Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages
Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages
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DOI：
10.1111/j.0887-378x.2004.00327.x
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发表时间：
2004-01-01
影响因子：
6.6
通讯作者：
Braslow, J
中科院分区：
文献类型：
--
作者：
Kravitz, RL;Duan, NH;Braslow, J
循证医学是科学证据在临床实践中的应用。本文讨论了将全球证据（以人口平均值衡量的“平均效应”）应用于局部问题（可能偏离人口平均值的个体患者或群体）的困难。它认为，临床试验中大多数治疗方法的益处或危害可能具有误导性，无法揭示某些治疗方法的实质性益处、许多治疗方法的微小益处和少数治疗方法的危害的潜在复杂混合物。治疗效果的异质性反映了患者在疾病风险、对治疗的反应性、对不良反应的脆弱性和对不同结局的效用方面的多样性。认识到这些因素，研究人员可以设计研究，更好地描述谁将从医学治疗中受益，临床医生和政策制定者可以更好地利用结果。
Evidence-based medicine is the application of scientific evidence to clinical practice. This article discusses the difficulties of applying global evidence ("average effects" measured as population means) to local problems (individual patients or groups who might depart from the population average). It argues that the benefit or harm of most treatments in clinical trials can be misleading and fail to reveal the potentially complex mixture of substantial benefits for some, little benefit for many, and harm for a few. Heterogeneity of treatment effects reflects patient diversity in risk of disease, responsiveness to treatment, vulnerability to adverse effects, and utility for different outcomes. Recognizing these factors, researchers can design studies that better characterize who will benefit from medical treatments, and clinicians and policymakers can make better use of the results.