title: Cost-utility analysis of deferiprone for the treatment of β-thalassaemia patients with chronic iron overload: a UK perspective.
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.
  
  
  OBJECTIVE
  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.
authors.affiliation: Abacus International, 6 Talisman Business Centre, Talisman Road, Bicester, Oxfordshire, OX26 6HR, UK. abentley@abacusint.com
authors.forename: Anthony
authors.initials: A
authors.lastname: Bentley
authors.forename: Samantha
authors.initials: S
authors.lastname: Gillard
authors.forename: Michael
authors.initials: M
authors.lastname: Spino
authors.forename: John
authors.initials: J
authors.lastname: Connelly
authors.forename: Fernando
authors.initials: F
authors.lastname: Tricta
mesh_terms: D003362:Cost-Benefit Analysis / Q000191:economics; D000077543:Deferiprone; D016527:Drug Costs; D017048:Health Care Costs; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000191:economics* / Q000627:therapeutic use; D019190:Iron Overload / Q000150:complications / Q000188:drug therapy* / Q000191:economics*; D018803:Models, Economic; D011728:Pyridones / Q000009:adverse effects / Q000191:economics* / Q000627:therapeutic use*; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D006113:United Kingdom; D017086:beta-Thalassemia / Q000150:complications* / Q000188:drug therapy / Q000191:economics*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone