Journal of Data Science

One crucial aspect of precision medicine is to allow physicians to recommend the most suitable treatment for their patients. This requires understanding the treatment heterogeneity from a patient-centric view, quantified by estimating the individualized treatment effect (ITE). With a large amount of genetics data and medical factors being collected, a complete picture of individuals’ characteristics is forming, which provides more opportunities to accurately estimate ITE. Recent development using machine learning methods within the counterfactual outcome framework shows excellent potential in analyzing such data. In this research, we propose to extend meta-learning approaches to estimate individualized treatment effects with survival outcomes. Two meta-learning algorithms are considered, T-learner and X-learner, each combined with three types of machine learning methods: random survival forest, Bayesian accelerated failure time model and survival neural network. We examine the performance of the proposed methods and provide practical guidelines for their application in randomized clinical trials (RCTs). Moreover, we propose to use the Boruta algorithm to identify risk factors that contribute to treatment heterogeneity based on ITE estimates. The finite sample performances of these methods are compared through extensive simulations under different randomization designs. The proposed approach is applied to a large RCT of eye disease, namely, age-related macular degeneration (AMD), to estimate the ITE on delaying time-to-AMD progression and to make individualized treatment recommendations.

Traditional methods for evaluating a potential treatment have focused on the average treatment effect. However, there exist situations where individuals can experience significantly heterogeneous responses to a treatment. In these situations, one needs to account for the differences among individuals when estimating the treatment effect. Li et al. (2022) proposed a method based on random forest of interaction trees (RFIT) for a binary or categorical treatment variable, while incorporating the propensity score in the construction of random forest. Motivated by the need to evaluate the effect of tutoring sessions at a Math and Stat Learning Center (MSLC), we extend their approach to an ordinal treatment variable. Our approach improves upon RFIT for multiple treatments by incorporating the ordered structure of the treatment variable into the tree growing process. To illustrate the effectiveness of our proposed method, we conduct simulation studies where the results show that our proposed method has a lower mean squared error and higher optimal treatment classification, and is able to identify the most important variables that impact the treatment effect. We then apply the proposed method to estimate how the number of visits to the MSLC impacts an individual student’s probability of passing an introductory statistics course. Our results show that every student is recommended to go to the MSLC at least once and some can drastically improve their chance of passing the course by going the optimal number of times suggested by our analysis.