Cost-per-responder analysis of TAR-200 versus other Food and Drug Administration-approved novel and generic treatments among patients with Bacillus Calmette-Guérin-unresponsive, high-risk, non-muscle-invasive bladder cancer with carcinoma in situ in the United States.

Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS) is aggressive and treatment options are suboptimal. TAR-200, a novel intravesical drug-releasing system, received United States (US) Food and Drug Administration (FDA) approval on 09/09/2025 for this population. An economic model compared the cost per responder for US patients with BCG-unresponsive HR-NMIBC with CIS treated with TAR-200 versus other FDA-approved treatments.

A 15-month cost-per-responder model was developed from a Medicare payer perspective (2025 USD). Patients treated with TAR-200 monotherapy were compared to those treated with pembrolizumab, nadofaragene firadenovec (NF), nogapendekin alfa inbakicept (NAI)+BCG (with/without reinduction), or valrubicin based on published clinical trial data. Model inputs included costs for initial/subsequent treatment, medical visits, and radical cystectomy (RC). Outcomes comprised the total cost per patient achieving and sustaining complete response (CR) for ≥12 months, based on overall CR rates and digitized Kaplan-Meier curves and swimmer plots for the 12-month duration of response. Patients experiencing non-response received subsequent treatment or underwent an RC.

At 15 months, the proportion of patients achieving and sustaining CR for ≥12 months was 43.5% for TAR-200, 18.8% for pembrolizumab, 21.9% for NF, 26.8% for NAI+BCG (36.6% with reinduction), and 10.1% for valrubicin. The total cost per patient achieving and sustaining CR for ≥12 months was $1,892,569 for TAR-200, resulting in cost savings of $698,262 versus pembrolizumab, $406,840 versus NF, $832,346 versus NAI+BCG, and $1,541,999 versus valrubicin. Considering NAI+BCG reinduction, cost savings of $162,599 per patient achieving and sustaining CR for ≥12 months were observed for TAR-200 versus NAI+BCG.

Model inputs were based on trial publications, possibly limiting generalizability.

TAR-200 demonstrated the highest proportion of patients achieving and sustaining CR for ≥12 months, yielding substantial cost savings per responder compared to other FDA-approved treatments for BCG-unresponsive HR-NMIBC with CIS.
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Authors

Williams Williams, Cho Cho, Morrison Morrison, Joshi Joshi, Pilon Pilon, Gwyn Gwyn, Libchaber Libchaber, Wong Wong, Lee Lee, Desai Desai, Singhal Singhal
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