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

2 citations · 3 across the 5 of their papers we have counts for

collaborators

5 papers

stat.ME2025

Collaborative Indirect Treatment Comparisons with Multiple Distributed Single-arm Trials

Yuru Zhu, Huiyuan Wang, Haitao Chu +2

When randomized controlled trials are impractical or unethical to simultaneously compare multiple treatments, indirect treatment comparisons using single-arm trials offer valuable…

cs.AI2025

Empowering Clinical Trial Design through AI: A Randomized Evaluation of PowerGPT

Yiwen Lu, Lu Li, Dazheng Zhang +20

Sample size calculations for power analysis are critical for clinical research and trial design, yet their complexity and reliance on statistical expertise create barriers for many…

cs.HC2024

OrigamiPlot: An R Package and Shiny Web App Enhanced Visualizations for Multivariate Data

Yiwen Lu, Jiayi Tong, Yuqing Lei +8

We introduce OrigamiPlot, an open-source R package and Shiny web application designed to enhance the visualization of multivariate data. This package implements the origami plot, a…

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…