Policies and availability of orphan medicines in outpatient care in 24 European countries
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To assess pricing and reimbursement policies specific to orphan medicines and the availability and distribution settings of ten recently authorised medicinal products suitable for outpatient care with orphan status and centralised marketing authorisation in Europe, and whether patients receive these products free of charge or have to pay some or all of the costs themselves.
Web survey to authorities and representatives of third party payers in the Pharmaceutical Pricing and Reimbursement Information (PPRI) network in April 2016.
In most of the 24 countries, special policies were not implemented in the assessment of reimbursement status (22 countries) or in the pricing (20 countries) of orphan medicines. An average of five of the ten recently authorised products per country were available for outpatient care. Products were dispensed from community pharmacies in eight countries and from health care units in five countries. In four countries, both distribution settings were used. When products were dispensed from community pharmacies, patients typically paid some of the price themselves. Products dispensed from health care units were often free of charge for patients.
Most European countries had not implemented pricing and reimbursement policies specific to orphan medicines. The availability of orphan products varied between countries. It is important to discuss whether orphan medicines should be considered as a separate group in the reimbursement regulations in order to secure patient access to these medicines.
KeywordsOrphan medicines Availability Pricing Reimbursement Europe
We are grateful to the PPRI network for their participation and co-operation in this study.
All authors have made a substantial contribution to (1) the conception or design of the work (the acquisition, analysis or interpretation of data for the work), (2) drafting the work or revising it critically for important intellectual content and (3) final approval of the version to be published. All authors agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.
Compliance with ethical standards
Conflict of interest
The authors (KS, RA, JT) were funded by a research grant from the Social Insurance Institution of Finland (Kela) (grant number: 12/26/2015).
Research involving human participants and/or animals
This article does not contain any studies with human participants or animals performed by any of the authors.
For this type of study, informed consent is not required.
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