4 papers
Arbitrated Indirect Treatment Comparisons
Yixin Fang, Weili He
Matching-adjusted indirect comparison (MAIC) has been increasingly employed in health technology assessments (HTA). By reweighting subjects from a trial with individual participant…
Decentralized Clinical Trials in the Era of Real-World Evidence: A Statistical Perspective
Jie Chen, Junrui Di, Nadia Daizadeh +10
There has been a growing trend that activities relating to clinical trials take place at locations other than traditional trial sites (hence decentralized clinical trials or DCTs),…
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…