Pragmatic Clinical Trials

In general, several major characteristics distinguish pragmatic trials and traditional clinical trials. First, PCTs involve the deliberate comparison of clinically relevant alternative treatments, chosen based on the most common decision-making scenarios. Traditional RCTs do not include highly relevant comparisons, leaving decision makers to make comparisons across trials, where assumptions, research methods, patient populations, and other crucial characteristics may be different. Second, PCTs are typically designed to be generalizable to real patient populations by including patients with common co-morbidities and from a variety of demographic backgrounds. Third, PCTs select clinically-relevant outcomes that are intended to address the primary issues and concerns of patients, clinicians and payers. Many RCTs include outcomes that are of primary interest to regulators, and pay less attention to the post-regulatory decision makers that will also use those studies to guide their choices. These clinically-relevant outcomes may include more quality-of-life information and may involve longer follow-up periods than are typical for traditional clinical trials. Importantly, the selection of the most useful and relevant outcomes requires direct consultation with decision makers during study protocol development. In fact, one of the keys to the successful design of clinical trials that are more useful for decision making is the greater engagement of decision makers in trial design to ensure that it provides answers to the critical questions affecting policy and practice.

Relevant Documents

• Background document, authored by Jodi Segal, MD, MPH, Johns Hopkins University School of Medicine
• Summary of May 2009 meeting