Patient and Stakeholder Engagement in Designing Pragmatic Clinical Trials

Abstract

The desire for patient-centeredness and more pragmatic clinical trials is increasing. Pragmatic clinical trials are conducted in normal practices to answer whether interventions work in real-world settings, whereas explanatory clinical trials are conducted under stricter settings with tighter control to answer whether an intervention can work. While both explanatory and pragmatic trials can have patient-centered elements, pragmatic trials more directly answer patient and policymaker questions.

To ensure trials are truly reflective of what is meaningful to patients, patients and stakeholders should be engaged during the entire trial process—from planning the trial through conducting the trial to disseminating the results. Identifying, recruiting, training, continually engaging, and compensating an advisory committee of patients and stakeholders to serve as a resource and guide through the trial process is one way to increase the patient-centeredness of a trial. Patients can help design recruitment and retention strategies, codevelop endpoints, review consent forms, and identify appropriate dissemination channels. Patient involvement throughout the trial benefits not only the research team but the patients themselves as well as future patients. This chapter explores how to increase patient-centeredness in clinical trials by engaging patients and stakeholders throughout the pragmatic clinical trial process.