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Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding

Author

Listed:
  • Andrea B. Apolo

    (National Cancer Institute, National Institutes of Health)

  • Christine Michaels-Igbokwe

    (Evidera)

  • Nicholas I. Simon

    (National Cancer Institute, National Institutes of Health)

  • David J. Benjamin

    (Hoag Family Cancer Institute)

  • Mallory Farrar

    (Pfizer Inc)

  • Zsolt Hepp

    (Pfizer Inc)

  • Lisa Mucha

    (Astellas Pharma, Inc)

  • Sebastian Heidenreich

    (Evidera)

  • Katelyn Cutts

    (Evidera)

  • Nicolas Krucien

    (Evidera)

  • Natasha Ramachandran

    (Evidera)

  • John L. Gore

    (University of Washington)

Abstract

Objectives Patient preferences have the potential to influence the development of new treatments for locally advanced/metastatic urothelial carcinoma (la/mUC), and therefore we explored how patients with la/mUC value different attributes of first-line treatments. Methods An online preference survey and multidimensional thresholding (MDT) exercise were developed following a targeted literature review and qualitative interviews with physicians, patients with la/mUC, and their caregivers. Treatment attributes included two benefits (overall response rate [ORR], pain related to bladder cancer [scored 0−100; 100 being the worst pain possible]) and four treatment-related risks (peripheral neuropathy, severe side effects, mild to moderate nausea, mild to moderate skin reactions). A Dirichlet regression was used to estimate average preference weights. Marginal utility and the reduction in ORR that patients would accept in exchange for a 10-point decrease or a 10% decrease in other attributes were calculated. Results A total of 100 patients were recruited and self-completed the survey and MDT. Mean patient age was 64.9 years (standard deviation, 7.6), 54% were female, and 38% identified as white. All included treatment attributes had a statistically significant impact on preferences. Changes in ORR had the largest impact, followed by cancer-related pain and treatment-related risks. Patients were willing to accept an 8.4% decrease in ORR to reduce their pain level by 10 points or a 7.8% decrease in ORR to reduce the risk of peripheral neuropathy by 10%. For a 10% decrease in severe side effects, mild to moderate nausea, or skin reaction, patients would accept decreases in ORR of 5.5%, 3.7%, or 3.4%, respectively. Conclusions Of the attributes tested, changes in ORR were most important to patients. Patients made tradeoffs between treatment attributes indicating that a lower ORR may be acceptable for an improvement in other attributes such as reduced cancer-related pain or the risk of treatment-related adverse events.

Suggested Citation

  • Andrea B. Apolo & Christine Michaels-Igbokwe & Nicholas I. Simon & David J. Benjamin & Mallory Farrar & Zsolt Hepp & Lisa Mucha & Sebastian Heidenreich & Katelyn Cutts & Nicolas Krucien & Natasha Rama, 2025. "Patient Preferences for First-Line Treatment of Locally Advanced or Metastatic Urothelial Carcinoma: An Application of Multidimensional Thresholding," The Patient: Patient-Centered Outcomes Research, Springer;International Academy of Health Preference Research, vol. 18(1), pages 77-87, January.
    • Handle: RePEc:spr:patien:v:18:y:2025:i:1:d:10.1007_s40271-024-00709-3
    DOI: 10.1007/s40271-024-00709-3
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    References listed on IDEAS

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    1. Arne Risa Hole, 2007. "A comparison of approaches to estimating confidence intervals for willingness to pay measures," Health Economics, John Wiley & Sons, Ltd., vol. 16(8), pages 827-840, August.
    2. Tommi Tervonen & Francesco Pignatti & Douwe Postmus, 2019. "From Individual to Population Preferences: Comparison of Discrete Choice and Dirichlet Models for Treatment Benefit-Risk Tradeoffs," Medical Decision Making, , vol. 39(7), pages 879-885, October.
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