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Treatment switch to nonacog beta pegol factor IX in hemophilia B: A Canadian cost-consequence analysis based on real-world factor IX consumption and clinical outcomes - PubMed

  • ️Sun Jan 01 2023

Treatment switch to nonacog beta pegol factor IX in hemophilia B: A Canadian cost-consequence analysis based on real-world factor IX consumption and clinical outcomes

Alfonso Iorio et al. Res Pract Thromb Haemost. 2023.

Abstract

Background: The Canadian Bleeding Disorders Registry (CBDR) is a source of real-world data for Canadian patients with hemophilia B. Nonacog beta pegol (N9-GP), an extended half-life (EHL) recombinant factor IX (FIX) concentrate, was awarded a Canadian Blood Services contract in 2018 and subsequently made available across Canada (except Québec) to adult patients. For most patients already on another EHL FIX treatment, a switch to N9-GP occurred.

Objectives: This study estimates the impact on treatment costs of a switch from a prior FIX to N9-GP based on annualized bleed rates and FIX consumption volumes before and after N9-GP switch from the CBDR.

Methods: Real-world data from the CBDR for total FIX consumption and annualized bleed rates were used to inform a deterministic 1-year cost-consequence model. The model considered that the EHL to N9-GP switches were from eftrenonacog alfa and the standard half-life switches were from nonacog alfa. Because FIX prices are confidential in Canada, the model assumed cost parity for annual prophylaxis with each FIX based on the product monograph recommended dosing regimen to calculate an estimated price per international unit for each product.

Results: The switch to N9-GP resulted in improvements in real-world annualized bleed rates and therefore reductions in annual breakthrough bleed treatment costs. Switching to N9-GP also resulted in reduced real-world annual FIX consumption for prophylaxis. Overall, annual treatment costs were 9.4% and 10.5% lower after the switch to N9-GP from nonacog alfa and eftrenonacog alfa, respectively.

Conclusion: N9-GP improves clinical outcomes and may be cost-saving vs nonacog alfa and eftrenonacog alfa.

Keywords: Canada; costs; factor IX; hemophilia B; nonacog beta pegol.

© 2023 Published by Elsevier Inc.

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Figures

Figure 1
Figure 1

Flow diagram showing the enrolment of patients from the CBDR into the analysis. CBDR, Canadian Bleeding Disorders Registry; N9-GP, nonacog beta pegol.

Figure 2
Figure 2

Real-world mean annualized bleed rates before and after switching from an SHL FIX, nonacog alfa or from an EHL FIX, eftrenonacog alfa, to N9-GP. EHL, extended half-life; FIX, factor IX; N9-GP, nonacog beta pegol; SHL, standard half-life.

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