The clinical, humanistic and economic consequences associated with haemophilia and inhibitors are considerable. Primary treatment for mild-to-moderate bleeding disorders in such patients is recombinant factor VIIa (rFVIIa) or activated prothrombin complex concentrate (APCC). The aims of this study were to identify, review and evaluate the quality of the published literature on the relative cost-effectiveness of rFVIIa and APCC in treating haemophilia patients with inhibitors. The review concentrates on model type, design and assumptions, and results. The results of this study suggest that rFVIIa may be the cost-effective alternative to treatment with APCC. In seven out of the nine studies, rFVIIa had the lower average treatment cost. The difference in average treatment cost to resolve a bleed, between rFVIIa and APCC in these seven studies, ranged from $3000 to $17 000. The adapted modelling framework is similar in all the economic models reviewed, suggesting clinical acceptability of the approach used. The estimates of efficacy varied between the models, especially for APCC. The efficacy for APCC derived from retrospective studies was lower than reported in the literature. Sensitivity analysis was undertaken in the majority of the economic analyses and the results were found to be robust to realistic parameter variations. Only one of the studies was a cost-utility study, showing the lack of measuring health status within this area. This systematic review showed that models based on different sources of data produced fairly similar robust results despite differences in the estimates of efficacy, average dosage required, and unit costs. However, ideally there should be a systematic approach to identifying the relevant data
A systematic review of the cost-effectiveness of rFVIIa and APCC in the treatment of minor/moderate bleeding episodes for haemophilia patients with inhibitors
Knight, C., Dano, AM., & Kennedy-Martin, T. (2009). A systematic review of the cost-effectiveness of rFVIIa and APCC in the treatment of minor/moderate bleeding episodes for haemophilia patients with inhibitors. Haemophilia, 15(2), 405-419.