TY - JOUR
T1 - Utility of routinely collected electronic health records data to support effectiveness evaluations in inflammatory bowel disease
T2 - A pilot study of tofacitinib
AU - Rudrapatna, Vivek Ashok
AU - Glicksberg, Benjamin Scott
AU - Butte, Atul Janardhan
N1 - Publisher Copyright:
© 2021 BMJ Publishing Group Limited. Published by BMJ.
PY - 2021/5/19
Y1 - 2021/5/19
N2 - 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 to 4/2019) were queried to identify tofacitinib-treated IBD patients. Disease activity variables at baseline and follow-up were manually abstracted according to a preregistered protocol. The proportion of patients meeting the endpoints of recent randomised 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 tumour necrosis factor 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/Conclusion 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.
AB - 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 to 4/2019) were queried to identify tofacitinib-treated IBD patients. Disease activity variables at baseline and follow-up were manually abstracted according to a preregistered protocol. The proportion of patients meeting the endpoints of recent randomised 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 tumour necrosis factor 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/Conclusion 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.
KW - computing methodologies
KW - medical informatics
UR - http://www.scopus.com/inward/record.url?scp=85106565240&partnerID=8YFLogxK
U2 - 10.1136/bmjhci-2021-100337
DO - 10.1136/bmjhci-2021-100337
M3 - Article
C2 - 34011632
AN - SCOPUS:85106565240
SN - 2058-4555
VL - 28
JO - BMJ Health and Care Informatics
JF - BMJ Health and Care Informatics
IS - 1
M1 - e100337
ER -