Cost-effectiveness analysis of Glybera for the treatment of lipoprotein lipase deficiency
Objectives Glybera is the first gene therapy drug for lipoprotein lipase deficiency (LPLD),
which was approved by European Commission in 2012. Before the approval of Glybera, no
effective treatment was available for LPLD. Patients with LPLD have to restrictively control fat
intaken and are still more likely to suffer recurrent acute pancreatitis and eruptive
xanthomas. Although Glybera can effectively improve the health conditoin of patients with
LPLD, it is still controversial to price the gene therapy at 1.1 million euros. This study …
which was approved by European Commission in 2012. Before the approval of Glybera, no
effective treatment was available for LPLD. Patients with LPLD have to restrictively control fat
intaken and are still more likely to suffer recurrent acute pancreatitis and eruptive
xanthomas. Although Glybera can effectively improve the health conditoin of patients with
LPLD, it is still controversial to price the gene therapy at 1.1 million euros. This study …
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