Pragmatic clinical trials (PCTs) are randomized controlled trials designed to inform clinical and health policy decisions by evaluating the risks and benefits of health interventions in real world, community settings. They are often contrasted with explanatory trials, which evaluate how well medical products work under optimal circumstances in a carefully chosen, treatment-adherent patient population to maximize the chance of observing a biological effect of a new treatment. In contrast to explanatory trials, PCTs generally include a broader range of patients by reducing the number of inclusion /exclusion criteria, generally include a broader range of study sites (versus only including academic medical centers) and generally include outcomes that are aligned with the evidence needs of post-regulatory decision makers. PCTs address practical questions about the risks, benefits, and costs of an intervention as they would occur in routine clinical practice.
The Center for Medical Technology Policy (CMTP) is a recognized thought leader in helping to define1,2, standardize3, and improve PCTs.4,5 We have a broad range of experience with ensuring PCTs are designed and implemented to be efficient, timely, credible and relevant to patients, clinicians and policymakers.
Selected CMTP publications on pragmatic clinical trials:
- Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA 2003;290:1624-32.
- Thorpe KE, Zwarenstein M, Oxman AD, Treweek S, Furberg CD, Altman DG, Tunis S, Bergel E, Harvey I, Magid DJ, Chalkidou K. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. CMAJ 2009;180:E47-57.
- Zwarenstein M, Treweek S, Gagnier JJ, Altman DG, Tunis S, Haynes B, Oxman AD, Moher D; CONSORT group; Pragmatic Trials in Healthcare (Practihc) group. Improving the reporting of pragmatic trials: an extension of the CONSORT statement. BMJ 2008;337:a2390.
- Helfand M, Tunis S., Whitlock EP, Pauker SG, Basu A, Chilengerian J, Harrell FE, Meltzer DO, Montori VM, Shepard DS, Kent DM and the Methods Work Group of the National CTSA Strategic Goal Committee on Comparative Effectiveness Research. A CTSA agenda to advance methods for comparative effectiveness research. Clinical and Translational Science Journal 2011;4:10.
- Luce BR, Kramer JM, Goodman SN, Connor JT, Tunis S, Whicher D, Schwartz JS. Rethinking randomized clinical trials for comparative effectiveness research: the need for transformational change. Ann Intern Med 2009;151:206-9.