Compounding of Orphan Drugs as an indicator of systemic obstacles in pharmacy practice: the experience of the Russian Federation

The aim. To identify existing obstacles hindering the introduction of extemporaneous orphan drugs into circulation in the Russian Federation, in order to develop recommendations for improving legislative regulation and organizing the activities of pharmacies with the right to manufacture drugs. Materials and methods. The study was performed using a comprehensive approach, including logical, comparative, structural-functional, and conceptual analysis. Information retrieval was conducted in international scientific indexes, search engines (PubMed, Google Scholar), and legal reference systems (ConsultantPlus, GARANT, Kontur.Normativ). Regulatory legal acts of the Russian Federation and foreign countries, modern scientific publications, as well as the practical experience of Russian pharmacies manufacturing drugs for the treatment of orphan diseases were considered. Results. The main obstacles in the development of pharmacy compounding of orphan drugs were identified: low availability of active pharmaceutical ingredients, limitation of the mechanism for establishing the shelf life of extemporaneous dosage forms, prohibition of the manufacture of registered drugs, heterogeneity of pharmacies in terms of equipment and competencies, lack of stable demand, restriction of dispensing compounded drugs for outpatient treatment, and uncertainty of requirements for the therapeutic effectiveness of extemporaneusly compounded drugs. Based on the analysis, proposals were formulated to improve the regulation of mechanisms for admitting substances to the market, using flexible approaches to establishing shelf life, differentiating requirements for pharmacies depending on their capabilities, and legally establishing the rights to manufacture certain categories of drugs, simplifying the dispensing of extemporaneous dosage forms, and involving federal and regional institutions in the formation of stable demand for pharmacy drugs. Conclusion . Overcoming the identified obstacles is of strategic importance not only for the pharmacotherapy of orphan diseases, but also for the development of pharmacy compounding in general. The implementation of the proposed solutions will create a sustainable system capable of providing patients with vital medicines regardless of market conditions and the political situation. Global practice confirms that key competition in the field of orphan drugs unfolds precisely at the level of regulatory systems, and in this perspective, the development of manufacturing pharmacies can become a tool for the Russian Federation to protect the interests of patients, increase access to therapy, and strengthen drug sovereignty.