Proposed Method for Economic Evaluation Based on Basket Trials: A Case Study of Pembrolizumab Publisher Pubmed



M Nosrati MARZIEH ; H Vatankhah HOSSEIN ; M Asfia MAHDI ; M Sharifnia MOHAMMADHOSSEIN ; F Shahi FARHAD ; M Hasanzad MANDANA ; A Abdollahi Asl AKBAR ; M Abdollahi MOHAMMAD ; S Nikfar SHEKOUFEH
Source: Expert Review of Pharmacoeconomics and Outcomes Research Published:2025

Abstract

Background: Precision medicine’s emphasis on genetic profiles rather than diseases has reshaped oncology trials, prioritizing biomarker-specific approaches named basket trials. They involve heterogeneous populations, necessitating new economic evaluation methods, as traditional approaches fail to capture this diversity. This study introduced a methodology for economic evaluations based on basket trials, exemplified through pembrolizumab’s cost-effectiveness analysis using Keynote-158 data as a case study and compared the results to disease-specific methods. Research design and methods: A regression-based approach was developed to aggregate costs and QALYs and calculate transition probabilities for economic evaluations in populations sharing a biomarker and various cancers. The proposed method involved five steps: designing a general model, selecting comparator arms, comparing Kaplan-Meier curves and conducting network analysis to evaluate treatment responses, calculating disease-specific coefficients to aggregate QALYs, and aggregating costs using weighted averages. Result: All the steps have been followed for conducting cost-effectiveness of Pembrolizumab in patients with dMMR/MSI-H biomarker. The results revealed significant differences in ICERs calculated by the proposed method versus disease-specific evaluations. Conclusion: This study introduces a biomarker-focused model for estimating ICERs across diverse diseases, enabling informed decision-making in healthcare resource allocation. Further research is recommended to broaden the model’s applicability and address additional population heterogeneity factors. © 2025 Elsevier B.V., All rights reserved.