Cost of patients with hemophilia A treated with standard half-life or extended half-life FVIII in Spain

References

• World Federation of Hemophilia. Guidelines for the management of hemophilia. 2nd ed. Montréal: Blackwell Publishing; 2012 [cited 2019 Oct 15]. Available from https://www1.wfh.org/publications/files/pdf-1472.pdf
   Google Scholar
• Mannucci PM, Tuddenham EG. The hemophilias-from royalgenes to gene therapy. N Engl J Med. 2001;344:1773–1779.
   PubMed Web of Science ®Google Scholar
• Berntorp E, Collins P, D’Oiron R, et al. Identifying non-responsive bleeding episodes in patients with haemophilia and inhibitors: a consensus definition. Haemophilia. 2011;17:e202–10.
   PubMed Web of Science ®Google Scholar
• Hay CR, Brown S, Collins PW, et al. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom haemophilia centre doctors organisation. Br J Haematol. 2006;133:591–605.
   PubMed Web of Science ®Google Scholar
• Ljung R. Aspects of prophylactic treatment of hemophilia. Thromb J. 2016;14:30.
   PubMed Web of Science ®Google Scholar
• Chhabra A, Fogarty PF, Tortella BJ, et al. Real-world analysis of dispensed international units of coagulation factor VIII and resultant expenditures for hemophilia A patients: a comparison between standard half-life and extended half-life products. Manag Care. 2018;27:39–50.
   PubMedGoogle Scholar
• Mahlangu J, Young G, Hermans C, et al. Defining extended half-life rFVIII – a critical review of the evidence. Haemophilia. 2018;24:348–358.
   PubMed Web of Science ®Google Scholar
• Rota M, Cortesi PA, Steinitz-Trost KN, et al. Meta-analysis on incidence of inhibitors in patients with haemophilia A treated with recombinant factor VIII products. Blood Coagul Fibrinolysis. 2017;28:627–637.
   PubMed Web of Science ®Google Scholar
• Afstyla, lonoctocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/afstyla-epar-product-information_en.pdf
   Google Scholar
• Refacto AF, moroctocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/refacto-af-epar-product-information_en.pdf
   Google Scholar
• Advate, octocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/advate-epar-product-information_en.pdf
   Google Scholar
• Helixate NexGen, octocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/helixate-nexgen-epar-product-information_en.pdf
   Google Scholar
• Kogenate Bayer, octocog alfa. Annex I, summary of product characteristics. European Medicines Agency. [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/kogenate-bayer-epar-product-information_en.pdf
   Google Scholar
• Kovaltry, octocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/kovaltry-epar-product-information_en.pdf.
   Google Scholar
• Nuwiq, simoctocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2019 Oct 16]. Available from: https://www.ema.europa.eu/en/documents/product-information/nuwiq-epar-product-information_en.pdf.
   Google Scholar
• NovoEight, turoctocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2020 Feb 28]. Available from: https://www.ema.europa.eu/en/documents/product-information/novoeight-epar-product-information_en.pdf
   Google Scholar
• Elocta, efmoroctocog alfa. Annex I, summary of product characteristics. European Medicines Agency; [cited 2020 Feb 28]. Available from: https://www.ema.europa.eu/en/documents/product-information/elocta-epar-product-information_en.pdf
   Google Scholar
• Adynovi, rurioctocog alfa pegol. Annex I, summary of product characteristics. European Medicines Agency; [cited 2020 Feb 28]. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/adynovi
   Google Scholar
• Jivi, damoctocog alfa pegol. Annex I, summary of product characteristics. European Medicines Agency; [cited 2010 Feb 28]. Available from: https://www.ema.europa.eu/en/documents/product-information/jivi-epar-product-information_en.pdf
   Google Scholar
• Esperoct, turoctocog alfa pegol. Annex I, summary of product characteristics. European Medicines Agency; [cited 2020 May 12]. Available from: https://www.ema.europa.eu/en/documents/product-information/esperoct-epar-product-information_en.pdf
   Google Scholar
• State Official Newsletter, Order SCB/1244/2018 [Spanish]. Ministry of Health, Consumption, and Social Welfare. Number 286, Section 1, p115,287; 2018 Nov 23.[cited 2019 Oct 16]. Available from: https://www.boe.es/boe/dias/2018/11/27/pdfs/BOE-A-2018-16150.pdf
   Google Scholar
• Álvarez MT, Parra R, Jiménez V, et al. Estudio retrospectivo de la práctica clínica habitual de la hemofilia A y B en dos centros en España (P-305). XXXII Congreso SEHH-SETH; Santiago de Compostela, Spain; 2016 Oct 20-22.
   Google Scholar
• Briggs A, Claxton K, Sculpher M. Decision modelling for health economic evaluation. Oxford (UK): Oxford University Press; 2006.
   Google Scholar
• Rubio-Terrés C, Rubio-Rodríguez D. Probabilistic analysis: sensitivity analysis or main result? [editorial]. Pharmacoecon Open. 2016;1(2):1000e102.
   Google Scholar
• Mayoralas S, Huerta A, Parrondo J, et al. Monte-Carlo simulation to estimate the health care cost avoided with fluticasone furoate/vilanterol due to exacerbation rate reduction in Spanish COPD patients. Value Health. 2014;17:A603.
   PubMed Web of Science ®Google Scholar
• Isla D, De Castro J, Juan O, et al. Costs of adverse events associated with erlotinib or afatinib in first-line treatment of advanced EGFR-positive non-small cell lung cancer. ClinicoEcon Outcomes Res. 2017;9:31–38.
   PubMed Web of Science ®Google Scholar
• Anguita P, González C, Cañete M, et al. Cost of adverse effects associated with enzalutamide or apalutamide in the treatment of prostate cancer resistant to non-metastatic castration in Spain. Rev Esp Econ Salud.2019;14:794-805.
   Google Scholar
• Manito N, Rubio-Rodríguez D, González J, et al. Economic analysis of intermittent outpatient treatment with levosimendan of heart failure in Spain. Rev Esp Cardiol. 2019. in press. DOI:10.1016/j.recesp.2019.06.019.
   Web of Science ®Google Scholar
• IQVIA, The Human Data Science company. EMH Report. Madrid: November 2018.
   Google Scholar
• Peyvandi F, Garagiola I, Boscarino M, et al. Real-life experience in switching to new extended half-life products at European haemophilia centres. Haemophilia. 2019;25:946–952.
   PubMed Web of Science ®Google Scholar
• Croteau SE, Cheng D, Cohen AJ, et al. Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. haemophilia treatment centres for patients with moderate and severe haemophilia. Haemophilia. 2019;25:668–675.
   PubMed Web of Science ®Google Scholar
• Bowen K, Borchardt M, Gleason PP Incremental cost of switching to extended half-life (EHL) coagulation factor products to treat hemophilia among 15 million commercially insured members [Poster]. Presented at AMCP; Boston, MA; 2018 Apr [cited 2019 Dec 23]. Available from: https://www.primetherapeutics.com/content/dam/corporate/Documents/Newsroom/Pressreleases/2018/document-amcpspring18-hemophilia.pdf
   Google Scholar
• Aledort L, Milligan S, Watt M, et al. A retrospective observational study of rurioctocog alfa pegol in clinical practice in the United States. J Manag Care Spec Pharm. 2020;26:492–503.
   PubMed Web of Science ®Google Scholar
• Chhabra A, Spurden D, Fogarty PF, et al. Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B. Blood Coagul Fibrinolysis. 2020;31:186–192.
   PubMed Web of Science ®Google Scholar
• Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia. 2017;23:105–114.
   PubMed Web of Science ®Google Scholar
• Refletion paper on the extrapolation of results from clinical studies conducted outside the USA to the EU-population. Committee for Medicinal Products for Human Use (CHMP): EMEA/CHMP/EWP/692702/2008. London; 2009 Oct 22.
   Google Scholar
• Note for guidance on ethnic factors in the acceptability of foreign clinical data (CPMP/ICH/289/95). CHMP/ICH/289/95. 1998 Sep.
   Google Scholar
• Álvarez T, Monzón E, Fernández-Bello I, et al. Real life experience in clinical practice with recombinant coagulation FVIII-Fc fusion protein. Blood. 2019;134(Suppl. 1):4929. .
   Google Scholar
• Roggeri DP, Zanon E, Roggeri A. Recently approved recombinant factor VIII (rFVIII) for the replacement treatment in patients with hemophilia A in Italy. Farmeconomia Health Econ Ther Pathways. 2017;18:55–60.
   Google Scholar
• Bullement A, McMordie ST, Hatswell AJ, et al. Cost-effectiveness analysis of recombinant factor VIII Fc-fusion protein (rFVIIIFc) for the treatment of severe hemophilia A in Italy incorporating real-world dosing and joint health data. Pharmacoecon Open. 2020;4:133-42.
   PubMed Web of Science ®Google Scholar
• Iorio A, Krishnan S, Myrén KJ, et al. Indirect comparisons of efficacy and weekly factor consumption during continuous prophylaxis with recombinant factor VIII Fc fusion protein and conventional recombinant factor VIII products. Haemophilia. 2017;23:408–416.
   PubMed Web of Science ®Google Scholar
• Dunn AL, Ahuja SP, Mullins ES. Real-world experience with use of antihemophilic factor (recombinant), PEGylated for prophylaxis in severe haemophilia A. Haemophilia. 2018;24:e84–e92.
   PubMed Web of Science ®Google Scholar
• Vepsäläinen K, Riikonen P, Lassila R, et al. Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A: a nationwide real-world study with 700 person-years. Haemophilia. 2018;24:436–444.
   PubMed Web of Science ®Google Scholar
• Tortella BJ, Alvir J, McDonald M, et al. Real-world analysis of dispensed IUs of coagulation factor IX and resultant expenditures in hemophilia B patients receiving standard half-life versus extended half-life products and those switching from standard half-life to extended half-life products. J Manag Care Spec Pharm. 2018;24:643–653.
   PubMed Web of Science ®Google Scholar