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

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 Ramachandran and John L. Gore
Additional contact information
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

The Patient: Patient-Centered Outcomes Research, 2025, vol. 18, issue 1, No 7, 77-87

Abstract: 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.

Date: 2025
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s40271-024-00709-3 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:patien:v:18:y:2025:i:1:d:10.1007_s40271-024-00709-3

Ordering information: This journal article can be ordered from
http://www.springer.com/economics/journal/40271

DOI: 10.1007/s40271-024-00709-3

Access Statistics for this article

The Patient: Patient-Centered Outcomes Research is currently edited by Christopher I. Carswell

More articles in The Patient: Patient-Centered Outcomes Research from Springer, International Academy of Health Preference Research
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().