OBJECTIVES

The incidence of Chronic Myeloid Leukemia (CML) in China has increased rapidly. The first-line imatinib (400 mg QD), as the first generation of tyrosine kinase inhibitor, can make CML reach a 10-year overall survival rate of 85-90% and a progression-free-survival rate of 92%. However, some patients caught imatinib-resistance (ImR) or imatinib-intolerance (ImI) in the first-line therapy, who had to begin second-line therapy replacing with nilotinib or high-dose imatinib (600/800 mg QD). Therefore, CML has become a chronic disease, aggravating the burden of both patients and society. This study assessed whether nilotinib or high-dose imatinib for the second-line treatment of ImR/ImI CML Chinese patients is more cost-effective from the perspective of both clinical medical cost and the medical insurance system.

METHODS

This study presented a cost-effectiveness analysis of nilotinib versus high-dose imatinib for ImR/ImI CML patients in China. The study used a model to simulate disease burden based on overall survival rate and a progression-free-survival rate over a 5-yeat time horizon. Results were presented as an incremental cost per quality adjusted life year (QALY) gained. Sensitivity analyses were performed.

RESULTS

In the second-line treatment of CML patients, nilotinib plan was associated with lower costs (RMB 5.97 million) and improved QALYs (170.7), over 5 years, resulting in incremental cost-effectiveness ratio of RMB 154,330.8/QALY gained. Sensitivity analysis showed that the results of this study were reliable and stable. At the used threshold of RMB 139,593/QALY, the new policy was cost-effective.

CONCLUSIONS

Treatment of ImR/ImI CML Chinese patients by nilotinib plan is more cost-effective based on the used threshold of RMB 154,330.8/QALY. The results implying that nilotinib is an absolute advantage as a second-line treatment for ImR/ImI CML patients, and if it could be included in the China’s medical insurance directory, Chinese medical insurance fund will be greatly saved in the payment for CML treatment.