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 former.

54% (UC) and 62% (CD) of patients had complete capture of disease activity at baseline (month -6 to 0), while only 32% (UC) and 69% (CD) of patients had complete follow-up data (month 2 to 8). Using data imputation, we estimated the proportion achieving the trial primary endpoints as being similar to the published estimates for both UC (16%, p-value=0.5) and CD (38%, p-value=0.8).

Discussion/Conclusions: This pilot study reproduced trial-based estimates of tofacitinib efficacy despite its use in a different cohort but revealed substantial missingness in routinely collected data. Future work is needed to strengthen EHR data and enable real-world evidence in complex diseases like IBD.

Methods
This data was primarily collected by manual review of the electronic health records according to the following protocol: dx.doi.org/10.17504/protocols.io.2bqgamw

It was subsequently de-identified and certified as such by the UCSF CTSI prior to broad release.