Cost-utility analysis of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria
Objective To evaluate the cost-utility of eculizumab versus supportive therapy in the treatment of paroxysmal nocturnal hemoglobinuria(PNH).Methods From the perspective of whole Chinese society,a partitioned survival model was constructed to simulate the total cost,quality-adjusted life years(QALYs)and incremental cost-effectiveness ratio(ICER)of patients over a lifetime(40 years)with a discount rate of 5％.One-way sensitivity analysis and probabilistic sensitivity analysis were used to verify the robustness of the model.Results The base-case results showed that compared with supportive therapy,patients who used eculizumab could obtaine 8.50 more QALYs and an incremental cost of 639 784 CNY,the ICER was 75 297 CNY/QALY,which was lower than the GDP per capita in 2023.The one-way sensitivity analysis showed that the annual spontaneous remission rate had the most significant impact on the final result.The probabilistic sensitivity analysis showed that the probability of cost-effective of eculizumab was 99.98％when the willingness-to-pay threshold was 3 times of GDP per capital.Conclusion From the perspective of the Chinese society,eculizumab was a cost-effective treatment regimen compared to supportive therapy in the treatment of PNH patients.
eculizumabparoxysmal nocturnal hemoglobinuriacost-utility analysispartition survival model
NETL
NSTL
万方数据
