Overview
- Provides patient prospective and narratives
- Addresses relevant operational and developmental processes in drug development
- Provides comments from prominent CEOs in the biopharmaceutical and rare disease industries
Access this book
Tax calculation will be finalised at checkout
Other ways to access
Table of contents (26 chapters)
-
Front Matter
Keywords
About this book
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 capital providers, patient advocacy groups, patients, and caregivers.
Reviews
Editors and Affiliations
About the editor
Dr. Huml has over 30 years in the healthcare and biopharmaceutical industries with over 15 years of experience in the rare disease field. Importantly, he has two children with a rare disease. Bringing a unique and passionate drug development perspective to the table, Dr. Huml has published two books with Springer Publishing and several peer-reviewed papers through Springer affiliations, such as Drug Information Association’s (DIA’s) Journal of Therapeutic Strategy & Regulatory Science. Dr. Huml has assembled a team of rare disease experts for this book proposal that span the entire spectrum of rare disease drug development from selecting patients for clinical trials through approval and even post marketing, including commercialization activities.
Bibliographic Information
Book Title: Rare Disease Drug Development
Book Subtitle: Clinical, Scientific, Patient, and Caregiver Perspectives
Editors: Raymond A. Huml
DOI: https://doi.org/10.1007/978-3-030-78605-2
Publisher: Springer Cham
eBook Packages: Medicine, Medicine (R0)
Copyright Information: The Editor(s) (if applicable) and The Author(s), under exclusive license to Springer Nature Switzerland AG 2021
Hardcover ISBN: 978-3-030-78604-5Published: 09 November 2021
Softcover ISBN: 978-3-030-78607-6Published: 10 November 2022
eBook ISBN: 978-3-030-78605-2Published: 08 November 2021
Edition Number: 1
Number of Pages: XXIV, 409
Number of Illustrations: 28 b/w illustrations, 8 illustrations in colour
Topics: Medicine/Public Health, general, Biomedicine, general, Pharmacology/Toxicology, Immunology, Behavioral Therapy, Drug Safety and Pharmacovigilance