Budget impact analysis of extended half-life recombinant factor IX (rFIXFc) in the treatment of haemophilia B
- PMID: 36627960
- PMCID: PMC9677589
- DOI: 10.33393/grhta.2020.2113
Budget impact analysis of extended half-life recombinant factor IX (rFIXFc) in the treatment of haemophilia B
Abstract
Introduction: Prophylaxis with factor IX (FIX) concentrates, produced by recombinant DNA technology (rFIX) or human plasma-derived concentrates, is the treatment of choice for haemophilia B (HB); rFIX covalently fused to the Fc domain of human immunoglobulin G1 (rFIXFc) allows for prophylaxis/treatment with one infusion every 7-14 days. The purpose of this study is to quantify the financial impact of prophylaxis with rFIXFc vs. other approved rFIX and reimbursed for treatment of HB in Italy.
Methods: The number of patients was estimated according to Italian epidemiological data and use of rFIX. Dose and frequency of administration used for weekly prophylaxis were those recommended in the Summary of Product Characteristics (SPC), while clinical trials and literature data were used to calculate bleeding rates and management. Drug costs were calculated using regional ex-factory net prices. In the model, a reference scenario (Reference) vs. an alternative scenario (Alternative) were created to account for introduction of rFIXFc, estimating an increasing trend of the market share of rFIXFc in a 3-year timeframe. The analysis was developed in the perspective of the National Health Service and included healthcare costs related to rFIX for prophylaxis and resolution of bleeding events.
Results: The model estimated an overall cumulative expenditure (years 1-3) of €209,453,646 for the Reference and €207,465,568 for Alternative scenarios, with calculated cumulative savings of €1,988,068.
Conclusions: The increasing use of rFIXFc as a substitute for other rFIX concentrates in the treatment of HB can represent a financially viable choice for the Italian National Health Service while ensuring effective control of bleeding.
Keywords: Budget impact analysis; Haemophilia B; rFIXFc.
Copyright © 2020, The Authors.
Conflict of interest statement
Conflict of Interest: AA and BP are employees of Certara Italy S.r.l.; MME reports personal fees from Swedish Orphan Biovitrum (Sobi), Bayer Healthcare, CSL Behring, Grifols, Kedrion, Novo Nordisk, Octapharma, Pfizer, Roche; CA and TC are employees of Sobi S.r.l., Milan, Italy.
Figures
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References
-
- World Federation of Haemophilia. Guidelines for the management of Haemophilia. [accessed 12/06/2020.];2013 https://www1.wfh.org/publications/files/pdf-1472.pdf Available at:
-
- ORPHANET. Emofilia B. [accessed 12/06/20]; https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Lng=IT&Expert=98879 Available at:
-
- Abbonizio F, Hassan HJ, Riccioni R, Arcieri R. Registro Nazionale delle Coagulopatie Congenite. Roma:: Istituto Superiore di Sanità 2019; Associazione Italiana Centri Emofilia (AICE), Giampaolo. Rapporto 2017. (Rapporti ISTISAN 19/8).
-
- Budde U, Drewke E. Von Willebrand factor multimers in virus-inactivated plasmas and F VIII concentrates. Beitr Infusionsther Transfusionsmed. 1994;32:408–414. - PubMed
-
- Chin S, Williams B, Gottlieb P et al. Virucidal short wavelength ultraviolet light treatment of plasma and factor VIII concentrate: protection of proteins by antioxidants. Blood. 1995 1 Dec;86(11):4331–4336. - PubMed
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