Abstract
<jats:p>The aim of this review is to analyze current approaches to the organization of healthcare for patients with rare diseases in the USA, European countries, Japan, China, and Russia, including legal and regulatory frameworks, criteria for defining orphan diseases and drugs, financing models, early access programs, and patient registry practices. The review demonstrates that all countries strive to ensure access to innovative treatments, accelerate diagnosis, and support patients with high-cost conditions. Common trends include the development of specialized registries, centralized financing, measures to stimulate research and development of orphan drugs, and the involvement of patient and advocacy organizations in policy-making. Analysis of international experience highlights directions for improving the Russian system of orphan disease care, including expanding registries, enhancing equitable drug provision, integrating innovative technologies, and strengthening coordination between federal and regional mechanisms.</jats:p>