Different Scenarios of Providing Drugs for Patients with Primary Immunodeficiency in Russia: Analysis of Costs and Effectiveness

Abstract

The only source of financing for immunoglobulin replacement therapy needed by patients with primary immunodeficiencies (PID) in Russian Federation is the compulsory health insurance system: payment is based on diagnosis-related groups (DRG), including the DRG for immunoglobulin treatment at day care and inpatient setting. PID are not included either into regional program for rare diseases, or into federal program of 12 high-cost diseases. This leads to limited coverage of patients with effective drug treatment. Furthermore, currently in Russian Federation there is no neonatal screening for PID, which could facilitate early detection of the disease and timely start of treatment. Goal of the study: to conduct the analysis of cost and effectiveness of different scenarios of drug provision for the patients with PID, which differ in coverage of patients with therapy and by presence/absence of neonatal screening. Materials and methods. The model compared three scenarios of drug provision for patients with PID: a) current practice, when in accordance with available data not all patients get necessary immunoglobulin replacement therapy, b) new scenario 1 – financing of drugs for all registered patients with PID when large-scale neonatal screening for PID is absent; c) new scenario 2 – financing of drugs for all registered patients with PID when large-scale neonatal screening is implemented. In each scenario direct medical costs and number of prevented deaths was calculated. Comparison of scenarios was planned to be made by calculation of additional costs per prevented death due to PID, or by assessment of difference of costs and effectiveness (number of prevented deaths) with identification of a dominant scenario. Results. Both new scenarios including financing of drugs for all registered patients with PID lead to the prevention of significant number of deaths and also to cost savings if compared with current practice. Total costs for current, 1st new and 2nd new scenario for 15 year was^ 2 285,54, 616,49 and 1 318,61 billions of rubles respectively. Also when comparing 1st and 2nd new scenarios with current practice we expect prevention of 243 and 2076 deaths respectively. Conclusion. Most effective approach is the introduction of large-scale neonatal screening for PID combined with the new source of financing: this scenario saves less money, but helps to prevent highest number of deaths compared with current practice. This scenario can prevent deaths of patients who would not have been diagnosed with PID in the absence of screening, and also helps to save healthcare system budget by centralization of government procurement of drugs.