Lifetime Cost-Utility Analyses of Deferasirox in Beta-Thalassaemia Patients with Chronic Iron Overload

Karnon, Jonathan; Tolley, Keith; Vieira, J.; Chandiwana, David

doi:10.1007/s40261-012-0008-2

Cited by 17 publications

(22 citation statements)

References 21 publications

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“…A Markov structure was used based on the relevant clinical data (cardiac mortality and morbidity); this structure has been used in previous economic evaluations of iron chelation regimens [29, 30]. An annual cycle was chosen given the availability of annual mortality data; such a cycle has also previously been used in economic evaluations of iron chelation regimens [28].…”

Section: Methods: Economic Modelmentioning

confidence: 99%

“…Previous economic evaluations have focused on DFO and DFX, and have reported that DFX is cost effective compared with DFO, although none have used published adverse event (AE) rates [3, 28, 29]. However, DFP has been included in cost-effectiveness analysis in only one study, from a Thai perspective, which did not consider combination therapy [30].…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

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

Bentley¹,

Gillard²,

Spino

et al. 2013

PharmacoEconomics

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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.

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Section: Methods: Economic Modelmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

Bentley¹,

Gillard²,

Spino

et al. 2013

PharmacoEconomics

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“…This paper expands on the findings in the Karnon 2008 UK analysis to consider cost-utility of DFO and DFX when modeled over the long term and to include additional brief commentary on DFO-DFP combination therapy [32]. This updated analysis found that the higher drug costs for DFX were more than offset by the administration costs of DFO, and that DFX was cost-effective compared with either DFO monotherapy or DFO-DFP combination.…”

Section: Karnon Et Al Clinical Drug Investigation 2012mentioning

confidence: 82%

“…Overall, the authors concluded that DFX dominated DFO (i.e., resulted in lower net costs and higher quality-adjusted life-years) in the base case; this conclusion was robust to the multiple sensitivity analyses that were reported in the paper. The choice of a 1-year time horizon may make the findings less relevant in the context of a lifetime treatment; the investigators addressed this limitation in their follow-up paper [32], which is also discussed in this article. The limitations inherent in short time horizons might be less of a factor for decision makers in markets such as the US, where many private insurers experience high turnover rates.…”

Section: Karnon Et Al Current Medical Research and Opinions 2008mentioning

confidence: 99%

Cost-utility of chelators in transfusion-dependent β-thalassemia major patients: a review of the pharmacoeconomic literature

Lee

Riedemann²,

Tricta³

2014

Expert Review of Pharmacoeconomics & Outcomes Research

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In the inherited hematologic disorder β-thalassemia major, patients receive regular, lifelong blood transfusions, which carry excess iron that the body is unable to eliminate. Chelation therapy (deferoxamine, deferiprone, deferasirox or deferoxamine-deferiprone combination) is required to reduce iron accumulation in target organs and the associated morbidity and mortality. Each chelation regimen has a distinct safety/efficacy profile and particular costs associated with its use. This review aims to provide an overview of published cost-utility analyses of currently used chelation regimens, and to comment on the potential relevance of their findings in the USA market, where deferiprone has recently been introduced.

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“…[18][19][20][21][22] The common results for the US 18 and UK 19,21 healthcare system perspectives on the cost effectiveness of oral DFX vs infusional DFO suggest that DFX is cost effective compared to standard chelation with DFO, while a different conclusion is reached by Luangasanatip et al 20 in Thailand. The two studies that perform a cost analysis of DFP 20,22 both suggest that DFP is the most cost-effective cure for the treatment of iron overload in b-thalassemia patients.…”

Section: Introductionmentioning

confidence: 99%

Iron Chelating Agents for Iron Overload Diseases

Crisponi

Nurchi

Zoroddu

2014

Thalassemia Reports

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Although iron is an essential element for life, an excessive amount may become extremely toxic both for its ability to generate reactive oxygen species, and for the lack in humans of regulatory mechanisms for iron excretion. Chelation therapy has been introduced in clinical practice in the seventies of last century to defend thalassemic patients from the effects of iron overload and, in spite of all its limitations, it has dramatically changed both life expectancy and quality of life of patients. It has to be considered that the drugs in clinical use present some disadvantages too, this makes urgent new more suitable chelating agents. The requirements of an iron chelator have been better and better defined over the years and in this paper they will be discussed in detail. As a final point the most interesting ligands studied in the last years will be presented.

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Lifetime Cost-Utility Analyses of Deferasirox in Beta-Thalassaemia Patients with Chronic Iron Overload

Cited by 17 publications

References 21 publications

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

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

Cost-utility of chelators in transfusion-dependent β-thalassemia major patients: a review of the pharmacoeconomic literature

Iron Chelating Agents for Iron Overload Diseases

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