Description
A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposed
to give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidence
inside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges.
Key Features:
- Rare disease.
- Drug development.
- Innovative clinical trial design.
- Regulatory approval.
- Real-world evidence.
Author: Bo Yang
Publisher: CRC Press
Published: 02/13/2023
Pages: 222
Binding Type: Hardcover
Weight: 1.11lbs
Size: 9.21h x 6.14w x 0.56d
ISBN13: 9780367518349
ISBN10: 0367518341
BISAC Categories:
- Mathematics | Probability & Statistics | General
- Medical | Pharmacology
- Medical | Pharmacy
About the Author
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.
This title is not returnable