Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A

Abstract Introduction Prophylaxis with factor VIII (FVIII) is the standard of care for individuals with severe hemophilia A, but it is associated with a substantial economic burden. This analysis compared the efficacy, dosing, and annual treatment costs of simoctocog alfa (Nuwiq, a recombinant FVIII [rFVIII]) with five extended half-life (EHL) rFVIII concentrate products. Methods Matching-adjusted indirect comparisons (MAICs) were performed to compare simoctocog alfa with efanesoctocog alfa (ALTUVIIIO) and turoctocog alfa pegol (Esperoct), and results were integrated with a previously published MAIC comparing simoctocog alfa with efmoroctocog alfa (ELOCTATE), damoctocog alfa pegol (JIVI), and rurioctocog alfa pegol (ADYNOVATE). These MAIC-adjusted populations were used to develop a cost model from a United States payer perspective. Results Annual total costs per person were $147,058 to $492,090 lower with simoctocog alfa compared with EHL rFVIII products, corresponding to a 21% to 46% cost reduction, primarily driven by lower drug acquisition costs. Outcomes were comparable or favorable to simoctocog alfa versus turoctocog alfa pegol, efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol. In comparison with efanesoctocog alfa (Group A), no significant differences were observed in the proportion of individuals with zero bleeds, whereas treated total annualized bleeding rate (ABR) and treated spontaneous ABR favored efanesoctocog alfa. Conclusion This indirect comparison suggests that personalized prophylaxis with simoctocog alfa may offer economic advantages versus EHL rFVIII products in individuals with severe hemophilia A. Clinical outcomes were broadly comparable across comparators, although treated total and spontaneous ABRs were significantly lower with efanesoctocog alfa (Group A).