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Subgroup analysis based on structured mixed-effects models for longitudinal data.

Juan ShenAnnie Qu
Published in: Journal of biopharmaceutical statistics (2020)
In recent years, subgroup analysis has emerged as an important tool to identify unknown subgroup memberships. However, subgroup analysis is still under-studied for longitudinal data. In this paper, we propose a structured mixed-effects approach for longitudinal data to model subgroup distribution and identify subgroup membership simultaneously. In the proposed structured mixed-effects model, the heterogeneous treatment effect is modeled as a random effect from a two-component mixture model, while the membership of the mixture model is incorporated using a logistic model with respect to some covariates. One advantage of our approach is that we are able to derive the estimation of the treatment effects through an EM-type algorithm which keeps the subgroup membership unchanged over time. Our numerical studies and real data example demonstrate that the proposed model outperforms other competing methods.
Keyphrases
  • electronic health record
  • big data
  • phase iii
  • machine learning
  • cross sectional
  • data analysis
  • combination therapy
  • replacement therapy