Economic evaluation of rFVIIa high initial dose compared to rFVIIa standard initial dose in patients with haemophilia with inhibitors using the Czech HemoRec registry
Abstract
Introduction: In the treatment of bleeds in haemophilia patients with inhibitors, a high initial dose of recombinant Factor VIIa (rFVIIa) provides at least equal efficacy and a similar safety profile to a standard initial dose. However, no pharmacoeconomic comparison between these dosing regimens has previously been performed.
Here, we assess the pharmacoeconomics of high (> 120 mu g/kg) versus standard ({= 120 mu g/kg) initial rFVIIa dose in inhibitor patients and the impact of time to treatment initiation on costs and outcomes. Methods: In a retrospective analysis, observational data on bleed characteristics, rFVIIa treatment, hospitalizations and outcomes were extracted from the Czech Republic HemoRec registry.
Crude comparisons and generalized linear regression modelling (GLM; correcting for patient differences) were performed to compare costs and outcomes between the high and standard initial dosing groups. Results: Of 314 rFVIIa-treated bleeding episodes (12 inhibitor patients), most were spontaneous joint bleeds and 67.5% were treated with a high initial dose.
In the crude comparison, high initial rFVIIa dosing was associated with a lower mean number of doses needed to achieve haemostasis compared with standard dosing (p < 0.001), but higher total dose and costs (p {= 0.008). However, regression analyses revealed that high initial dose was associated with similar costs (p = 0.891) and a shorter time to bleeding resolution (p = 0.014).
Increasing time to treatment initiation increased both time to bleeding resolution and total costs. Conclusion: Compared with a standard dose, a high initial rFVIIa dose may improve treatment outcomes without increasing costs.
Early treatment initiation may reduce treatment costs.