Orphan Diseases: Regulation of Drug Provision for Patients in Russia

In conditions of budget deficit, the issues of drug provision for patients suffering from life-threatening and chronic progressive rare (orphan) diseases require constant monitoring by regulatory authorities. In this regard, the purpose of the work was to analyze the main aspects of the legal regulation of drug provision for persons with rare (orphan) diseases. The lack of funds in the regions which could fully meet the needs of patients with rare (orphan) diseases in the provision of medicines, served as a weighty argument for the launch of the state federal program for high-costnosologies and the centralization of public procurement of medicines. The key tool for improving drug care was the approval of lists of orphan diseases and the introduction of the federal register of patients eligible for preferential drug provision into the unified information and analytical healthcare system. An important and defining moment of federalization of procurement of medicines was the creation of a federal state institution under the Ministry of Health "Federal Center for Planning and Organization of Drug Provision for Citizens". The main additional mechanism and alternative solution for providing medicines to children under 18 with severe life-threatening and chronic diseases, including rare (orphan) diseases, was the charitable support fund "Circle of Good" created by presidential decree. The financial and organizational approach and the decision of the regulator to federalize the procurement of orphan medicines under the high-cost nosology program makes it possible to increase the equal availability of medicines to persons with rare (orphan) diseases in all subjects of the Russian Federation and to reduce the costs of budget allocations.