Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands
Mfumbilwa, Zakile A; Wilschut, Janneke A; Simons, Martijn J H G; Ramaekers, Bram; Joore, Manuela; Retèl, Valesca; der Welle, Christine M Cramer-van; Schramel, Franz M N H; van de Garde, Ewoudt M W; Coupé, Veerle M H
(2023) Scientific Reports, volume 13, issue 1
(Article)
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
... read more
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.
show less
Download/Full Text
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, General
ISSN: 2045-2322
Publisher: NLM (Medline)
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).
(Peer reviewed)