Shulph Ink
Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives
Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives
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Excellent- More about Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives
This book provides a comprehensive overview of rare disease drug development, offering insights from various perspectives and addressing key challenges and processes. It emphasizes the role of patient advocacy groups and advocates for improved clinical trials and market access strategies.
Format: Hardback
Length: 409 pages
Publication date: 09 November 2021
Publisher: Springer Nature Switzerland AG
This comprehensive book offers a thorough exploration of the intricate landscape of rare disease drug development, providing a diverse range of insights from a multitude of perspectives. It delves into the challenges faced by individuals living with rare diseases and their caregivers, emphasizing the critical role of patient advocacy groups in navigating the complexities of clinical trials and investment decisions. The book also sheds light on innovative approaches to clinical trial design and the optimization of rare disease registries. By examining the drug development processes across various disciplines, such as oncology, hematology, pediatrics, and gene therapy, it provides a comprehensive understanding of the complexities involved in bringing new treatments to patients with rare disorders. Furthermore, the book addresses the operational aspects of drug development, including regulatory approval processes, development accelerations, and market access strategies.
The authors argue for the importance of real-world data and evidence generation in orphan medicinal drug development, highlighting the need for more robust data to inform decision-making and improve patient outcomes.
Rare Disease Drug Development is an invaluable resource for biopharmaceutical R&D professionals, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers seeking to gain a deeper understanding of the challenges and opportunities in rare disease drug development. Its comprehensive coverage, expert insights, and thoughtful analysis make it a must-read for anyone involved in this field.
Weight: 870g
Dimension: 160 x 241 x 23 (mm)
ISBN-13: 9783030786045
Edition number: 1st ed. 2021
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