OBJECTIVES: Pragmatic clinical trials (PCTs) are gaining interest among decision-makers as transferability and generalisability of clinical trial results to real-world settings are increasingly scrutinised. The aim of this study was to identify how PCT design has evolved over the years.

METHODS: Data on PCTs were retrieved from clinicaltrials.gov. Trials were identified using the free search term “pragmatic” and study type “interventional studies”. PCT characteristics were assessed for all identified trials (1996-2017), and for three subgroups defined according to the year of launch (2003-2007, 2008-2012, and 2013-2017).

RESULTS: In total, 497 PCTs were identified. The oldest trial reported in the database was from 1996; however, the number of PCTs started to increase after 2000. The highest increase in the number of newly-launched PCTs was in 2015 (98%) and the number of newly-registered trials was highest in 2016 (108). Most PCTs were sponsored by universities (51.1%) and medical centres (21.1%). Industry sponsored only a minor proportion of the trials (3.6%). Cardiovascular, musculoskeletal and psychiatric areas were most commonly studied (~10% each). Trials were mainly conducted for interventions classified as “other” (38.0%), “behavioural” (32.4%) and “drugs” (22.1%); 93% of PCTs were reported as randomised clinical trials (RCTs). Parallel assignment dominated (85%) in PCTs design, but an increase in crossover assignment was observed over time. There was an increasing trend for open-label design, representing 54% of all trials. On average, PCTs enrolled 5,423 patients (range: 2-933,789). The average trial duration was 2.8 years (range: 0.2-14.8), albeit more recent trials tended to be shorter.

CONCLUSIONS: Unlike that of academics, manufacturer interest in PCTs has grown little, possibly reflecting the complexity of balancing internal and external validity in RCTs. Several initiatives are underway to assess, from a broad perspective, the challenges and potential solutions to introducing a greater degree of pragmatism in health technology development plans.