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Cost Effectiveness of Once-Daily Oral Chelation Therapy with Deferasirox versus Infusional Deferoxamine in Transfusion-Dependent Thalassaemia Patients

US Healthcare System Perspective

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Abstract

Background

Deferasirox is a recently approved once-daily oral iron chelator that has been shown to reduce liver iron concentrations and serum ferritin levels to a similar extent as infusional deferoxamine.

Objective

To determine the cost effectiveness of deferasirox versus deferoxamine in patients with ß-thalassaemia major from a US healthcare system perspective.

Methods

A Markov model was used to estimate the total additional lifetime costs and QALYs gained with deferasirox versus deferoxamine in patients with ß-thalassaemia major and chronic iron overload from blood transfusions. Patients were assumed to be 3 years of age at initiation of chelation therapy and to receive prescribed dosages of deferasirox and deferoxamine that have been shown to be similarly effective in such patients. Compliance with chelation therapy and probabilities of iron overload-related cardiac disease and death by degree of compliance were estimated using data from published studies. Costs ($US, year 2006 values) of deferoxamine administration and iron overload-related cardiac disease were based on analyses of health insurance claims of transfusion-dependent thalassaemia patients. Utilities were based on a study of patient preferences for oral versus infusional chelation therapy, as well as published literature. Probabilistic and deterministic sensitivity analyses were employed to examine the robustness of the results to key assumptions.

Results

Deferasirox resulted in a gain of 4.5 QALYs per patient at an additional expected lifetime cost of $US126 018 per patient; the cost per QALY gained was $US28 255. The cost effectiveness of deferasirox versus deferoxamine was sensitive to the estimated costs of deferoxamine administration and the quality-of-life benefit associated with oral versus infusional therapy. Cost effectiveness was also relatively sensitive to the equivalent daily dose of deferasirox, and the unit costs of deferasirox and deferoxamine, and was more favourable in younger patients.

Conclusion

Results of this analysis of the cost effectiveness of oral deferasirox versus infusional deferoxamine suggest that deferasirox is a cost effective iron chelator from a US healthcare perspective.

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Notes

  1. The use of trade names is for product identification purposes only and does not imply endorsement.

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Acknowledgements

Funding for this study was provided by Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA. TE Delea and O Sofrygin are employees of Policy Analysis Inc. (PAI), an independent contract research organisation, which has received research support and consulting fees from Novartis. TE Delea has also received speaker honorarium from Novartis. SK Thomas and J-F Baladi are employees of Novartis and own stock and/or stock options in Novartis. PD Phatak has received consulting fees from PAI and honoraria from Novartis, and has served on advisory boards for Novartis. TD Coates has received consulting fees, research funding and honoraria from Novartis, has participated on advisory boards for Novartis, and is a member of the University Childrens Medical Group Speakers Bureau.

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Authors and Affiliations

  1. Policy Analysis Inc. (PAI), Four Davis Court, Brookline, Massachusetts, 02445, USA

    Thomas E. Delea & Oleg Sofrygin

  2. Novartis Pharmaceuticals Corp., East Hanover, New Jersey, USA

    Simu K. Thomas & Jean-Francois Baladi

  3. Hematology/Oncology Unit Rochester General Hospital, Rochester, New York, USA

    Pradyumna D. Phatak

  4. Center for Cancer and Blood Diseases, Childrens Hospital of Los Angeles, Los Angeles, California, USA

    Thomas D. Coates

Authors
  1. Thomas E. Delea
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  2. Oleg Sofrygin
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  3. Simu K. Thomas
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  4. Jean-Francois Baladi
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  5. Pradyumna D. Phatak
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  6. Thomas D. Coates
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Correspondence to Thomas E. Delea.

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Delea, T.E., Sofrygin, O., Thomas, S.K. et al. Cost Effectiveness of Once-Daily Oral Chelation Therapy with Deferasirox versus Infusional Deferoxamine in Transfusion-Dependent Thalassaemia Patients. Pharmacoeconomics 25, 329–342 (2007). https://doi.org/10.2165/00019053-200725040-00005

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