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Titlebook: Innovative Designs and Analyses for Small Population Clinical Trials; Development Strategi Jingjing Ye,Lei Nie Book 2024 The Editor(s) (if

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發(fā)表于 2025-3-28 16:20:23 | 只看該作者
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Introduction,e discussed. The rest of the book will dive deep into providing a systematic framework that require a collaborative infrastructure including natural histories, diagnosis criteria, identification of clinical endpoints and engagement with the stakeholders. Each of the book chapters will discuss the cl
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發(fā)表于 2025-3-28 23:44:12 | 只看該作者
The Case Study of NCI-COG Pediatric MATCH Trial,al, is discussed in detail. The NCI-COG Pediatric MATCH trial overcomes many challenges and has created a collaborative framework for efficient collection, processing, and sequencing of refractory pediatric cancers. The study successfully facilitates evaluation of molecular-targeted agents in biomar
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Use of Real-World Data (RWD) and Real-World Evidence (RWE), and natural history studies have emerged as valuable resources, aiding in clinical trial planning and endpoint modeling. However, challenges such as data bias and variability require careful consideration. Case examples, such as the approval of Lutathera for neuroendocrine tumors, demonstrate the u
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