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Full 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.
Contents
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
