2 citations · 4 across the 4 of their papers we have counts for
4 papers · 1 filter
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…
Sensitivity analysis for causality in observational studies for regulatory science
Iván Díaz, Hana Lee, Emre Kıcıman +3
Recognizing the importance of real-world data (RWD) for regulatory purposes, the United States (US) Congress passed the 21st Century Cures Act1 mandating the development of Food an…