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“Pragmatic” clinical trials: from whose perspective?
  1. Paul J Karanicolas1,
  2. Victor M Montori2,
  3. Holger Schünemann3,
  4. Gordon H Guyatt3
  1. 1
    University of Western Ontario; London, Ontario, Canada
  2. 2
    Mayo Clinic; Rochester, Minnesota, USA
  3. 3
    McMaster University, Hamilton, Ontario, Canada

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    Over the past 40 years, methodologists have become increasingly enthusiastic about conducting “pragmatic” clinical trials, which aim to simulate real-world settings as much as possible.1 In contrast to explanatory trials that are conducted under idealised circumstances, successful pragmatic trials will have the intended benefit of directly informing healthcare decision making. We wholeheartedly support the notion that, to the extent possible, trials should inform real-world decisions. In this editorial, we will, however, argue that the current conceptualisation of pragmatic trials sometimes serves the needs of only a small proportion of healthcare decision makers. Furthermore, a truly pragmatic or practical trial requires that clinical trialists carefully define the real-world context to which they hope their results apply, and design their trials accordingly.2

    Authors’ descriptions of pragmatic trials have varied slightly, but most agree that such trials should enrol all patients to whom healthcare providers might offer the intervention, allow clinicians to administer the intervention and co-interventions without restrictions, and measure patient-important outcomes. In this discussion, we will focus on one tenet of pragmatic trials: that these trials should include patients who do not take the intervention as prescribed, presumably in the same proportion as they are likely to be seen in the community. We will interpret the results of a recent self-described pragmatic trial of nortriptyline as an adjunct to nicotine replacement for smoking cessation3 from 3 perspectives. We show that although it may be pragmatic for some, it certainly isn’t for all healthcare decision makers.

    Perspective 1: the policy-maker

    The policy-maker is an employee of a third-party payer, who must decide which pharmaceutical products his organisation will fund for its members. His organisation is currently deciding whether to approve nortriptyline as an adjunct to nicotine …

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