Drug Development for Rare Diseases (Chapman & HallCRC Biostatistics Series) 1st Edition by Bo Yang , Yang Song, Yijie Zhou
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Drug Development for Rare Diseases (Chapman & HallCRC Biostatistics Series) 1st Edition by Bo Yang , Yang Song, Yijie Zhou

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233
File size
9.19 MB
Format
Digital PDF
Category
eBook[PDF]
About this ebook
The book Drug Development for Rare Diseases (Chapman & Hall/CRC Biostatistics Series) 1st Edition, edited by Bo Yang, Yang Song, and Yijie Zhou, provides a comprehensive statistical and strategic overview of therapeutic design for small patient populations. [1, 2, 3]
The primary topics and chapters covered in the book include: [1]
  • Introduction to Rare Disease Therapy Development: An foundational overview of the rare disease landscape and the unique framework required for orphan drug development. [1]
  • Challenges and Opportunities in Rare Disease Drug Development: Insights into the complexities of clinical development, small sample sizes, and the high unmet needs of patients. [1]
  • Regulatory Strategies and Considerations: Evaluating the evolving global regulatory environment, framework requirements, and accelerated approval pathways. [1]
  • Clinical Trial Design and Analysis Considerations: Deep-dive into innovative, flexible, and statistically robust trial methodologies tailored specifically for small patient cohorts. [1]
  • Use of Real-World Evidence (RWE) to Support Drug Development: Exploring how external data sources, natural history studies, and real-world data (RWD) augment or substitute for traditional randomized clinical trials. [1, 2]
  • Clinical Development of Pediatric Programs within Rare Diseases: Specialized approaches for evaluating rare conditions that uniquely manifest in, or require extrapolation for, infants and children. [1, 2, 3]
  • Use of Modeling and Simulation: Implementing mechanistic modeling, exposure-response calculations, and virtual simulations to optimize dosage and forecast trial outcomes when physical data is scarce. [1, 2, 3]
  • Case Studies of Rare Disease Drug Development: Practical, real-world examples demonstrating how these regulatory strategies and innovative designs have been successfully applied in the industry. [1]

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Topics

Case Studies of Rare Disease Drug Development Challenges and Opportunities in Rare Disease Drug Development Clinical Development of Pediatric Programs within Rare Diseases Clinical Trial Design and Analysis Considerations optimize dosage and forecast trial outcomes when physical data is scarce rare disease drug development Regulatory Strategies and Considerations Use of Real-World Evidence (RWE) to Support Drug Development
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