Identifying treatment responders using counterfactual modeling and potential outcomes

International audienceIndividualizing treatment according to patients' characteristics is central for personalized or precision medicine. There has been considerable recent research in developing statistical methods to determine optimal personalized treatment strategies by modeling the outcome of patients according to relevant covariates under each of the alternative treatments, and then relying on so-called predicted individual treatment effects. In this paper, we use potential outcomes and principal stratification frameworks and develop a multinomial model for left and right-censored data to estimate the probability that a patient is a responder given a set of baseline covariates. The model can apply to RCT or observational study data. This method is based on the monotonicity assumption, which implies that no patients would respond to the control treatment but not to the experimental one. We conduct a simulation study to evaluate the properties of the proposed estimation method. Results showed that the predictions of the probability of being a responder were well calibrated even if we observed variability and a small bias when many parameters were estimated. We finally applied the method to a cohort study on the selection of patients for additional radiotherapy after resection of a soft-tissue sarcoma