Design and implementation of pragmatic clinical trials using the electronic medical record and an adaptive design. Issue 1 (11th June 2018)

• Record Type: Journal Article
• Authors: Simon, Kelly Claire; Tideman, Samuel; Hillman, Laura; Lai, Rebekah; Jathar, Raman; Ji, Yuan; Bergman-Bock, Stuart; Castle, James; Franada, Tiffani; Freedom, Thomas; Marcus, Revital; Mark, Angela; Meyers, Steven; Rubin, Susan; Semenov, Irene; Yucus, Chad; Pham, Anna; Garduno, Lisette; Szela, Monika; Frigerio, Roberta; Maraganore, Demetrius M
• Is Part Of: JAMIA open. Volume 1:Issue 1(2018)
• Page Start: 99
• Page End: 106
• Publication Date: 2018-06-11
• Subjects: pragmatic clinical trials -- precision medicine -- clinical decision support -- electronic medical records -- sub-group based adaptive designs; Medical informatics -- Periodicals; 610.285
• DOI: 10.1093/jamiaopen/ooy017
• Languages: English
• ISSNs: 2574-2531

Abstract

Abstract: Objectives: To demonstrate the feasibility of pragmatic clinical trials comparing the effectiveness of treatments using the electronic medical record (EMR) and an adaptive assignment design. Methods: We have designed and are implementing pragmatic trials at the point-of-care using custom-designed structured clinical documentation support and clinical decision support tools within our physician's typical EMR workflow. We are applying a subgroup based adaptive design (SUBA) that enriches treatment assignments based on baseline characteristics and prior outcomes. SUBA uses information from a randomization phase (phase 1, equal randomization, 120 patients), to adaptively assign treatments to the remaining participants (at least 300 additional patients total) based on a Bayesian hierarchical model. Enrollment in phase 1 is underway in our neurology clinical practices for 2 separate trials using this method, for migraine and mild cognitive impairment (MCI). Results: We are successfully collecting structured data, in the context of the providers' clinical workflow, necessary to conduct our trials. We are currently enrolling patients in 2 point-of-care trials of non-inferior treatments. As of March 1, 2018, we have enrolled 36% of eligible patients into our migraine study and 63% of eligible patients into our MCI study. Enrollment is ongoing and validation of outcomes has begun. Discussion: This proof of concept article demonstrates the feasibility of conducting pragmatic trials using the EMR and an adaptive design. Conclusion: The demonstration of successful pragmatic clinical trials based on a customized EMR and adaptive design is an important next step in achieving personalized medicine and provides a framework for future studies of comparative effectiveness.