Background: Patients with transfusional iron overload have relied on treatment with deferoxamine, a standard chelating agent. Deferoxamine is administered by intravenous or subcutaneous infusion over an 8∼12 hour period 5∼7 times per week; however, administration of deferoxamine may lead to poor compliance and reduced quality of life in patients. The use of deferasirox, a once daily oral chelation agent, was recently approved. We conducted an economic evaluation of these two iron-chelating medications in transfusion-dependent patients. Methods: The efficacy of oral deferasirox and infusion deferoxamine was assumed equal based on clinical trials of non-inferiority with the administration of 20mg/kg/day deferasirox versus 40mg/kg/day deferoxamine. Depending on the methods utilized for measuring administration time, travel time and convenience between the use of infusion and oral therapy, either cost analysis or cost-utility analysis was undertaken, respectively. Cost analysis included determination of direct medical costs (drug costs and administration costs), non-medical costs (travel costs), and indirect costs (productivity loss associated medical utilization). For cost utility analysis, the cost per QALYs (quality-adjusted life years) was calculated based on costs subtracting indirect costs (productivity loss) and gains of QALYs between the two agents. Results: Deferasirox therapy resulted in a cost savings per patient of 23,471,777 Korean won based on cost analysis. Based on cost utility analysis, the cost per QALYs gained was −398,576 Korean won (4,527,819 Korean won savings with a gain of 11.5 QALYs per patient). Conclusion: The results of cost analysis and cost utility analysis of the use of oral deferasirox versus infusion deferoxamine showed that deferasirox is a more economical and potentially socially beneficial iron-chelating agent in Korea. (Korean J Hematol 2008;43:89-97.)

Keywords: Deferasirox, Deferoxamine, Iron chelating agents, Cost analysis, Cost utility analysis