Cost-utility of chelators in transfusion-dependent β-thalassemia major patients: a review of the pharmacoeconomic literature

Abstract

In the inherited hematologic disorder β-thalassemia major, patients receive regular, lifelong blood transfusions, which carry excess iron that the body is unable to eliminate. Chelation therapy (deferoxamine, deferiprone, deferasirox or deferoxamine–deferiprone combination) is required to reduce iron accumulation in target organs and the associated morbidity and mortality. Each chelation regimen has a distinct safety/efficacy profile and particular costs associated with its use. This review aims to provide an overview of published cost-utility analyses of currently used chelation regimens, and to comment on the potential relevance of their findings in the USA market, where deferiprone has recently been introduced.

Keywords::

• cost-utility
• deferasirox
• deferiprone
• deferoxamine
• iron chelation
• thalassemia major

Acknowledgements

The authors would like to thank J Ashkenas at SCRIPT for editorial support, and M Spino at ApoPharma for reviewing and commenting on the manuscript.

Financial & competing interests disclosure

Financial support for creation of this manuscript and pricing data for chelators in the USA market were provided by ApoPharma. TA Lee was contracted by SCRIPT/ApoPharma as a consultant on this manuscript. F Tricta is an employee of ApoPharma. S von Riedemann is an employee of SCRIPT, a medical communications agency contracted by ApoPharma. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• β-Thalassemia major is a severe chronic anemia treated with regular red blood cell transfusions; chelation therapy is required to reduce morbidity and mortality associated with transfusional iron overload.

• Three chelators are currently available for use as monotherapies: parenteral deferoxamine (DFO) and oral deferasirox (DFX) and deferiprone (DFP); combination therapy with DFO and DFP is also widely used.

• All three monotherapies and DFO–DFP combination have demonstrated efficacy at reducing body iron load, as measured by serum ferritin and liver iron concentration.

• Regimens containing DFP (monotherapy or combination with DFO) appear to have a preferential effect for removing cardiac iron (CMR T2*), improving heart function (left ventricular ejection fraction) and reducing cardiac-related mortality.

• In spite of its higher drug cost, DFX was found to be cost-effective compared with DFO in all pharmacoeconomic analyses of the two agents as monotherapies, due largely to the high costs and disutility associated with parenteral DFO administration, and also to a lack of significant efficacy differences between DFO and DFX.

• In cost-utility analyses that evaluated all three monotherapies and assumed equal efficacy on blood, liver and cardiac iron, DFP was found to be cost-effective compared with DFX due to its lower drug cost; this finding was even stronger in analyses that incorporated the cardiac effects of DFP. DFP monotherapy was also cost-effective compared with DFO–DFP combination, due largely to the costs associated with DFO administration.

• The pharmacoeconomic analyses discussed here were conducted in markets worldwide, where DFX is significantly more expensive than DFP. In the USA, the prices of DFX and the newly introduced DFP are similar; the analyses from other markets may or may not hold true in the USA and therefore further analyses should be performed to confirm the cost-utility of DFP in the USA.