Document Detail

Lifetime cost-utility analyses of deferasirox in beta-thalassaemia patients with chronic iron overload: a UK perspective.
MedLine Citation:
PMID:  23026844     Owner:  NLM     Status:  MEDLINE    
BACKGROUND AND OBJECTIVES: Regular blood transfusions for beta-thalassaemia patients lead to the accumulation of iron deposits in the body. In order to remove such deposits, iron chelation therapy is required. Subcutaneously administered deferoxamine has been the gold standard chelation therapy for over 40 years. Deferasirox is a newer chelation therapy that is taken orally once daily. The objective of this study was to estimate the long-term costs and quality-adjusted life-years (QALYs) associated with deferoxamine and deferasirox in a cohort of transfusion-dependent beta-thalassaemia patients from a UK health service perspective.
METHODS: A 50-year annual cycle state transition model comprised three core health states: alive without cardiac complications, alive with cardiac complications, and dead, as well as representing other chronic complications of iron overload: diabetes, hypogonadism, hypoparathyroidism and hypothyroidism. The model was calibrated to identify sets of convergent input parameter values that predicted observed overall survival by mean lifetime compliance with chelation therapy. A pivotal non-inferiority trial informed the main estimates of the effectiveness of deferasirox, which were applied to the calibrated model. Using cost values for the year 2011, costs and utilities were summed over patients' lifetimes to estimate lifetime costs and QALY gains.
RESULTS: Mean lifetime treatment costs for patients receiving deferoxamine were £70,000 higher than deferasirox. Drug acquisition costs were £100,000 higher for deferasirox, but administration costs associated with deferoxamine were £170,000 higher. Higher compliance associated with oral deferasirox administration led to fewer complications. Combined with the quality-of-life effects of an oral mode of administration, an average gain of 4.85 QALYs for deferasirox was estimated. In the base case, deferasirox dominates deferoxamine, i.e., costs less and patients gain more QALYs. The key parameter is the proportion of deferoxamine patients using balloon infusers. Sensitivity analyses showed that even when the proportion of patients using balloon infusers is decreased from 79 to 25 %, the incremental cost per QALY gained remains well under £20,000.
CONCLUSION: Higher drug acquisition costs for deferasirox are offset by the avoidance of infusion-related equipment costs. Combined with health benefits derived from an oral mode of administration and improved compliance, deferasirox has a high probability of being a cost-effective intervention compared with deferoxamine.
Jonathan Karnon; Keith Tolley; Joao Vieira; David Chandiwana
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Publication Detail:
Type:  Comparative Study; Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Clinical drug investigation     Volume:  32     ISSN:  1179-1918     ISO Abbreviation:  Clin Drug Investig     Publication Date:  2012 Dec 
Date Detail:
Created Date:  2012-11-27     Completed Date:  2013-05-01     Revised Date:  2013-06-03    
Medline Journal Info:
Nlm Unique ID:  9504817     Medline TA:  Clin Drug Investig     Country:  New Zealand    
Other Details:
Languages:  eng     Pagination:  805-15     Citation Subset:  IM    
University of Adelaide, Adelaide, Australia.
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MeSH Terms
Administration, Oral
Benzoates / administration & dosage,  economics,  therapeutic use*
Blood Transfusion / economics,  methods
Cohort Studies
Cost-Benefit Analysis
Deferoxamine / administration & dosage,  economics,  therapeutic use*
Drug Costs
Great Britain
Injections, Subcutaneous
Iron Chelating Agents / administration & dosage,  economics,  therapeutic use*
Iron Overload / complications,  drug therapy,  economics
Medication Adherence
Models, Economic
Quality of Life
Quality-Adjusted Life Years
Survival Rate
Triazoles / administration & dosage,  economics,  therapeutic use*
beta-Thalassemia / complications,  drug therapy*,  economics
Reg. No./Substance:
0/Benzoates; 0/Iron Chelating Agents; 0/Triazoles; 70-51-9/Deferoxamine; V8G4MOF2V9/deferasirox

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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