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Cost-utility analysis of deferiprone for the treatment of β-thalassaemia patients with chronic iron overload: a UK perspective.

Bentley A, Gillard S, Spino M, Connelly J, Tricta F - Pharmacoeconomics (2013)

Bottom Line: 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.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.Use of DFP in these patients could therefore result in substantial cost savings.

View Article: PubMed Central - PubMed

Affiliation: Abacus International, 6 Talisman Business Centre, Talisman Road, Bicester, Oxfordshire, OX26 6HR, UK. abentley@abacusint.com

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.

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Related in: MedlinePlus

Schematics of the Markov model used for each scenario. No schematic is shown for Scenario 3, as in this scenario the patient is alive without cardiac disease and remains in this state. DFP deferiprone
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Fig1: Schematics of the Markov model used for each scenario. No schematic is shown for Scenario 3, as in this scenario the patient is alive without cardiac disease and remains in this state. DFP deferiprone

Mentions: The cardiac benefit in morbidity and mortality of DFP over DFX is an assumption based on the available evidence (see Sect. 4.2.1); three alternative efficacy-based scenarios were therefore also considered using Markov-type models: the base case excluding a cardiac morbidity benefit but maintaining the mortality benefit over 5 years (Scenario 1) (Fig. 1); the base case excluding a cardiac mortality benefit but maintaining the cardiac morbidity benefit over 5 years (Scenario 2); and a 1-year Markov-type model where all treatments are assumed to have an equal effect on cardiac morbidity and mortality (Scenario 3). The 1-year approach is used in Scenario 3 as this is consistent with other economic evaluations which have assumed equal efficacy for all treatments [3, 28]. As DFO patients may receive treatment via a battery-operated pump instead of a balloon infuser, a scenario was also considered where all DFO and combination therapy patients received treatment via a pump (Scenario 4).Fig. 1


Cost-utility analysis of deferiprone for the treatment of β-thalassaemia patients with chronic iron overload: a UK perspective.

Bentley A, Gillard S, Spino M, Connelly J, Tricta F - Pharmacoeconomics (2013)

Schematics of the Markov model used for each scenario. No schematic is shown for Scenario 3, as in this scenario the patient is alive without cardiac disease and remains in this state. DFP deferiprone
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC3757270&req=5

Fig1: Schematics of the Markov model used for each scenario. No schematic is shown for Scenario 3, as in this scenario the patient is alive without cardiac disease and remains in this state. DFP deferiprone
Mentions: The cardiac benefit in morbidity and mortality of DFP over DFX is an assumption based on the available evidence (see Sect. 4.2.1); three alternative efficacy-based scenarios were therefore also considered using Markov-type models: the base case excluding a cardiac morbidity benefit but maintaining the mortality benefit over 5 years (Scenario 1) (Fig. 1); the base case excluding a cardiac mortality benefit but maintaining the cardiac morbidity benefit over 5 years (Scenario 2); and a 1-year Markov-type model where all treatments are assumed to have an equal effect on cardiac morbidity and mortality (Scenario 3). The 1-year approach is used in Scenario 3 as this is consistent with other economic evaluations which have assumed equal efficacy for all treatments [3, 28]. As DFO patients may receive treatment via a battery-operated pump instead of a balloon infuser, a scenario was also considered where all DFO and combination therapy patients received treatment via a pump (Scenario 4).Fig. 1

Bottom Line: 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.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.Use of DFP in these patients could therefore result in substantial cost savings.

View Article: PubMed Central - PubMed

Affiliation: Abacus International, 6 Talisman Business Centre, Talisman Road, Bicester, Oxfordshire, OX26 6HR, UK. abentley@abacusint.com

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.

Show MeSH
Related in: MedlinePlus