Examining Endpoint Concordance in Clinical Trials and Real-World Clinical Practice to Advance Real-World Evidence...

Real-world evidence (RWE) is increasingly contributing to more informed decisions regarding the optimal access to and use of therapeutics to improve patient outcomes. However, in many cases, a disconnect between evidence derived from clinical trials and the RWE that follows market approval impedes the potential value and widespread adoption of RWE to optimize patient care. Collaborators with the Learning Ecosystems Accelerator for Patient-centered, Sustainable innovation (LEAPS), a major project of the Tufts Medical Center [formally Massachusetts Institute of Technology (MIT)] NEW Drug Development ParadIGmS (NEWDIGS) initiative, propose assessing the relationship between efficacy endpoints used in randomized controlled trials (RCTs) and effectiveness measures that inform treatment decisions within real-world clinical settings as one way to bridge this divide and further leverage RWE to improve care and patient outcomes. This commentary outlines elements of an endpoint concordance study using Rheumatoid Arthritis as a case study. The authors describe the ways in which better understanding of the relationship between effectiveness and RCT endpoints could improve the confidence in and adoption of RWE by both contextualizing existing RWE as well as identifying ways in which to improve the value of RWE in improving care and outcomes.