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

ABSTRACT

Introduction: 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.

KEYWORDS:

• Pharmaceutical expenditures
• Bulgaria
• cost-effectiveness
• orphan medicinal products

Authors’ contributions

MK collected, analyzed the available data and actively participated in writing the manuscript. KT performed critical analysis and approved the final manuscript. GP contributed to the new design of the study. He also analyzed the data, read and approved the final manuscript. MM and AS read and approved the final version of manuscript.

Declaration of Interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose

Additional information

Funding

This paper is not funded.