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Orphan drugs: pricing, reimbursement and patient access

Orphan drugs: pricing, reimbursement and patient access Purpose – This article aims to provide a description of principal aspects of policy and practice associated with orphan drugs and treatments of rare diseases, and give perspectives for 2011 on new and emerging approaches for addressing patient access. Design/methodology/approach – Information has been synthesized from a wide variety of sources including the authors' experience and familiarity with this topic, having over the past ten years developed market access strategies for many orphan drugs. This is supplemented by pertinent information from conferences, meetings and articles published over the last 20 years and from policy documents released by relevant regulatory bodies. Findings – While a small number of people suffer from any one rare disease, approximately 30 million in the EU and 25 million in the USA are affected by such diseases. Annual orphan drug treatment costs may approach $500,000. High costs coupled with relatively sparse supportive clinical data and difficulty in meeting standard cost‐effectiveness benchmarks raise significant issues for payers in allocating finite budgetary resources. Since the US (1983) and European (European Parliament, 2000) orphan drug regulations were approved, hundreds of agents have been granted orphan status although many fewer have achieved marketing authorization. Agencies have introduced a slew of incentives to help sponsors develop and market orphan medicinal products. The nature and focus of the programs are undergoing a metamorphosis as access to funding is becoming as important as supporting development. Originality/value – The article represents a contemporary examination of orphan drug policy and practice, bringing together historic elements which influenced practice up to 2010 and the latest, emerging trends and approaches which stakeholders are now embracing and which will shape procedures from 2011. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png International Journal of Pharmaceutical and Healthcare Marketing Emerald Publishing

Orphan drugs: pricing, reimbursement and patient access

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References (27)

Publisher
Emerald Publishing
Copyright
Copyright © 2011 Emerald Group Publishing Limited. All rights reserved.
ISSN
1750-6123
DOI
10.1108/17506121111190121
Publisher site
See Article on Publisher Site

Abstract

Purpose – This article aims to provide a description of principal aspects of policy and practice associated with orphan drugs and treatments of rare diseases, and give perspectives for 2011 on new and emerging approaches for addressing patient access. Design/methodology/approach – Information has been synthesized from a wide variety of sources including the authors' experience and familiarity with this topic, having over the past ten years developed market access strategies for many orphan drugs. This is supplemented by pertinent information from conferences, meetings and articles published over the last 20 years and from policy documents released by relevant regulatory bodies. Findings – While a small number of people suffer from any one rare disease, approximately 30 million in the EU and 25 million in the USA are affected by such diseases. Annual orphan drug treatment costs may approach $500,000. High costs coupled with relatively sparse supportive clinical data and difficulty in meeting standard cost‐effectiveness benchmarks raise significant issues for payers in allocating finite budgetary resources. Since the US (1983) and European (European Parliament, 2000) orphan drug regulations were approved, hundreds of agents have been granted orphan status although many fewer have achieved marketing authorization. Agencies have introduced a slew of incentives to help sponsors develop and market orphan medicinal products. The nature and focus of the programs are undergoing a metamorphosis as access to funding is becoming as important as supporting development. Originality/value – The article represents a contemporary examination of orphan drug policy and practice, bringing together historic elements which influenced practice up to 2010 and the latest, emerging trends and approaches which stakeholders are now embracing and which will shape procedures from 2011.

Journal

International Journal of Pharmaceutical and Healthcare Marketing – Emerald Publishing

Published: Nov 21, 2011

Keywords: Rare diseases; Orphan drugs; Pricing; Reimbursement; Market access; Health policy; Diseases; Pharmaceuticals industry

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