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Development of a Decision Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma

Author

Listed:
  • Saskia de Groot

    (Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, The Netherlands
    Erasmus School of Health Policy and Management, Erasmus University Rotterdam, The Netherlands)

  • Hedwig M. Blommestein

    (Erasmus School of Health Policy and Management, Erasmus University Rotterdam, The Netherlands)

  • Brenda Leeneman

    (Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, The Netherlands
    Erasmus School of Health Policy and Management, Erasmus University Rotterdam, The Netherlands)

  • Carin A. Uyl-de Groot

    (Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, The Netherlands
    Erasmus School of Health Policy and Management, Erasmus University Rotterdam, The Netherlands)

  • John B. A. G. Haanen

    (Division of Medical Oncology, Netherlands Cancer Institute, Amsterdam, The Netherlands)

  • Michel W. J. M. Wouters

    (Scientific Bureau, Dutch Institute for Clinical Auditing, Leiden, The Netherlands
    Department of Surgical Oncology, Netherlands Cancer Institute, Antoni van Leeuwenhoek, Amsterdam, The Netherlands
    Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands)

  • Maureen J. B. Aarts

    (Department of Medical Oncology, GROW School for Oncology and Developmental Biology, Maastricht University Medical Center+, Maastricht, The Netherlands)

  • Franchette W. P. J. van den Berkmortel

    (Department of Medical Oncology, Zuyderland Medical Center Sittard, Sittard-Geleen, The Netherlands)

  • Willeke A. M. Blokx

    (Department of Pathology, Division of Laboratories, Pharmacy and Biomedical Genetics, University Medical Center Utrecht, Utrecht, The Netherlands)

  • Marye J. Boers-Sonderen

    (Department of Medical Oncology, Radboud University Medical Center, Nijmegen, The Netherlands)

  • Alfons J. M. van den Eertwegh

    (Department of Medical Oncology, Amsterdam UMC, VU University Medical Center, Cancer Center Amsterdam, Amsterdam, The Netherlands)

  • Jan Willem B. de Groot

    (Isala Oncology Center, Isala, Zwolle, The Netherlands)

  • Geke A. P. Hospers

    (Department of Medical Oncology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands)

  • Ellen Kapiteijn

    (Department of Medical Oncology, Leiden University Medical Center, Leiden, The Netherlands)

  • Olivier J. van Not

    (Scientific Bureau, Dutch Institute for Clinical Auditing, Leiden, The Netherlands
    Department of Medical Oncology, University Medical Center Utrecht, Utrecht, The Netherlands)

  • Astrid A. M. van der Veldt

    (Department of Medical Oncology and Radiology & Nuclear Medicine, Erasmus Medical Center, Rotterdam, The Netherlands)

  • Karijn P. M. Suijkerbuijk

    (Department of Medical Oncology, University Medical Center Utrecht, Utrecht, The Netherlands)

  • Pieter H. M. van Baal

    (Erasmus School of Health Policy and Management, Erasmus University Rotterdam, The Netherlands)

Abstract

Background A decision model for patients with advanced melanoma to estimate outcomes of a wide range of treatment sequences is lacking. Objectives To develop a decision model for advanced melanoma to estimate outcomes of treatment sequences in clinical practice with the aim of supporting decision making. The article focuses on methodology and long-term health benefits. Methods A semi-Markov model with a lifetime horizon was developed. Transitions describing disease progression, time to next treatment, and mortality were estimated from real-world data (RWD) as a function of time since starting treatment or disease progression and patient characteristics. Transitions were estimated separately for melanoma with and without a BRAF mutation and for patients with favorable and intermediate prognostic factors. All transitions can be adjusted using relative effectiveness of treatments derived from a network meta-analysis of randomized controlled trials (RCTs). The duration of treatment effect can be adjusted to obtain outcomes under different assumptions. Results The model distinguishes 3 lines of systemic treatment for melanoma with a BRAF mutation and 2 lines of systemic treatment for melanoma without a BRAF mutation. Life expectancy ranged from 7.8 to 12.0 years in patients with favorable prognostic factors and from 5.1 to 8.7 years in patients with intermediate prognostic factors when treated with sequences consisting of targeted therapies and immunotherapies. Scenario analyses illustrate how estimates of life expectancy depend on the duration of treatment effect. Conclusion The model is flexible because it can accommodate different treatments and treatment sequences, and the duration of treatment effects and the transitions influenced by treatment can be adjusted. We show how using RWD and data from RCTs can harness advantages of both data sources, guiding the development of future decision models. Highlights The model is flexible because it can accommodate different treatments and treatment sequences, and the duration of treatment effects as well as the transitions that are influenced by treatment can be adjusted. The long-term health benefits of treatment sequences depend on the place of different therapies within a treatment sequence. Assumptions about the duration of relative treatment effects influence the estimates of long-term health benefits. We show how the use of real-world data and data from randomized controlled trials harness the advantages of both data sources, guiding the development of future decision models.

Suggested Citation

  • Saskia de Groot & Hedwig M. Blommestein & Brenda Leeneman & Carin A. Uyl-de Groot & John B. A. G. Haanen & Michel W. J. M. Wouters & Maureen J. B. Aarts & Franchette W. P. J. van den Berkmortel & Will, 2025. "Development of a Decision Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma," Medical Decision Making, , vol. 45(3), pages 302-317, April.
    • Handle: RePEc:sae:medema:v:45:y:2025:i:3:p:302-317
    DOI: 10.1177/0272989X251319338
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    References listed on IDEAS

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    1. Mark J. Sculpher & Karl Claxton & Mike Drummond & Chris McCabe, 2006. "Whither trial‐based economic evaluation for health care decision making?," Health Economics, John Wiley & Sons, Ltd., vol. 15(7), pages 677-687, July.
    2. Briggs, Andrew & Sculpher, Mark & Claxton, Karl, 2006. "Decision Modelling for Health Economic Evaluation," OUP Catalogue, Oxford University Press, number 9780198526629.
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