Direct Cost Analysis About The Three Chelators For The Treatment Of Thalassemia Patients With Chronic Iron Overload: An Italian Perspective From The MIOT Network
Authors: Pepe Alessia , Meloni Antonella , Rossi Giuseppe , Carollo Antonella , Santamaria Vincenzo , Scaccetti Augusto , Roccamo Gaetano , Maggio Aurelio , Lombardi Massimo 
Published: October 2019

Abstract

Introduction
In thalassemia major (TM) three iron chelators in monotherapy are available to treat chronic iron overload due to blood transfusions: subcutaneous desferrioxamine (DFO) introduced in the 1960s, oral deferiprone introduced in 1999 and oral deferasirox (DFX) licensed in 2006. Nowadays pharmacoeconomics analysis are frequently required by the health authorities due to the actual economic crisis. The aim should be to ensure to the whole community the sustainability for health care of proved quality. The objective of this study was to determine the costs of the three chelators in monotherapy in a cohort of 193 TM patients followed prospectively for 18 ± 3 months.

Methods
Within the MIOT (Myocardial Iron Overload in Thalassemia) network, we evaluated prospectively 193 TM patients who had been received one chelator alone between the 2 Magnetic Resonance scans and we calculated the direct costs (drug, administration and monitoring) for each patient treated with DFX, DFO and DFP. We used the cost values for the year 2007. For the drugs we considered the cost ex-factory. For the oral chelators the administration cost was considered null. For the DFO we calculated the costs for the administration (pump, infusion set, syringes and gauzes) using the tariffs applied in Veneto Region, Italy. For the monitoring costs we considered the exams suggested in the technical sheet for each drug; we considered the tariffs by the Veneto Region, Italy. In Italy Veneto Region was proved to be one of the most upright region in the health costs management. In the analysis we considered the drug cost for the standard dosage reported in the technical sheet: 40 mg/kg/d for DFO, 75 mg/kg/d for DFP and 30 mg/kg/d for DFX. Based on the mean weight of the patients we referred the drug cost to a patient of 60 Kg.

Results
In the clinical practice the dose of DFX was 26±7 mg/kg/d, DFP was 73±13 mg/kg/d and DFO was 41±6 mg/kg for 5.5 d/wk. Excellent/good levels of compliance were similar in the 3 groups (DFX 99%, DFP 95%; DFO 96%, P=0.6). The cost/mg was € 0.006 for Generic DFO, € 0.003 for Ferriprox® (DFP) and € 0.047 for Exjade® (DFX). For 18 months of treatment the total costs for DFO were € 10.465,8 (administration and monitoring costs € 3.965,1 + drug cost 6.500,7), for DFP were € 8.292,9 (administration and monitoring costs € 460,8 + drug cost 7.832,2), for DFX were € 46.461,2 (administration and monitoring costs € 211,14 + drug cost 46.249,8). The details about the administration and monitoring costs for DFO, DFP and DFX are reported in the table.

Conclusion
In this analysis, for managing chronic iron overload the direct costs for oral DFP appeared to be the less expensive. The limit of this study is that a cost-utility analysis taking into account efficacy, adverse events and route of administration was not performed.

Disclosures:
Pepe: ApoPharma inc, Novartis, Chiesi: Speakers Bureau.