1st Edition
Drug Development for Rare Diseases
1. Introduction to Rare Disease Therapy Development
By Glen Laird
2. Challenges and Opportunities in Rare Disease Drug Development
By Glen Laird
3. Developing Drugs for Rare Diseases – Regulatory Strategies and Considerations
By Joanne Palmisano
4. Clinical Trial Design and Analysis 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. Use of Real-World Evidence to Support Drug Development
By Lina Titievsky and Nataliya Volkova
6. Clinical Development of Pediatric Program within Rare Diseases
By Jingjin Ye, Lian Ma, Amy Barone, Nicholas Richardson, Emily Wearne, Kerry Jo Lee and Charu Gandotra
7. Use of Modeling and Simulation in Support of Drug Development for 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
Biography
Bo Yang is the Vice President, Biometrics and Real World Evidence at Vertex Pharmaceuticals, USA.
Yang Song is the Executive Director, Biostatistics Group Head of Pipeline Development at Vertex Pharmaceuticals, USA.
Yijie Zhou is the Executive Director, Real World Statistics and Analytics at Vertex Pharmaceuticals, USA.
