Особенности регулирования обращения орфанных лекарственных препаратов в разных регионах мира

Вестник Университета имени О.Е. Кутафина. 2020. № 5(69). С. 26–41.

Абашидзе А. Х., Маличенко В. С.

Orphan medications became a significant share of drug expendi-
tures in every country. The high cost of therapy is caused by the low preva-
lence 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 sys-
tem 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 prac-
tices. 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.

Keywords: лекарственное обеспечение, право на здоровье, орфанные заболевания