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 aimed to explore the availability and access to orphan drugs for rare cancers in Bulgaria for the period of 2020–2023. We cross-compared data from both the European Union and national public sources 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. We juxtaposed the main characteristics of oncological and non-oncological orphan drugs 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 delay between market authorization and inclusion in the Bulgarian Positive Drug List 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. We call for targeted European and national policies to address this major inequality.
罕见癌症的定义为年发病率低于十万分之六。与罕见疾病类似，由于诊断复杂性和创新疗法获取延迟，罕见癌症患者面临着严重的健康不平等问题。在保加利亚等东南欧国家，这一状况因公共资源和专业知识的匮乏而进一步恶化，凸显了对罕见癌症开展额外政策与转化研究的迫切需求。本研究旨在探讨2020-2023年间保加利亚罕见癌症孤儿药的供应与获取情况。通过交叉比对欧盟及本国公共数据源，我们评估了罕见癌症孤儿药的可及品种数量、市场授权至纳入报销目录的时间延迟、公共支出动态以及全国范围内的区域差异，并对比了肿瘤类与非肿瘤类孤儿药的核心特征。结果显示：在欧洲药品管理局授权的50种肿瘤孤儿药中，保加利亚罕见癌症患者仅能获取15种；市场授权至纳入国家正面药品目录的中位延迟时间为760天；2020-2023年间罕见癌症孤儿药总支出达74,353,493欧元，其预算影响占公共药品总支出的比例从0.24%上升至3.77%。罕见癌症患者作为脆弱群体，常面临治疗途径受限甚至完全无法获取治疗的困境。我们呼吁制定针对性的欧洲及国家政策，以应对这一重大不平等问题。
肿瘤(癌症)患者之家