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

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

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stat.AP2024★ 2 cited

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★ 1 cited

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…