Reimbursed Orphan Medicines in Bulgaria and the Share of Biotechnology-Derived Products

Abstract

ABSTRACTRare diseases are life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000people in the European Union. Most of the people suffering from rare diseases are actually affected by less frequently occurring diseases affecting one in 100,000 people or fewer. Almost 80% of the rare diseases have identified genetic origins and lots of them are treated with biotechnology-derived medicinal products. The aim of our study was to evaluate the access to orphan medicines in Bulgaria based on the analysis of the Positive Drug List (PDL) and the share of biotechnology-derived products reimbursed for rare diseases in Bulgaria. Only 21 out of 56 medicines with European orphan designation and European marketing authorisation are available and funded in Bulgaria. 29% of them are biotechnology-derived. Another 17 (out of 47) orphan medicines with European marketing authorisation and without prior orphan designations in the EU are reimbursed and 59% of them are biotechnology-derived. Thus approximately just 37% of the orphan medicines (both with and without prior orphan designation) are available and funded in Bulgaria. Evidently the centralised marketing authorisation is not supported by the national regulatory requirements for price setting and inclusion into the PDL, which are necessary for guaranteeing medicines availability on the national market. The regulators and payers still do not ensure balance between the needs ofpatients and resources allocation.