Drug Development for Rare Diseases

Bo Yang, Yang Song, Yijie Zhou

$263

Hardback

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English

Chapman & Hall/CRC
13 February 2023

Clinical trials; Probability & statistics; Biology, life sciences; Pharmaceutical technology

Series: Chapman & Hall/CRC Biostatistics Series

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.

Edited by: Bo Yang, Yang Song, Yijie Zhou
Imprint: Chapman & Hall/CRC
Country of Publication: United Kingdom
Dimensions: Height: 234mm, Width: 156mm,
Weight: 589g
ISBN: 9780367518349
ISBN 10: 0367518341
Series: Chapman & Hall/CRC Biostatistics Series
Pages: 222
Publication Date: 13 February 2023
Audience: General/trade , ELT Advanced
Format: Hardback
Publisher's Status: Active

1. Introduction to Rare Disease Therapy Development 2. Challenges and Opportunities in Rare Disease Drug 3. Developing Drugs for Rare Diseases: Regulatory Strategies 4. Clinical Trial Design and Analysis Considerations for Rare Diseases 5. Use of Real-World Evidence to Support Drug Development 6. Clinical Development of Pediatric Program within Rare Diseases 7. Use of Modeling and Simulation in Support of Drug Development for Rare Diseases 8. Case Studies of Rare Disease Drug Development

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.

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Drug Development for Rare Diseases

Bo Yang, Yang Song, Yijie Zhou

$263

Hardback

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