High costs associated with the management of orphan diseases determine the need in health-economic studies of the orphan medicines. Authors conducted systematic literature review and assessed the quality of health-economic studies of orphan drugs in Russia in 2015-2021 years. In total, 14 healtheconomic studies were evaluated. The following methodological issues of health-economic studies were identified: the bias in effectiveness comparison, the biases in the methodology of cost calculation, the choice of modeling horizon, the bias in patient population size calculation, restricted sensitivity analysis, the comparison of incremental cost-effectiveness ratio with cost-effectiveness threshold. Authors conclude that there are a series of methodological biases in the healtheconomic studies, which prevent correct assessment of the burden of orphan diseases in Russia and it’s influence on the healthcare system.
Orphan diseases, clinical and economic research, high-cost nosology program