Cited by 33Statistical Methods and Bayesian InferenceAdvanced Causal Inference TechniquesStatistical Methods in Clinical Trials

Missing Data Matter: An Empirical Evaluation of the Impacts of Missing EHR Data in Comparative Effectiveness Research

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Zhou, Yizhao, Shi, Jiasheng, Stein, Ronen, Liu, Xiaokang, Baldassano, Robert N, Forrest, Christopher B, Chen, Yong, Huang, Jing·2023·Journal of the American Medical Informatics Association

Abstract

Objectives: The impacts of missing data in comparative effectiveness research (CER) using electronic health records (EHRs) may vary depending on the type and pattern of missing data. In this study, we aimed to quantify these impacts and compare the performance of different imputation methods. Materials and Methods: We conducted an empirical (simulation) study to quantify the bias and power loss in estimating treatment effects in CER using EHR data. We considered various missing scenarios and used the propensity scores to control for confounding. We compared the performance of the multiple imputation and spline smoothing methods to handle missing data. Results: When missing data depended on the stochastic progression of disease and medical practice patterns, the spline smoothing method produced results that were close to those obtained when there were no missing data. Compared to multiple imputation, the spline smoothing generally performed similarly or better, with smaller estimation bias and less power loss. The multiple imputation can still reduce study bias and power loss in some restrictive scenarios, eg, when missing data did not depend on the stochastic process of disease progression. Discussion and Conclusion: Missing data in EHRs could lead to biased estimates of treatment effects and false negative findings in CER even after missing data were imputed. It is important to leverage the temporal information of disease trajectory to impute missing values when using EHRs as a data resource for CER and to consider the missing rate and the effect size when choosing an imputation method.

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  • Automated tools can track sources and issue real-time alerts for deviations from quality standards, supporting compliance with regulatory requirements for accurate and secure management.

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Authors
Zhou, Y., Shi, J., Stein, R., Liu, X., Baldassano, R. N., Forrest, C. B., Chen, Y., & Huang, J.
Affiliations
  • Children's Hospital of Philadelphia (US)
  • University of Pennsylvania (US)
Corresponding author
Zhou, Yizhao
Venue
Journal of the American Medical Informatics Association
Published
2023-04-17
Volume
30
Issue
7
Pages
1246-1256
ISSN
  • 1067-5027 (ISSN-L)
  • 1527-974X
OpenAlex
W4366085127
Cited by
33
References
70
Citations by year
2026: 11 · 2025: 14 · 2024: 6 · 2023: 2
FWCI
6.71
Type
journal article
Language
en
Open access
Locations
  • Journal of the American Medical Informatics Association (publishedVersion)
  • PubMed Central (publishedVersion)
  • PubMed (publishedVersion)
Funders
  • Patient-Centered Outcomes Research Institute (PCORI) Project Program Awards (ME-2018C3-14899)
  • Eunice Kennedy Shriver National Institute of Child Health (R01HD099348)
Topics
  • Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods and Bayesian Inference
  • Physical Sciences › Mathematics › Statistics and Probability › Advanced Causal Inference Techniques
  • Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
Keywords
missing data, electronic health records, comparative effectiveness research, multiple imputation, smoothing splines, imputation method comparison, propensity score, disease progression, temporal data
MeSH terms
Computer Simulation, Data Interpretation, Statistical, Research Design, Bias, Comparative Effectiveness Research, Propensity Score

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