Policies and availability of orphan medicines in outpatient care in 24 European countries
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CitationSarnola, K. Ahonen, R. Martikainen, JE. Timonen, J. (2018). Policies and availability of orphan medicines in outpatient care in 24 European countries. EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY, Published Online: 09 April 2018, 10.1007/s00228-018-2457-x.
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.