Yuxin Wang

Yuxin Wang, Maresa Schröder, Dennis Frauen et al.

Constructing confidence intervals (CIs) for the average treatment effect (ATE) from patient records is crucial to assess the effectiveness and safety of drugs. However, patient records typically come from different hospitals, thus raising the question of how multiple observational datasets can be effectively combined for this purpose. In our paper, we propose a new method that estimates the ATE from multiple observational datasets and provides valid CIs. Our method makes little assumptions about the observational datasets and is thus widely applicable in medical practice. The key idea of our method is that we leverage prediction-powered inferences and thereby essentially `shrink' the CIs so that we offer more precise uncertainty quantification as compared to naïve approaches. We further prove the unbiasedness of our method and the validity of our CIs. We confirm our theoretical results through various numerical experiments. Finally, we provide an extension of our method for constructing CIs from combinations of experimental and observational datasets.

Yuxin Wang, Dennis Frauen, Jonas Schweisthal et al.

Dropout is common in clinical studies, with up to half of patients leaving early due to side effects or other reasons. When dropout is informative (i.e., dependent on survival time), it introduces censoring bias, because of which treatment effect estimates are also biased. In this paper, we propose an assumption-lean framework to assess the robustness of conditional average treatment effect (CATE) estimates in survival analysis when facing censoring bias. Unlike existing works that rely on strong assumptions, such as non-informative censoring, to obtain point estimation, we use partial identification to derive informative bounds on the CATE. Thereby, our framework helps to identify patient subgroups where treatment is effective despite informative censoring. We further develop a novel meta-learner that estimates the bounds using arbitrary machine learning models and with favorable theoretical properties, including double robustness and quasi-oracle efficiency. We demonstrate the practical value of our meta-learner through numerical experiments and in an application to a cancer drug trial. Together, our framework offers a practical tool for assessing the robustness of estimated treatment effects in the presence of censoring and thus promotes the reliable use of survival data for evidence generation in medicine and epidemiology.