Cost-utility analysis of deferasirox compared to standard therapy with desferrioxamine for patients requiring iron chelation therapy in the United Kingdom

ABSTRACT

Objective: The primary objective of the study was to evaluate the cost-utility of deferasirox (Exjade) compared to standard therapy using desferrioxamine (Desferal) for the control of iron overload in patients receiving frequent blood transfusions. The perspective adopted was that of the National Health Service in the UK.

Methods: Phase II/III clinical trials have shown deferasirox in the recommended doses of 20–30 mg/kg per day to have similar efficacy to desferrioxamine at equivalent doses in the control of chronic iron overload. The main difference between them is in the mode of administration. Desferrioxamine is administered parenterally as a slow subcutaneous infusion typically infused 8–12 hours a day for 5–7 days a week. In comparison, deferasirox provides 24 hour chelation via a once daily oral tablet dispersed in water or juice. An excel based economic model was developed to evaluate the annual healthcare costs and quality of life, or utility, benefits associated with differences in mode of administration, using β-thalassaemia as the reference case. A community utility study using time trade-off methods was performed to determine utility outcomes associated with iron chelation therapy (ICT) mode of administration.

Results: In the reference case (patient mean weight 42 kg), deferasirox “dominated” desferrioxamine, i.e. resulted in lower net costs and higher quality adjusted life years (QALYs). Drug dose and cost is patient weight related. Incremental cost per QALY gained was £7775 for patients with a mean weight of 62 kg.

Conclusions: The cost-utility analysis did not take drug compliance into account. However, Deferasirox is cost-effective compared to standard iron chelation therapy with desferrioxamine, due to the cost and quality of life benefits derived from a simpler and more convenient oral mode of administration.

Key words: :

• β-thalassaemia
• Cost-utility
• Iron chelation therapy
• QALYs
• Time trade-off

Acknowledgements

Declaration of interest: The School of Health and Related Research, University of Sheffield and Mapi Values UK received financial sponsorship from Novartis Pharmaceuticals UK Ltd for the conduct of this study.

We wish to acknowledge Claire Gait, previously of Mapi Values UK, who was project manager for the UK utility study. We would also like to thank two anonymous reviewers for their comments.

Notes

* Desferal and Exjade are registered trade names of Novartis AG, CH-4002, Basel, Switzerland.