A conceptual health economic modelling framework to assess the cost-effectiveness of molecular target–driven treatment regimens in oncology
DOI:
https://doi.org/10.2340/1651-226X.2026.45088Keywords:
Costs and cost analysis, medical oncology, models, economic, reimbursement mechanismsAbstract
Background and purpose: Molecularly targeted cancer therapies challenge conventional health economic evaluation frameworks that are structured around tumour-specific indications, comparators, and trial designs. Existing models often rely on pooled estimates from heterogeneous early-phase evidence or single-indication analyses, creating uncertainty for reimbursement decision-makers. We propose a conceptual modelling framework that aligns cost-effectiveness analyses with the biological rationale of precision oncology, evaluating therapies according to shared molecular alterations across tumour types.
Patient/material and methods: We examined the methodological limitations of conventional partitioned survival models (PSMs) commonly applied in oncology and evaluated their suitability for tumour-agnostic indications. Based on the collected literature, we developed a dynamic, modular PSM framework that integrates multiple tumour sites expressing a common biomarker. The framework supports pooled and tumour-specific analysis of cost-effectiveness and enables progressive disaggregation of subgroups as additional evidence becomes available.
Results: The proposed modelling approach facilitates transparent synthesis of heterogeneous evidence across tumour types using epidemiologically informed weighting, while preserving the ability to estimate tumour-specific cost-effectiveness where data permit. It addresses key challenges in tumour-agnostic evaluation, including variation in standard of care, treatment effects, and resource use across cancer sites. The modular design promotes internal consistency, reduces duplication of analytical effort, and enables iterative re-assessment of both overall and subgroup-specific cost-effectiveness.
Interpretation: A dynamic, weighted multi-site modelling framework represents a coherent and adaptable extension of current health-technology assessment-practice for tumour-agnostic therapies. By structuring evidence around molecular targets, the framework can improve transparency and robustness of cost-effectiveness estimates, thereby supporting more equitable and efficient reimbursement decisions in the context of precision oncology.
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Copyright (c) 2026 Oskar Frisell, Eline Aas, Pia Sofie Henkel, Gro Live Fagereng, Kjetil Taskén, Ebba Hallersjö Hult, Peter Lindgren, Katarina Steen Carlsson
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