gms | German Medical Science

Background/research question: Pragmatic trials provide decision-oriented, real-world evidence that is highly applicable and generalizable. Conversely, explanatory trials conducted in highly controlled settings are often considered less applicable and generalizable to routine care. It is widely assumed that effects in the “real-world” (and thus pragmatic trials) are different to effects obtained under artificial, controlled, research conditions (and thus explanatory trials). However, it is unknown which features of pragmatism, generalizability, and applicability are responsible. The PragMeta project aims at determining the impact of pragmatism on treatment effect estimates in a wide and collaborative meta-epidemiological project characterizing hundreds of randomized trials.

Methods: We built a public database (www.PragMeta.org) to collect relevant data from trials with identical clinical questions (i.e., on population, intervention, comparator, and outcome) but having different aspects of generalizability, applicability, and pragmatism. We identify trials that are self-labelled as or have prominent features of being pragmatic (e.g., use of routinely collected data) and then used forward citation to identify citing systematic reviews including corresponding trials on the same research question. By using the PRagmatic Explanatory Continuum Indicator Summary tool (PRECIS-2), we assess the degree of pragmatism and then its impact on treatment estimates.

The PragMeta database covers published trials of various topics that focus on specific therapeutic areas (e.g., multiple sclerosis), outcomes (e.g., patient-reported outcomes), or specific trial features (e.g., use of routinely collected data); among others. We invite other groups to collaborate, contribute, and/or use the PragMeta database.

Preliminary/expected results, outlook: At the conference, the status of PragMeta will be summarized and use cases of the database presented to illustrate its potential for meta-research on pragmatic evidence.

Our results will inform a better understanding of the pragmatic-explanatory continuum and the generation and interpretation of real-world evidence. It will give practical guidance for all stakeholders developing, funding, conducting, assessing, or otherwise using clinical trials. Ultimately it may help to generate, report, and use evidence that is more relevant for patients, clinicians, and other key health care decision-makers.

Competing interests: RC2NB (Research Center for Clinical Neuroimmunology and Neuroscience Basel) is supported by Foundation Clinical Neuroimmunology and Neuroscience Basel. All authors declare no competing interests.