书目名称 | Rare Disease Drug Development | 副标题 | Clinical, Scientific | 编辑 | Raymond A. Huml | 视频video | http://file.papertrans.cn/822/821231/821231.mp4 | 概述 | Provides patient prospective and narratives.Addresses relevant operational and developmental processes in drug development.Provides comments from prominent CEOs in the biopharmaceutical and rare disea | 图书封面 |  | 描述 | .This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. ..A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors‘ case forreal-world data and evidence generation in orphan medicinal drug development. ..Rare Disease Drug Development. is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party | 出版日期 | Book 2021 | 关键词 | Rare disease; drug development; caregivers; orphan drug; disease-modifying treatments | 版次 | 1 | doi | https://doi.org/10.1007/978-3-030-78605-2 | isbn_softcover | 978-3-030-78607-6 | isbn_ebook | 978-3-030-78605-2 | copyright | The Editor(s) (if applicable) and The Author(s), under exclusive license to Springer Nature Switzerl |
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