Recent issues on accessibility to orphan drugs and policy implications in Turkey

Rare diseases are conditions which affects a small percentage of the population, life-threatening, serious genetic, chronic, complex and needed special care. It is estimated that one in 15 people worldwide can be affected by rare diseases. According to The U.S. Food and Drug Administration (U.S. FDA), diseases that are affecting less than 200.000 people are defined as rare diseases. It is estimated that rare diseases affecting not more than 1 person per 2000 in the EU population. Orphan drug is a type of medicine which is developed specially to treat a rare medical condition. It is very high cost to develop orphan drugs for patients in very small populations. Therefore, the government sector support is important to encourage pharmaceutical companies to develop orphan drugs. This study has been conducted to contribute to determining the deficiencies and development goals, and therefore, preparing action plans, by setting out the market access of the policies introduced on the Rare Diseases in Turkey and of orphan drugs used in the treatment of rare diseases in full detail. In Turkey, national 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. Depending on the status of the non-registered orphan drug, either it might be reimbursed by the payer or it might be compensated by the patients. Based on The European Medicines Agency (EMA) publications, 105 pharmaceuticals are approved with orphan drug status by December 2018. Among approved 105 EMA orphan drugs, 36 of them are not available in Turkey, only 69 of them are accessible. 32 out of 69 accessible orphan drugs are licensed products by Turkish Ministry of Health. Among 32 licensed orphan drugs, only 26 of them are reimbursed by national payer. Within 69 accessible orphan drugs, 37 of them are at the non-registered product status and only 19 out of 37 are reimbursed through national payer. The rest of these reimbursed drugs could only be compensated by patients. It is estimated that in Turkey, orphan drugs have 276 million € sales in 2018. It is very important to have an orphan drug incentive policy in order to ensure early access to drugs used in rare diseases. First it must prepare a regulation on orphan drugs with Turkey.