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Rare Disease Drug Development = Clinical, Scientific, Patient, and Caregiver Perspectives /

Record Type:	Language materials, printed : Monograph/item
Title/Author:	Rare Disease Drug Development/ edited by Raymond A. Huml.
Reminder of title:	Clinical, Scientific, Patient, and Caregiver Perspectives /
other author:	Huml, Raymond A.
Description:	XXIV, 409 p. 36 illus., 8 illus. in color.online resource. :
Contained By:	Springer Nature eBook
Subject:	Medicine. -
Online resource:	https://doi.org/10.1007/978-3-030-78605-2
ISBN:	9783030786052

Rare Disease Drug Development = Clinical, Scientific, Patient, and Caregiver Perspectives /
Rare Disease Drug DevelopmentClinical, Scientific, Patient, and Caregiver Perspectives /[electronic resource] :edited by Raymond A. Huml. - 1st ed. 2021. - XXIV, 409 p. 36 illus., 8 illus. in color.online resource.

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 for real-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 capital providers, patient advocacy groups, patients, and caregivers.

ISBN: 9783030786052

Standard No.: 10.1007/978-3-030-78605-2doiSubjects--Topical Terms:

644133
Medicine.

LC Class. No.: R1

Dewey Class. No.: 610

Rare Disease Drug Development = Clinical, Scientific, Patient, and Caregiver Perspectives /
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