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: K Tolley, N Oliver, E Miranda, K Migliaccio-Walle, D Bozkaya, Q Li
• Journal: Journal of medical economics (J Med Econ) Vol. 13 Issue 3 Pg. 559-70 ( 2010) ISSN: 1941-837X [Electronic] England
• PMID: 20812793 (Publication Type: Comparative Study, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Iron Chelating Agents
• Siderophores
• Triazoles
• Deferoxamine
• Deferasirox

Topics

• Benzoates (economics, therapeutic use)
• Cost-Benefit Analysis
• Deferasirox
• Deferoxamine (economics, therapeutic use)
• Erythrocyte Transfusion (adverse effects, economics)
• Humans
• Insurance Claim Review
• Iron Chelating Agents (economics, therapeutic use)
• Iron Overload (blood, drug therapy, economics, etiology)
• Longitudinal Studies
• Myelodysplastic Syndromes (blood, complications, economics, therapy)
• Quality-Adjusted Life Years
• Siderophores (economics, therapeutic use)
• State Medicine (economics)
• Survival Analysis
• Triazoles (economics, therapeutic use)
• United Kingdom