Evidence-Based Medicine, Heterogeneity of Treatment Effects, and the Trouble with Averages

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...

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Bibliographic Details
Published in:The Milbank quarterly 2004-01, Vol.82 (4), p.661-687
Main Authors: KRAVITZ, RICHARD L., DUAN, NAIHUA, BRASLOW, JOEL
Format: Article
Language:English
Subjects:
Adverse effects
Clinical trials
Drug Approval - legislation & jurisprudence
Evidence based medicine
Experimentation
genomics
Health care
Health outcomes
Heterogeneity
Humans
Medical practice
Medical research
Original
Policy Making
Population characteristics
Practice Patterns, Physicians
Randomized controlled trials
Randomized Controlled Trials as Topic
Research design
Research methodology
Sample size
Side effects
Sociology
Sociology of health and medicine
Statistics
Strokes
Treatment Outcome
United States
United States Food and Drug Administration
USA
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Summary: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.
ISSN:0887-378X
1468-0009
DOI:10.1111/j.0887-378X.2004.00327.x