Cost-effectiveness and cost-utility of optimized mercaptopurine treatment versus placebo in ulcerative colitis patients: The randomized controlled OPTIC trial.

6‐mercaptopurine IBD TDM costs inflammatory bowel disease of therapeutic drug monitoring thiopurines

Journal

United European gastroenterology journal
ISSN: 2050-6414
Titre abrégé: United European Gastroenterol J
Pays: England
ID NLM: 101606807

Informations de publication

Date de publication:
13 Oct 2024
Historique:
received: 09 05 2024
accepted: 21 08 2024
medline: 14 10 2024
pubmed: 14 10 2024
entrez: 14 10 2024
Statut: aheadofprint

Résumé

We assessed the cost-effectiveness and cost-utility of therapeutic drug monitoring (TDM)-guided mercaptopurine treatment compared with placebo in ulcerative colitis (UC) patients failing 5-aminosalicylates. Data were gathered alongside the randomized controlled OPTIC trial (EudraCT: 2015-005260-41). The evaluation was performed from a health care and societal perspective as cost-effectiveness and cost-utility analyses with a time horizon of one year. Volumes and costs of in-hospital care, out-of-hospital care, out-of-pocket expenses and productivity loss were assessed. The main outcomes were the extra costs per additional patient who achieved clinical remission and endoscopic improvement at 52 weeks (responders) and extra costs per quality-adjusted life-year (QALY) gained. In total, 59 patients were randomized to the intervention (n = 29) and control (n = 30) group. Non-significant differences in costs were €63 (-€1267 to €1434; P = 0.93) in favour of placebo from a health care perspective and -€742 (-€3683 to €2016; P = 0.64) in favour of mercaptopurine from a societal perspective. The higher proportion of responders and a non-significant QALY difference of 0.0475 (-0.024-0.117) (P = 0.184) favouring patients on mercaptopurine treatment resulted in €165 extra costs per additional responder and €1326 extra costs per QALY gained from a health care perspective. From a societal perspective, dominance over placebo was observed with cost savings of €1937 per additional responder and €15,621 per QALY gained. The probability of optimised mercaptopurine treatment being cost-effective was 0.80 at a willingness to pay per additional QALY of €20,000. TDM-based mercaptopurine treatment in UC patients failing 5-aminosalicylates is a cost-effective strategy from a societal perspective.

Sections du résumé

BACKGROUND AND AIMS OBJECTIVE
We assessed the cost-effectiveness and cost-utility of therapeutic drug monitoring (TDM)-guided mercaptopurine treatment compared with placebo in ulcerative colitis (UC) patients failing 5-aminosalicylates.
METHODS METHODS
Data were gathered alongside the randomized controlled OPTIC trial (EudraCT: 2015-005260-41). The evaluation was performed from a health care and societal perspective as cost-effectiveness and cost-utility analyses with a time horizon of one year. Volumes and costs of in-hospital care, out-of-hospital care, out-of-pocket expenses and productivity loss were assessed. The main outcomes were the extra costs per additional patient who achieved clinical remission and endoscopic improvement at 52 weeks (responders) and extra costs per quality-adjusted life-year (QALY) gained.
RESULTS RESULTS
In total, 59 patients were randomized to the intervention (n = 29) and control (n = 30) group. Non-significant differences in costs were €63 (-€1267 to €1434; P = 0.93) in favour of placebo from a health care perspective and -€742 (-€3683 to €2016; P = 0.64) in favour of mercaptopurine from a societal perspective. The higher proportion of responders and a non-significant QALY difference of 0.0475 (-0.024-0.117) (P = 0.184) favouring patients on mercaptopurine treatment resulted in €165 extra costs per additional responder and €1326 extra costs per QALY gained from a health care perspective. From a societal perspective, dominance over placebo was observed with cost savings of €1937 per additional responder and €15,621 per QALY gained. The probability of optimised mercaptopurine treatment being cost-effective was 0.80 at a willingness to pay per additional QALY of €20,000.
CONCLUSIONS CONCLUSIONS
TDM-based mercaptopurine treatment in UC patients failing 5-aminosalicylates is a cost-effective strategy from a societal perspective.

Identifiants

pubmed: 39400525
doi: 10.1002/ueg2.12661
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : ZonMw
ID : 836041002
Pays : Netherlands

Informations de copyright

© 2024 The Author(s). United European Gastroenterology Journal published by Wiley Periodicals LLC on behalf of United European Gastroenterology.

Références

de Boer NKH, Peyrin‐Biroulet L, Jharap B, Sanderson JD, Meijer B, Atreya I, et al. Thiopurines in inflammatory bowel disease: new findings and perspectives. J Crohns Colitis. 2018;12(5):610–620. https://doi.org/10.1093/ecco‐jcc/jjx181
Löwenberg M, Volkers A, van Gennep S, Mookhoek A, Montazeri N, Clasquin E, et al. Mercaptopurine for the treatment of ulcerative colitis: a randomized placebo‐controlled trial. J Crohns Colitis. 2023;17(7):1055–1065. https://doi.org/10.1093/ecco‐jcc/jjad022
de Raad voor Volksgezondheid & Zorg. Zinnige en duurzame zorg: transparante keuze in de zorg voor een houdbaar zorgstelsel. Advies Uitgebracht Door de RVZ Aan de Minister van Volksgezondheid, Welzijn en Sport Zoetermeer: RVZ. 2006.
Zaap J, Knies S, van der Meijden C, Staal P, van der Heiden L. Kosteneffectiviteit in de praktijk, 26. Diemen: Zorginstituut Nederland; 2015. juni.
Husereau D, Drummond M, Augustovski F, de Bekker‐Grob E, Briggs AH, Carswell C, et al. Consolidated health economic evaluation reporting standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR CHEERS II good practies task force. Value Health. 2022;25(1):10–31. https://doi.org/10.1016/j.jval.2021.10.008
Bouwmans C, Hakkaart‐van Roijen L, Koopmanschap M, Krol M, Severens H, Brouwer W. iMTA medical consumption questionnaire. Rotterdam: Erasmus University; 2013.
Bouwmans C, Hakkaart‐van Roijen L, Koopmanschap M, Krol M, Severens H, Brouwer W. iMTA productivity cost questionnaire. Rotterdam: Erasmus University; 2013.
Hakkaart‐van Roijen L, Peeters S, Kanters T. [Revisited costing manual for economic evaluations in healthcare: methodology and reference prices]. Rotterdam: Erasmus University; 2024.
Amsterdam UMC. Unit costing sheet, 2022.
Koopmanschap MA, Rutten FFH. A practical guide for calculating indirect costs of disease. Pharmacoeconomics. 1996;10(5):460–466. https://doi.org/10.2165/00019053‐199610050‐00003
Unit costs for medication use. https://www.medicijnkosten.nl Accessed December 25, 2023.
Yearly general consumer price indices. https://www.cbs.nl/nl‐nl/cijfers/detail/83131NED Accessed December 15, 2023.
Versteegh M, Vermeulen K, Evers S, Wit G, Prenger R, Stolk E. Dutch tariff for the five‐level version of EQ‐5D. Value Health. 2016;19:343–352.
Barber JA, Thompson SG. Analysis of cost data in randomized trials: an application of the non‐parametric bootstrap. Stat Med. 2000;19(23):3219–3236. https://doi.org/10.1002/1097‐0258(20001215)19:23<3219::aid‐sim623>3.0.co;2‐p
Black WC. The CE plane: a graphic representation of cost‐effectiveness. Med Decis Making. 1990;10(3):212–214. https://doi.org/10.1177/0272989x9001000308
Fenwick E, Claxton K, Sculpher M. Representing uncertainty: the role of cost‐effectiveness acceptability curves. Health Econ. 2001;10(8):779–787. https://doi.org/10.1002/hec.635
Jeuring SFG, van den Heuvel TRA, Zeegers MP, Hameeteman WH, Romberg‐Camps MJL, Oostenbrug LE, et al. Epidemiology and long‐term outcome of inflammatory bowel disease diagnosed at elderly age. An increasing distinct entity? Inflamm Bowel Dis. 2016;22(6):1425–1434. https://doi.org/10.1097/mib.0000000000000738
Timmer A, Patton PH, Chande N, McDonald JWD, MacDonald JK. Azathioprine and 6‐mercaptopurine for maintenance of remission in ulcerative colitis. Cochrane Database Syst Rev. 2016;5:CD000478. https://doi.org/10.1002/14651858.cd000478.pub4
Total number of patients in The Netherlands who received a prescription for azathioprine. https://www.gipdatabank.nl/databank?infotype=g&label=00‐totaal&tabel=B_01‐basis&geg=gebr&item=L04AX Accessed April 10, 2024.
Total number of patients in The Netherlands who received a prescription for mercaptopurine. https://www.gipdatabank.nl/databank?infotype=g&label=00‐totaal&tabel=B_01‐basis&geg=gebr&item=L01BB Accessed April 10, 2024.
De Groof EJ, Rossen NGM, Van Rhijn BD, Karregat EP, Boonstra K, Hageman I, et al. Burden of disease and increasing prevalence of inflammatory bowel disease in a population‐based cohort in The Netherlands. Eur J Gastro & Hep. 2016;28(9):1065–1072. https://doi.org/10.1097/meg.0000000000000660
Number of inhabitants in The Netherlands. https://www.cbs.nl/nl‐nl/visualisaties/dashboard‐bevolking/bevolkingsteller Accessed March 3, 2024.
Eurostat methodologies and working papers. Eurostat‐OECD Methodological manual on purchasing power parities. Luxembourg: Publications Office of the European Union, 2012.

Auteurs

Mark Löwenberg (M)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Department of Gastroenterology and Hepatology, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Amsterdam University Medical Center, VU University Amsterdam, Amsterdam, The Netherlands.

Marit van Barreveld (M)

Department of Epidemiology and Data Science, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Methodology, Amsterdam Public Health, University of Amsterdam, Amsterdam, The Netherlands.

Adriaan Volkers (A)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.

Sara van Gennep (S)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.

Marjolijn Duijvestein (M)

Department of Gastroenterology and Hepatology, Radboudumc, Nijmegen, The Netherlands.

Adriaan A van Bodegraven (AA)

Department of Gastroenterology, Zuyderland Medical Centre and Maastricht University Medical Center, Sittard, The Netherlands.

Melanie S Hulshoff (MS)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.

Jeroen M Jansen (JM)

Department of Gastroenterology and Hepatology, OLVG, Amsterdam, The Netherlands.

Dirk van Asseldonk (D)

Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands.

Rachel West (R)

Department of Gastroenterology and Hepatology, Franciscus Gasthuis, Rotterdam, The Netherlands.

Geert D'Haens (G)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Department of Gastroenterology and Hepatology, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Amsterdam University Medical Center, VU University Amsterdam, Amsterdam, The Netherlands.

Nanne de Boer (N)

Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Department of Gastroenterology and Hepatology, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Amsterdam University Medical Center, VU University Amsterdam, Amsterdam, The Netherlands.

Marcel G W Dijkgraaf (MGW)

Department of Epidemiology and Data Science, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Methodology, Amsterdam Public Health, University of Amsterdam, Amsterdam, The Netherlands.

Classifications MeSH