Academy of Mathematics and Systems Science, CAS Colloquia & Seminars | Speaker: | Prof. Zhiwei Zhang,National Cancer Institute, National Institutes of Health | Inviter: | | Title: | Data-adaptive efficient estimation strategies for biomarker studies embedded in randomized trials | Time & Venue: | 2021.11.18 09:00-10:00 腾讯会议 ID: 394872957 | Abstract: | Predictive and prognostic biomarkers are increasingly important in clinical research and practice. Biomarker studies are frequently embedded in randomized clinical trials with biospecimens collected at baseline and assayed for biomarkers, either in real time or retrospectively. In this work, we develop efficient estimation strategies for two study settings in terms of biomarker ascertainment: the complete-data setting in which the biomarker is measured for all subjects in the trial, and a two-phase sampling design in which the biomarker is measured retrospectively for a random subsample of subjects selected in an outcome-dependent fashion. In both settings, efficient estimating functions are characterized using semiparametric theory and approximated using data-adaptive machine learning methods, leading to estimators that are consistent, asymptotically normal, and (approximately) efficient under general conditions. The methods are evaluated in simulation studies and applied to from two biomarker studies, one in each setting. | | | |