在适应性试验中调查估计和考虑:系统性审查
Fran Piazza1, Hannah Wallace1, Rachel Phillips2
1Imperial Clinical Trials Unit, School of Public Health, Imperial College London, London, UK.
Trials
|February 6, 2026
概括
该估计和框架旨在澄清临床试验研究问题,在适应性设计试验中采用率最低. 需要更明确的指导,以便在这些复杂的研究设计中有效实施.
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
- 监管科学 监管科学
背景情况:
- 随机对照试验 (RCT) 对于评估治疗至关重要,但模两可的研究问题阻碍了解释.
- 估计和框架 (ICH E9 (R1)) 是为了标准化治疗效应的定义而引入的.
- 早期的审查表明,采用率很低,并缺乏关于间接事件的清晰度.
研究的目的:
- 评估当前在适应性设计临床试验中的估计和框架的应用.
- 确定适应性试验估计和框架的实施中的挑战和差距.
主要方法:
- 在2023年出版的第2至4阶段适应性设计RCT协议和统计分析计划的系统审查.
- 搜索了BMJ公开和试验期刊.
- 数据提取的重点是估计和规范,处理间流事件和中间分析.
主要成果:
- 在146个符合条件的项目中,只有3%的项目指定了初级估值;2%的项目详细说明了所有估值和属性.
- 64%的文章描述了间流动事件,但没有一个将它们与初级估计联系起来.
- 没有文章指定中期分析的估计或考虑的适应影响.
结论:
- 在适应性设计的RCT中,estimatand框架的使用不足.
- 广泛采用是必要的,以清楚报告和解释试验结果.
- 需要针对适应性设计应用估计和框架的具体指导.
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