Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands

Zakile A Mfumbilwa; Janneke A Wilschut; Martijn J H G Simons; Bram Ramaekers; Manuela Joore; Valesca Retèl; Christine M Cramer-van der Welle; Franz M N H Schramel; Ewoudt M W van de Garde; Veerle M H Coupé

doi:10.1038/s41598-023-29286-5

Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands

Zakile A Mfumbilwa
, Janneke A Wilschut
, Martijn J H G Simons
, Bram Ramaekers
, Manuela Joore
, Valesca Retèl
, Christine M Cramer-van der Welle
, Franz M N H Schramel
, Ewoudt M W van de Garde
, Veerle M H Coupé

Department of Epidemiology and Data Science
Maastricht University Medical Centre
University of Twente
Santeon Hospital Group

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

Recent discoveries in molecular diagnostics and drug treatments have improved the treatment of patients with advanced (inoperable) non-squamous non-small cell lung cancer (NSCLC) from solely platinum-based chemotherapy to more personalized treatment, including targeted therapies and immunotherapies. However, these improvements come at considerable costs, highlighting the need to assess their cost-effectiveness in order to optimize lung cancer care. Traditionally, cost-effectiveness models for the evaluation of new lung cancer treatments were based on the findings of the randomized control trials (RCTs). However, the strict RCT inclusion criteria make RCT patients not representative of patients in the real-world. Patients in RCTs have a better prognosis than patients in a real-world setting. Therefore, in this study, we developed and validated a diagnosis-treatment decision model for patients with advanced (inoperable) non-squamous NSCLC based on real-world data in the Netherlands. The model is a patient-level microsimulation model implemented as discrete event simulation with five health events. Patients are simulated from diagnosis to death, including at most three treatment lines. The base-model (non-personalized strategy) was populated using real-world data of patients treated with platinum-based chemotherapy between 2008 and 2014 in one of six Dutch teaching hospitals. To simulate personalized care, molecular tumor characteristics were incorporated in the model based on the literature. The impact of novel targeted treatments and immunotherapies was included based on published RCTs. To validate the model, we compared survival under a personalized treatment strategy with observed real-world survival. This model can be used for health-care evaluation of personalized treatment for patients with advanced (inoperable) NSCLC in the Netherlands.

Original language	English
Article number	2349
Number of pages	14
Journal	Scientific Reports
Volume	13
Issue number	1
DOIs	https://doi.org/10.1038/s41598-023-29286-5
Publication status	Published - Dec 2023

Bibliographical note

Funding Information:
This work was funded by the Netherlands Organization for Health Research and Development (ZonMw) (grant number 846001002), the Dutch Cancer Society (KWF), and the Dutch health-care insurance company Zilveren kruis Achmea. The funding sources had no involvement in the conduct of this research. The authors thank all members of the Technology Assessment of Next Generation Sequencing in Personalized Oncology (TANGO) consortium ( https://zenodo.org/communities/tango-wgs/?page=1&size=20 ).

Funding Information:
Valesca Retèl has received grants from Agendia B.V. and Intuitive Surgical outside the submitted work. Zakile A. Mfumbilwa, Janneke A. Wilschut, Martijn J.H.G. Simons, Bram Ramaekers, Manuela Joore, Christine M. Cramer-van der Welle, Franz M.N.H. Schramel, Ewoudt M.W. van de Garde, and Veerle M.H. Coupé declares no potential conflict of interest.

Publisher Copyright:
© 2023, The Author(s).

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

Humans
Antineoplastic Agents/adverse effects
Netherlands
Cost-Benefit Analysis
Lung Neoplasms/drug therapy
Carcinoma, Non-Small-Cell Lung/drug therapy
Antineoplastic Combined Chemotherapy Protocols/therapeutic use
Competing risks
Multicenter
Tyrosine kinase inhibitors
Survival outcomes
Cost-effectiveness analysis
Chemotherapy
Pembrolizumab
Simulation
Immunotherapy
1st-line therapy

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s41598-023-29286-5Final published version, 2.35 MBLicence: CC BY

Cite this

Mfumbilwa, Z. A., Wilschut, J. A., Simons, M. J. H. G., Ramaekers, B., Joore, M., Retèl, V., der Welle, C. M. C., Schramel, F. M. N. H., van de Garde, E. M. W., & Coupé, V. M. H. (2023). Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands. Scientific Reports, 13(1), Article 2349. https://doi.org/10.1038/s41598-023-29286-5

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title = "Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands",

abstract = "Recent discoveries in molecular diagnostics and drug treatments have improved the treatment of patients with advanced (inoperable) non-squamous non-small cell lung cancer (NSCLC) from solely platinum-based chemotherapy to more personalized treatment, including targeted therapies and immunotherapies. However, these improvements come at considerable costs, highlighting the need to assess their cost-effectiveness in order to optimize lung cancer care. Traditionally, cost-effectiveness models for the evaluation of new lung cancer treatments were based on the findings of the randomized control trials (RCTs). However, the strict RCT inclusion criteria make RCT patients not representative of patients in the real-world. Patients in RCTs have a better prognosis than patients in a real-world setting. Therefore, in this study, we developed and validated a diagnosis-treatment decision model for patients with advanced (inoperable) non-squamous NSCLC based on real-world data in the Netherlands. The model is a patient-level microsimulation model implemented as discrete event simulation with five health events. Patients are simulated from diagnosis to death, including at most three treatment lines. The base-model (non-personalized strategy) was populated using real-world data of patients treated with platinum-based chemotherapy between 2008 and 2014 in one of six Dutch teaching hospitals. To simulate personalized care, molecular tumor characteristics were incorporated in the model based on the literature. The impact of novel targeted treatments and immunotherapies was included based on published RCTs. To validate the model, we compared survival under a personalized treatment strategy with observed real-world survival. This model can be used for health-care evaluation of personalized treatment for patients with advanced (inoperable) NSCLC in the Netherlands.",

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author = "Mfumbilwa, \{Zakile A\} and Wilschut, \{Janneke A\} and Simons, \{Martijn J H G\} and Bram Ramaekers and Manuela Joore and Valesca Ret{\`e}l and \{der Welle\}, \{Christine M Cramer-van\} and Schramel, \{Franz M N H\} and \{van de Garde\}, \{Ewoudt M W\} and Coup{\'e}, \{Veerle M H\}",

note = "Funding Information: This work was funded by the Netherlands Organization for Health Research and Development (ZonMw) (grant number 846001002), the Dutch Cancer Society (KWF), and the Dutch health-care insurance company Zilveren kruis Achmea. The funding sources had no involvement in the conduct of this research. The authors thank all members of the Technology Assessment of Next Generation Sequencing in Personalized Oncology (TANGO) consortium ( https://zenodo.org/communities/tango-wgs/?page=1\&size=20 ). Funding Information: Valesca Ret{\`e}l has received grants from Agendia B.V. and Intuitive Surgical outside the submitted work. Zakile A. Mfumbilwa, Janneke A. Wilschut, Martijn J.H.G. Simons, Bram Ramaekers, Manuela Joore, Christine M. Cramer-van der Welle, Franz M.N.H. Schramel, Ewoudt M.W. van de Garde, and Veerle M.H. Coup{\'e} declares no potential conflict of interest. Publisher Copyright: {\textcopyright} 2023, The Author(s).",

year = "2023",

month = dec,

doi = "10.1038/s41598-023-29286-5",

language = "English",

volume = "13",

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AU - Mfumbilwa, Zakile A

AU - Wilschut, Janneke A

AU - Simons, Martijn J H G

AU - Ramaekers, Bram

AU - Joore, Manuela

AU - Retèl, Valesca

AU - der Welle, Christine M Cramer-van

AU - Schramel, Franz M N H

AU - van de Garde, Ewoudt M W

AU - Coupé, Veerle M H

N1 - Funding Information: This work was funded by the Netherlands Organization for Health Research and Development (ZonMw) (grant number 846001002), the Dutch Cancer Society (KWF), and the Dutch health-care insurance company Zilveren kruis Achmea. The funding sources had no involvement in the conduct of this research. The authors thank all members of the Technology Assessment of Next Generation Sequencing in Personalized Oncology (TANGO) consortium ( https://zenodo.org/communities/tango-wgs/?page=1&size=20 ). Funding Information: Valesca Retèl has received grants from Agendia B.V. and Intuitive Surgical outside the submitted work. Zakile A. Mfumbilwa, Janneke A. Wilschut, Martijn J.H.G. Simons, Bram Ramaekers, Manuela Joore, Christine M. Cramer-van der Welle, Franz M.N.H. Schramel, Ewoudt M.W. van de Garde, and Veerle M.H. Coupé declares no potential conflict of interest. Publisher Copyright: © 2023, The Author(s).

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N2 - Recent discoveries in molecular diagnostics and drug treatments have improved the treatment of patients with advanced (inoperable) non-squamous non-small cell lung cancer (NSCLC) from solely platinum-based chemotherapy to more personalized treatment, including targeted therapies and immunotherapies. However, these improvements come at considerable costs, highlighting the need to assess their cost-effectiveness in order to optimize lung cancer care. Traditionally, cost-effectiveness models for the evaluation of new lung cancer treatments were based on the findings of the randomized control trials (RCTs). However, the strict RCT inclusion criteria make RCT patients not representative of patients in the real-world. Patients in RCTs have a better prognosis than patients in a real-world setting. Therefore, in this study, we developed and validated a diagnosis-treatment decision model for patients with advanced (inoperable) non-squamous NSCLC based on real-world data in the Netherlands. The model is a patient-level microsimulation model implemented as discrete event simulation with five health events. Patients are simulated from diagnosis to death, including at most three treatment lines. The base-model (non-personalized strategy) was populated using real-world data of patients treated with platinum-based chemotherapy between 2008 and 2014 in one of six Dutch teaching hospitals. To simulate personalized care, molecular tumor characteristics were incorporated in the model based on the literature. The impact of novel targeted treatments and immunotherapies was included based on published RCTs. To validate the model, we compared survival under a personalized treatment strategy with observed real-world survival. This model can be used for health-care evaluation of personalized treatment for patients with advanced (inoperable) NSCLC in the Netherlands.

AB - Recent discoveries in molecular diagnostics and drug treatments have improved the treatment of patients with advanced (inoperable) non-squamous non-small cell lung cancer (NSCLC) from solely platinum-based chemotherapy to more personalized treatment, including targeted therapies and immunotherapies. However, these improvements come at considerable costs, highlighting the need to assess their cost-effectiveness in order to optimize lung cancer care. Traditionally, cost-effectiveness models for the evaluation of new lung cancer treatments were based on the findings of the randomized control trials (RCTs). However, the strict RCT inclusion criteria make RCT patients not representative of patients in the real-world. Patients in RCTs have a better prognosis than patients in a real-world setting. Therefore, in this study, we developed and validated a diagnosis-treatment decision model for patients with advanced (inoperable) non-squamous NSCLC based on real-world data in the Netherlands. The model is a patient-level microsimulation model implemented as discrete event simulation with five health events. Patients are simulated from diagnosis to death, including at most three treatment lines. The base-model (non-personalized strategy) was populated using real-world data of patients treated with platinum-based chemotherapy between 2008 and 2014 in one of six Dutch teaching hospitals. To simulate personalized care, molecular tumor characteristics were incorporated in the model based on the literature. The impact of novel targeted treatments and immunotherapies was included based on published RCTs. To validate the model, we compared survival under a personalized treatment strategy with observed real-world survival. This model can be used for health-care evaluation of personalized treatment for patients with advanced (inoperable) NSCLC in the Netherlands.

KW - Humans

KW - Antineoplastic Agents/adverse effects

KW - Netherlands

KW - Cost-Benefit Analysis

KW - Lung Neoplasms/drug therapy

KW - Carcinoma, Non-Small-Cell Lung/drug therapy

KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use

KW - Competing risks

KW - Multicenter

KW - Tyrosine kinase inhibitors

KW - Survival outcomes

KW - Cost-effectiveness analysis

KW - Chemotherapy

KW - Pembrolizumab

KW - Simulation

KW - Immunotherapy

KW - 1st-line therapy

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DO - 10.1038/s41598-023-29286-5

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SN - 2045-2322

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JO - Scientific Reports

JF - Scientific Reports

IS - 1

M1 - 2349

ER -

Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands

Abstract

Bibliographical note

UN SDGs

Keywords

Access to Document

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