Innovative Methods for Rare Disease Drug Development

The very limited sample size of patients with rare disease brings a lot of challenges in both design and analysis of clinical trials as compared to the other common disease. To promote rare diseases drug development, innovative thinking is not only encouraged by FDA but also needed for the pharmaceutical companies. This book is published in a very timely manner that enable the promoting of these innovative design and analysis. I believe that it will certainly not only inspire the statisticians working in rare disease, but also could shed some light on the solutions of unique situation for clinical trials with common disease. Therefore, I strongly recommend this book to all the statisticians who work on clinical trials, not only those in rare disease but also on other indications.

- Meijing Wu, Journal of Biopharmaceutical Statistics, August 2021

"I recommend this book to researchers who want to delve into the world of rare-disease trials, and in the meanwhile would encourage them to actively think about the problems also from a Bayesian perspective."

- Haiyan Zheng, International Society for Clinical Biostatistics, 72, 2021