JUSTIFICATION OF DIRECTIONS FOR IMPROVING THE EFFICIENCY OF PHARMACEUTICAL SUPPLY FOR PATIENTS WITH PHENYLKETONURIA

Abstract

Phenylketonuria (PKU) is a rare metabolic disease, and it is vital for patients to use special medical nutrition products (SMN) to achieve metabolic control. The main problems are the difficulty of adhering to a strict diet for children and adolescents, low affordability of SMH products, and a lack of budgetary funds. A marketing analysis of the assortment and prices of SMN products was conducted based on the study of distributors' price lists and online store offers. We analyzed tender documents and purchase prices for SMN products under the contracts concluded in 2022-2023, which are published in the Prozorro electronic system. The analysis of the medical and technical requirements for the procurement of SMN products allowed us to identify the key criteria indicated in the tender documents. The analysis showed that the procurement primarily involve foreign-made powdered amino acid mixtures (PAMs) prescribed by doctors individually depending on the patient's age and disease characteristics. An online survey of patients with PKU (or their family members caring for them) was conducted using a designed questionnaire with goggle forms (n = 156). This allowed us to determine consumer preferences and demand for SMN products of different groups (pasta, bread, sweets, etc.). The formulation and technology of low-protein SMN products for patients with PKU were scientifically substantiated. The research also elaborated the composition and technology of tablets containing a combination of large neutral amino acids (LNAA). The cost-effectiveness of the production of SMN products and the reasonability of their procurement to meet the needs of patients with PKU within the framework of regional programs have been proven.