Treatment options for patients with "bacillus Calmette-Guérin (BCG)-unresponsive" disease who are ineligible for or refuse radical cystectomy (RC) are expanding. Given the lack of direct comparative data, we conducted a cost-effectiveness analysis to guide treatment selection.
We developed a Markov decision analytic model to assess five treatments: RC, nadofaragene, nogapendekin, pembrolizumab, and gemcitabine/docetaxel. Cost effectiveness was evaluated over a 5-yr period using a willingness-to-pay threshold of $100 000 per quality-adjusted life year from the US Medicare perspective. Three index patients with BCG-unresponsive carcinoma in situ (CIS) were assessed.
For index patient 1, who is willing to try one line of therapy or proceed directly to RC, gemcitabine/docetaxel was the most cost-effective option. For index patient 2, open to two lines of therapy or upfront RC, RC was most cost effective. For index patient 3, willing to try up to two lines of US Food and Drug Administration (FDA)-approved therapy (pembrolizumab, nadofaragene, or nogapendekin) before RC, pembrolizumab was the most cost-effective option.
Current pricing of bladder-sparing treatments poses significant financial barriers for patients with BCG-unresponsive CIS. Gemcitabine/docetaxel is most cost effective when only one therapy line is considered before RC, although this varies by clinical scenario. Upfront RC is most cost effective for patients wanting to try up to two lines of therapy. For patients only willing to try FDA-approved options and unwilling to undergo upfront RC, pembrolizumab is the most cost-effective option. Our findings highlight the need for better treatment selection tools and more equitable pricing.
European urology. 2025 Oct 03 [Epub ahead of print]
Amanda A Myers, Ruchika Talwar, Zhigang Duan, Patrick Hensley, Yair Lotan, Stephen B Williams, Amy N Luckenbaugh, Roger Li, Wassim Kassouf, Andrea Necchi, Vignesh T Packiam, Neal Shore, Gary D Steinberg, J Alfred Witjes, Bogdana Schmidt, Sima Porten, Noah M Hahn, Kelly K Bree, Iakovos Toumazis, Hui Zhao, Ashish M Kamat
Department of Urology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA., Department of Urology, Vanderbilt University Medical Center, Nashville, TN, USA., Health Services Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA., Department of Urology, University of Kentucky, Lexington, KY, USA., Department of Urology, UT Southwestern Medical Center, Dallas, TX, USA., Department of Urology, The University of Texas Medical Branch, Galveston, TX, USA., Department of Urology, Moffitt Cancer Center, Tampa, FL, USA., Urology, McGill University Health Centre, Montreal, Canada., Department of Oncology, Vita-Salute San Raffaele University, Milan, Italy., Department of Urology, Rutgers Cancer Institute, New Brunswick, NJ, USA., Department of Urology, Carolina Urologic Research Center, Myrtle Beach, SC, USA., Department of Urology, Rush University Medical Center, Chicago, IL, USA., Department of Urology, Nijmegen Medical Centre, Radboud University, Nijmegen, The Netherlands., Department of Urology, University of Utah, Salt Lake City, UT, USA., Department of Urology, University of California San Francisco, San Francisco, CA, USA., Department of Oncology and Urology, Johns Hopkins University, Baltimore, MD, USA., Department of Urology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address: .