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Validation of the Economic and Health Outcomes Model of Type 2 Diabetes Mellitus (ECHO-T2DM)

Author

Listed:
  • Michael Willis

    (The Swedish Institute for Health Economics)

  • Pierre Johansen

    (The Swedish Institute for Health Economics)

  • Andreas Nilsson

    (The Swedish Institute for Health Economics)

  • Christian Asseburg

    (The Swedish Institute for Health Economics)

Abstract

Background The Economic and Health Outcomes Model of Type 2 Diabetes Mellitus (ECHO-T2DM) was developed to address study questions pertaining to the cost-effectiveness of treatment alternatives in the care of patients with type 2 diabetes mellitus (T2DM). Naturally, the usefulness of a model is determined by the accuracy of its predictions. A previous version of ECHO-T2DM was validated against actual trial outcomes and the model predictions were generally accurate. However, there have been recent upgrades to the model, which modify model predictions and necessitate an update of the validation exercises. Objectives The objectives of this study were to extend the methods available for evaluating model validity, to conduct a formal model validation of ECHO-T2DM (version 2.3.0) in accordance with the principles espoused by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Society for Medical Decision Making (SMDM), and secondarily to evaluate the relative accuracy of four sets of macrovascular risk equations included in ECHO-T2DM. Methods We followed the ISPOR/SMDM guidelines on model validation, evaluating face validity, verification, cross-validation, and external validation. Model verification involved 297 ‘stress tests’, in which specific model inputs were modified systematically to ascertain correct model implementation. Cross-validation consisted of a comparison between ECHO-T2DM predictions and those of the seminal National Institutes of Health model. In external validation, study characteristics were entered into ECHO-T2DM to replicate the clinical results of 12 studies (including 17 patient populations), and model predictions were compared to observed values using established statistical techniques as well as measures of average prediction error, separately for the four sets of macrovascular risk equations supported in ECHO-T2DM. Sub-group analyses were conducted for dependent vs. independent outcomes and for microvascular vs. macrovascular vs. mortality endpoints. Results All stress tests were passed. ECHO-T2DM replicated the National Institutes of Health cost-effectiveness application with numerically similar results. In external validation of ECHO-T2DM, model predictions agreed well with observed clinical outcomes. For all sets of macrovascular risk equations, the results were close to the intercept and slope coefficients corresponding to a perfect match, resulting in high R 2 and failure to reject concordance using an F test. The results were similar for sub-groups of dependent and independent validation, with some degree of under-prediction of macrovascular events. Conclusion ECHO-T2DM continues to match health outcomes in clinical trials in T2DM, with prediction accuracy similar to other leading models of T2DM.

Suggested Citation

  • Michael Willis & Pierre Johansen & Andreas Nilsson & Christian Asseburg, 2017. "Validation of the Economic and Health Outcomes Model of Type 2 Diabetes Mellitus (ECHO-T2DM)," PharmacoEconomics, Springer, vol. 35(3), pages 375-396, March.
  • Handle: RePEc:spr:pharme:v:35:y:2017:i:3:d:10.1007_s40273-016-0471-3
    DOI: 10.1007/s40273-016-0471-3
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    References listed on IDEAS

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    1. Thornton, P. K. & Hansen, J. W., 1996. "A note on regressing real-world data on model output," Agricultural Systems, Elsevier, vol. 50(4), pages 411-414.
    2. Adam Lundqvist & Katarina Steen Carlsson & Pierre Johansen & Emelie Andersson & Michael Willis, 2014. "Validation of the IHE Cohort Model of Type 2 Diabetes and the Impact of Choice of Macrovascular Risk Equations," PLOS ONE, Public Library of Science, vol. 9(10), pages 1-12, October.
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    1. Michael Laxy & Verena Maria Schöning & Christoph Kurz & Rolf Holle & Annette Peters & Christa Meisinger & Wolfgang Rathmann & Kristin Mühlenbruch & Katharina Kähm, 2019. "Performance of the UKPDS Outcomes Model 2 for Predicting Death and Cardiovascular Events in Patients with Type 2 Diabetes Mellitus from a German Population-Based Cohort," PharmacoEconomics, Springer, vol. 37(12), pages 1485-1494, December.
    2. Michael Willis & Christian Asseburg & April Slee & Andreas Nilsson & Cheryl Neslusan, 2021. "Macrovascular Risk Equations Based on the CANVAS Program," PharmacoEconomics, Springer, vol. 39(4), pages 447-461, April.
    3. Joan Gil & Antoni Sicras-Mainar & Eugenio Zucchelli, 2018. "Uncontrolled diabetes and health care utilisation: panel data evidence from Spain," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 19(6), pages 785-795, July.
    4. Pierre Johansen & Jonas Håkan-Bloch & Aiden R. Liu & Peter G. Bech & Sofie Persson & Lawrence A. Leiter, 2019. "Cost Effectiveness of Once-Weekly Semaglutide Versus Once-Weekly Dulaglutide in the Treatment of Type 2 Diabetes in Canada," PharmacoEconomics - Open, Springer, vol. 3(4), pages 537-550, December.
    5. Sandra Lopes & Pierre Johansen & Mark Lamotte & Phil McEwan & Anamaria-Vera Olivieri & Volker Foos, 2020. "External Validation of the Core Obesity Model to Assess the Cost-Effectiveness of Weight Management Interventions," PharmacoEconomics, Springer, vol. 38(10), pages 1123-1133, October.
    6. Nasuh C. Büyükkaramikli & Maureen P. M. H. Rutten-van Mölken & Johan L. Severens & Maiwenn Al, 2019. "TECH-VER: A Verification Checklist to Reduce Errors in Models and Improve Their Credibility," PharmacoEconomics, Springer, vol. 37(11), pages 1391-1408, November.

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