Prediction of treatment outcome in clinical trials under a personalized medicine perspective

Abstract

A central problem in most data-driven personalized medicine scenarios is the estimation of heterogeneous treatment effects to stratify individuals into subpopulations that differ in their susceptibility to a particular disease or response to a specific treatment. In this work, with an illustrative example on type 2 diabetes we showed how the increasing ability to access and analyzed open data from randomized clinical trials (RCTs) allows to build Machine Learning applications in a framework of personalized medicine. An ensemble machine learning predictive model is first developed and then applied to estimate the expected treatment response according to the medication that would be prescribed. Machine learning is quickly becoming indispensable to bridge science and clinical practice, but it is not sufficient on its own. A collaborative effort is requested to clinicians, statisticians, and computer scientists to strengthen tools built on machine learning to take advantage of this evidence flow.