Innovative Methods for Rare Disease Drug Development

ISBN-13: 9780367502904 / Angielski / Miękka / 2022 / 320 str.

Opis

Innovative Methods for Rare Diseases Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives.

Recenzje

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

Spis treści

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