OBJECTIVES: To analyze and compare the access of patients with rare diseases (RDs) to pharmacotherapy by monitoring the legislative procedures and the number of reimbursed orphan medicinal products (OMPs) in Bulgaria, Romania and Greece.

METHODS: A comparative analysis of the current legislation in these countries was performed. In addition, the national programs (NPs) of the three countries, the trends in the development of reimbursement and pricing policies for OMPs, the availability of national registers, the policy for neonatal screening and the development of centers of expertise for the last 10 years were analyzed.

RESULTS: Since joining the European Union (EU), all three countries have harmonized their legislation with the European one. There has been an increase in the number of national registries for RD and centers of expertise. The total number of medicines for RDs with orphan status authorized for use in the EU is 179 (163% increase in comparison with 2012). Out of them, 42 are included in the Positive drug list (PDL) in Bulgaria, 100 in Greece, and 61 in Romania. The percentage increase in availability compared to 2012 is 100%, 146.44% and 69.44%, respectively. The number of OMPs that have been authorized but lost orphan status is 103 (37.33% increase compared to 2012). In Bulgaria 89 are reimbursed, in Greece – 86, and in Romania – 39. The percentage increase in availability is 122.50%, 65.38% and -11.36%, respectively. The therapeutic group 'L - Antineoplastic and immune modulating agents' prevails among the OMPs included in all three countries.

CONCLUSIONS: The study found an increased number of reimbursed OMPs compared to 2012. The access to therapy has improved, as a result of hard work in the field of legislation, the goals set in the NPs, and the creation of organizations that aim to address the needs of patients with RDs.