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.
• Rare disease.
• Drug development.
• Innovative clinical trial design.
• Regulatory approval.
• Real-world evidence.
1. Introduction to Rare Disease Therapy Development
By Glen Laird
2. Challenges and Opportunities in Rare Disease Drug Development
By Glen Laird
3. Developing Drugs for Rare Diseases – Regulatory Strategies and Considerations
By Joanne Palmisano
4. Clinical Trial Design and Analysis Considerations for Rare Diseases.
By Lanju Zhang, Xiaolong Luo, Lingyun Liu, Ina Jazic, Bingming Yi, Ntin Nair, Xiaopeng Miao, Glen Laird and Xioayan Liu
5. Use of Real-World Evidence to Support Drug Development
By Lina Titievsky and Nataliya Volkova
6. Clinical Development of Pediatric Program within Rare Diseases
By Jingjin Ye, Lian Ma, Amy Barone, Nicholas Richardson, Emily Wearne, Kerry Jo Lee and Charu Gandotra
7. Use of Modeling and Simulation in Support of Drug Development for Rare Diseases
By Brenda Cirincione and Mark Peterson
8. Case Studies of Rare Disease Drug Development.
By Yimeng Lu, Chenkun Wang, Tu Xu, Jason Yuan and Tina Liu