ORPHAN DRUG REGULATION IN VARIOUS REGIONS OF THE WORLD

Abstract

Orphan medications became a significant share of drug expenditures in every country. The high cost of therapy is caused by the low prevalence of diseases and the need to compensate the investment of producers in long-term development and registration procedures. The article highlights the main stages of the formation of legislation regulating the circulation of orphan medicines in the United States, covering state guarantees for the development of «orphan medicines», and imperfections of the existing system which influence the cost and availability of medicines. On the example of the EU, the author examines the unified approaches of EU countries to determining the orphan status of medicines, which is especially relevant in the context of convergence and harmonization of national legal systems regulating the circulation of medicines.

The purpose of this article is to analyze various approaches to regulating orphan medicinal products in order to determine the most effective practices. The author identifies the main barriers in the provision of medicines to patients with orphan diseases in the Russian Federation, analyzes the main directions of the development of the drug supply system and outlines possible directions for expanding access to necessary treatment methods.