Orphan Drugs, 1st Edition

  • Published By:
  • ISBN-10: 1908818395
  • ISBN-13: 9781908818393
  • DDC: 615.19
  • Grade Level Range: College Freshman - College Senior
  • 334 Pages | eBook
  • Original Copyright 2013 | Published/Released June 2014
  • This publication's content originally published in print form: 2013

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This authoritative book familiarizes the reader with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before orphan drug legislation enacted in the US in 1983, there was a limited interest in developing treatments for very small patient groups. Similar levels of investment are needed to bring a drug to the market for small and large patient groups. 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 and their specific needs and concerns: patients and patient organizations, researchers and treating physicians, industry, regulatory and reimbursement bodies. They will also understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients and to minimize the gap between known diseases and orphan designations, and approved drugs and paid drugs. The book provides short practical case stories from patients and researchers, as well as industry representatives and other authorities on the challenges they encountered in developing orphan drugs or getting access to orphan drugs.

Table of Contents

Front Cover.
Half Title Page.
Other Frontmatter.
Title Page.
Copyright Page.
List of Figures and Tables.
About the Authors.
1: Introduction.
2: Orphan Drugs and Orphan Drug Legislation.
3: Characteristics of Rare Diseases.
4: Patient Network and Advocacy Groups.
5: Organisations and Networks Dedicated to Rare Diseases and Orphan Drugs.
6: Policies and Research Funding.
7: Designing Robust Clinical Trials for Orphan Drugs.
8: Market Access Procedures for Orphan Drugs.