4 papers · 1 filter
Externally Controlled Trials: A Review of Design and Borrowing Through a Causal Lens
Ke Zhu, Rima Izem, Peng Yang +8
Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pe…
Adaptive Targeted Maximum Likelihood Estimation of the Mean Potential Outcome under a Treatment Rule
Yichen Xu, Mark J. van der Laan
Estimating the mean counterfactual outcome under a treatment rule is a central problem in causal inference and policy evaluation. Standard estimators, including inverse probability…
A CV-TMLE global test approach to improve power in rare disease clinical studies with multiple-component endpoints
Tianyue Zhou, Susan Gruber, Hana Lee +3
Rare disease trials face unique statistical challenges due to limited patient populations and heterogeneous clinical manifestations among patients. Multiple endpoints are often nec…
Investigating Targeting Strategies and Truncation in TMLE for the Average Treatment Effect under Practical Positivity Violations
Yichen Xu, Susan Gruber, Mark J. van der Laan
Estimating average treatment effects from observational data is challenging under practical violations of the positivity assumption. Targeted Maximum Likelihood Estimators (TMLEs)…