Budget Impact Analysis of anakinra in the treatment of patients with Still’s Disease
Analisi di budget impact di anakinra nel trattamento della Malattia di Still in Italia
Keywords:Adult onset Still’s disease, Anakinra, Budget impact, Italian National Health Service, Still’s disease, Systemic juvenile idiopathic arthritis
Background: Anakinra, canakinumab and tocilizumab are all effective alternative treatment choice in patients with Still’s disease including both systemic juvenile idiopathic arthritis (SJIA) adult onset Still’s disease (AOSD) compared to canakinumab and tocilizumab.
Objective: Aim of this study was to estimate the budget impact of the use of anakinra compared to canakinumab and tocilizumab in the treatment of patients with AOSD or SJIA.
Methods: Considering the perspective of the Italian National Health Service (iNHS), a budget impact model (BIM) was developed to estimate the drugs costs of anakinra, canakinumab and tocilizumab up to 12 months. The BIM showed the difference of drug expenditure generated by the base case calculated for current prescription volumes, and for different prescription volume scenarios with increased anakinra prescription. Key variables were tested in the sensitivity analysis.
Results: Compared to the current scenario for SJIA, an increase in the market share of anakinra (40% or 50%) would lead to a reduction in the drug expenditure sustained by iNHS (-€1,118,005 [-12.7%] or -€2,054,502 [-23.4%]). Compared to the current scenario for AOSD, an increase in the market share of anakinra (40% or 50%) would lead to a reduction in the drug expenditure sustained by iNHS (-€4,024,585 [-13.5%] or -€8,049,169 [-27.0%]).
Conclusion: According to the present analysis, the use of anakinra, as an alternative to canakinumab or tocilizumab in patients with AOSD or SJIA, could represent a cost-saving option for the iNHS.
Sfriso P, Bindoli S, Galozzi P. Adult-onset Still’s disease: molecular pathophysiology and therapeutic advances. Drugs. 2018;78(12):1187-95.
Zhou S, Qiao J, Bai J et al. Biological therapy of traditional therapy-resistant adult-onset Still’s disease: an evidence-based review. Ther Clin Risk Manag. 2018;14: 167-71.
Sfriso P, Priori R, Valesini G et al. Adult onset Still’s disease: an Italian multicenter retrospective observational study of manifestations and treatments in 245 patients. Clin Rheumatol. 2016;35(7):1683-9.
Arthur VL, Shuldiner E, Remmers EF et al. IL1RN variation influences both disease susceptibility and response to recombinant human interleukin-1 receptor antagonist therapy in systemic juvenile idiopathic arthritis. Arthritis Rheumatol. 2018;70(8):1319-30.
Nirmala N, Brachat A, Feist E et al. Gene-expression analysis of adult-onset Still’s disease and systemic juvenile idiopathic arthritis is consistent with a continuum of a single disease entity. Pediatr Rheumatol Online J. 2015;13:50.
Castañeda S, Blanco R, González-Gay MA. Adult-onset Still’s disease: advances in the treatment. Best Pract Res Clin Rheumatol. 2016;30(2):222-38.
Pascual V, Allantaz F, Arce E et al. Role of interleukin-1 (IL-1) in the pathogenesis of systemic onset juvenile idiopathic arthritis and clinical response to IL-1 blockade. J Exp Med. 2005;201(9):1479-86.
Kimura Y, Weiss JE, Haroldson KL et al. Pulmonary hypertension and other potentially fatal pulmonary complications in systemic juvenile idiopathic arthritis. Arthritis Care Res (Hoboken). 2013;65(5):745-52.
Néel A, Wahbi A, Tessoulin B et al. Diagnostic and management of life-threatening adult-onset Still disease: a French nationwide multicenter study and systematic literature review. Crit Care. 2018;22(1):88.
Ruscitti P, Cipriani P, Masedu F et al. Adult-onset Still’s disease: evaluation of prognostic tools and validation of the systemic score by analysis of 100 cases from three centers. BMC Med. 2016;14(1):194.
Schulert GS, Grom AA. Pathogenesis of macrophage activation syndrome and potential for cytokine-directed therapies. Annu Rev Med. 2015;66:145-59.
Bracaglia C, Prencipe G, De Benedetti F. Macrophage activation syndrome: different mechanisms leading to a one clinical syndrome. Pediatr Rheumatol. 2017;15(1):5.
Ringold S, Weiss PF, Beukelman T et al. 2013 update of the 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: recommendations for the medical therapy of children with systemic juvenile idiopathic arthritis and tuberculosis screening among children receiving biologic medications. Arthritis Rheum. 2013;65(10):2499-512.
Mimura T, Kondo Y, Ohta A et al. Evidence-based clinical practice guideline for adult Still’s disease. Mod Rheumatol. 2018 Sep;28(5):736-57.
Ter Haar NM, Tak T, Mokry M et al. Reversal of sepsis-like features of neutrophils by interleukin-1 blockade in patients with systemic-onset juvenile idiopathic arthritis. Arthritis Rheumatol. 2018;70(6):943-56.
Lyseng-Williamson KA. Anakinra in Still’s disease: a profile of its use. Drugs Ther Perspect. 2018;34(12):543-53.
Canakinumab RCP. https://ec.europa.eu/health/documents/community-register/2016/20160801135455/anx_135455_it.pdf Accesso Marzo 2020.
Tocilizumab RCP. https://www.ema.europa.eu/en/documents/product-information/roactemra-epar-product-information_it.pdf Accesso Marzo 2020.
Mauskopf JA, Sullivan SD, Annemans L et al. Principles of good practice for budget impact analysis: report of the ISPOR Task Force on Good Research Practices—budget impact analysis. Value Health. 2007;10(5):336-47.
Sullivan SD, Mauskopf JA, Augustovski F et al. Budget impact analysis principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force. Value Health. 2014;17(1):5-14.
Gerfaud-Valentin M, Jamilloux Y, Iwaz J et al. Adult-onset Still’s disease. Autoimmunity Reviews. 2014;13:708-22.
Data on file SOBI. Kineret Mapping Research.
Data on file SOBI. Expert Opinion from national Advisory Board.
ISTAT Popolazione 2016. http://dati.istat.it/Index.aspx?DataSetCode=DCIS_POPRES1 Accesso Agosto 2016.
Modesto C, Rodriguez B, Bou R et al. Incidence and prevalence of juvenile idiopathic arthritis in Catalonia (Spain). Scand J Rheumatol. 2010;39(6):472-9.
Vitale A, Insalaco I, Sfriso P et al. A snapshot on the on-label and off-label use of the interleukin-1 inhibitors in Italy among rheumatologists and pediatric rheumatologists: a nationwide multi-center retrospective observational study. Front Pharmacol. 2016 Oct 24;7:380.
Jamilloux Y, Gerfaud-Valentin M, Martinon F et al. Pathogenesis of adult-onset Still’s disease: new insights from the juvenile counterpart. Immunol Res. 2015;61:53-62.
Anakinra RCP. https://www.ema.europa.eu/en/documents/product-information/kineret-epar-product-information_it.pdf. Accesso Dicembre 2019.
Nordström D1, Knight A, Luukkainen R et al. Beneficial effect of interleukin 1 inhibition with anakinra in adult-onset Still’s disease. An open, randomized, multicenter study. J Rheumatol. 2012 Oct;39(10):2008-11.
Kontzias A, Efthimiou P. The use of Canakinumab, a novel IL-1β long-acting inhibitor, in refractory adult-onset Still’s disease. Semin Arthritis Rheum. 2012 Oct;42(2):201-5.
Quartier P, Allantaz F, Cimaz R et al. A multicentre, randomised, double-blind, placebo-controlled trial with the interleukin-1 receptor antagonist anakinra in patients with systemic-onset juvenile idiopathic arthritis (ANAJIS trial). Ann Rheum Dis. 2011;70:747-54.
Ruperto N, Brunner HI, Quartier P et al. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med. 2012;367:2396-406.
De Benedetti F, Brunner HI, Ruperto N et al. Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis. N Engl J Med. 2012 Dec 20;367(25):2385-95.
Otten MH, Anink J, Spronk S, van Suijlekom-Smit LW. Efficacy of biological agents in juvenile idiopathic arthritis: a systematic review using indirect comparisons. Ann Rheum Dis. 2013 Nov;72(11):1806-12.
Sota J, Vitale A, Insalaco I et al. Safety profile of the interleukin-1 inhibitors anakinra and canakinumab in real-life clinical practice: a nationwide multicenter retrospective observational study. Clin Rheumatol. 2018 Aug;37(8):2233-40.
Donath MY, Dinarello CA, Mandrup-Poulsen T. targeting innate immune mediators in type 1 and type 2 diabetes. Nat Rev Immunol. 2019 Dec;19(12):734-46.
Bettiol A, Lopalco G, Emmi G et al. Unveiling the efficacy, safety, and tolerability of anti-interleukin-1 treatment in monogenic and multifactorial autoinflammatory diseases. Int J Mol Sci. 2019 Apr 17;20(8):1898.
Nota AIFA RoActemra, 27 giugno 2019. http://www.agenziafarmaco.gov.it/content/nota-informativa-importante-su-roactemra%C2%AE-tocilizumab-27062019
Center for Disease Control and Prevention (CDC). https://www.cdc.gov/growthcharts/clinical_charts.htm Accesso Dicembre 2019.
How to Cite
Copyright (c) 2020 The authors
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Authors contributing to Global & Regional Health Technology Assessment agree to publish their articles under the CC-BY-NC 4.0 license, which allows third parties to re-use the work without permission as long as the work is properly referenced and the use is non-commercial.