Drug Development for Rare Diseases

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.

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.