most citedUse of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective

2 citations · 3 across the 4 of their papers we have counts for

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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…