Caring for the Individual Patient: Understanding Heterogeneous Treatment Effects

A Special Publication from the National Academy of Medicine


Evidence-based medicine arose from a clear need and represents a major advance in the science of clinical decision-making. Despite broad acceptance of evidence-based medicine, however, a fundamental issue remains unresolved: evidence is derived from groups of people, yet medical decisions are made by and for individuals. Despite persistent assertions from clinicians that determining the best therapy for each patient is a more complicated endeavor than just picking the best treatment on average, traditional approaches have been overly reliant on the average effects estimated from the outcomes of clinical trials.

This Special Publication is based on a workshop, held by the NAM, that considered patient and stakeholder perspectives on the importance of understanding heterogeneous treatment effects (HTE) and best practices for implementing clinical programs that take HTE into account. For evidence to be more applicable to individual patients, we need to combine methods for strong causal inference (first and foremost, randomization) with methods for prediction that permit inferences about which particular patients are likely to benefit and which are not. Better population-based outcomes will only be realized when we understand more completely how to treat patients as the unique individuals they are.

Workshop Planning Committee

  • David M. Kent (Chair), Tufts Medical Center
  • Thomas Concannon, RAND Corporation
  • Robert Golub, Journal of the American Medical Association
  • Sheldon Greenfield, University of California Irvine School of Medicine
  • Rodney Hayward, University of Michigan
  • A. Cecile Janssens, Emory University Rollins School of Public Health
  • Muin J. Khoury, Centers for Disease Control and Prevention
  • Peter Rothwell, University of Oxford
  • Ewout Steyerberg, Leiden University Medical Center
  • Andrew J. Vickers, Memorial Sloan Kettering Cancer Center

Related Meetings

Evidence and the Individual Patient: Understanding Heterogeneous Treatment Effects for Patient-Centered Care  |  May 31, 2018


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