Assessment of the Pharmacoeconomical Effectiveness of Second Stage Personalized Rehabilitation in Children with Primary Immunodeficiencies: the Experience of One Center

Primary immunodeficiencies as a heterogeneous group of orphan diseases are of particular interest for the pharmaco-economical assessment of applied medical interventions due to diversity of their clinical presentation and complexity of standardizing approaches. In connection with increasing detectability due to neonatal screening and the potential for its expansion, it is increasingly important not only to treat, but to rehabilitate such patients as well in order to maintain a high quality of their lives, and therefore social and economic activity. The purpose of the research is a pharmaco-economical assessment of personalized bidirectional rehabilitation technology at the second stage in children with primary immunodeficiencies with the study of clinical and economic features of the method before including it in existing sources of financing for medical care. Materials and methods. A sample of patients from published data on evaluating the clinical component of the method was used based on determining the quality of life indicator. In this paper, data from calculations of direct expenses for providing care to one patient are used to calculate the costs as part of implementing the 2019-55-7 clinical approbation protocol. As an integral criterion of clinical and economic efficiency, QALY was calculated for one year of life, a pharmacoeconomical analysis of discounting at rates of 3 and 5%, a cost-effectiveness analysis, an incremental cost-effectiveness analysis, and a comparison of the results with the threshold of willingness to pay for the end of 2023 were carried out. Research results. The QALY for one year of life was 0.75. According to discounting results, since 2019, the amount of expenses has decreased from 603,071 to 452,303 rubles at the rate of 5% and up to 512 604 rubles at the rate of 3%. According to the results of the cost-effectiveness analysis, the cost of one unit of efficiency (QALY) in 2019 was 804,095 rubles, in 2024 – 683,480 rubles at the rate of 3% and 603,071 rubles at the rate of 5%. According to the results of the incremental cost-effectiveness analysis, the cost of one additional unit of efficiency (QALY) cost 6,884,372 rubles in 2019, in 2024 it is 5,851,716 rubles for the rate of 3% and 5,136,279 rubles for a 5% rate. Conclusions. When compared with the threshold of willingness to pay, the method is borderline acceptable. A significant factor contributing the most to the total cost of expenses is the cost of purchasing inventory. Additional criteria of clinical efficacy are required to recognize the method as pharmaco-economically effective. The transfer of catamnesis to the pre-rehabilitation stage, exclusion of provision with medicines at the second stage of rehabilitation at the expense of the medical organization's funds serve as ways to reduce the cost of the method.