Ανάλυση κόστους-αποτελεσματικότητας του σχήματος “Enfortumab Vedotin και Pembrolizumab” σε σύγκριση με πλατινούχο  χημειοθεραπεία, σε προχωρημένο ουροθηλιακό καρκίνο

Μεταξώτης, Γεώργιος

Cost-effectiveness analysis of the “Enfortumab Vedotin plus Pembrolizumab” combination versus platinum-based chemotherapy in advanced urothelial carcinoma

Master Thesis

Author

Μεταξώτης, Γεώργιος

Date

2026

Abstract

Urothelial cancer represents a particularly significant threat to public health, and the mortality of the disease in an advanced or metastatic stage remains at high levels on a global scale. For decades, platinum-based chemotherapy constituted the standard first-line treatment, offering limited survival. According to the findings of the study of interest EV-302, the combination of enfortumab vedotin and pembrolizumab (EV+P) demonstrated superior progression-free survival rates as well as better overall survival outcomes. Specifically, the median PFS was 12.5 months for the EV+P group and 6.3 months for the chemotherapy group, while the median overall survival reached 31.5 months for the EV+P combination and 16.1 months for chemotherapy, indicating a 53% reduction in the risk of death in favor of the immuno-targeted therapy. Furthermore, the median duration of response was significantly longer with the EV+P combination, as it was 7.0 months in the chemotherapy group, whereas it was not yet estimable in the EV+P group at the time of the analysis, indicating a continuous clinical response in this patient population. Enfortumab vedotin is an antibody-drug conjugate (ADC) that targets nectin-4, while pembrolizumab is a monoclonal antibody that targets the programmed cell death protein 1 (PD-1) receptor, thereby blocking its interaction with its ligands. Thus, the inhibitory signaling is lifted, and the antineoplastic immune response is reactivated for the eradication of cancer cells. Within this framework, the Food and Drug Administration (FDA) approved the use of this combination for patients with locally advanced or metastatic disease. Objective: The purpose of this study is to estimate the cost and effectiveness of the innovative combination of enfortumab vedotin and pembrolizumab (EV+P) versus standard platinum-based chemotherapy as a first-line treatment in adult patients with previously untreated, locally advanced or metastatic urothelial cancer. This analysis was conducted from the perspective of the healthcare payer (National Health System). Method: To conduct the cost-effectiveness analysis, a partitioned survival model (PSM) was developed with three health states: "progression-free survival" (PFS), "progressed disease" (PD), and "death". The population was modeled based on data from the EV-302 study, and the remaining data required for the analysis were sourced from international and Greek literature, as well as official state pricing sources. The model followed patients in monthly time-cycles over a period covering the life expectancy of the patients (lifetime horizon). In addition, an annual discount rate of 3.5% was applied. Following the completion of the base case analysis, a sensitivity analysis was performed to assess the robustness of the outcome against parameter uncertainties in the model data. Results: In the base case analysis, selecting the EV+P combination for the treatment of advanced urothelial cancer resulted in increased costs, with the annual discounted cost difference per patient amounting to approximately €18,345 compared to chemotherapy. Furthermore, treatment with the EV+P combination demonstrated a significant gain in life years and an increase in quality-adjusted life years by 0.47 compared to the chemotherapy option. From the ratio of the difference in costs and QALYs, the calculated incremental cost-effectiveness ratio (ICER) is approximately €39,009/QALY. The sensitivity analysis showed that the clinical effectiveness parameter of overall survival (OS EV+P extrapolated) and the total cost of the regimen exert the greatest influence on the ICER value. Conversely, factors such as adverse event management costs and utility in the progressed disease state (Utility PD) have a minimal impact on the outcome. Conclusions: According to the findings of this analysis, the selection of the EV+P combination as a first-line treatment versus chemotherapy in patients with advanced urothelial cancer appears to constitute a potentially cost-effective intervention, given that the ICER (€39,009/QALY) falls within the range of 1–3 times the per capita GDP (€23,500–€70,500) utilized as an indicative threshold for the National Health System.

Postgraduate Studies Programme

Οικονομικά και Διοίκηση της Υγείας

Department

Σχολή Οικονομικών, Επιχειρηματικών και Διεθνών Σπουδών. Τμήμα Οικονομικής Επιστήμης

Number of pages

Language

Greek

URI

https://dione.lib.unipi.gr/xmlui/handle/unipi/19566

Collections

Τμήμα Οικονομικής Επιστήμης

Show full item record

Except where otherwise noted, this item's license is described as
Αναφορά Δημιουργού-Μη Εμπορική Χρήση-Όχι Παράγωγα Έργα 3.0 Ελλάδα