Cost-Utility Analysis of Primary Prophylaxis versus Treatment On-Demand for Individuals with Severe Haemophilia

Abstract

Despite the high costs of treatment, primary prophylaxis was cost effective compared with treatment on-demand in some scenarios. Primary prophylaxis is more likely to be cost effective for individuals with severe haemophilia B compared with individuals with severe haemophilia A/severe von Willebrands disease. Further research is required to assess the relationship between methods of clotting factor infusion and health-related quality-of-life.

Cost effectiveness of haemophilia treatment: a cross-national assessment

“…The lifetime cost for prophylaxis, however, was larger and accounted for s1 514 970 in a scenario with factor infusion every 56 h. The ICER for individuals receiving factor VIII was s76 060 per QALY gained. If patients were treated with factor IX, ICER was s13 490 per QALY [14]. As a result, these ICERs were lower than in our study.…”

“…We identified only one prior economic study comparing the cost effectiveness of on-demand versus primary prophylactic treatment using a decision-analytic Markov model to simulate long-term consequences [14]. In this study, lifetime healthcare costs of on-demand treatment were s426 550.…”

“…In a cost utility study Miners et al [14] compared the lifetime costs and utilities of treating severe haemophiliacs on-demand versus primary prophylaxis. In the absence of randomized controlled studies, Miners et al [14] used Markov modelling to construct a theoretical model of lifetime costs of treatment. In comparison with Miners et al we used resource utilization data from a clinical trial (observation period was 6 months) and extrapolated these to 1 year.…”

“…Improvements in outcomes, however, were achieved at considerable cost. To our knowledge, only two such studies have extrapolated mean lifetime costs of on-demand and prophylactic treatment [13,14]. One, a long-term US model, measured only avoided bleeds as an outcome and did not measure quality-adjusted life years (QALYs) gained [13].…”

“…One, a long-term US model, measured only avoided bleeds as an outcome and did not measure quality-adjusted life years (QALYs) gained [13]. The other, a Markov model by Miners et al [14] appears to be a more sophisticated attempt at weighing the longterm costs and benefits of primary prophylaxis. It is, however, based on multiple assumptions that should be critically evaluated.…”