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Activity Number: 100 - Pragmatic Randomized Clinical Trials: Challenges and Impact on Clinical Practice and Health Policies
Type: Invited
Date/Time: Monday, July 29, 2019 : 8:30 AM to 10:20 AM
Sponsor: Health Policy Statistics Section
Abstract #300306 Presentation
Title: Key Elements in the Design of Pragmatic Randomized Clinical Trials
Author(s): Valentina Bayer*
Companies: Boehringer Ingelheim
Keywords: pragmatic clinical trials; randomization; real-world evidence; large simple trials

Pragmatic randomized clinical trials (PrCTs) help patients, clinicians, payers and health policy decision makers to make decisions by providing real-world evidence about healthcare interventions. Randomization is a major component of PrCTs, which ensures a solid statistical foundation for comparing interventions. Several characteristics set PrCTs apart from traditional randomized clinical trials: minimal inclusion/exclusion criteria leading to a heterogeneous population, usual care setting with fewer scheduled visits, multiple data sources (e.g., clinician-collected, electronic health records, insurance claims, mobile devices), real-world interventions and real-world outcomes. The result is a simpler trial design with increased generalizability, but challenges arise, such as: impact on enrollment, larger sample size (e.g., cluster randomization), smaller treatment effect and higher variability in the heterogeneous population, fewer visits (which may render an interim analysis infeasible and reduce adherence) and bias from open label design. This talk will review key design elements of PrCTs, their challenges and impact on clinical practice.

Authors who are presenting talks have a * after their name.

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