Cost-effectiveness Analysis of Treatments for Bacillus Calmette-Guérin–unresponsive Carcinoma in Situ of the Bladder

Background and objective

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.

Methods

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.

Key findings and limitations

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.

Conclusions and clinical implications

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.