目前对共变量调整和分层分析的实践 - 基于ASA瘤估计和工作组条件和边际影响工作组的调查结果
Jiawei Wei1, Sarwar I Mozumder2, Liming Li3
1Novartis Institutes for Biomedical Research Co., Shanghai, China. jiawei.wei@novartis.com.
BMC medical research methodology
|November 4, 2025
概括
一项关于临床试验中共变量调整的调查显示,在理解统计模型如何针对不同的估计值方面存在差距,特别是对于不可合的措施. 提供了建议,以澄清实践,提高分析的一致性.
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
- 药学研究 药学研究
背景情况:
- 美国食品和药物管理局 (FDA) 指南 (2023) 提倡对临床试验效率进行共变量调整.
- 在非线性模型中保持目标估计和估计与共变量调整存在挑战,根据ICH E9 (R1) 附录.
- 美国癌症评估协会的瘤评估工作组调查了在评估框架内对共变量调整的当前做法和挑战.
研究的目的:
- 评估临床试验中共变量调整的当前做法和挑战.
- 了解估计框架在统计分析中的应用.
- 识别有关统计分析模型和估计的知识差距.
主要方法:
- 在2023年6月至7月期间,向临床试验环境 (工业和学术界) 的生物统计学家分发了一项在线调查.
- 该调查包括19个问题,并向50多个组织的联系人传播.
- 收集和分析了这些回复,以总结与共变量调整和分层分析相关的经验和挑战.
主要成果:
- 收到来自12个国家的122个回复,表明全球参与.
- 调查结果突出显示,对不同估计值的统计模型的理解存在持续的差距,特别是对于不可合的指标.
- 实践包括在分层因素之外添加共变量,并将小层聚合在一起,以应对估计挑战.
结论:
- 调查发现需要加强对统计分析模型和估计模型的澄清和培训.
- 调查结果强调了解决共变量调整和分层分析实践中的误解的重要性.
- 建议建议促进广泛应用的统计方法的一致性和清晰度.
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