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 |
Abstract
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.
References
- Bulgarian Health Act (2004) State Gazette, 70/10 Aug. 2004.
- Bulgarian Law on Medicinal Products in Human Medicine (2007) State Gazette, 31/13 Apr. 2007.
- Bulgarian National Plan for Rare Diseases (2009–2013) (genetic, birth defects and noninherited diseases), Ministry of Health, Republic of Bulgaria.
- Council Recommendation (2009) Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) Official Journal of the European Communities.
- Denis A., Mergaert L., Fostier C., Cleemput I., Simoens S. (2010) Health Policy, 97(2-3), 173-179.
- Dental and Pharmaceutical Benefits Agency of Sweden (2011) Medical products database (Accessed: Dec. 2011)
- EURORDIS Proposal for the practical implementation of policy principles to improve access to orphan drugs in the EU (2009) EURORDIS(Accessed: Feb. 2012)
- Heemstra H.E. (2010) EJHP Practice, 16(4), 25-27.
- International Classification of Diseases (2011) (Accessed: Dec. 2011)
- International Monetary Fund (2011) (Accessed: Jan. 2012)
- List of orphan drugs in Europe (2011) Orphanet Report Series, Orphan Drugs collection, July 2011.
- Medicines for rare diseases. European Medicines Agency (Accessed: Apr. 2012)
- Ministry of Health in Bulgaria (2011) The Positive Drug List (Accessed: Dec. 2011)
- National Organisation for Rare Diseases of Serbia (2011) (Accessed: Jan. 2012)
- Policies for rare diseases and orphan drugs: KCE reports 112C (2009) Belgian Health Care Knowledge Centre (Accessed: Mar. 2012)
- Rare Diseases Sweden (2011) (Accessed: Dec. 2011)
- Regulation (1999) Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan drugs.
- Republic Fund of Health Insurance of Serbia (2011) The List of Medicines (Accessed: Dec. 2011)
- Rinaldi A. (2005) EMBO Reports, 6(6), 507-510.
- Schneider C.K., et al. (2010) Nat. Rev. Drug Discov., 9(3), 195-201.
- Serbian Games of Chance Act (2011) Official Gazette, 88/2011.
- Serbian Health Insurance Act (2011) Official Gazette, 107/2005, 109/2005 and 57/2011.
- Serbian Law on Healthcare (2011) Official Gazette, 107/2005, 72/2009, 88/2010, 99/2010 and 57/2011.
- Serbian Law on Medicines and Medical Devices (2010) Official Gazette, 30/2010.
- Simoens S. (2011) Orphanet J. Rare Dis., 6, 42.
- Stefanov R., Taruscio D. (2009) Ital. J. Public Health, 7(6), 289-293.
- Stoimenova A., Manova M., Savova A., Angelovska B., Petrova G. (2011) Biotechnol. Biotech. Eq., 25(2), 2418-2423.
- Tejada P. (2009) Bulgaria at the forefront of rare disease policy (Accessed: Jan. 2012)
- The National Board of Health and Welfare of Sweden (2011) (Accessed: Nov. 2011)
- Wastfelt M., Fadeel B., Henter J.I. (2006) J. Intern. Med., 260(1), 1-10.
- Westermark K., et al. (2011) Nat. Rev. Drug Discov., 10, 341-349.