Orphan Drug Development for Rare Diseases (e-book)

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Orphan Drug Development for Rare Diseases (e-book)

Table of Contents


Lead Editors

Sundar Ramanan, PhD, MBA and Sunny Kamlesh Dave, MPharm

Product Description

RAPS latest global regulatory reference guide, Orphan Drug Development for Rare Diseases, covers every aspect of orphan drug development. Topics include history, incentives, benefits, patient engagement, orphan drug designation, marketing authorization, and market access. Real-world case studies and examples are presented.

Orphan Drug Development for Rare Diseases explores challenges facing the developer and provides strategies to overcome them. The strategies involve adopting the applicable regulatory guidance, involving patients, and reviewing precedent and case studies. Each chapter emphasizes the critical role that patients and patient advocacy groups play in each stage of orphan drug development. A number of diverse rare disease organizations are described.

Who should buy this book?

Orphan Drug Development for Rare Diseases will be helpful for regulatory professionals at all levels. It demonstrates key learnings for those new to drug development to those seeking advanced treatments and cures for rare diseases. Academic institutions, non-profit organizations, small and large pharmaceutical companies, and pharmaceutical professionals involved in drug development also will benefit.

Understanding development and regulatory processes is crucial to advancing therapies for rare diseases, which involves complex and unique incentives, requirements, market considerations, and opportunities for patient engagement
Eva A. Temkin, JD
Partner, King & Spalding

Published by RAPS © 2021. E-book. 160 pages. ISBN: 978-1-947493-76-6

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