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JAMA Guide to Statistics and Methods
September 20, 2016

Pragmatic TrialsPractical Answers to “Real World” Questions

Author Affiliations
  • 1Patient Centered Outcomes Research Institute (PCORI), Washington, DC
  • 2Geisel School of Medicine at Dartmouth, Hanover, New Hampshire
  • 3Department of Emergency Medicine, Harbor-UCLA Medical Center, Torrance, California
  • 4Department of Emergency Medicine, David Geffen School of Medicine at the University of California-Los Angeles
  • 5Berry Consultants, LLC, Austin, Texas
JAMA. 2016;316(11):1205-1206. doi:10.1001/jama.2016.11409

The concept of a “pragmatic” clinical trial was first proposed nearly 50 years ago as a study design philosophy that emphasizes answering questions of most interest to decision makers.1 Decision makers, whether patients, physicians, or policy makers, need to know what they can expect from the available diagnostic or therapeutic options when applied in day-to-day clinical practice. This focus on addressing real-world effectiveness questions influences choices about trial design, patient population, interventions, outcomes, and analysis. In this issue of JAMA, Gottenberg et al2 report the results of a trial designed to answer the question “If a biologic agent for rheumatoid arthritis is no longer effective for an individual patient, should the clinician recommend another drug with the same mechanism of action or switch to a biologic with a different mechanism of action?” Because the authors included some pragmatic elements in the trial design, this study illustrates the issues that clinicians should consider in deciding whether a trial result is likely to apply to their patients.

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