Generation of Individualized Treatment Decision Tree Algorithm with Application to Randomized Control Trials and Electronic Medical Record Data

摘要

With new treatments and novel technology available, personalized medicine has become a key topic in the new era of healthcare. Traditional statistical methods for personalized medicine and subgroup identification primarily focus on single treatment or two arm randomized control trials (RCTs). With restricted inclusion and exclusion criteria, data from RCTs may not reflect real world treatment effectiveness. However, electronic medical records (EMR) offers an alternative venue. In this paper, we propose a general framework to identify individualized treatment rule (ITR), which connects the subgroup identification methods and ITR. It is applicable to both RCT and EMR data. Given the large scale of EMR datasets, we develop a recursive partitioning algorithm to solve the problem (ITR-Tree). A variable importance measure is also developed for personalized medicine using random forest. We demonstrate our method through simulations, and apply ITR-Tree to datasets from diabetes studies using both RCT and EMR data. Software package is available at https://github.com/jinjinzhou/ITR.Tree.