Orphan medicinal products’ access to the Bulgarian pharmaceutical market – challenges and obstacles

Abstract

ABSTRACTIntroduction: Demonstrating the value for money of orphan and ultra-orphan medicinal products is a challenge for every pharmaceutical company.Methods: A regulatory and pharmacoeconomic study was performed in Bulgaria. A retrospective analysis of the number of reimbursed orphan medicinal products (OMPs) in June 2017, as well as their expenditures for the period 2013–2016, was performed. The average growth rate was calculated as a compound annual growth rate (CAGR). A literature review about global requirements for orphan medicines was combined with critical analysis for the Bulgarian settings.Results and conclusion: Pharmacoeconomic requirements about OMPs inclusion in the positive drug list (PDL) in Bulgaria are implemented, but there is a need for more specific criteria. The OMPs included in the PDL in 2017 represent 22.34% of all OMPs in the European Union. The main tendency observed was of increasing pharmacotherapy expenditures: from 8.77 million euro in 2013 to 26.5 million euro in 2016. Although oncology OMPs have the highest cost, their annual growth rate is quite stable (CAGR = 35%). The reimbursement decision for all OMPs should be made on the basis of some specific criteria, a comparison with a higher threshold than the conventional one, and provision of strong evidence for significant therapeutic benefits of innovative OMPs.