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A conceptual health economic modelling framework to assess the cost-effectivenesof molecular target–driven treatment regimens in oncology

Frisell, Oskar ; Aas, Eline ; Henkel, Pia Sofie ; Fagereng, Gro Live ; Taskén, Kjetil ; Hult, Ebba Hallersjö ; Lindgren, Peter and Carlsson, Katarina Steen LU orcid (2026) In Acta Oncologica 65. p.141-147
Abstract

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... (More)

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-agnos-tic 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 adapt-able extension of current health-technology assessment-practice for tumour-agnostic therapies. By struc-turing 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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author
; ; ; ; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Costs and cost analysis, economic, medical oncology, models, reimbursement mechanisms
in
Acta Oncologica
volume
65
pages
7 pages
publisher
Taylor & Francis
external identifiers
  • pmid:41711494
  • scopus:105030632762
ISSN
0284-186X
DOI
10.2340/1651-226X.2026.45088
language
English
LU publication?
yes
additional info
Publisher Copyright: © 2026 The Author(s). Published by MJS Publishing on behalf of Acta Oncologica. This is an Open Access article distributed under the terms of the Creative Commons Attribution 4.0 International.
id
8fd86bad-5662-4689-b52b-01eafcf64aff
date added to LUP
2026-04-15 14:39:15
date last changed
2026-04-16 03:00:02
@article{8fd86bad-5662-4689-b52b-01eafcf64aff,
  abstract     = {{<p>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-agnos-tic 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 adapt-able extension of current health-technology assessment-practice for tumour-agnostic therapies. By struc-turing 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.</p>}},
  author       = {{Frisell, Oskar and Aas, Eline and Henkel, Pia Sofie and Fagereng, Gro Live and Taskén, Kjetil and Hult, Ebba Hallersjö and Lindgren, Peter and Carlsson, Katarina Steen}},
  issn         = {{0284-186X}},
  keywords     = {{Costs and cost analysis; economic; medical oncology; models; reimbursement mechanisms}},
  language     = {{eng}},
  pages        = {{141--147}},
  publisher    = {{Taylor & Francis}},
  series       = {{Acta Oncologica}},
  title        = {{A conceptual health economic modelling framework to assess the cost-effectivenesof molecular target–driven treatment regimens in oncology}},
  url          = {{http://dx.doi.org/10.2340/1651-226X.2026.45088}},
  doi          = {{10.2340/1651-226X.2026.45088}},
  volume       = {{65}},
  year         = {{2026}},
}