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PharmacoEconomics
Published in: PharmacoEconomics 9/2013

Open Access 01-09-2013 | Original Research Article

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

Authors: Anthony Bentley, Samantha Gillard, Michael Spino, John Connelly, Fernando Tricta

Published in: PharmacoEconomics | Issue 9/2013

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Abstract

Background

Patients 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.

Objectives

This 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.

Methods

A 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.

Results

DFP 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.

Conclusions

In 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.
Appendix
Available only for authorised users
Footnotes
1
The registration trial for DFX failed to meet the primary endpoint of maintenance or reduction of LIC; however, this was attributed to the fact that patients received proportionally lower doses of DFX relative to DFO. The authors conclude that there was a clear demonstration of iron excretion related to the dose administered [31].
 
2
Data were adjusted for use in the model by converting the 5-year probability of the event into a 5-year rate, which was then adjusted to an annual rate and converted back into an annual probability. This is achieved using the following formula: 1 − EXP(−(−LN(1 − (Probability of event over time period considered, X))/(Time period considered, X/Time period required, Y))).
 
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Metadata
Title
Cost–Utility Analysis of Deferiprone for the Treatment of β-Thalassaemia Patients with Chronic Iron Overload: A UK Perspective
Authors
Anthony Bentley
Samantha Gillard
Michael Spino
John Connelly
Fernando Tricta
Publication date
01-09-2013
Publisher
Springer International Publishing
Published in
PharmacoEconomics / Issue 9/2013
Print ISSN: 1170-7690
Electronic ISSN: 1179-2027
DOI
https://doi.org/10.1007/s40273-013-0076-z

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