OBJECTIVES : European Medicine Agency(EMA) defines rare diseases as life-threatening or chronically debilitating diseases. It is estimated that rare diseases affecting not more than 1 person per 2000 in the EU population. By 2018, 105 products were designated with orphan status in EMA. This study has been conducted to understand the patient access policies to orphan medicines and their estimated financial impact in Turkey.

METHODS : Systematic literature review has been conducted from the publicly available publications of EMA, IQVIA Turkey, Turkish Medicines and Medical Device Agency (TITCK) and Social Security Institution(SGK). Descriptive analysis have been applied to understand the accessibility condition and estimated financial impact of EMA defined orphan drugs in Turkey. The period selected for this study corresponds from 2016 to 2018.

RESULTS : Based on EMA, 105 pharmaceuticals are approved with orphan drug status by December 2018. In Turkey, local regulations allow patients to access registered orphan drugs either through national reimbursement system of payer or through out of pocket payment contribution by patients. Non-registered orphan drugs can be accessible through national patient accessibility programs. Among 105 EMA orphan drugs, 36 of them are not available in Turkey. Within 69 accessible orphan drugs, 37 of them are at the non-registered product status.19 out of 37 are reimbursed through national payer and 18 out of 37 are compensated by patients. 32 out of 69 accessible orphan drugs are licensed products. Among 32 licensed orphan drugs, 26 of them are reimbursed by SGK. It is estimated that in Turkey, orphan drugs have 276 million € sales in 2018.

CONCLUSIONS : National payer reimburses 45 of these accessible orphan drugs. In order to enhance the access to adequate treatment for the patients suffering from rare diseases, reimbursement pathway has to be shaped accompanied with budget allocation to recognize the added value of orphan drugs.