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.