A comparative study of European rare disease and orphan drug markets |
| |
Affiliation: | 1. Yellow Window Management Consultants, Antwerp, Belgium;2. Belgian Health Care Knowledge Centre, Brussels, Belgium;3. Research Centre for Pharmaceutical Care and Pharmaco-economics, Katholieke Universiteit Leuven, Onderwijs en Navorsing 2, P.O. Box 521, Herestraat 49, 3000 Leuven, Belgium;1. European Organisation for Rare Diseases, 96 rue Didot, 75014 Paris, France;2. NHS England, London, UK;3. University of Southern Denmark, Unit for Health Promotion Research, Niels Bohrs Vej 9, DK-6700 Esbjerg, Denmark;1. Laboratory of Pharmacology and Pharmaceutical Care, Faculty of Pharmacy, University of Coimbra, Pólo das Ciências da Saúde, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal;2. Coimbra Institute for Clinical and Biomedical Research (iCBR), Faculty of Medicine, University of Coimbra, Pólo das Ciências da Saúde, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal;3. Faculty of Pharmacy, University of Coimbra, Pólo das Ciências da Saúde, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal;4. Bluepharma, Indústria Farmacêutica SA, São Martinho do Bispo, 3045-016 Coimbra, Portugal |
| |
Abstract: | ObjectivesThis article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom.MethodsInformation was derived from the international literature, analysis of legal texts, and a survey completed by national experts.ResultsThese 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.ConclusionsExtensive 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: | |
本文献已被 ScienceDirect 等数据库收录! |
|