Presentation Details
| Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States Craig M.Kessler1, Fernando F.Corrales-Medina2. 1Lombardi Comprehensive Cancer Center, Georgetown University Medical Center, Washington DC, DC, USA.2University of Miami-Miller School of Medicine, and University of Miami-Hemophilia Treatment Center, Miami, FL, USA |
Abstract
Background: In rare diseases such as hemophilia A, direct head-to-head comparisons of treatments are not feasible. Matching-adjusted indirect comparison (MAIC) is a statistical method allowing an alternative approach to evaluate treatment efficacy between clinical studies. In MAIC, study populations are re-weighted based on clinically relevant patient characteristics to better match each other. Treatment decisions in hemophilia A are not only impacted by efficacy, but also by the cost, particularly as there is a need for life-long prophylaxis, which comes with a high economic burden. Objectives: Here we use MAIC to compare the efficacy of pharmacokinetic-guided, personalized prophylaxis with simoctocog alfa (Nuwiq®, a recombinant factor VIII (rFVIII)) versus prophylaxis with efanesoctocog alfa (Altuviiio®; a novel extended half-life Fc-fusion rFVIII) and evaluate their cost in patients with severe hemophilia A. Methods: For the MAIC, individual patient-level data (IPD) were extracted from NuPreviq (phase IIIb study on simoctocog alfa) and aggregate published data from XTEND-1 (phase III study on once-weekly efanesoctocog alfa). Baseline age and body weight were used to re-weight IPD from 65 patients from NuPreviq to match the aggregate data of 133 patients from XTEND-1 (Group A). The following efficacy outcomes for the matched populations were examined: total annualized bleeding rate (ABR) and percentage of patients with zero bleeds (including treated and untreated bleeds); weekly consumption of FVIII; and percentage of bleeds treated with a single infusion. Annual cost of the two regimens was based on the match-adjusted patient populations and included both direct and indirect costs: prophylaxis, on-demand treatment of breakthrough bleeds, non-drug related costs (e.g. hospitalization, emergency services, outpatient care) and societal costs (absence from work). Costs of prophylaxis and on-demand treatment were based on wholesaler acquisition costs (simoctocog alfa: $1.90/IU; efanesoctocog alfa: $5.11/IU). Non-drug related costs were based on Centers for Medicare & Medicaid Services. Results: No patients were excluded from the index study based on the key inclusion criteria for the comparator study. After matching, the effective sample size for simoctocog alfa was 35.3. The percentage of patients with zero bleeds was similar between matched simoctocog alfa population and efanesoctocog alfa (64.1% vs 55.5%; p=0.291, Figure 1). Similarly, there was no statistical difference in mean total ABR between simoctocog alfa and efanesoctocog alfa (1.5 vs 1.1, respectively; p=0.342, Figure 1). The mean weekly dose was significantly higher in patients treated with simoctocog alfa versus efanesoctocog alfa (98.3 IU/kg vs 52.2 IU/kg; p<0.001). Despite the higher dose required, the annual estimated cost of treatment per patient with simoctocog alfa was lower than with efanesoctocog alfa ($792,046 vs $1,116,488). Over 5 years, this translates to a difference of $1,622,209 per patient between the two regimens (Figure 2). Cost was largely driven by cost of prophylaxis as breakthrough bleed related- and other costs were similar between the regimens. Conclusions: Based on MAIC, prophylaxis with simoctocog alfa resulted in similar zero bleed rates and total ABR as with efanesoctocog alfa, albeit with a higher weekly dose. Despite higher dosing, simoctocog alfa endured an estimated 29% lower cost burden than efanesoctocog alfa.
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