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Innovative Methods for Rare Disease Drug Development


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Table of Contents

Preface. 1. Introduction. 2. Basic Considerations. 3. Hypotheses Testing for Clinical Evaluation. 4. Endpoint Selection in Clinical Trials. 5. Strategy for Margin Selection. 6. Probability of Inconclusiveness. 7. Probability Monitoring Procedure for Sample Size. 8. Real-World Data and Real-World Evidence. 9. Innovative Approaches for Rare Diseases Drug Development. 10. The n-of-1 Trial Design and Its Application. 11. Two-Stage Adaptive Seamless Trial Design. 12. Master Protocol - Platform Trial Design. 13. Gene Therapy for Rare Diseases. 14. Clinical Development for NASH Program

About the Author

Shein-Chung Chow, Ph.D. is currently a Professor at Duke University School of Medicine, Durham, NC. He was previously the Associate Director at the Office of Biostatistics, Center for Drug Evaluation and Research, United States Food and Drug Administration (FDA). Dr. Chow has also held various positions in the pharmaceutical industry such as Vice President at Millennium, Cambridge, MA, Executive Director at Covance, Princeton, NJ, and Director and Department Head at Bristol-Myers

Squibb, Plainsboro, NJ. He was elected Fellow of the American Statistical Association and an elected member of the ISI (International Statistical Institute). Dr. Chow is Editor-in-Chief of the Journal of Biopharmaceutical Statistics and Biostatistics Book Series, Chapman and Hall/CRC Press, Taylor & Francis, New York. Dr. Chow is the author or co-author of over 300 methodology papers and 30 books.


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

"I recommended this book not only to statisticians and physicians working on drug development but also to scientists/professionals involving in patients' access in personalized medicine. The book is directly not only useful for trial study design for rare disease for regulatory approval but also helpful on providing statistical concepts for other type of studies tackling many statistical issues such as limited numbers of research objects."Min-Hua Jen, Eli Lilly, Bracknell UK, Journal of the Royal Statistical Society: Series A, 2022.

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