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
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
Este título pertence ao(s) assunto(s) indicados(s). Para ver outros títulos clique no assunto desejado.
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