181 citations · 310 across the 17 of their papers we have counts for
20 papers
Machine learning to optimize precision in the analysis of randomized trials: A journey in pre-specified, yet data-adaptive learning
Laura B. Balzer, Mark J. van der Laan, Maya L. Petersen
Covariate adjustment is an approach to improve the precision of trial analyses by adjusting for baseline variables that are prognostic of the primary endpoint. Motivated by the SEA…
A cautionary note for plasmode simulation studies in the setting of causal inference
Pamela A Shaw, Susan Gruber, Brian D. Williamson +5
Plasmode simulation has become an important tool for evaluating the operating characteristics of different statistical methods in complex settings, such as pharmacoepidemiological…
The impact of job stability on monetary poverty in Italy: causal small area estimation
Katarzyna Reluga, Dehan Kong, Setareh Ranjbar +2
Job stability - encompassing secure contracts, adequate wages, social benefits, and career opportunities - is a critical determinant in reducing monetary poverty, as it provides ho…
Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective
Jie Chen, Susan Gruber, Hana Lee +13
Real-world data (RWD) and real-world evidence (RWE) have been increasingly used in medical product development and regulatory decision-making, especially for rare diseases. After o…
Challenges and Possible Strategies to Address Them in Rare Disease Drug Development: A Statistical Perspective
Jie Chen, Lei Nie, Shiowjen Lee +13
Developing drugs for rare diseases presents unique challenges from a statistical perspective. These challenges may include slowly progressive diseases with unmet medical needs, poo…
Evaluating the Robustness of Targeted Maximum Likelihood Estimators via Realistic Simulations in Nutrition Intervention Trials
Haodong Li, Sonali Rosete, Jeremy Coyle +9
Several recently developed methods have the potential to harness machine learning in the pursuit of target quantities inspired by causal inference, including inverse weighting, dou…