从试验试验中估计的治疗效果是不可靠的
Jesse D Troy1, Megan L Neely1, Gina-Maria Pomann1
1Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina, USA.
Journal of pain and symptom management
|September 4, 2023
概括
试点试验对于评估治疗疗效是不可靠的,因为采样变化. 计算机模拟表明,这些小型研究往往产生误导性的结果,错误地表明危害或益处,不应该指导治疗开发决策.
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
- 翻译研究是翻译研究.
背景情况:
- 试点试验是小规模的研究,旨在为更大规模的疗效试验提供信息.
- 康索特指南定义了可行性试验,而不是疗效评估.
- 采样变异性对试验试验有效性估计的可靠性构成重大挑战.
研究的目的:
- 为了证明采样变异对试点试验的疗效估计的影响,使用计算机模拟.
- 为了说明为什么试点试验设计不适合评估治疗前景.
主要方法:
- 一个双臂并行组试验的计算机模拟与生存结果 (N=20每组).
- 在两个场景下进行的模拟:治疗有效 (危险比率[HR]=0.75) 和无效 (HR=1).
主要成果:
- 在治疗有效的模拟中 (HR=0.75),约20%的试验错误地暗示了危害 (HR>1).
- 在治疗无效的模拟中 (HR=1),50%的试验错误地表明受益.
- 在模拟中观察到的结果分布在真正的治疗效果周围.
结论:
- 从试点试验中估计的治疗效果非常容易导致误导性结论.
- 由于存在错误发现的高风险,试点试验不应用于对新疗法的未来开发决策.
- 采样变化的统计现象破坏了试验试验对疗效评估的可靠性.
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