Statistica Sinica 24 (2014), 1461-1485
Abstract: Biosignatures such as brain scans, mass spectrometry, or gene expression profiles might one day be used to guide treatment selection and improve outcomes. This article develops a way of estimating optimal treatment policies based on data from randomized clinical trials by interpreting patient biosignatures as functional predictors. A flexible functional regression model is used to represent the treatment effect and construct the estimated policy. The effectiveness of the estimated policy is assessed by furnishing prediction intervals for the mean outcome when all patients follow the policy. The validity of these prediction intervals is established under mild regularity conditions on the functional regression model. The performance of the proposed approach is evaluated in numerical studies.
Key words and phrases: Empirical processes, functional data analysis, inverse treatment probability weighting, locally efficient estimation.