 (TwiCs) 的研究
R Gal1, R Kessels2, K Luijken3
1Division of Imaging and Oncology, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.
Global epidemiology
|October 14, 2024
概括
本研究为在群组内试验 (TwiCs) 研究中定义估值提供了指导. 它解决了TwiCs中独特的间流事件,以确保精确估计治疗效果.
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
- 流行病学 流行病学
背景情况:
- 估计框架标准化了临床研究中的治疗效果定义.
- 队列内试验 (TwiCs) 设计提出了独特的方法挑战.
- 清晰的估计和定义对于研究设计和分析至关重要.
研究的目的:
- 描述专门为TwiCs研究制定估计值的考虑因素.
- 为TwiCs应用估计和框架提供实际指导.
- 用现实世界TwiCs的例子来说明估计和定义.
主要方法:
- 对两个TwiCs研究 (SPONGE和UMBRELLA Fit) 进行估计和框架的追溯应用.
- 确定和分析关键的估计和属性:目标人群,治疗,终点,间流事件和总结量.
- 专注于在TwiCs背景下定义估值的独特考虑因素.
主要成果:
- 鉴定了TwiCs独特的间流事件:干预拒绝,时间错位和对照臂治疗启动.
- 探索处理这些事件的策略,例如定义目标人群或考虑治疗政策.
- 展示了如何定义TwiCs研究的具体估计值.
结论:
- 在制定估算时,TwiCs的设计需要特别考虑.
- 提供的例子为未来的TwiCs研究提供了有价值的指导.
- 一致的估计和定义提高了TwiCs研究的有效性和可解释性.
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