Cost-effectiveness of defibrotide for treatment of severe veno-occlusive disease: it is time for evidence based economic evaluations

Abstract

Rodriguez et al present in this issue a cost-effectiveness analysis of Defibrotide (Defitelio R ) for the treatment of severe veno-occlusive disease in the context of hematopoietic stem cell transplantation (HSCT) [1]. According to their analysis, Defibrotide might produce 1.214 QALYs compared to best supportive care, with a total cost of €33,708. They conclude that Defibrotide offers a cost-effective therapy, with ratios of €27,757/QALY and €25,007/LY gained [1]. Their complex analysis follows the rules and roles of economic evaluations for novel drugs, and the authors admit some limitations. Still, they suggest that Defibrotide is cost effective within a

Low Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease in adults undergoing allogenic stem cell transplantation with prophylactic ursodiol and low-dose heparin

“…Expert groups suggest that DF prophylaxis can be considered for adult patients at very high risk of SOS [5], respectively is recommended in children at high risk for SOS [15,16]. The high costs of DF are controversial [20]. A recent study evaluated the cost-effectiveness of DF compared to best supportive care (BSC) for the treatment of SOS with MOF after HSCT [21].…”

“…In an editorial about this study, Gratwohl criticizes that the study is based on data which was partly collected over 20 years ago and adapted to today's health system. Also, he questions the high price of DF, which was inexpensive when the drug was approved in the 1980ies for prophylaxis of thrombotic complications, and only since being granted orphan drug status by the European medicine agency (EMA), in 2004, became this expensive [20].…”