Series: Pragmatic trials and real world evidence: Paper 1. Introduction

• Published in: Journal of clinical epidemiology Vol. 88; pp. 7 - 13
• Main Authors: Zuidgeest, Mira G.P., Goetz, Iris, Groenwold, Rolf H.H., Irving, Elaine, van Thiel, Ghislaine J.M.W., Grobbee, Diederick E.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.08.2017; Elsevier Limited
• Subjects: Blood pressure; Clinical medicine; Clinical trials; Data collection; Data Collection - methods; Design; Epidemiologic Research Design; Epidemiology; Feasibility studies; Generalizability; Humans; Observational Studies as Topic - methods; Patient safety; Patient Selection; Physicians; Population studies; Pragmatic trial; Randomized Controlled Trials as Topic - methods; Real-world evidence; Routine clinical practice; Trial conduct; Trial design; Pragmatic trial; Routine clinical practice; Generalizability; Trial conduct; Trial design; Real-world evidence
• ISSN: 0895-4356; 1878-5921; 1878-5921
• DOI: 10.1016/j.jclinepi.2016.12.023

This is the introductory paper in a series of eight papers. In this series, we integrate the theoretical design options with the practice of conducting pragmatic trials. For most new market-approved treatments, the clinical evidence is insufficient to fully guide physicians and policy makers in choosing the optimal treatment for their patients. Pragmatic trials can fill this gap, by providing evidence on the relative effectiveness of a treatment strategy in routine clinical practice, already in an early phase of development, while maintaining the strength of randomized controlled trials. Selecting the setting, study population, mode of intervention, comparator, and outcome are crucial in designing pragmatic trials. In combination with monitoring and data collection that does not change routine care, this will enable appropriate generalization to the target patient group in clinical practice. To benefit from the full potential of pragmatic trials, there is a need for guidance and tools in designing these studies while ensuring operational feasibility. This paper introduces the concept of pragmatic trial design. The complex interplay between pragmatic design options, feasibility, stakeholder acceptability, validity, precision, and generalizability will be clarified. In this way, balanced design choices can be made in pragmatic trials with an optimal chance of success in practice.