Utility of routinely collected electronic health records data to support effectiveness evaluations in Inflammatory Bowel Disease: A pilot study of Tofacitinib

Objectives: Electronic health records (EHR) are receiving growing attention from regulators, biopharmaceuticals, and payors as a potential source of real-world evidence. However, their suitability for the study of diseases with complex activity measures is unclear. We sought to evaluate the use of EHR data for estimating treatment effectiveness in inflammatory bowel disease (IBD), using tofacitinib as a use case.

Methods: Records from the University of California, San Francisco (6/2012-4/2019) were queried to identify tofacitinib-treated IBD patients. Disease activity variables at baseline and follow-up were manually abstracted according to a pre-registered protocol. The proportion of patients meeting the endpoints of recent randomized trials in ulcerative colitis (UC) and Crohn’s disease (CD) was assessed.

Results: 86 patients initiated tofacitinib. Baseline characteristics of the real-world and trial cohorts were similar, except for universal failure of TNF-inhibitors in the for...