外部比较者和估计者
1IQVIA, Biostatistics, Frankfurt, Germany.
Frontiers in drug safety and regulation
|September 22, 2025
概括
对于临床试验至关重要的估计和框架,需要适应混合研究中的外部比较器. 可能需要新的属性来解决观察数据中的挑战,并确保准确的治疗效果估计.
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
- 观测研究 观测研究
背景情况:
- ICH E9(R1) 附录提供了一个估计和框架,以清楚地定义治疗效应.
- 该框架规定了诸如治疗条件,人群,终点,间流事件的处理和人群层次总结等属性.
研究的目的:
- 探索混合临床/观察研究,特别是使用外部比较器 (ECs) 对现有的估计和框架的影响.
- 在将框架应用于EC时,识别潜在的挑战和对估计和属性的必要修改.
主要方法:
- 在外部比较器的背景下对ICH E9(R1) 估计和框架进行概念分析.
- 确定与治疗条件,人口定义,终点可比性,间流事件和EC研究中的人口层次摘要相关的特定挑战.
主要成果:
- 外部比较器在定义治疗条件,种群 (超越经典的治疗意图) 和确保终点可比性方面提出了独特的挑战.
- 处理间流事件和假设治疗策略的适用性变得更加关键,特别是对于长期终点.
- 在ECs中的数据异质性可能会挑战人口层次总结的假设,例如比例危险.
结论:
- 目前的估计和框架需要仔细考虑和潜在的扩展,以充分解决混合研究中的外部比较器的复杂性.
- 建议将基线定义和边际估计器作为未来修订的潜在额外估计和属性.
- 调整估计和框架对于在涉及外部数据源的研究中进行可靠和可靠的治疗效果估计至关重要.
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