The study of the state and problems of pharmaceutical provision of orphan patients with medicines under managed entry agreements
DOI:
https://doi.org/10.24959/nphj.25.187Keywords:
medicines; rare (orphan) diseases; managed entry agreements; public procurement; health technology assessmentAbstract
Aim. To conduct a comprehensive study of the state and problems of providing orphan patients with innovative medicines purchased under managed entry agreements (MEA).
Materials and methods. The analysis, generalization, and systematization of regulatory and legal acts, scientific publications, analytical and reporting data, information from the official websites of authorized bodies (the Ministry of Health of Ukraine, the central procurement organization SE “Medical Procurement of Ukraine,” State Expert Center of the Ministry of Health of Ukraine) and the “Prozorro” electronic procurement system regarding the purchase of orphan drugs under the terms of MEA were carried out.
Results. The analysis of global experience has shown that MEAs (financial or clinical outcome-based) are used to expand access to innovative high-cost medicines both at the state level and for individual patients. According to the results of the studies conducted it has been found that as of September 2025, 23 names of drugs (17 INNs) are purchased in Ukraine under the conditions of MEA; among them, 13 INNs are for the treatment of rare diseases (mucopolysaccharidosis, Fabry disease, Gaucher disease, hemophilia A, primary (congenital) immunodeficiencies, spinal muscular atrophy (SMA)). All these medicines have previously undergone a state health technology assessment (HTA), including a comparative evaluation of their effectiveness, safety, and impact on the budget, and have been recognized as the optimal option for providing therapy for rare diseases. The analysis of “Prozorro” data shows that some medicines were purchased in a decentralized manner prior to the signing of the MEA. Currently, due to changes in the legislation, a transparent multi-level MEA mechanism is being created with the possibility of attracting various sources of funding, which will increase the effectiveness, targeting, and availability of pharmaceutical care.
Conclusions. It has been proven that MEAs (also known as risk-sharing agreements) are a promising direction for improving the system of pharmaceutical provision for orphan patients who require continuous, high-cost treatment. The flexibility of the terms of MEAs makes it possible to expand the patients’ access to medicines, use rationally the limited financial resources, and collect real data on the clinical effectiveness of medicines. Key problems in the procurement of medicines under MEAs, such as insufficient transparency of the terms of agreements, lack of a patient registry, and a limited analytical base for evaluating clinical outcomes, have been identified.
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