OBJECTIVES : Once patients with hemophilia A (HA) developed inhibitors to blood clotting factor VIII (FVIII), the regular treatments based on FVIII would have less or no effect. In China, emicizumab prophylaxis therapy and recombinant activated FVII (rFVIIa) on-demand therapy are the main treating options for patients with inhibitors, since the activated prothrombin complex concentrate (aPCC) is not available, and prothrombin complex concentrate (PCC) has very limited effect on bleed controlling. This study aims at comparing the cost-effectiveness of emicizumab prophylaxis therapy and rFVIIa on-demand therapy for HA patients with inhibitors in China.

METHODS : A two-state-plus-two-event Markov model from Chinese healthcare system perspective using annual cycles was constructed to evaluate the lifelong cost and effectiveness associated with the abovementioned therapies. Patients are either alive or dead (two states), and the alive patients can experience treated bleeds and arthroplasties (two events). The bleeding rates, adverse event rates and utility values associated with the two therapies were sourced from HAVEN 1 & 2 trials and a non-interventional trial. Prophylactic therapy is administrated from one year old to lifelong for the emicizumab group, while on-demand therapy is given upon each treated bleed and the arthroplasties for both groups. The unit prices of emicizumab and rFVIIa were CNY 270 per mg and CNY 5,500 per 1.2mg, respectively. Other model parameters were from literature review and a survey with HA patients. Costs and quality-adjusted life-years (QALYs) were discounted by 5% annually.

RESULTS : Emicizumab prophylaxis therapy was associated with 4.96 more QALYs gained and CNY 10.73 million less in costs compared with rFVIIa on-demand therapy. Patients on emicizumab prophylaxis therapy were estimated to avoid 1,259 episodes of on-demand treatments and 2 arthroplasties over the whole lifetime.

CONCLUSIONS : Emicizumab prophylaxis therapy is a dominant option over rFVIIa on-demand therapy for HA with inhibitors in China, with increased QALYs and less total costs.