Rare Diseases and Drug Development: Overcoming Challenges in Clinical Trials

In the United States, a disease is considered rare if it affects fewer than 200,000 people. With over 7,000 rare diseases identified, approximately 30 million Americans – or 10% of the population – are impacted. This complex and diverse disease area poses significant challenges for patients, caregivers, regulators, drug developers, and other stakeholders.

This book provides a comprehensive overview of the common issues facing rare disease drug developers, highlighting the unique challenges of clinical development in small populations. It delves into the strategies employed in the evolving regulatory environment, and explains the generation and utilization of various data and evidence, both within and beyond clinical trials.

Through recent examples, this book demonstrates the challenges faced by rare disease drug developers and the innovative strategies used to overcome them. Key topics include:

• Rare diseases and their impact on public health
• Innovative clinical trial design for small population groups
• Regulatory approval pathways for rare disease treatments
• Real-world evidence and its role in rare disease drug development

This book serves as a valuable resource for industry professionals, researchers, and stakeholders seeking to better understand the complexities of rare disease drug development and the strategies employed to address these challenges.

Publication Details:

Publisher: Chapman and Hall/CRC

Publication Date: February 13, 2023

Language: English

Hardcover: 222 pages

ISBN-10: 0367518341

ISBN-13: 978-0367518349