Abstract
Background: The high costs and uncertainties surrounding the effectiveness and safety of new health technologies necessitate continuous evaluation to balance access and sustainability within Brazil’s Unified Health System.
Aims: Cost-effectiveness analysis comparing etranacogene dezaparvovec versus factor IX prophylaxis in people with hemophilia B from the Brazilian healthcare system perspective.
Methods: A cost-effectiveness analysis was conducted using TreeAge Pro software. A decision tree model compared the costs and effectiveness of etranacogene dezaparvovec versus factor IX prophylaxis in preventing bleeding in adult male with severe or moderately severe hemophilia B over a three-year horizon. A deterministic sensitivity analysis identified variables impacting incremental cost-effectiveness ratio.
Results: etranacogene dezaparvovec incurred a cost of USD 2,335,747 (BRL 10.810.993) versus USD 46,451 (BRL 232.256) for plasma-derived factor IX prophylaxis, with an incremental cost of USD 2,115,747 (BRL 10.578.737). The effectiveness (number of bleeding events) was 2.86 for etranacogene dezaparvovec and 10.95 for factor IX prophylaxis, with etranacogene dezaparvovec avoiding 8.09 additional bleeding events. The incremental cost-effectiveness ratio was USD 261,436 (BRL1.307.179) per bleeding event avoided. Sensitivity analysis revealed treatment duration as the most impactful variable on incremental cost-effectiveness ratio, contributing to 80% of model uncertainty.
Discussion: International studies suggest that etranacogene dezaparvovec is cost-effective and dominant in multiple contexts, particularly when compared to extended half-life recombinant factor IX (rFIX-EHL), offering greater quality-adjusted life years for people with hemophilia B at lower long-term costs. However, a lack of robust data underscores the need for ongoing monitoring and careful evaluation before incorporation by the health system. It should also be noted that high pricing of new treatments like etranacogene dezaparvovec poses challenges for Brazil’s Unified Health System sustainability and equitable access.