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

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

Owen Alan Edwards Cooper  1 , Ola Ghatnekar  2 , Natalia Piglowska  1 , Charlie A Smith  1 , Paul Swinburn  3 , James W F Catto  4 , Günter Niegisch  5 , Jørn Skibsted Jakobsen  2
Affiliations

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

Owen Alan Edwards Cooper et al. Pharmacoecon Open. 2023 May.

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.

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Conflict of interest statement

Owen Alan Edwards Cooper, Natalia Piglowska, Charlie Smith and Paul Swinburn are employees of Evidera PPD, a CRO paid by Ferring Pharmaceuticals A/S to conduct this study. Ola Ghatnekar and Jørn Skibsted Jakobsen are employees of Ferring Pharmaceuticals A/S. James Catto received honoraria for membership of advisory boards for Ferring Pharmaceuticals A/S. Günter Niegisch received consulting fees from Ferring Pharmaceuticals A/S.

Figures

Fig. 1
Fig. 1
Box plots of NMIBC health state utility scores. The box plot shows minimum, lower quartile, median, mean, upper quartile and maximum utility scores for each of the health states including outliers (defined as 1.5 times the interquartile range larger than the third quartile or 1.5 times the interquartile range smaller than the first quartile). MIBC muscle invasive bladder cancer, NMIBC non-muscle invasive bladder cancer
See this image and copyright information in PMC

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