COST-UTILITY ANALYSIS OF DEFERASIROX FOR THE TREATMENT OF IRON OVERLOAD DUE TO FREQUENT BLOOD TRANSFUSIONS IN THE CHILDREN AND ADOLESCENTS

Author(s)

Walczak J*1;Sołtys E1;Obrzut G1, Laczewski T2 1Arcana Institute, Cracow, Poland, 2Novartis Poland Sp. z o.o., Warsaw, Poland

OBJECTIVES: To estimate the cost-utility of deferasirox (DSX) treatment of iron overload due to frequent blood transfusions in children and adolescents in Polish conditions. METHODS: Clinical effectiveness data were taken from previously performed systematic review of deferasirox for the treatment of pediatric patients (age ≤ 18 years) with iron overload due to frequent blood transfusions. The way of administration of chelation therapy (parenteral versus oral) significantly affects patients’ functionality and quality of life. Therefore it was decided to perform a cost-utility analysis. Analogous to other economic studies, it was assumed that patient survival was the same for both compared interventions: deferasirox and deferoxamine (DFO). In the analysis a simple decision model was developed. The calculations were performed using Monte Carlo microsimulation technique (100,000 trials). Only direct medical costs were included in the analysis: costs of drugs and their administration, costs of monitoring and costs of blood transfusions. The time horizon of the analysis was one-year period. Two perspectives were considered: a public payer (National Health Fund, NHF) and the patient and NHF perspective. The measure of effects was QALY (quality adjusted life years). RESULTS: The results showed that DSX compared to current standard treatment of iron overload due to frequent blood transfusions (DFO) in the children and adolescents is more costly however also more effective therapy from both considered perspectives. The ICUR (incremental cost-utility ratio) of replacing DFO by DSX was 26,180 PN/QALY from NHF perspective. The results from both patient and NHF perspective was similar. CONCLUSIONS: With reference to the acceptability threshold in Poland the oral chelation therapy in the population of children and adolescents with iron overload due to frequent blood transfusions with deferasirox is cost effective intervention when compared with deferoxamine.

Conference/Value in Health Info

2013-11, ISPOR Europe 2013, The Convention Centre Dublin

Value in Health, Vol. 16, No. 7 (November 2013)

Code

PSY44

Topic

Economic Evaluation

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis

Disease

Systemic Disorders/Conditions

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