Cost-Utility Analysis of Primary Prophylaxis versus Treatment On-Demand for Individuals with Severe Haemophilia
Alexander H. Miners
Caroline A. Sabin
Keith H. Tolley
Christine A. Lee
Objective: To assess the cost effectiveness of primary prophylaxis with clotting factor instead of treatment following a bleed (on-demand) for individuals with severe haemophilia. Design: Different data sources on the clinical effects and costs of treatments were combined using a Markov model. Setting: English treatment centres. Perspective: UK societal. Participants: Hypothetical cohorts of 100 individuals with severe haemophilia A or B or severe von Willebrands disease. Interventions: Primary prophylaxis treatment on-demand with clotting factor. Outcome measures: Costs, quality-adjusted life-years (QALYs) and incremental cost per QALY in UK pounds (Lstg , 1999/2000 values). Results: The baseline results showed that treating individuals with severe haemophilia A/severe von Willebrands disease or severe haemophilia B with primary prophylaxis instead of treatment on-demand cost an additional Lstg 46 500 and Lstg 8600 per QALY gained, respectively. However, the results were extremely sensitive to a number of factors including the clotting factor unit cost, the time between prophylactic doses and the discount rate. Conclusions: 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.
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