Proposed method for economic evaluation based on basket trials: a case study of pembrolizumab

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.

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.

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.

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.