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 (£, 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 £46 500 and £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.
Clotting Factor Major Surgery Bolus Dose Primary Prophylaxis Utility Weight
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The authors would like to thank Martin Buxton for comments on an earlier draft of the manuscript.
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