Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages

Richard L Kravitz, Naihua Duan, Joel Braslow

Research output: Contribution to journalArticle

313 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)661-687
Number of pages27
JournalMilbank Quarterly
Volume82
Issue number4
DOIs
StatePublished - 2004

Keywords

  • Evidence-based medicine
  • Genomics
  • Population characteristics
  • Research design
  • Statistics
  • United States Food And Drug Administration

ASJC Scopus subject areas

  • Nursing(all)
  • Health(social science)
  • Health Professions(all)
  • Health Policy

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