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