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Availability of orphan medicines varies between European countries

There are differences in the availability of orphan medicines between European countries, a study has found.

Orphan medicines are pharmaceutical products developed specifically for the treatment, prevention or diagnostics of rare diseases, which affect some 30 million people in Europe.

Factors limiting the availability of orphan medicines include them not entering the markets, or their financial burden on patients or society being too high.

Researchers from the University of Eastern Finland looked at the availability and distribution channels of ten orphan medicines used in outpatient care in 24 European countries. On average, five of the ten medicines were available on the markets, but there was variation between the countries.

All ten medicines were available only in three countries: the Netherlands, Malta and Poland. Five to nine medicines were available in ten countries: Austria, Finland, Germany, Iceland, Italy, Norway, Slovakia, Spain, Sweden and the United Kingdom. Latvia, Lithuania, Turkey and Belarus, did not have any of the medicines studied available.

The study revealed that typically, rare medicines were dispensed to patients at pharmacies. Other dispensaries, such as hospitals or health centres, were also quite commonly used. When orphan medicines were dispensed to patients at pharmacies, a part of the price was often paid by the patient, whereas hospitals and health centres typically dispensed the medicine free of charge.

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The study also looked at whether the European countries studied had implemented policies to regulate decisions relating to the pricing and reimbursement of rare medicines specifically. Typically, no policies applicable to rare medicines alone were in place. Instead, decisions on rare medicines were made on the same grounds as decisions on other medicines.

However, 13 countries reported some type of a special arrangement. For example, in Latvia and Russia rare medicines come under a separate budget, and countries such as Spain, Lithuania and Hungary have set special conditions on the reimbursability of rare medicines.

The study was conducted in April 2016 via a survey sent to the members of a network of authorities dealing with pharmaceutical product pricing and reimbursement in Europe, and 24 countries responded. It was funded by the Social Insurance Institution of Finland, Kela, and the University of Eastern Finland.

Researchers concluded that “most European countries had not implemented pricing and reimbursement policies specific to orphan medicines”.

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.”


  • Research article: Sarnola Kati, Ahonen Riitta, Martikainen Jaana E, Timonen Johanna: Policies and availability of orphan medicines in outpatient care in 24 European countries. European Journal of Clinical Pharmacology. DOI: 10.1007/s00228-018-2457-x