2 papers
stat.AP2024
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…
stat.AP2024
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…