Update della valutazione dell'impatto clinico ed economico dell'utilizzo di Advate nella gestione in profilassi del paziente con emofilia A

Translated title of the contribution: [Autom. eng. transl.] Update of the evaluation of the clinical and economic impact of the use of Advate in the prophylaxis management of the patient with hemophilia A

Maria Lucia Specchia, Gualtiero Ricciardi, Chiara De Waure, S Capri, F Kheiraoui

Research output: Working paper

Abstract

[Autom. eng. transl.] The implementation of the latest evidence regarding the pharmacokinetic, efficacy and safety aspects and the economic and organizational implications of the use of Advate allows us to conclude that: - Advate continues to be shown to be effective in controlling bleeding episodes with 69- 70% of cases that are successfully managed with a single infusion; - the haemostatic efficacy of Advate is considered excellent or good in over 90% of cases and prophylaxis can reduce the rate of bleeding per year by more than 99% compared to on-demand treatment, - the development of inhibitors remains low with an overall risk of 0.37% in previously treated patients enrolled in clinical trials; - based on the results of a study of consumption of FL-FVIII and BDD-FVIII, conducted in the United States, a potential saving for the Italian reality of 3-16% was extrapolated in the case of a comparison between treatment with Advate and a base of ReFacto AF. Such differences would even increase to 41-57% by applying the formulas, published in the literature, which link the drug's half-life, frequency of administration and plasma FVIII levels; - recent evidence shows that standard and modulated prophylaxis on the patient's pharmacokinetics are able to return the same results in terms of hemostatic efficacy with a potential benefit in terms of indirect costs and quality of life, to be associated with the reduced frequency of administration , which, however, should be further demonstrated; - some pharmacoeconomic evidence suggests that the different risk of inhibitor development may have a significant impact on patient management costs, with potential savings in the use of low immunogenicity products, such as Advate. This is all the more true if one refers to the recent evidence that would estimate the risk of developing de novo inhibitors about 7 times higher with BDDFVIII compared to FL-FVIII; - the additional services made available free of charge by the Company (nursing, physiotherapy and psychological care at home; multimedia telemedicine platform; home delivery of drugs) would amount to an economic value of about € 31,000 in pediatric patients and about € 25,000 in adults. This value, now included in the total cost borne by the NHS for the management of the patient with Advate, accounts for 23-36% and 8-12% of the total cost, respectively in children and adults.
Translated title of the contribution[Autom. eng. transl.] Update of the evaluation of the clinical and economic impact of the use of Advate in the prophylaxis management of the patient with hemophilia A
Original languageItalian
Number of pages14
Publication statusPublished - 2012

Keywords

  • assessment
  • clinica impact
  • economic aspects
  • haemophilia A

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