Cost per Number Needed to Treat (NNT) for upadacitinib in the treatment of bio-exposed patients with moderately-severely active ulcerative rectocolitis

Authors

  • Flavio Caprioli Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milano - Italy Gastroenterology and Endoscopy Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico di Milano, Milano - Italy
  • Massimo Claudio Fantini epartment of Medical Science and Public Health, University of Cagliari, Cagliari - Italy Azienda Ospedaliero-Universitaria (AOU) di Cagliari, Cagliari - Italy https://orcid.org/0000-0003-2870-3827
  • Francesca Marando AbbVie, Roma - Italy
  • Dario Scaduto AbbVie, Roma - Italy https://orcid.org/0009-0004-8863-0249
  • Roberto Ravasio HEOR and RWE Lead, PharmaLex Italy S.p.A. formerly MA Provider, Milano - Italy https://orcid.org/0000-0002-5477-1957

DOI:

https://doi.org/10.33393/grhta.2024.2658

Keywords:

Bio-exposed, Cost per NNT, Italian NHS, Ulcerative Rectocolitis, Upadacitinib

Abstract

Background: Only limited information is available on cost efficacy of the advanced therapies for the treatment of ulcerative rectocolitis. We evaluated the efficacy and the treatment costs of these advanced therapies in the treatment of bio-exposed (primary failure to a biological agent) patients with moderately to severely active ulcerative rectocolitis.

Methods: Data from a previous network meta-analysis (NMA) in the treatment of bio-exposed patients with moderately to severely active ulcerative rectocolitis was used. The analysis involved three therapies approved in Italy for the treatment of moderately to severely active ulcerative rectocolitis: upadacitinib (UPA), ustekinumab (UST) and vedolizumab (VDZ). According to the NMA results, the analysis was conducted considering two different timepoints: induction and maintenance. The pharmacoeconomic comparison between advanced therapies was carried out to estimate the respective cost for the NNT (Number Needed to Treat) compared to placebo using the clinical response (CRes) for the induction and clinical remission (CRem) and endoscopic improvement (EI) for the maintenance. Only the ex-factory price of each advanced therapy was considered. The cost per NNT was adopted as a cost-effectiveness indicator.

Results: Independently of the timepoint considered (induction or maintenance) and the clinical (CRem and CRes) or endoscopic endpoint (EI) evaluated, UPA had always the lower cost per NNT than UST and VDZ. For example, considering the induction and the CRes, the cost per NNT for upadacitinib (45 mg) was € 7,862.07 compared to € 30,459.38 for ustekinumab (6 mg/kg) and to € 67,868.00 for vedolizumab.

Conclusion: Upadacitinib is a cost-effective therapeutic option compared to ustekinumab and vedolizumab in the treatment of bio-exposed patients with moderately to severely active ulcerative rectocolitis.

Downloads

Download data is not yet available.

References

Burr NE, Gracie DJ, Black CJ, Ford AC. Efficacy of biological therapies and small molecules in moderate to severe ulcerative colitis: systematic review and network meta-analysis. Gut. 2021 Dec 22:gutjnl-2021-326390. https://doi.org/10.1136/gutjnl-2021-326390 PMID: 34937767

Ungaro R, Mehandru S, Allen PB, Peyrin-Biroulet L, Colombel J-F. Ulcerative colitis. Lancet. 2017;389(10080):1756-1770. https://doi.org/10.1016/S0140-6736(16)32126-2 PMID:27914657 DOI: https://doi.org/10.1016/S0140-6736(16)32126-2

Lynch WD and Hsu R. Ulcerative Colitis. StatPearls. Last update June 5, 2023. https://www.ncbi.nlm.nih.gov/books/NBK459282/

Cohen RD, Yu AP, Wu EQ, Xie J, Mulani PM, Chao J. Systematic review: the costs of ulcerative colitis in Western countries. Aliment Pharmacol Ther. 2010;31(7):693-707. https://doi.org/10.1111/j.1365-2036.2010.04234.x PMID:20064142 DOI: https://doi.org/10.1111/j.1365-2036.2010.04234.x

Paschos P, Katsoula A, Salanti G, Giouleme O, Athanasiadou E, Tsapas A. Systematic review with network meta-analysis: the impact of medical interventions for moderate-to-severe ulcerative colitis on health-related quality of life. Aliment Pharmacol Ther. 2018;48(11-12):1174-1185. https://doi.org/10.1111/apt.15005 PMID:30378141 DOI: https://doi.org/10.1111/apt.15005

Gray JR, Leung E, Scales J. Treatment of ulcerative colitis from the patient’s perspective: a survey of preferences and satisfaction with therapy. Aliment Pharmacol Ther. 2009;29(10):1114-1120. https://doi.org/10.1111/j.1365-2036.2009.03972.x PMID:19222412 DOI: https://doi.org/10.1111/j.1365-2036.2009.03972.x

Peyrin-Biroulet L, Van Assche G, Sturm A, et al. Treatment satisfaction, preferences and perception gaps between patients and physicians in the ulcerative colitis CARES study: A real world-based study. Dig Liver Dis. 2016;48(6):601-607. https://doi.org/10.1016/j.dld.2016.01.013 PMID:27012447 DOI: https://doi.org/10.1016/j.dld.2016.01.013

Turner D, Ricciuto A, Lewis A, et al; International Organization for the Study of IBD. STRIDE-II: An Update on the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) Initiative of the International Organization for the Study of IBD (IOIBD): Determining Therapeutic Goals for Treat-to-Target strategies in IBD. Gastroenterology. 2021 Apr;160(5):1570-1583. https://doi.org/10.1053/j.gastro.2020.12.031. PMID: 33359090 DOI: https://doi.org/10.1053/j.gastro.2020.12.031

Yoon H, Jangi S, Dulai PS, et al. Incremental Benefit of Achieving Endoscopic and Histologic Remission in Patients With Ulcerative Colitis: A Systematic Review and Meta-Analysis. Gastroenterology. 2020;159(4):1262-1275.e7. https://doi.org/10.1053/j.gastro.2020.06.043 PMID:32585306 DOI: https://doi.org/10.1053/j.gastro.2020.06.043

European Medicines Agency (EMA). Riassunto delle caratteristiche di prodotto Entyvio. https://www.ema.europa.eu/en/documents/product-information/entyvio-epar-product-information_it.pdf

European Medicines Agency (EMA). Riassunto delle caratteristiche di prodotto Stelara. https://www.ema.europa.eu/en/documents/product-information/stelara-epar-product-information_it.pdf

Roda G, Jharap B, Neeraj N, Colombel JF. Loss of response to anti-TNFs: definition, epidemiology, and management. Clin Transl Gastroenterol. 2016;7(1):e135. https://doi.org/10.1038/ctg.2015.63 PMID:26741065 DOI: https://doi.org/10.1038/ctg.2015.63

Harris C, Cummings JRF. JAK1 inhibition and inflammatory bowel disease. Rheumatology (Oxford). 2021;60(suppl 2):ii45-ii51. https://doi.org/10.1093/rheumatology/keaa896 PMID:33950226 DOI: https://doi.org/10.1093/rheumatology/keaa896

Agenzia Italiana del Farmaco (AIFA). Riassunto delle caratteristiche di prodotto Rinvoq. https://farmaci.agenziafarmaco.gov.it/aifa/servlet/PdfDownloadServlet?pdfFileName=footer_004726_048399_RCP.pdf&sys=m0b1l3

Danese S, Vermeire S, Zhou W, et al. OP24 Efficacy and safety of upadacitinib induction therapy in patients with Moderately to Severely Active Ulcerative Colitis: results from the phase 3 U-ACHIEVE study. J Crohns Colitis. 2021;15(suppl 1):S022-S024. https://doi.org/10.1093/ecco-jcc/jjab075.023 DOI: https://doi.org/10.1093/ecco-jcc/jjab075.023

Danese S, Vermeire S, Zhou W, et al. Upadacitinib as induction and maintenance therapy for moderately to severely active ulcerative colitis: results from three phase 3, multicentre, double-blind, randomised trials. Lancet. 2022;399(10341):2113-2128. https://doi.org/10.1016/S0140-6736(22)00581-5 PMID:35644166 DOI: https://doi.org/10.1016/S0140-6736(22)00581-5

A Study to Evaluate the Long-Term Safety and Efficacy of Upadacitinib (ABT-494) in Participants With Ulcerative Colitis (UC). https://clinicaltrials.gov/study/NCT03006068

Market Research. AbbVie Data on file

Panaccione R, Collins EB, Melmed GY, et al. Efficacy and Safety of Advanced Therapies for Moderately to Severely Active Ulcerative Colitis at Induction and Maintenance: An Indirect Treatment Comparison Using Bayesian Network Meta-analysis. Crohns Colitis 360. 2023;5(2):otad009. https://doi.org/10.1093/crocol/otad009 PMID:36998249 DOI: https://doi.org/10.1093/crocol/otad009

Schroeder KW, Tremaine WJ, Ilstrup DM. Coated oral 5-aminosalicylic acid therapy for mildly to moderately active ulcerative colitis. A randomized study. N Engl J Med. 1987;317(26):1625-1629. https://doi.org/10.1056/NEJM198712243172603 PMID:3317057 DOI: https://doi.org/10.1056/NEJM198712243172603

Salanti G, Ades AE, Ioannidis JP. Graphical methods and numerical summaries for presenting results from multiple-treatment meta-analysis: an overview and tutorial. J Clin Epidemiol. 2011;64(2):163-171. https://doi.org/10.1016/j.jclinepi.2010.03.016 PMID:20688472 DOI: https://doi.org/10.1016/j.jclinepi.2010.03.016

Agenzia Italiana del Farmaco (AIFA). Linee guida per la compilazione del Dossier a supporto della domanda di rimborsabilità e prezzo di un medicinale. https://www.aifa.gov.it/documents/20142/0/AIFA_Linee+Guida_v.+16.9.2020+per+consultazione+pubblica.pdf/64f8d5b5-69df-a799-9ae7-36a5743d5f17

Chiappetta MF, Viola A, Mastronardi M, et al. One-year effectiveness and safety of ustekinumab in ulcerative colitis: a multicenter real-world study from Italy. Expert Opin Biol Ther. 2021;21(11):1483-1489. https://doi.org/10.1080/14712598.2021.1981855 PMID:34521307 DOI: https://doi.org/10.1080/14712598.2021.1981855

Panaccione R, Lee WJ, Clark R, et al. Dose Escalation Patterns of Advanced Therapies in Crohn’s Disease and Ulcerative Colitis: A Systematic Literature Review. Adv Ther. 2023;40(5):2051-2081. https://doi.org/10.1007/s12325-023-02457-6 PMID:36930430 DOI: https://doi.org/10.1007/s12325-023-02457-6

Zingone F, Barberio B, Compostella F, et al. Good efficacy and safety of vedolizumab in Crohn’s disease and ulcerative colitis in a real-world scenario. Therap Adv Gastroenterol. 2020;13:1756284820936536. https://doi.org/10.1177/1756284820936536 PMID:32695231 DOI: https://doi.org/10.1177/1756284820936536

Macaluso FS, Fries W, Renna S, et al; Sicilian Network for Inflammatory Bowel Disease (SN-IBD). Effectiveness and safety of vedolizumab in biologically naïve patients: A real-world multi-centre study. United European Gastroenterol J. 2020;8(9):1045-1055. https://doi.org/10.1177/2050640620948802 PMID:32772830 DOI: https://doi.org/10.1177/2050640620948802

Elenco dei medicinali di classe a) rimborsabili dal Servizio sanitario nazionale (SSN) ai sensi dell'articolo 48, comma 5, lettera c), del decreto-legge 30 settembre 2003, n. 269, convertito, con modificazioni, nella legge 24 novembre 2006, n. 326. (Prontuario farmaceutico nazionale 2006). (GU Serie Generale n.156 del 07-07-2006 - Suppl. Ordinario n. 161) https://www.gazzettaufficiale.it/atto/serie_generale/caricaDettaglioAtto/originario?atto.dataPubblicazioneGazzetta=2006-07-07&atto.codiceRedazionale=06A06166

G.U. serie generale n° 154 del 30 giugno 2021. https://www.gazzettaufficiale.it/eli/gu/2021/06/30/154/sg/pdf

Greco T, Biondi-Zoccai G, Saleh O, et al. The attractiveness of network meta-analysis: a comprehensive systematic and narrative review. Heart Lung Vessel. 2015;7(2):133-142. PMID:26157739

Greco T, Landoni G, Biondi-Zoccai G, D’Ascenzo F, Zangrillo A. A Bayesian network meta-analysis for binary outcome: how to do it. Stat Methods Med Res. 2016;25(5):1757-1773. https://doi.org/10.1177/0962280213500185 PMID:23970014 DOI: https://doi.org/10.1177/0962280213500185

Lasa JS, Olivera PA, Danese S, Peyrin-Biroulet L. Efficacy and safety of biologics and small molecule drugs for patients with moderate-to-severe ulcerative colitis: a systematic review and network meta-analysis. Lancet Gastroenterol Hepatol. 2022;7(2):161-170. https://doi.org/10.1016/S2468-1253(21)00377-0 PMID:34856198 DOI: https://doi.org/10.1016/S2468-1253(21)00377-0

Burr NE, Gracie DJ, Black CJ, Ford AC. Efficacy of biological therapies and small molecules in moderate to severe ulcerative colitis: systematic review and network meta-analysis. Gut. 2021 Dec 22:gutjnl-2021-326390. https://doi.org/10.1136/gutjnl-2021-326390 PMID:34937767 DOI: https://doi.org/10.1136/gutjnl-2021-326390

Ahuja D, Murad MH, Ma C, Jairath V, Singh S. Comparative Speed of Early Symptomatic Remission With Advanced Therapies for Moderate-to-Severe Ulcerative Colitis: A Systematic Review and Network Meta-Analysis. Am J Gastroenterol. 2023;118(9):1618-1625; Online ahead of print. https://doi.org/10.14309/ajg.0000000000002263 PMID:36976548 DOI: https://doi.org/10.14309/ajg.0000000000002263

Published

2024-03-07

How to Cite

Caprioli, F., Fantini, M. C., Marando, F., Scaduto, D., & Ravasio, R. (2024). Cost per Number Needed to Treat (NNT) for upadacitinib in the treatment of bio-exposed patients with moderately-severely active ulcerative rectocolitis. Global and Regional Health Technology Assessment, 11(1), 55–67. https://doi.org/10.33393/grhta.2024.2658

Issue

Section

Original Research Articles

Categories

Received 2023-09-08
Accepted 2024-02-14
Published 2024-03-07

Metrics

Most read articles by the same author(s)

1 2 3 4 > >>