The system of drug provision for patients with rare diseases in the Russian Federation: institutional analysis, key problems and development prospects

this article is devoted to a comprehensive institutional analysis of the system of drug provision for patients with rare diseases in the Russian Federation. The purpose of the study is to identify the key structural elements of the system, reveal systemic problems, and determine promising areas for its improvement. The work examines the multi-level structure of the organization of medical care and drug provision at both the federal and regional levels, as well as the activities of specialized funds, particularly the «Circle of Kindness» Foundation. An analysis of the regulatory framework governing the circulation and financing of orphan drugs was conducted. The methodological basis of the study consisted of systemic and institutional approaches, content analysis of regulations and scientific literature, as well as SWOT analysis. The study revealed the main problems of the system: financial and organizational fragmentation, the presence of a "therapeutic gap" when patients reach adulthood, regional disparities in the availability of treatment, as well as regulatory barriers and imperfections in the patient registry system. The need for further centralization of procurement and financing, the creation of a united source for all patients, regardless of age, and the improvement of mechanisms for pharmacoeconomic assessment and registration of orphan drugs is substantiated.

orphan diseases, drug provision, institutional analysis, healthcare system, financing, government regulation, «Circle of Kindness» Foundation, rare diseases, Russian Federation