Analysis of patient access to orphan drugs in Turkey

Abstract Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care have a low prevalence. An estimated one in 15 people worldwide affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed Anatomical Therapeutic Chemical (ATC) codes of drugs European Medicines Agency (EMA) Turkey considers be “orphan” pharmaceuticals. Methods The included this analysis were obtained from list orphan published EMA. Orphan drugs’ accessibility licensing status Health Implementation Communiqué Social Security Institution (SGK) List Abroad Active Substance Licensed Products Pharmaceuticals Medical Devices (T?TCK). Descriptive was applied determine identified EMA Turkey. Results Based on EMA, 105 pharmaceuticals approved with “orphan drug” except for lost drug decommissioned Union withdrawn Community Register January 2020. Of list, 34 inaccessible 71 available drugs, 23 (32%) 48 (68%) unlicensed 17 (74%) products (35%) covered reimbursement. When ATC examined, most common group found “L—Antineoplastic Immunomodulatory” agents. Conclusion incentive policy is very important ensure early access used treat Considering capacity prices Turkey, it can said many patients difficulty their treatment. It obvious such must prepare regulation