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QJM Advance Access originally published online on October 3, 2005
QJM 2005 98(11):829-836; doi:10.1093/qjmed/hci128
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© The Author 2005. Published by Oxford University Press on behalf of the Association of Physicians. All rights reserved. For Permissions, please email: journals.permissions@oxfordjournals.org

Commentary

Drugs for exceptionally rare diseases: do they deserve special status for funding?

D.A. Hughes, B. Tunnage and S.T. Yeo

From the Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, University of Wales, Bangor, UK

Address correspondence to Dr D.A. Hughes, Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, Dean Street, University of Wales, Bangor, Gwynedd LL57 1UT. email: d.a.hughes{at}bangor.ac.uk

Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, some EU healthcare systems assess the cost-effectiveness of therapies when deciding if they should be funded. As ultra-orphan drugs are invariably cost-ineffective, factors in addition to cost-effectiveness need to be considered if ultra-orphan drugs are to be provided by public health services. Health service funding of ultra-orphan drugs, which varies across the EU and within the UK, has led to geographical inequities in patients' access to treatment. In some instances, support for these drugs would appear to have been approved on the basis that diseases that are rare and severe are a special case. We explore whether ultra-orphan drugs merit special status by considering efficiency, effectiveness and equity criteria. Mechanisms are discussed for creating a policy that would reduce geographical inequalities in provision across Europe.


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