Description
- Reviews critical issues (e.g., endpoint/margin selection, sample size requirement and complex innovative design).
- Provides better understanding of statistical concepts and methods which may be used in regulatory review and approval.
- Clarifies controversial statistical issues in regulatory review and approval.
- Makes recommendations to accurately and reliably evaluate rare diseases regulatory submissions.
- Proposes innovative study designs and statistical methods for rare diseases drug development including n-of-1 trial design, adaptive trial design, and master protocols such as platform trials.
- Provides insight regarding current regulatory guidance on rare diseases drug development such as gene therapy.
Book Information
ISBN 9780367502102
Author Shein-Chung Chow
Format Hardback
Page Count 306
Imprint CRC Press
Publisher Taylor & Francis Ltd
Weight(grams) 580g
