20 September 2016 - This JAMA Guide to Statistics article compares pragmatic randomized controlled trials, which focus on important challenges that patients, physicians, and policy makers face in day-to-day life with explanatory trials that seek to test a hypothesis.

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. 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.

Read JAMA Guide to Statistics article