Analysis of the availability of health technologies for the treatment of phenylketonuria in Ukraine and the world

Abstract

Phenylketonuria (PKU) is a rare (orphan) disease characterized by a severe, chronic, progressive course. Patients' quality of life depends on timely detection (screening) and proper treatment - the use of special medical food products, and therefore, their availability and affordability. The goal (objective) of the study is to analyze the available health technologies for the treatment of PKU in Ukraine and the world. Materials and methods. Research objects: scientific publications, guidelines, statistical data, marketing information. Methods: analysis, comparison, generalization, system analysis. Results of the research. Guidelines and protocols for the treatment of patients with PKU/HFA define a diet as a key component, taking into account the age and features of the course of the disease. The analysis of providing patients with PKU with special medical food products in different countries shows significant differences in the approaches to their assignment and reimbursement. The quantity, availability, range and composition of these products vary significantly. In the UK, they are included in the national formulary, are available on prescription and are subject to reimbursement. In Ukraine, local authorities provide PKU patients with special medical food products by procurement through the ProZorro electronic system in accordance with the calculated need. In the domestic market, there is a wide range of specialized products for patients with PKU/HFA, mainly of imported production, which have a high cost and, accordingly, low accessibility for the population. Conclusions. The introduction of reference centers and extended neonatal screening, the creation of a register of such patients, the development of domestic production of products for PKU/HPA, and the creation of effective procurement and reimbursement mechanisms will contribute to increasing the effectiveness of treatment, the quality and life expectancy of patients.