Innovative Methods for Rare Disease Drug Development

by Shein-Chung Chow

Book Overview

In the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects fewer than 200,000 persons. For the approval of "orphan" drug products for rare diseases, the traditional approach of power analysis for sample size calculation is not feasible because there are only limited number of subjects available for clinical trials. In this case, innovative approaches are needed for providing substantial evidence...

• Format: Paperback
• Language: English
• ISBN: 0367502909
• ISBN13: 9780367502904
• Release Date: May 2022
• Publisher: CRC Press
• Length: 306 Pages
• Weight: 0.99 lbs.
• Dimensions: 0.7" x 6.1" x 9.2"