临床试验中估计值的灵敏度,不完全符合标准
Heng Chen1, Daniel F Heitjan2,3
1Biostatistics, Gilead Sciences Inc., Foster City, CA 94404, USA.
The international journal of biostatistics
|June 26, 2023
概括
临床试验中的治疗意图 (ITT) 估计对合规性敏感. 我们提出了一个模型,其中合规者平均因果效应 (CACE) 是强大的,并且在某些条件下等于人口平均因果效应.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 流行病学 流行病学
背景情况:
- 临床试验中的不合规性使因果效应估计变得复杂.
- 治疗意图 (ITT) 估计对治疗分配是有效的,但对合规水平敏感.
- 合规者平均因果效应 (CACE) 估计了合规子组中的影响,但可以根据试验情况而有所不同.
研究的目的:
- 提出一种新的模型来理解临床试验中的合规行为.
- 调查CACE强大且与人口平均因果效应 (POCE) 一致的条件.
- 在各种试验环境中评估CACE对合规率的敏感性.
主要方法:
- 开发一种与试验特征相互作用的潜在原始合规模型.
- 模拟研究来评估CACE灵敏度.
- 该模型应用于维生素A补充试验和外围止痛元分析中的真实世界数据.
主要成果:
- 拟议的模型阐明了潜在合规性和试验因素之间的相互作用.
- 当潜在的合规性独立于个体治疗效果时,CACE在试验中是强大的,并且等于POCE.
- 在模拟和经验分析中证明了CACE的潜在敏感性.
结论:
- 拟议的潜在变量模型为理解合规性及其对因果效应估计的影响提供了一个框架.
- 在特定假设下,CACE可以是一个强大而可靠的估计,相当于POCE.
- 这些发现强调了在解释临床试验结果时考虑合规机制的重要性.
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