Why carry out this study?

Recombinant coagulation factor IX Fc fusion protein (rFIXFc) is approved for the treatment and prophylaxis of bleeding in patients with haemophilia B on the basis of the results from clinical trials, which demonstrated significantly lower rates of bleeding with rFIXFc prophylaxis compared with on-demand treatment.

Cost-effectiveness is an important consideration for the management of haemophilia B because of the requirement of lifelong treatment with prophylaxis or on-demand strategies.

This study aimed to evaluate the cost-effectiveness of rFIXFc when used as prophylaxis for patients with haemophilia B without inhibitors from an Italian healthcare perspective.

What was learned from the study?

Over a lifelong time horizon, rFIXFc prophylaxis dominated rFIX on-demand on the basis of greater total quality-adjusted life years (incremental, 3.993 per patient) and lower total costs (incremental, − €1,255,885 per patient).

The improved health outcomes and lower costs associated with rFIXFc prophylaxis were underscored by estimated fewer bleeds and surgeries, as well as lower costs for bleeding management, surgery and workdays lost compared with rFIX on-demand.

This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers regarding the optimal treatment strategy for patients with haemophilia B.