Cost-effectiveness of Pembrolizumab for Patients with Advanced, Unresectable, or Metastatic Urothelial Cancer Ineligible for Cisplatin-based Therapy

Patterson, K.; Prabhu, Vimalanand S.; Xu, Ruifeng; Li, Haojie; Meng, Yang; Zarabi, Natalie; Zhong, Yichen; Batteson, Rachael; Pellissier, James M.; Keefe, Stephen Michael; Grivas, Petros; Wit, Ronald de

doi:10.1016/j.euo.2018.09.009

Cited by 20 publications

(20 citation statements)

References 13 publications

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“…Pembrolizumab is predicted to provide an incremental benefit of 2.58 life-years compared with carboplatin plus gemcitabine, longer than the life-year gains predicted in the previous analyses using shorter follow-up data (2.11 and 1.44 life-year gains, respectively). 13,14 Our results reflect the durable treatment effect observed in the trial and confirm the sustainable survival benefit of pembrolizumab in this population.…”

Section: Discussionsupporting

confidence: 75%

“…The survival outcomes of pembrolizumab were modeled using the final analysis data cut of the KEYNOTE-052 trial, which provides an additional 18 months of follow-up from randomization compared with the data used in previous cost-effectiveness analyses. 13,14 The longer followup reduces the uncertainty of extrapolating the survival beyond the trial observation period, and thus allows for a more robust prediction of long-term survival outcomes. Pembrolizumab is predicted to provide an incremental benefit of 2.58 life-years compared with carboplatin plus gemcitabine, longer than the life-year gains predicted in the previous analyses using shorter follow-up data (2.11 and 1.44 life-year gains, respectively).…”

Section: Discussionmentioning

confidence: 99%

“…[19][20][21][22] The approach was detailed in previous publications. 13,14 The constant HRs were assumed for the base case, which represented a stable estimation of the relative treatment effect observed within the trial follow-up and a reliable extrapolation beyond that. The calculated OS and PFS HRs for carboplatin plus gemcitabine versus pembrolizumab were 2.34 and 1.72, respectively.…”

Section: Modeling Pfs Os and Tot For Carboplatin Plus Gemcitabinementioning

confidence: 99%

“…2 Two prior costeffectiveness studies of first-line pembrolizumab were performed in the UK and Sweden by using country-specific cost and resource data, and based on interim analyses of KEYNOTE-052 (median follow-up of 13.7 months; range, 8.6-22.7 months from the date of first dose to database cutoff date). 13,14 This study utilizes the final analyses of KEYNOTE-052 (median follow-up of 32.2 months; range, 27.2-41.3 months), with an additional 18 months of followup compared with the data used in previous studies. 9 This study reduces the uncertainty of survival extrapolation and provides a more reliable estimate of pembrolizumab's long-term cost effectiveness versus chemotherapy in the first-line setting.…”

Section: Introductionmentioning

confidence: 99%

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Cost-effectiveness of Pembrolizumab versus Carboplatin-based Chemotherapy as First-line Treatment of PD-L1–positive Locally Advanced or Metastatic Urothelial Carcinoma Ineligible for Cisplatin-based Therapy in the United States

Hale

Patterson

Lai

et al. 2021

Clinical Genitourinary Cancer

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A partitioned survival cost-effectiveness model informed by the final analyses from KEYNOTE-052 predicted long-term health and cost outcomes for cisplatin-ineligible patients with urothelial cancer with programmed death-ligand 1-positive tumors receiving either pembrolizumab or carboplatin plus gemcitabine. Over 20 years, treatment with pembrolizumab resulted in a mean gain of 2.58 life years and was shown to be cost-effective over carboplatin plus gemcitabine. Introduction: Pembrolizumab has been approved in the United States (US) for the first-line treatment of patients with advanced or metastatic urothelial carcinoma, who are ineligible for cisplatin-containing chemotherapy and with tumors expressing programmed death-ligand 1 (PD-L1) (Combined Positive Score ! 10), or ineligible for any platinumcontaining chemotherapy regardless of PD-L1 status. Long-term KEYNOTE-052 data continue to demonstrate pembrolizumab's meaningful, durable, and well-tolerated antitumor activity. This study evaluates the costeffectiveness of pembrolizumab versus carboplatin plus gemcitabine as first-line treatment for cisplatin-ineligible patients who have PD-L1epositive tumors from a US third-party healthcare payer's perspective. Patients and Methods: A partitioned survival model containing 3 health states (progression-free, progressed, and death) was developed. A simulated treatment comparison and a network meta-analysis were conducted to estimate the comparative efficacy of pembrolizumab versus carboplatin-based chemotherapy. Overall survival, progression-free survival, time on treatment, adverse events, and utilities were modeled using the final analyses of the KEYNOTE-052 trial and 4 studies for carboplatin plus gemcitabine. Cost data were estimated using US standard sources and real-world data. Deterministic, probabilistic, and scenario analyses were conducted to assess the robustness of the results. Results: Over 20 years, pembrolizumab resulted in a mean gain of 2.58 life-years, 2.01 quality-adjusted life-years, and additional costs of $158,561, leading to an incremental cost-effectiveness ratio of $78,925/qualityadjusted life-year compared with carboplatin plus gemcitabine. Conclusion: This study suggests that pembrolizumab is cost-effective compared with carboplatin plus gemcitabine as a first-line therapy for patients with advanced or metastatic urothelial carcinoma who are PD-L1epositive.

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Section: Discussionsupporting

confidence: 75%

Section: Discussionmentioning

confidence: 99%

Section: Modeling Pfs Os and Tot For Carboplatin Plus Gemcitabinementioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Cost-effectiveness of Pembrolizumab versus Carboplatin-based Chemotherapy as First-line Treatment of PD-L1–positive Locally Advanced or Metastatic Urothelial Carcinoma Ineligible for Cisplatin-based Therapy in the United States

Hale

Patterson

Lai

et al. 2021

Clinical Genitourinary Cancer

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“…Quality-of-life data on patients receiving pembrolizumab in the KEYNOTE-240 trial were not published; therefore, we extrapolated these data from previously published studies of pembrolizumab. 20 This process was appropriate because the utility score was similar to that reported in patients with HCC who received nivolumab or regorafenib in the second-line treatment setting. 5,10 We also assumed the decline of quality of life on progression of HCC, which is consistent with previous literature and real-life experience.…”

Section: Limitationsmentioning

confidence: 99%

Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma

et al. 2021

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IMPORTANCE Immune checkpoint inhibitors have been approved for use as a second-line therapy for hepatocellular carcinoma (HCC) in patients who previously received sorafenib. Pembrolizumab has shown substantial antitumor activity and a favorable toxicity profile as a second-line treatment of HCC. However, considering the high cost of pembrolizumab, there is a need to assess its value by considering both the clinical efficacy and cost.OBJECTIVE To evaluate the cost-effectiveness of pembrolizumab vs placebo as second-line therapy in patients with HCC from the US payer perspective. DESIGN, SETTING, AND PARTICIPANTSA Markov model was developed to compare the lifetime cost and efficacy of pembrolizumab as a second-line treatment of HCC with those of placebo using outcome data from the KEYNOTE-240 randomized placebo-controlled trial, which included 413 patients with advanced HCC previously treated with sorafenib and randomized patients to receive pembrolizumab plus best supportive care or placebo plus best supportive care in a 2:1 ratio. MAIN OUTCOMES AND MEASURESLife-years, quality-adjusted life-years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER) were estimated at a willingness-to-pay threshold of $150 000 per QALY. One-way and probabilistic sensitivity analyses were performed to account for the parameter of uncertainty. A cost-threshold analysis was also performed. The study was conducted from January 31 to July 29, 2020.

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A cost‐effectiveness analysis of avelumab plus best supportive care versus best supportive care alone as first‐line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma in Taiwan

Su,

Xiao,

Lin

et al. 2023

Cancer Reports

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BackgroundPatients with locally advanced or metastatic urothelial carcinoma have limited treatment options and a poor prognosis. The JAVELIN Bladder 100 trial showed that avelumab as first‐line maintenance plus best supportive care significantly prolonged overall survival and progression‐free survival versus best supportive care alone in patients with locally advanced or metastatic urothelial carcinoma that had not progressed with first‐line platinum‐containing chemotherapy.AimsWe assessed whether avelumab plus best supportive care is a cost‐effective treatment option versus best supportive care alone in this patient group in Taiwan.Methods and ResultsA partitioned survival model was used to estimate the costs and effects of avelumab plus best supportive care versus best supportive care alone over a 20‐year time horizon from the perspective of Taiwan's National Health Insurance Administration. Patient‐level data from JAVELIN Bladder 100 on efficacy, safety, utility, and time on treatment were analyzed to provide parameters for the model. Log‐normal and Weibull distributions were used for overall survival and progression‐free survival, respectively. Costs of healthcare resources, drug acquisition, adverse events, and progression were identified through publicly available data sources and clinician interviews. The model estimated total costs, life years, and quality‐adjusted life years. In the modeled base case, avelumab plus best supportive care increased survival versus best supportive care alone by 0.79 life years (2.93 vs. 2.14) and 0.61 quality‐adjusted life years (2.15 vs. 1.54). The incremental cost‐effectiveness ratio for avelumab plus best supportive care versus best supportive care alone was NT$1 827 680. Most (78%) of the probabilistic sensitivity analyses fell below three times the gross domestic product per capita. Scenario analysis indicated that life year and quality‐adjusted life year gains were most sensitive to alternative survival extrapolations for both avelumab plus best supportive care and best supportive care alone.ConclusionAvelumab first‐line maintenance therapy combined with best supportive care was determined as a cost‐effective treatment strategy for patients in Taiwan diagnosed with locally advanced or metastatic urothelial carcinoma that had not progressed with platinum‐containing chemotherapy.

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Cost-effectiveness of Pembrolizumab for Patients with Advanced, Unresectable, or Metastatic Urothelial Cancer Ineligible for Cisplatin-based Therapy

Cited by 20 publications

References 13 publications

Cost-effectiveness of Pembrolizumab versus Carboplatin-based Chemotherapy as First-line Treatment of PD-L1–positive Locally Advanced or Metastatic Urothelial Carcinoma Ineligible for Cisplatin-based Therapy in the United States

Cost-effectiveness of Pembrolizumab versus Carboplatin-based Chemotherapy as First-line Treatment of PD-L1–positive Locally Advanced or Metastatic Urothelial Carcinoma Ineligible for Cisplatin-based Therapy in the United States

Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma

A cost‐effectiveness analysis of avelumab plus best supportive care versus best supportive care alone as first‐line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma in Taiwan

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