Functional additive models for optimizing individualized treatment rules

Biometrics. 2023 Mar;79(1):113-126. doi: 10.1111/biom.13586. Epub 2021 Nov 22.

Abstract

A novel functional additive model is proposed, which is uniquely modified and constrained to model nonlinear interactions between a treatment indicator and a potentially large number of functional and/or scalar pretreatment covariates. The primary motivation for this approach is to optimize individualized treatment rules based on data from a randomized clinical trial. We generalize functional additive regression models by incorporating treatment-specific components into additive effect components. A structural constraint is imposed on the treatment-specific components in order to provide a class of additive models with main effects and interaction effects that are orthogonal to each other. If primary interest is in the interaction between treatment and the covariates, as is generally the case when optimizing individualized treatment rules, we can thereby circumvent the need to estimate the main effects of the covariates, obviating the need to specify their form and thus avoiding the issue of model misspecification. The methods are illustrated with data from a depression clinical trial with electroencephalogram functional data as patients' pretreatment covariates.

Keywords: functional additive regression; individualized treatment rules; sparse additive models; treatment effect-modifiers.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Humans
  • Models, Statistical*
  • Precision Medicine*
  • Randomized Controlled Trials as Topic*