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Estimation and visualization of heterogeneous treatment effects for multiple outcomes

Statistics in medicine(2023)

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Abstract
We consider two-arm comparison in clinical trials. The objective is to identify a population with characteristics that make the treatment effective. Such a population is called a subgroup. This identification can be made by estimating the treatment effect and identifying the interactions between treatments and covariates. For a single outcome, there are several ways available to identify the subgroups. There are also multiple outcomes, but they are difficult to interpret and cannot be applied to outcomes other than continuous values. In this paper, we thus propose a new method that allows for a straightforward interpretation of subgroups and deals with both continuous and binary outcomes. The proposed method introduces latent variables and adds Lasso sparsity constraints to the estimated loadings to facilitate the interpretation of the relationship between outcomes and covariates. The interpretation of the subgroups is made by visualizing treatment effects and latent variables. Since we are performing sparse estimation, we can interpret the covariates related to the treatment effects and subgroups. Finally, simulation and real data examples demonstrate the effectiveness of the proposed method.
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Key words
latent variables,multiple outcomes,multivariate regression,subgroup identification,treatment effects
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