Cost–Utility Analysis of Deferiprone for the Treatment of β-Thalassaemia Patients with Chronic Iron Overload: A UK Perspective

Abstract

BackgroundPatients with β-thalassaemia major experience chronic iron overload due to regular blood transfusions. Chronic iron overload can be treated using iron-chelating therapies such as desferrioxamine (DFO), deferiprone (DFP) and deferasirox (DFX) monotherapy, or DFO–DFP combination therapy.ObjectivesThis study evaluated the relative cost effectiveness of these regimens over a 5-year timeframe from a UK National Health Service (NHS) perspective, including personal and social services.MethodsA Markov model was constructed to evaluate the cost effectiveness of the treatment regimens over 5 years. Based on published randomized controlled trial evidence, it was assumed that all four treatment regimens had a comparable effect on serum ferritin concentration (SFC) and liver iron concentration (LIC), and that DFP was more effective for reducing cardiac morbidity and mortality. Published utility scores for route of administration were used, with subcutaneously administered DFO assumed to incur a greater quality of life (QoL) burden than the oral chelators DFP and DFX. Healthcare resource use, drug costs (2010/2011 costs), and utilities associated with adverse events were also considered, with the effect of varying all parameters assessed in sensitivity analysis. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Assumptions that DFP conferred no cardiac morbidity, mortality, or morbidity and mortality benefit were also explored in scenario analysis.ResultsDFP was the dominant strategy in all scenarios modelled, providing greater QALY gains at a lower cost. Sensitivity analysis showed that DFP dominated all other treatments unless the QoL burden associated with the route of administration was greater for DFP than for DFO, which is unlikely to be the case. DFP had >99 % likelihood of being cost effective against all comparators at a willingness-to-pay threshold of £20,000 per QALY.ConclusionsIn this analysis, DFP appeared to be the most cost-effective treatment available for managing chronic iron overload in β-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-013-0076-z) contains supplementary material, which is available to authorized users.

Not Simply More of the Same

“…2 Heterogeneity has sometimes been handled by combining it with parameter uncertainty in a PSA. [27][28][29] Using the single-loop PSA, one is able to consider many different types of patients without a significant increase in calculation time. However, because of a larger number of parameters being sampled, the amount of uncertainty increases and the shape of the CE plane also changes compared with disregarding heterogeneity.…”

Healthcare costs and outcomes of managing β‐thalassemia major over 50 years in the United Kingdom

“…Unit resource costs (Table S2, available as supporting information in the online version of this paper) were obtained from relevant NHS tariffs at 2013/14 prices. Costs from previous years were uprated to 2013/14 prices using the hospital and community health services index .…”

Economic Evaluation of Severe Anaemia: Review-Based Recommendations and a Conceptual Framework

“…A summary of studies is presented in Table 1. [6][7][8][9][10][11][12][13][14][15][16][17][18][19][20][21][22][23] Diseases Seven studies evaluated therapy for β-thalassaemia, one for AIHA, one for chemotherapy-induced anaemia, one for chronic heart failure-related anaemia, two for SAA, and six for CKD-related anaemia.…”