Causal machine learning for predicting treatment outcomes

Causal machine learning (ML) offers flexible, data-driven methods for predicting treatment outcomes. Here, we present how methods from causal ML can be used to understand the effectiveness of treatments, thereby supporting the assessment and safety of drugs. A key benefit of causal ML is that allows for estimating individualized treatment effects, as well as personalized predictions of potential patient outcomes under different treatments. This offers granular insights into when treatments are effective, so that decision-making in patient care can be personalized to individual patient profiles. We further discuss how causal ML can be used in combination with both clinical trial data as well as real-world data such as clinical registries and electronic health records. We finally provide recommendations for the reliable use of causal ML in medicine. First published in Nature Medicine, 30, 958-968 (2024) by Springer Nature. Assessing the effectiveness of treatments is crucial to ensure patient safety and personalize patient care. Recent innovations in machine learning (ML) offer new, data-driven methods to estimate treatment effects from data. This branch in ML is commonly referred to as causal ML as it aims to predict a causal quantity, namely, the patient outcomes due to treatment [1]. Causal ML can be used in order to estimate treatment effects from both experimental data obtained through randomized controlled trials (RCTs) and observational data obtained from clinical registries, electronic health records, and other real-world data (RWD) sources to generate clinical evidence. A key strength of causal ML is that it allows to estimate individualized treatment effects, as well as to make personalized predictions of potential patient outcomes under different treatments.