Kostadin Kostadinov, Ivelina Popova-Sotirova, Yuliyana Marinova, Nina Musurlieva, Georgi Iskrov and Rumen Stefanov, as part of the Institute for Rare Diseases and Medical University – Plovdiv examine the availability and access to orphan drugs for rare cancers in Bulgaria, aiming to respond to the need for its improvement. This research provides valuable insights into the challenges faced by patients with rare cancers in accessing treatment and has been published in the prestigious international journal “Cancers” (Impact factor: 5.2).
Rare cancers are defined by an annual incidence of fewer than 6 per 100,000. Bearing similarities to rare diseases, they are associated with substantial health inequalities due to diagnostic complexity and delayed access to innovative therapies. This situation is further aggravated in Southeastern European countries like Bulgaria, where limited public resources and expertise underscore the need for additional policy and translational research on rare cancers.
This study aims to explore the availability and access to orphan drugs for rare cancers in Bulgaria for the period of 2020–2023. The data from both the European Union and national public sources is cross-compared to evaluate the number of available and accessible orphan drugs for rare cancers, the delay from market authorization to reimbursement, the dynamics of public expenditures, and regional disparities in access across the country. The main characteristics of oncological and non-oncological orphan drugs are juxtaposed as well.
Only 15 out of 50 oncological orphan drugs that were authorized by the European Medicine Agency were accessible for rare cancer patients in Bulgaria. The median external delay defined as the time interval between EMA authorization and PDL inclusion of the respected cancer orphan drugs was 760 days. The total expenditures for all orphan drugs for rare cancers amounted to EUR 74,353,493 from 2020 to 2023. The budgetary impact of this group rose from 0.24% to 3.77% of total public medicinal product expenditures for the study period.
Rare cancer patients represent a vulnerable population that often faces limited to no access to treatment. This article calls for targeted European and national policies to address this major inequality. Read the full article here.