Understanding the Rare Disease Market and its Dynamics
- Elizabeth Hernberg-Ståhl, Late Phase Solutions Europe AB, Sweden
- Miroslav Reljanović
The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey.
After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs.
The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs.
Researchers and scientists; Specialists physicians managing patients with rare diseases; Managers and project leaders in the pharmaceutical industry; Patient organizations
- Published: October 2013
- Imprint: Woodhead Publishing
- ISBN: 978-1-907568-09-1