Drug Development for Rare Diseases

Drug Development for Rare Diseases

Zhou, Yijie; Yang, Bo; Song, Yang

Taylor & Francis Ltd

02/2023

222

Dura

Inglês

9780367518349

15 a 20 dias

590

Descrição não disponível.
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
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clinical trial;pharmaceutical statistics;biostatistics;Rare Diseases;Odd;Surrogate Endpoint;Basket Trial;Pediatric Drug Development;Conditional Marketing Authorization;Rare Pediatric Disease;PBPK Model;BLA;Single Arm Trials;SMA Type;Accelerated Approval;Mathematical Expressions;Exondys 51;Group Sequential Design;Interim Analysis;Adaptive Enrichment Design;Sample Size Re-estimation;Single Arm Clinical Studies;Sample Size Reestimation;Rare Disease Registries;Randomized Withdrawal Period;Natural History Studies;OD;Rare Disease Population