2 papers
stat.ME2026
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…
stat.ME2026
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…