The availability of orphan medicines varies from country-to-country, says new European study

A new study from the University of Eastern Finland, published in the European Journal of Clinical Pharmacology, has highlighted that there are differences in the availability of orphan medicines between different European countries.

Orphan medicines are those developed specifically for the treatment, prevention or diagnostics of rare diseases, which are considered to be life-threatening or permanently disabling medical conditions that affect less than 5 in 10,000 of the general population.

This study, which 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, focused on the availability and distribution channels of 10 orphan medicines used in outpatient care in 24 European countries.

On average, half of the medicines were available on the markets, but there was variation when looking at each country individually. All 10 medicines were only available in three of the countries included in the study — the Netherlands, Malta and Poland. Five to nine medicines were available in 10 countries: Austria, Finland, Germany, Iceland, Italy, Norway, Slovakia, Spain, Sweden and the UK. Four countries, namely Latvia, Lithuania, Turkey and Belarus, did not have any of the medicines studied available.

Typically, rare medicines were dispensed to patients at pharmacies with hospitals or health centres 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.

Additionally, the study evaluated whether the European countries studied had implemented policies to regulate decisions relating to the pricing and reimbursement of rare medicines specifically. Generally, no policies that solely applied to rare medicines were in place; instead, decisions on these medicines were made on the same grounds as decisions on other medicines.

However, 13 countries reported some type of a special arrangement for rare medicines. In Latvia and Russia, for example, 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.

This study was funded by the Social Insurance Institution of Finland, Kela, and the University of Eastern Finland.