Pragmatic Clinical Trials
Background
Systematic reviews of clinical literature often find that meaningful clinical evidence on given technologies is lacking or uninformative. Pragmatic clinical trials (PCTs) are designed to provide meaningful evidence on medical technologies to a range of health care decision makers, including patients, providers, payers, and policymakers. 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.
Scope and Methods
In May 2009, CMTP convened a "think tank" of approximately fifty experts to discuss PCT design and develop recommendations for a guidance document (see Meeting Summary below). This meeting was complemented by a series of semi-structured interviews with meeting attendees and other expert stakeholders. The guidance document and meeting summary will undergo several rounds of review by meeting participants and the general public before being finalized.
In addition, CMTP has continued to work with an international consortium to refine tools to help trial designers develop more pragmatic clinical trials. Our work in this area has resulted in several major publications (Zwarenstein et al. 2008, Thorpe et al., 2009, Luce et al. 2009), including the development of the Pragmatic-Explanatory Continuum Indicator (PRECIS), which provides a practical visual tool to assess the extent to which trials are more or less pragmatic.
Documents
- Effectiveness Guidance Document -- Released September 14, 2010
- Meeting Summary -- Summary of May 2009 Think Tank, including list of participants.
- The Prospect of Early Pharmaceutical Pragmatic Clinical Trials - Background document authored by Jodi Segal, MD, MPH, Johns Hopkins University School of Medicine
