Drug development for rare diseases. (2022)

• Record Type: Book
• Further Information: Note: Edited by Bo Yang, Yang Song, Yijie Zhou.
• Editors: Yang, Bo; Song, Yang; Zhou, Yijie
• Contents: 1. Introduction. By Glen Laird 2. Challenges for Rare Disease Drug Development. By Glen Laird 3. Regulatory Landscape. By Joanne Palmisano 4. Clinical Trial Design Considerations for Rare Diseases. By Lanju Zhang, Xiaolong Luo, Lingyun Liu, Ina Jazic, Bingming Yi, Ntin Nair, Xiaopeng Miao, Glen Laird and Xioayan Liu 5. Statistical Analyses Methodologies and Considerations for Rare Disease Data. By Lina Titievsky and Nataliya Volkova 6. Real World Evidence vs. Clinical Trials. By Jingjin Ye, Lian Ma, Amy Barone, Nicholas Richardson, Emily Wearne, Kerry Jo Lee and Charu Gandotra 7. Clinical Development of Pediatric Program within Rare Diseases. By Brenda Cirincione and Mark Peterson 8. Case Studies of Rare Disease Drug Development. By Yimeng Lu, Chenkun Wang, Tu Xu, Jason Yuan and Tina Liu
• Edition: 1st
• Publisher Details: Boca Raton : Chapman & Hall/CRC
• Publication Date: 2022
• Extent: 1 online resource, illustrations (black and white, and colour)
• Subjects: 616; Rare diseases; Drug development
• Languages: English
• ISBNs: 9781000820287; 9781000820225
• Related ISBNs: 9780367518349
• Notes: Note: Description based on CIP data; resource not viewed.
• Access Rights: Legal Deposit; Only available on premises controlled by the deposit library and to one user at any one time; The Legal Deposit Libraries (Non-Print Works) Regulations (UK).
• Access Usage: Restricted: Printing from this resource is governed by The Legal Deposit Libraries (Non-Print Works) Regulations (UK) and UK copyright law currently in force.