Cost analysis comparison of rurioctocog alfa pegol compared to turoctocog alfa pegol in the prophylaxis of Haemophilia A in Italy

Authors

  • Federica Demma Takeda Italia S.p.A., Roma - Italy
  • Pierpaola Arpa Takeda Italia S.p.A., Roma - Italy

DOI:

https://doi.org/10.33393/ao.2022.2405

Keywords:

Cost, Hemophilia A, Once-weekly, Prophylaxis, Rurioctocog alfa pegol, Turoctocog alfa pegol

Abstract

 

Background: Hemophilia-A is an X-linked genetic disorder arising from a mutation to the X chromosome and causing either a quantitative or qualitative deficiency in blood clotting factor-VIII (FVIII). Several recombinant extended half-life FVIII products (rFVIII-EHL) are in use in Italy for the treatment of hemophilia-A. 

Objective: To estimate the annual average cost of the prophylactic treatment of a patient with hemophilia-A in Italy with rurioctocog alfa  pegol or turoctocog alfa pegol, with particular regard to potential dose adjustments as provided in the SPCs. 

Methods: The comparison between the two rFVIII-EHL, expressed in terms of average annual cost per patient treated, was conducted from the NHS perspective. The average annual consumption (IU/kg) of the two rFVIII-EHL was derived from the clinical trial of reference for in label prophylactic treatment. Adjustments of doses and administration intervals allowed in the SPCs were considered on the basis of the literature. The published ex-factory price considered for both rFVIII-EHL was € 0.72. A scenario analysis focused on possible dosing adjustments was conducted. 

Results: With regard to the average consumption associated with the posology in label, the average annual treatment cost is almost the same between the two rFVIII-EHL. Whereas taking into account the possible dose adjustments based on once-weekly administration, rurioctocog alfa pegol shows a considerably lower average annual treatment cost compared to turoctocog alfa pegol (– € 42,719). 

Conclusions: Rurioctocog alfa pegol would be the more sustainable option, based on the annual average cost, for the NHS in the prophylactic treatment of hemophilia-A. 

Downloads

Download data is not yet available.

References

Coppola A, Franchini M, Castaman G, et al; AICE ad hoc Working Group. Treatment Regimens with Bypassing Agents in Patients with Hemophilia A and Inhibitors: A Survey from the Italian Association of Hemophilia Centers (AICE). Semin Thromb Hemost. 2018;44(6):551-560. https://doi.org/10.1055/s-0038-1648230 PMID:29723892 DOI: https://doi.org/10.1055/s-0038-1648230

Roggeri DP, Zanon E, Biasoli C, Roggeri A. Extended Half-life rFVIII for the Treatment of Hemophilia A: Drugs Consumption and Patients’ Perspective. Farmeconomia. Health economics and therapeutic pathways 2020; 21(1): 59-68. https://doi.org/10.7175/fe.v21i1.1472 DOI: https://doi.org/10.7175/fe.v21i1.1472

Center for Disease Control and prevention. Data & Statistics on hemophilia. Online: https://www.cdc.gov/ncbddd/hemophilia/data.html (Accessed March 2022)

Osservatorio Malattie Rare. Online https://www.osservatoriomalattierare.it/malattie-rare/emofilie (Accessed March 2022)

Giordano P, Franchini M, Lassandro G, Faienza MF, Valente R, Molinari AC. Issues in pediatric haemophilia care. Ital J Pediatr. 2013;39(1):24. https://doi.org/10.1186/1824-7288-39-24 PMID:23601343 DOI: https://doi.org/10.1186/1824-7288-39-24

Associazione Italiana Ematologia Oncologica Pediatrica (AIEOP). Online https://www.aieop.org/web/famiglie/schede-malattia/emofilia/ (Accessed March 2022)

Franchini M, Mannucci PM. Inhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice. Br J Clin Pharmacol. 2011;72(4):553-562. https://doi.org/10.1111/j.1365-2125.2010.03899.x PMID:21204915 DOI: https://doi.org/10.1111/j.1365-2125.2010.03899.x

Björkman S, Folkesson A, Jönsson S. Pharmacokinetics and dose requirements of factor VIII over the age range 3-74 years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol. 2009;65(10):989-998. https://doi.org/10.1007/s00228-009-0676-x PMID:19557401 DOI: https://doi.org/10.1007/s00228-009-0676-x

Jivi® (damoctocog alfa pegol) Riassunto delle caratteristiche di prodotto. Online https://www.ema.europa.eu/en/documents/product-information/jivi-epar-product-information_it.pdf (Accessed March 2022)

Elocta® (efmoroctocog alfa) Riassunto delle caratteristiche di prodotto. Online https://www.ema.europa.eu/en/documents/product-information/elocta-epar-product-information_it.pdf (Accessed March 2022)

Adynovi® (rurioctocog alfa pegol) Riassunto delle caratteristiche di prodotto. Online https://www.ema.europa.eu/en/documents/product-information/adynovi-epar-product-information_it.pdf (Accessed March 2022)

Esperoct® (turoctocog alfa pegol) Riassunto delle caratteristiche di prodotto. Online https://www.ema.europa.eu/en/documents/product-information/esperoct-epar-product-information_it.pdf (Accessed March 2022)

Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015;126(9):1078-1085. https://doi.org/10.1182/blood-2015-03-630897 PMID:26157075 DOI: https://doi.org/10.1182/blood-2015-03-630897

Giangrande P, Andreeva T, Chowdary P, et al; Pathfinder™2 Investigators. Clinical evaluation of glycoPEGylated recombinant FVIII: efficacy and safety in severe haemophilia A. Thromb Haemost. 2017;117(2):252-261. https://doi.org/10.1160/TH16-06-0444 PMID:27904904 DOI: https://doi.org/10.1160/TH16-06-0444

Guidelines ATC classification and DDD assignment 2021 WHO. Online https://www.whocc.no/filearchive/publications/2021_guidelines_web.pdf (Accessed March 2022)

Chowdary P, Mullins ES, Konkle BA, et al. Long-term safety and efficacy results from the phase 3b, open-label, multicentre Continuation study of rurioctocog alfa pegol for prophylaxis in previously treated patients with severe haemophilia A. Haemophilia. 2020;26(4):e168-e178. https://doi.org/10.1111/hae.14052 PMID:32597029 DOI: https://doi.org/10.1111/hae.14052

Curry N, Albayrak C, Escobar M, et al. Once-weekly prophylaxis with glycoPEGylated recombinant factor VIII (N8-GP) in severe haemophilia A: safety and efficacy results from pathfinder 2 (randomized phase III trial). Haemophilia. 2019;25(3):373-381. https://doi.org/10.1111/hae.13712 PMID:30817066 DOI: https://doi.org/10.1111/hae.13712

Chowdary P, Mullins E, Konkle BA, et al. Outcomes With an Extended Prophylactic Treatment Schedule of Rurioctocog Alfa Pegol in a Phase 3b, Open-label, Multicenter, CONTINUATION Study in Previously Treated Patients With Severe Hemophilia A. Presented at the 27th Congress of the International Society on Thrombosis and Haemostasis (ISTH); July 6–10, 2019; Melbourne, Australia

AGENZIA ITALIANA DEL FARMACO DETERMINA 3 gennaio 2020 Riclassificazione del medicinale per uso umano «Adynovi», ai sensi dell'articolo 8, comma 10, della legge 24 dicembre 1993, n. 537. (Determina n. 38/2020). (20A00280) (GU Serie Generale n.14 del 18-01-2020). Online https://www.gazzettaufficiale.it/eli/id/2020/01/18/20A00280/sg

AGENZIA ITALIANA DEL FARMACO DETERMINA 6 luglio 2020 Riclassificazione del medicinale per uso umano «Esperoct» ai sensi dell'art. 8, comma 10, della legge 24 dicembre 1993, n. 537. (Determina n. DG 705/2020). (20A03694) (GU Serie Generale n.178 del 16-07-2020). Online https://www.gazzettaufficiale.it/eli/id/2020/07/16/20A03694/sg

Pierluigi Russo, Andrea Marcellusi, Matteo Zanuzzi et all. Drug Prices and Value of Oncology Drugs in Italy June 2021Value in Health 24(28). https://doi.org/10.1016/j.jval.2021.04.1278 DOI: https://doi.org/10.1016/j.jval.2021.04.1278

Reding MT, Ng HJ, Poulsen LH, et al. Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII. J Thromb Haemost. 2017;15(3):411-419. https://doi.org/10.1111/jth.13597PMID:27992112 DOI: https://doi.org/10.1111/jth.13597

Suiter TM. First and next generation native rFVIII in the treatment of hemophilia A. What has been achieved? Can patients be switched safely? Semin Thromb Hemost. 2002;28(3):277-284. https://doi.org/10.1055/s-2002-32663 PMID:12098089 DOI: https://doi.org/10.1055/s-2002-32663

Wells JR, Gater A, Marshall C, Tritton T, Vashi P, Kessabi S. Exploring the Impact of Infusion Frequency in Hemophilia A: Exit Interviews with Patients Participating in BAY 94-9027 Extension Studies (PROTECT VIII). Patient. 2019;12(6):611-619. https://doi.org/10.1007/s40271-019-00374-x PMID:31313270 DOI: https://doi.org/10.1007/s40271-019-00374-x

Kearney S, Raffini LJ, Pham TP, et al. Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII. Patient Prefer Adherence. 2019;13:497-513. https://doi.org/10.2147/PPA.S196103 PMID:31040652 DOI: https://doi.org/10.2147/PPA.S196103

Published

2022-06-06

How to Cite

Demma, F., & Arpa, P. (2022). Cost analysis comparison of rurioctocog alfa pegol compared to turoctocog alfa pegol in the prophylaxis of Haemophilia A in Italy. AboutOpen, 9(1), 36–42. https://doi.org/10.33393/ao.2022.2405

Issue

Section

Original research articles

Categories

Received 2022-03-25
Accepted 2022-05-06
Published 2022-06-06

Metrics