Nebojša Pavlović1, Bojan Stanimirov1, Maja Stojančević1, Milica Paut-Kusturica1, Assena Stoimenova2, Svetlana Goločorbin-Kon1 and Momir Mikov1

(1) University of Novi Sad, Faculty of Medicine, Novi Sad, Serbia
(2) Medical University – Sofia, Faculty of Pharmacy, Sofia, Bulgaria


In the European Union (EU), rare diseases are defined as life-threatening or chronically debilitating diseases with prevalence lower than five in 10,000 inhabitants. Although individually rare, together, rare diseases affect a significant part of the population (27–36 million people in the EU). Therefore, patient access to orphan medicines is receiving increasing political attention in the EU.

In order to assess the differences in availability of reimbursed orphan medicines among Serbia, Bulgaria and Sweden, National Reimbursement Lists were reviewed and identified orphan medicines were crossed with the List of orphan drugs in Europe, published in July 2011, available from Orphanet. The analysis of regulatory traits was based mainly on a review of the official documents setting out legislation regarding rare diseases and orphan medicines in the studied countries.

Only 6.5 % (4 out of 61) of the authorised orphan medicines in Europe with prior orphan designation and 25.0 % (17 out of 68) without prior orphan designation were available and reimbursed in Serbia. In the Bulgarian Positive Drug List 44.3 % (27 out of 61) of the drugs with prior orphan designation and 50.0 % (34 out of 68) without prior orphan designation were identified. The share of reimbursed orphan medicines was the highest in Sweden among the observed countries – 52.5 % (32 out of 61) of the medicines with orphan designation and 60.3 % (41 out of 61) without prior orphan designation. According to the first level of the ATC Classification System, most of the reimbursed orphan medicines in the three studied countries belonged to the group L: “Antineoplastic and immunomodulating agents”, while the most common indications for authorised and reimbursed orphan medicines were “Neoplasms” (C00-D48), with 19 available orphan drugs in Serbia, 26 in Sweden and 31 in Bulgaria.

Inequities in the access to orphan medicines among Serbia, Bulgaria and Sweden may be explained by the differences in the approaches for registration, pricing and reimbursement of orphan medicines. The low share of reimbursed orphan drugs in Serbia may be due to incomplete compliance with EU legislation and existence of domestic procedure for authorisation as well. The EU legislation and policy on treatment of rare diseases obviously facilitate the penetration of orphan drugs on the EU market, but apparently there is also considerable budget impact on the availability of reimbursed orphan medicines.


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