一再 发表于 2025-3-21 17:37:35

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Ornament 发表于 2025-3-21 20:34:11

Master Protocol,e described in detail, separately for exploratory and confirmatory settings. The master protocol has been implemented successfully and improved the patient care in treating patients with rare cancers and pediatric cancers. One of those successful examples is discussed in the chapter.

Magisterial 发表于 2025-3-22 00:27:12

Operation and Other Considerations,sease classification, and patient monitoring. Additionally, translational relevance of preclinical models to pediatric and rare diseases and potential pharmacodynamic endpoints are included in the considerations.

争议的苹果 发表于 2025-3-22 07:23:51

Case Studies,ents who receive lung transplants. The third case is Belimumab’s approval that has applied Bayesian analysis to borrow adult data into pediatric to support approval in children with system lupus erythematosus (SLE).

Offensive 发表于 2025-3-22 10:23:09

Book 2024a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patie

Misnomer 发表于 2025-3-22 15:43:55

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责怪 发表于 2025-3-22 19:02:05

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参考书目 发表于 2025-3-22 23:55:08

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妨碍 发表于 2025-3-23 05:07:25

Diagnostic Criteria and Endpoint,tive multi-stake holders’ collaboration to best utilize the available information or generate the fit-for-purpose evidence. The statistical evaluation of the diagnosis and the endpoints are provided. Successful implementations in case studies are included.

habile 发表于 2025-3-23 06:42:39

Introduction,que rare diseases, affecting 300 million people worldwide, about 72% of which are genetic and about 70% of which are exclusively pediatric onset. More than 90% of rare diseases are without an FDA-approved treatment. Rare diseases are numerous, heterogenous in nature, and geographically disparate. Gi
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查看完整版本: Titlebook: Innovative Designs and Analyses for Small Population Clinical Trials; Development Strategi Jingjing Ye,Lei Nie Book 2024 The Editor(s) (if