title: Cost effectiveness of deferasirox compared to desferrioxamine in the treatment of iron overload in lower-risk, transfusion-dependent myelodysplastic syndrome patients.
abstract: OBJECTIVE
  The study evaluated the cost effectiveness of deferasirox (Exjade * ) compared to non-proprietary desferrioxamine (DFO) for the control of transfusional iron overload in lower risk myelodysplastic syndromes (MDS) patients. A UK National Health Service perspective was adopted.
  
  
  METHODS
  Recent clinical evidence has demonstrated the efficacy and safety of deferasirox in transfusion-dependent MDS patients with elevated serum ferritin levels. An economic model was used to extrapolate the clinical benefits of iron chelation therapy (ICT) in a cohort of lower risk MDS patients. Costs for drug acquisition, drug administration and monitoring, and quality of life (utility) outcomes associated with mode of drug administration were derived from a variety of sources. The incremental cost per QALY gained for deferasirox was estimated. Costs and outcomes were discounted at 3.5% in line with UK standards.
  
  
  RESULTS
  The base-case cost effectiveness of deferasirox versus DFO was estimated to be £20,822 per QALY gained, the key driver being the additional quality of life benefits associated with a simpler mode of administration for deferasirox. A mean survival benefit for both forms of ICT of 4.5 years was estimated. The results were sensitive to drug dose, days of DFO administration, and patient weight.
  
  
  CONCLUSIONS
  In the UK, a cost per QALY below £20,000-30,000 is considered cost effective. Hence, the results from this economic analysis suggest deferasirox is cost effective in lower risk, transfusion-dependent, MDS patients. Limitations with the analysis include a lack of comparative randomised controlled trial evidence, in particular to differentiate survival and clinical outcomes for deferasirox and DFO.
authors.affiliation: Tolley Health Economics Ltd., Buxton, Derbyshire, UK. keith@tolleyhealtheconomics.com
authors.forename: K
authors.initials: K
authors.lastname: Tolley
authors.forename: N
authors.initials: N
authors.lastname: Oliver
authors.forename: E
authors.initials: E
authors.lastname: Miranda
authors.forename: K
authors.initials: K
authors.lastname: Migliaccio-Walle
authors.forename: D
authors.initials: D
authors.lastname: Bozkaya
authors.forename: Q
authors.initials: Q
authors.lastname: Li
mesh_terms: D001565:Benzoates / Q000191:economics* / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D000077588:Deferasirox; D003676:Deferoxamine / Q000191:economics* / Q000627:therapeutic use*; D017707:Erythrocyte Transfusion / Q000009:adverse effects / Q000191:economics; D006801:Humans; D007345:Insurance Claim Review; D007502:Iron Chelating Agents / Q000191:economics / Q000627:therapeutic use; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000191:economics* / Q000209:etiology; D008137:Longitudinal Studies; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000150:complications / Q000191:economics* / Q000628:therapy; D019057:Quality-Adjusted Life Years; D017262:Siderophores / Q000191:economics / Q000627:therapeutic use; D013222:State Medicine / Q000191:economics; D016019:Survival Analysis; D014230:Triazoles / Q000191:economics* / Q000627:therapeutic use*; D006113:United Kingdom
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D017262:Siderophores; D014230:Triazoles; D003676:Deferoxamine; D000077588:Deferasirox