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.

About the Author
Raymond A. Huml, MS, DVM, RAC; Vice President of Medical & Scientific Strategy, Head of the Rare Disease Consortium for Syneos Health Clinical Services, Morrisville, North Carolina, USA
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.

Reviews
"The book explores various rare disease drug development processes in disciplines such as oncology, haematology, paediatrics, and gene therapy. ... each chapter offers interesting insights to anyone interested in drug development for rare diseases. Highly recommended." (Geoff Case, RARE Revolution Magazine, Issue 26, 2023)



Book Information
ISBN 9783030786045
Author Raymond A. Huml
Format Hardback
Page Count 409
Imprint Springer Nature Switzerland AG
Publisher Springer Nature Switzerland AG