A comparative study of European rare disease and orphan drug markets

Alain Denis, Lut Mergaert, Christel Fostier, Irina Cleemput and Steven Simoens

Health Policy, 2010, vol. 97, issue 2-3, 173-179

Abstract: Objectives This article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom.Methods Information was derived from the international literature, analysis of legal texts, and a survey completed by national experts.Results These countries adopted varying approaches towards regulating rare disease and orphan drug markets and, hence, the availability, pricing and reimbursement of orphan drugs vary between countries. Strategies to keep down prices include public procurement in Sweden, profit controls in the United Kingdom, and price comparisons with other countries. To gain reimbursement, the cost-effectiveness and/or budget impact of orphan drugs is considered in some countries. Other societal considerations, such as whether the drug treats a life-threatening disease, are sometimes taken into account.Conclusions Extensive government intervention exists in rare disease and orphan drug markets in the countries studied. Our recommendations are to define priorities for research on rare diseases and orphan drugs at the European level, to set up disease and patient registries with a view to investigating the long-term effectiveness and cost-effectiveness of orphan drugs, to assess the profitability of orphan drugs, and to take into account societal considerations when evaluating orphan drugs.

Keywords: Rare; disease; Orphan; drug; Europe; Health; policy; Comparative; analysis (search for similar items in EconPapers)
Date: 2010
References: View references in EconPapers View complete reference list from CitEc
Citations: View citations in EconPapers (8)

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Persistent link: https://EconPapers.repec.org/RePEc:eee:hepoli:v:97:y:2010:i:2-3:p:173-179

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