Abstract
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.
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Data sharing is not applicable to this article as no datasets were generated or analyzed for the described work.
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Funding
This work was conducted and funded by the New Drug Development Paradigms (NEWDIGS) initiative under Massachusetts Institute of Technology’s (MIT’s) Center for Biomedical Innovation (CBI). Since completion of the work, NEWDIGS has moved to Tufts Medical Center’s Center for Biomedical System Design. NEWDIGS members and collaborator partners are identified on the website (https://newdigs.tuftsmedicalcenter.org/about/members-and-collaboration-partners/). The opinions expressed herein are those of the author(s), and not necessarily those of the institution with whom they are affiliated, or those of NEWDIGS members and collaborators.
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Schaumberg, D., Larholt, K., Apgar, E. et al. Examining Endpoint Concordance in Clinical Trials and Real-World Clinical Practice to Advance Real-World Evidence Utilization. Ther Innov Regul Sci 57, 472–475 (2023). https://doi.org/10.1007/s43441-022-00492-z
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DOI: https://doi.org/10.1007/s43441-022-00492-z