Cost-Effectiveness Analysis of Pembrolizumab for Bacillus Calmette-Guérin-Unresponsive Carcinoma In Situ of the Bladder

Wymer, Kevin; Sharma, Vidit; Saigal, Christopher S.; Chamie, Karim; Litwin, Mark S.; Packiam, Vignesh T.; Mossanen, Matthew; Pagliaro, Lance C.; Borah, Bijan J.; Boorjian, Stephen A.

doi:10.1097/ju.0000000000001515

Cited by 24 publications

(17 citation statements)

References 26 publications

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“…As there is no universally accepted bladder-preserving therapy for BCG-unresponsive NMIBC, there is a need to standardize treatment used in the control arm. Recent FDA approval suggests pembrolizumab is an option; however, many consider its efficacy insufficient to offset the observed toxicity profile and the significant cost [13]. Intravesical gemcitabine/docetaxel has also been proposed as an alternative by some on the basis of "expert opinion," although its efficacy has not yet been established in prospective clinical trials.…”

Section: Resultsmentioning

confidence: 99%

Systematic Review and Meta-Analysis of Response Rates in BCG-unresponsive Non–Muscle-Invasive Bladder Cancer: a Consensus Statement From the International Bladder Cancer Group

Rose¹,

García‐Perdomo²,

Bivalacqua³

et al. 2022

SIUJ

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There is a critical need to establish reference response rates following bladder-sparing therapies administered in the setting of bacillus Calmete-Guerin (BCG)-unresponsive non–muscle-invasive bladder cancer (NMIBC). We sought to determine the efficacy of different interventions in recent trials accruing patients fulfilling the strict BCGunresponsive definition established by the US Food and Drug Administration. We performed a systematic review and meta-analysis for clinical trials in the BCG-unresponsive disease space to include published and presented results. The primary endpoints were complete response rate for CIS±Ta/T1 tumors, recurrence-free rate for patients with papillary-only disease, and disease-free rate in studies enrolling both papillary CIS tumors (Ta/T1/CIS). I2 was used for assessing heterogeneity. Eleven studies using 9 different therapeutic agents in a total of 909 patients with BCG-unresponsive NMIBC were identified. The resulting outcomes at 3, 6, and 12 months were 44%, 38%, and 25% complete response rate in CIS±Ta/T1 tumors; 73%, 58%, and 48% recurrence-free rate in papillary-only; and 48%, 22%, and 43% disease-free rate in combined Ta/T1/CIS, respectively. Relatively low levels of heterogeneity were observed amongst studies restricted to papillary-only or CIS±Ta/T1 tumors. Future randomized controlled studies are needed and will likely require stratification between papillary-only and CIS±Ta/T1 tumors. Introduction

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Section: Resultsmentioning

confidence: 99%

Systematic Review and Meta-Analysis of Response Rates in BCG-unresponsive Non–Muscle-Invasive Bladder Cancer: a Consensus Statement From the International Bladder Cancer Group

Rose¹,

García‐Perdomo²,

Bivalacqua³

et al. 2022

SIUJ

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“…Wymer et al provided the only study for NMI disease with the primary objective of comparing pembrolizumab to radical cystectomy (RC) and intravesical chemotherapy in BCG-unresponsive CIS tumors [ 13 ]. The inspiration was KEYNOTE-057, which prompted FDA approval of pembrolizumab in this population [ 6 ].…”

Section: Resultsmentioning

confidence: 99%

Cost-Effectiveness of Immune Checkpoint Inhibitors in Urothelial Carcinoma—A Review

Walia

Sweis

Agarwal

et al. 2021

Cancers

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Over the last decade, an increasing number of immune checkpoint inhibitors (ICIs) have been assessed for therapeutic efficacy in urothelial carcinoma (UC). The high cost has prompted multiple cost-effectiveness analyses for the various disease stages, with no established consensus. We reviewed the literature to assess the available cost-effectiveness studies and summarize their findings. Studies were filtered for a calculated incremental cost-effectiveness ratio (ICER) to standardize comparison. Over 2600 articles were narrowed to eight primary investigations: one for BCG-refractory non-muscle invasive (NMI), one for neoadjuvant therapy in muscle-invasive (MI), and six for advanced disease. Cost-effectiveness was not achieved for NMI disease. Atezolizumab met the willingness-to-pay (WTP) threshold as neoadjuvant therapy for MI disease compared to chemotherapy, but with multiple limitations on the interpretation. Of the six studies on advanced disease, the results were mixed. This was at least partially attributable to varied methodologies including extrapolated time horizons, inconsistent cost inputs, and different WTP thresholds. Overall, the aggregate results were not compelling enough to establish ICIs as cost-effective compared to conventional chemotherapy. Value may improve with continued investigation into long-term outcomes, refined patient selection, and pricing discounts.

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“…This translates to a Mayo progression score of at least 10 9 or an ASSURE score of at least 8.5. 15 Reserving Figure 2. The tornado diagram demonstrates the 4 variables out of over 100 model inputs that when modified converted pembrolizumab (Pembro) to be cost-effective in our model.…”

Section: Discussionmentioning

confidence: 99%

“…This translates to a Mayo progression score of at least 10 9 or an ASSURE score of at least 8.5. 15 Reserving adjuvant pembrolizumab for these higher-risk subsets would reduce the number-needed-to-treat post-nephrectomy, thereby reducing overall costs and toxicity while optimizing benefit at a population level.…”

Section: Discussionmentioning

confidence: 99%

Cost-effectiveness of Adjuvant Pembrolizumab After Nephrectomy for High-risk Renal Cell Carcinoma: Insights for Patient Selection From a Markov Model

et al. 2023

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Purpose:The KEYNOTE-564 trial demonstrated that adjuvant pembrolizumab after nephrectomy for clear cell renal cell carcinoma decreased the risk of disease progression and potentially overall mortality as well. Herein, we used a Markov model to weigh the costs, toxicities, and efficacy of pembrolizumab to further investigate its utility.Materials and Methods:Decision-analytic Markov modeling was used to conduct a cost-utility analysis of adjuvant pembrolizumab versus observation after nephrectomy for high-risk clear cell renal cell carcinoma, using data from KEYNOTE-564 to inform model probabilities. Primary outcomes were quality-adjusted life years, Medicare costs, and incremental cost-effectiveness ratios. The willingness-to-pay threshold utilized was $100,000/quality-adjusted life year.Results:At 5 years, adjuvant treatment with pembrolizumab resulted in 0.3 additional quality-adjusted life years at an additional cost of $99,484 relative to observation. Pembrolizumab was found not to be cost-effective at a 5-year time horizon (incremental cost-effectiveness ratio=$326,534). On sensitivity analysis, pembrolizumab became cost-effective if its per cycle cost was <$5,064 (base=$10,278) or its 5-year progression benefit was >18.8% (base 9%). Upon simulation, pembrolizumab was cost-effective for 29% of patients at 5 years. Specifically, we found that pembrolizumab would be cost-effective at 5 years for patients with at least a 59% 5 year risk of progression, which corresponds to a Mayo Progression-free Survival Score ≥10.Conclusions:At current prices, adjuvant pembrolizumab was found to be cost-effective only for the highest risk subset of clear cell renal cell carcinoma patients 5 years after treatment, including patients with complete metastasectomy, regional lymph node involvement, or ≥7cm pT3 tumors with sarcomatoid features. Longer-term trial data, including overall survival results, are necessary to confirm these extrapolations.

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Cost-Effectiveness Analysis of Pembrolizumab for Bacillus Calmette-Guérin-Unresponsive Carcinoma In Situ of the Bladder

Abstract: cost-effective relative to radical cystectomy at 5 years, further studies may validate its cost-effectiveness if recurrence and metastasis thresholds are met.

Cited by 24 publications

References 26 publications

Systematic Review and Meta-Analysis of Response Rates in BCG-unresponsive Non–Muscle-Invasive Bladder Cancer: a Consensus Statement From the International Bladder Cancer Group

Systematic Review and Meta-Analysis of Response Rates in BCG-unresponsive Non–Muscle-Invasive Bladder Cancer: a Consensus Statement From the International Bladder Cancer Group

Cost-Effectiveness of Immune Checkpoint Inhibitors in Urothelial Carcinoma—A Review

Cost-effectiveness of Adjuvant Pembrolizumab After Nephrectomy for High-risk Renal Cell Carcinoma: Insights for Patient Selection From a Markov Model

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