Cost–utility analysis of prophylaxis versus treatment on demand in severe hemophilia A

Abstract

Background:Individuals with severe hemophilia A have reduced blood levels of clotting factor VIII (FVIII) leading to recurrent bleeding into joints and muscles. Primary prophylaxis with clotting factor concentrates started early in childhood prevents joint bleeds, thus avoiding joint damage and improving people’s quality of life. There remain significant differences in the implementation of primary prophylaxis worldwide mainly due to the cost of prophylaxis compared with treatment on demand.Objective:To evaluate the cost-effectiveness of primary prophylaxis with FVIII concentrates versus secondary prophylaxis, versus treatment on demand, and versus a “hybrid” (primary prophylaxis followed by on-demand treatment in adults) in individuals with severe hemophilia A.Methods:A Markov model was developed and run using different sources of clinical, cost, and utility data. The model was populated with a hypothetical cohort of 100 individuals with severe hemophilia A. The perspective of the Italian National Health System was used.Results:The baseline results showed that primary and secondary prophylaxis is cost-effective compared both with treatment on demand and with a hybrid strategy. The incremental costs per quality-adjusted life-year gained for individuals with hemophilia A receiving primary and secondary prophylaxis were €40,229 to €40,236 versus an on-demand strategy. However, the sensitivity analyses performed showed that the results were sensitive to the unit cost of clotting FVIII, bleeding frequency, and the discount rate.Conclusion:Although primary prophylaxis is a costly treatment, our results show that it is cost-effective compared with treatment on demand.

Adult severe haemophilia A patients under long-term prophylaxis with factor VIII in routine clinical practice

“…This approach could be cost-effective when long-term benefits are considered (bleeding events avoided, osteomuscular status improvement, fewer adverse effects and reduced hospitalizations) [20]. However, prophylaxis for patients with frequent haemorrhagic episodes is likely more cost-effective than for patients with few haemorrhagic episodes.…”

Estimates of utility weights in hemophilia: implications for cost-utility analysis of clotting factor prophylaxis

“…Six CUAs of clotting factor prophylaxis in males with hemophilia have been published. Four reported ICERs <$75,000 US dollars or <50,000 GB pounds or euros [4–7]. Two other CUAs concluded that prophylaxis is very unlikely to be cost-effective by conventional criteria, with ICERs in excess of 1 million dollars or euros [8,9].…”

Modelling lifelong effects of different prophylactic treatment strategies for severe haemophilia A

“…None have included an analysis of the effects of treatment on joint status, but all reported that treatment costs are highly dependent on FVIII consumption and price. Interestingly, Colombo et al [15] corroborated the finding of reduced treatment costs following a switching scenario between prophylactic and on-demand treatment in adulthood.…”

“…The present model is the first to confirm this hypothesis. So far, several other groups have simulated life-long haemophilia treatment using computer models [13][14][15][16][17]. However, most of them used Markov modelling, and all were focused on economic evaluations, using cost per QALY as outcome parameters.…”

“…However, the long-term follow-up required [7,11]; and the low number of patients available render this design infeasible for the comparison of prophylactic strategies [12]. Studies modelling patient treatment and outcome are a method used to circumvent this problem [13][14][15][16]. Our group designed a micro simulation model based on data from existing cohorts [17].…”