Pharmacoeconomic analysis of the use of the kuvan drug product in phenylketonuric patients

Abstract

A pharmacoeconomic study of the treatment of phenylketonuria (PHU) treatment using Kuvan drug product combined with the diet or dietary therapy alone has been conducted. It has been established that the therapy is dominant in the first case when compared to the second case because it shows better therapeutic effectiveness and is characterized by a lower costeffectiveness ratio. It has also been determined that the addition of Kuvan to the dietary therapy requires additional costs of 11,569,761 RUB per one patient over 16 years. Despite the increase in the costs linked to the use of the new and only drug for the treatment of PKU, the overall effect on the budget will be decreased due to the low prevalence of the disease. Keywords: pharmacoeconomic analysis, effectiveness, costs, costeffectiveness analysis, budget impact analysis, phenylketonuria, phenylalanine, Kuvan, sapropterin, dietary therapy.Introduction Phenylketonuria (PKU) is a challenging issue for the public healthcare system in the Russian Federation (RF) and other countries worldwide.PKU is a genetic disease that is inherited in an autosomal recessive fashion and results in the disturbance of the amino acid metabolism. Here, up to 1% of the cases of PKU is represented by the atypical forms associated with the mutations in other genes responsible for the coding for enzymes providing the synthesis of the cofactor of phenylalanine hydroxylase tetrahydrobioptherin (BH4). The clinical presentation of the disease develops in 2-3 weeks after the birth already and the irreversible changes in the central nervous system (CNS) develop in the child by the age of six month. Progressive intellectual disability, epilepsy syndrome, and other psychoneurological disorders are characteristic for the PKU patients [14].The occurrence of the methods for the drug treatment of PKU is of particular interest because dietary therapy is currently the main method of medical treatment. In 2007, the Food and Drug Administration of the USA (FDA) approved the Kuvan® (sapropterin) drug product (DP) that is the first medication capable of providing the body with BH4 in the PKU patients, thus making it possible to reduce the incapacitation and mortality and improve the patients' quality of life.This DP was registered within the territory of the RF on July 15, 2014 [9]. The introduction of Kuvan into practice will lead to the growth in the costs and burden on the budget of the public healthcare system. Therefore, the need for carrying out the pharmacoeconomic analysis of the use of the dietary therapy alone in comparison with the use of Kuvan combined with the dietary therapy in PKU patients emerges. Research procedureThe pharmacoeconomic assessment of the treatment of PKU patients with the Kuvan innovative drug product combined with the dietary therapy in comparison with the dietary therapy alone was performed as part of this study. These treatment regimens were used in children from 4 years to their adulthood with the initial weight of 14 kg. It was taken into account in the ...