The cost effectiveness of pembrolizumab versus chemotherapy or atezolizumab as second-line therapy for advanced urothelial carcinoma in the United States

Slater, Rachael Louise; Lai, Yizhen; Zhong, Yichen; Li, Haojie; Meng, Yang; Moreno, Blanca Homet; Godwin, James Luke; Frenkl, Tara L.; Sonpavde, Guru; Mamtani, Ronac

doi:10.1080/13696998.2020.1770261

Cited by 14 publications

(13 citation statements)

References 18 publications

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“…Costs and utilities for model health states were derived from 3 key studies. [17][18][19] Since the prices for nadofaragene firadenovec and response rates that are similar to or better than currently available treatments, such as gemcitabine with or without docetaxel, there continues to be considerable uncertainty about their long-term efficacy. Because they are administered in the bladder, they appear to have few serious side effects.…”

Section: Limitations Of the Clinical Evidencementioning

confidence: 99%

The effectiveness and value of nadofaragene firadenovec, oportuzumab monatox, and pembrolizumab for BCG-unresponsive non-muscle-invasive bladder cancer

Beinfeld

Atlas

Touchette

et al. 2021

JMCP

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Section: Limitations Of the Clinical Evidencementioning

confidence: 99%

The effectiveness and value of nadofaragene firadenovec, oportuzumab monatox, and pembrolizumab for BCG-unresponsive non-muscle-invasive bladder cancer

Beinfeld

Atlas

Touchette

et al. 2021

JMCP

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“…For example, the cost-effectiveness of second-line Pembrolizumab was evaluated against chemotherapy or Atezolizumab in an American population, based on extrapolated results from KEYNOTE-045 and IMvigor-211. Results showed that despite the study limitations and at a $100.000 willingness-to-pay threshold scenario, pembrolizumab and atezolizumab had a 66% and 100% probability of being cost-effective against chemotherapy, respectively [ 27 ]. Pembrolizumab was also evaluated against carboplatin-based chemotherapy in a PD-L1 positive first-line scenario using data from KEYNOTE-052.…”

Section: Case Reportmentioning

confidence: 99%

Emerging biomarkers in metastatic urothelial carcinoma: tumour mutational burden, PD-L1 expression and APOBEC polypeptide-like signature in a patient with complete response to anti-programmed cell death protein-1 inhibitor

Queiroz

Souza

Gongora

et al. 2021

ecancer

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Immunotherapy has recently been incorporated into the treatment guidelines for metastatic urothelial carcinoma. Nevertheless, the role of prognostic and predictive biomarkers in this setting is not completely defined. To date, PD-L1 expression and a high tumour mutational burden (TMB) seem to predict better responses to immune checkpoint inhibitors, but patients without these biomarkers may still respond to immunotherapy. There are some caveats regarding these biomarkers, such as lack of standardisation of techniques, tumour heterogeneity and other factors influencing the tumour microenvironment. Genomic signatures are other promising emerging strategies. We hereby discuss the management of a 70-year-old man with a metastatic recurrence of urothelial carcinoma within 1 year after neoadjuvant chemotherapy and radical cystectomy. Tumour next-generation sequencing showed a high TMB and a CD274 (PD-L1) amplification. The patient was treated with pembrolizumab and achieved a complete response.

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“…This finding was reliant on short-term data obtained from the trial. In comparison, Slater et al analyzed results from the KEYNOTE-045 study at a median follow-up of >2-years [ 80 ]. The study reported that at a willingness-to-pay threshold of $100,000, pembrolizumab is a cost-effective option ($93,481/QALY gained) compared to chemotherapy in the U.S.…”

Section: Health Economic Factorsmentioning

confidence: 99%

“…As pembrolizumab and atezolizumab are the only ICIs evaluated in randomized controlled trial for this bladder cancer treatment setting, Slater et al, performed a cost-effective evaluation comparative study [ 80 ]. The analysis compared a >2-year follow-up for pembrolizumab’s KEYNOTE-045 trial with the data from atezolizumab’s IMvigor211 trial.…”

Section: Health Economic Factorsmentioning

confidence: 99%

Targeted Molecular Therapeutics for Bladder Cancer—A New Option beyond the Mixed Fortunes of Immune Checkpoint Inhibitors?

Bednova

Leyton

2020

IJMS

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The fact that there are now five immune checkpoint inhibitor (ICI) monoclonal antibodies approved since 2016 that target programmed cell death protein 1 or programmed death ligand-1 for the treatment of metastatic and refractory bladder cancer is an outstanding achievement. Although patients can display pronounced responses that extend survival when treated with ICIs, the main benefit of these drugs compared to traditional chemotherapy is that they are better tolerated and result in reduced adverse events (AEs). Unfortunately, response rates to ICI treatment are relatively low and, these drugs are expensive and have a high economic burden. As a result, their clinical efficacy/cost-value relationship is debated. Long sought after targeted molecular therapeutics have now emerged and are boasting impressive response rates in heavily pre-treated, including ICI treated, patients with metastatic bladder cancer. The antibody-drug conjugates (ADCs) enfortumab vedotin (EV) and sacituzumab govitecan (SG) have demonstrated the ability to provide objective response rates (ORRs) of 44% and 31% in patients with bladder tumor cells that express Nectin-4 and Trop-2, respectively. As a result, EV was approved by the U.S. Food and Drug Administration for the treatment of patients with advanced or metastatic bladder cancer who have previously received ICI and platinum-containing chemotherapy. SG has been granted fast track designation. The small molecule Erdafitinib was recently approved for the treatment of patients with advanced or metastatic bladder cancer with genetic alterations in fibroblast growth factor receptors that have previously been treated with a platinum-containing chemotherapy. Erdafitinib achieved an ORR of 40% in patients including a proportion who had previously received ICI therapy. In addition, these targeted drugs are sufficiently tolerated or AEs can be appropriately managed. Hence, the early performance in clinical effectiveness of these targeted drugs are substantially increased relative to ICIs. In this article, the most up to date follow-ups on treatment efficacy and AEs of the ICIs and targeted therapeutics are described. In addition, drug price and cost-effectiveness are described. For best overall value taking into account clinical effectiveness, price and cost-effectiveness, results favor avelumab and atezolizumab for ICIs. Although therapeutically promising, it is too early to determine if the described targeted therapeutics provide the best overall value as cost-effectiveness analyses have yet to be performed and long-term follow-ups are needed. Nonetheless, with the arrival of targeted molecular therapeutics and their increased effectiveness relative to ICIs, creates a potential novel paradigm based on ‘targeting’ for affecting clinical practice for metastatic bladder cancer treatment.

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The cost effectiveness of pembrolizumab versus chemotherapy or atezolizumab as second-line therapy for advanced urothelial carcinoma in the United States

Cited by 14 publications

References 18 publications

The effectiveness and value of nadofaragene firadenovec, oportuzumab monatox, and pembrolizumab for BCG-unresponsive non-muscle-invasive bladder cancer

The effectiveness and value of nadofaragene firadenovec, oportuzumab monatox, and pembrolizumab for BCG-unresponsive non-muscle-invasive bladder cancer

Emerging biomarkers in metastatic urothelial carcinoma: tumour mutational burden, PD-L1 expression and APOBEC polypeptide-like signature in a patient with complete response to anti-programmed cell death protein-1 inhibitor

Targeted Molecular Therapeutics for Bladder Cancer—A New Option beyond the Mixed Fortunes of Immune Checkpoint Inhibitors?

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