Comparative analysis of legislative requirements about patients' access to biotechnological drugs for rare diseases in central and Eastern European countries

摘要: The aim of the study was to compare access patients with rare diseases (RDs) biotechnological drugs in several Central and Eastern European countries (CEECs). We focused on legislative pricing reimbursement requirements, availability orphan medicinal products (BOMPs) for RDs, expenditures. A questionnaire-based survey conducted among experts from 10 CEECs: Bulgaria, Croatia, Estonia, Greece, Hungary, Poland, Romania, Slovakia, Serbia Macedonia. legal requirements BOMPs were collected. All medicines without prior designations extracted list products, 2017. status these 2017 public coverage included CEECs, as well share their costs relation total pharmaceutical spending period 2014 2016, defined. Our revealed that some differences amongst study. Union have developed implemented pharmacoeconomic guidelines or specific products. Cost-effectiveness analysis, cost-utility Markov models, meta-analysis discount levels results required only Poland Hungary. number reimbursed RDs a designation highest Hungary (17 40, respectively). Patient-based schemes available 11 out 17 BOMPs. Greece expenditure are approximately 214 million 180 EUR, respectively observed period. High proportion 2014-2016 is presented Bulgaria Slovakia. non-European CEECs face significant delay implementation guideline assessment similar best Greece. influence BOMP expenditures budget individual significant.