Orphan drugs in Europe

PubMed, the internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Drugs for exceptionally rare diseases: do they deserve special status for funding?“ (QJM 2005 Oct 3). Authors are Hughes DA, Tunnage B and Yeo ST from the Centre for Economics and Policy in Health, Institute of Medical and Social Care Research, University of Wales, Bangor, 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. Mechanisms are discussed for creating a policy that would reduce geographical inequalities in provision across Europe. To access the full abstract of the article, click here.