Elicitation of Health State Utilities Associated with Progression from Bacillus Calmette-Guerin (BCG) Unresponsive Non-muscle Invasive Bladder Cancer (NMIBC)

Owen Alan Edwards Cooper; Ola Ghatnekar; Natalia Piglowska; Charlie A Smith; Paul Swinburn; James W F Catto; Günter Niegisch; Jørn Skibsted Jakobsen

doi:10.1007/s41669-023-00392-4

Elicitation of Health State Utilities Associated with Progression from Bacillus Calmette-Guerin (BCG) Unresponsive Non-muscle Invasive Bladder Cancer (NMIBC)

Pharmacoecon Open. 2023 May;7(3):469-477. doi: 10.1007/s41669-023-00392-4. Epub 2023 Feb 3.

Authors

Owen Alan Edwards Cooper¹, Ola Ghatnekar², Natalia Piglowska¹, Charlie A Smith¹, Paul Swinburn³, James W F Catto⁴, Günter Niegisch⁵, Jørn Skibsted Jakobsen²

Affiliations

¹ Evidera by PPD, London, UK.
² Ferring Pharmaceuticals A/S, Copenhagen, Denmark.
³ Evidera by PPD, London, UK. Paul.Swinburn@evidera.com.
⁴ Department of Oncology and Metabolism, University of Sheffield, Sheffield, UK.
⁵ Medical Faculty and University Hospital Düsseldorf, Heinrich Heine University, Düsseldorf, Germany.

Abstract

Objectives: The treatments for high-grade non-muscle invasive bladder cancer (NMIBC) vary between bladder preserving intravesical approaches and radical cystectomy. The impact of these treatments on health-related quality of life may vary widely. The purpose of this study was to elicit the general public's perspective on quality of life, measured as utility scores associated with treatment for Bacillus Calmette-Guerin (BCG)-unresponsive NMIBC and disease progression, for supporting economic evaluation of newly developed treatments for NMIBC.

Materials and methods: Part I involved the development and testing of health states describing NMIBC, which was informed by a rapid review, expert input and a patient advisor. Part II involved elicitation of societal utility values for the different health states. Time trade-off (TTO) interviews were conducted with members of the UK general public. Five health states described different NMIBC scenarios including disease recurrence and progression. Participants ranked each health state, followed by the TTO valuation exercise. Descriptors included NMIBC symptom severity, impact and treatment characteristics.

Results: In total, 202 members of the general public participated. The mean age was 46 (standard deviation [SD] 14.6) years. Sample mean (SD) EQ-5D-5L visual analogue scale (VAS) and index scores were 83.2 (12.3) and 0.89 (0.18), respectively. Mean utilities were 0.781 for No High-Grade Recurrence, 0.586 for High-Grade Recurrence, 0.572 for > 1-Year Post-cystectomy and 0.283 for metastatic disease. The First Year Post-cystectomy path health state had a mean utility of 0.288. Pairwise comparisons found statistically significant differences between utilities (p < 0.001), except between High-Grade Recurrence and > 1-Year Post-cystectomy (p = 0.524). There were significant differences in utility scores by age and employment status.

Conclusion: This study provides utility scores for health states describing living with NMIBC, which is associated with a significant health-related quality-of-life burden. These values address an existing gap in available data and have the potential to be used in models evaluating the cost-effectiveness of both current and newly developed treatments for bladder cancer.