Observed versus modelled lifetime overall survival of targeted therapies and immunotherapies for advanced non-small cell lung cancer patients – A systematic review

Simons, Martijn; Ramaekers, Bram; Peeters, Andrea; Mankor, Joanne; Paats, Marthe S.; Aerts, Joachim; Harten, Wim H. van; Retèl, Valesca P.; Joore, Manuela A.

doi:10.1016/j.critrevonc.2020.103035

Cited by 9 publications

(13 citation statements)

References 60 publications

(107 reference statements)

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“…Secondly, currently there are no phase III RCTs of crizotinib when used in patients with a ROS1 mutation, the combination of dabrafenib and trametinib used in patients with BRAF v600E mutation, and larotrectinib when used patients with NTRK (1, 2, 3) mutation. In simulating the personalized strategy for subgroups ROS1, BRAF v600E , and NTRK (1,2,3) , it was assumed that crizotinib in ROS1 has the similar effectiveness as crizotinib in the ALK subgroup [33], dabrafenib and trametinib was equally effective in NSCLC BRAF v600E as in melanoma BRAF val600 [67], and for larotrectinib in NTRK (1,2,3) was assumed to have PF HR as that of prognostic value of EGFR classic [38]. There was no data to validate the model outputs of these subgroup.…”

Section: Limitationsmentioning

confidence: 99%

Modelling of Lung Cancer Therapy; Modelled And Real-World Impact of Targeted Therapy And Immunotherapy In The Netherlands

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Wilschut

Simons

et al. 2022

Preprint

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Recent discoveries in molecular diagnostics and lung cancer drugs have improved the treatment of patients with advanced (inoperable) non-squamous 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 cost-effectiveness for optimization of lung cancer care. Traditionally, cost-effectiveness models built to evaluate new lung cancer treatments were based on randomized control trial (RCT) findings. The strict RCT inclusion criteria make RCT patients not representative of real-world patients: Patients in RCT have better prognosis compared to real-world patients. Therefore, in this study we have 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 – 2014 in one of six Dutch’s teaching hospitals. To allow simulation of 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 against 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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Section: Limitationsmentioning

confidence: 99%

Modelling of Lung Cancer Therapy; Modelled And Real-World Impact of Targeted Therapy And Immunotherapy In The Netherlands

Mfumbilwa

Wilschut

Simons

et al. 2022

Preprint

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“…Parameter values for the decision tree and STM are listed in Table 1. Results from a recent systematic review were used to model the OS and PFS of targeted therapies and immunotherapies in NSCLC [13,[37][38][39][40][41][42][43][44][45][46][47][48][49][50][51]. Treatment effects that were not available in the systematic review were replaced with the OS and PFS of chemotherapy of patients with EGFR mutations.…”

Section: Identified Targets and Treatment Effectsmentioning

confidence: 99%

“…The clinical utility of a sequencing technology lies in its ability to accurately identify individuals with a given disease and to aid in personalised and targeted treatment selection. Targeted treatments have demonstrated significant improvement of progression-free survival (PFS) and overall survival (OS) for some patients with NSCLC [ 13 ]. Many actionable targets can be tested for (e.g.…”

Section: Introductionmentioning

confidence: 99%

Early Cost Effectiveness of Whole-Genome Sequencing as a Clinical Diagnostic Test for Patients with Inoperable Stage IIIB,C/IV Non-squamous Non-small-Cell Lung Cancer

et al. 2021

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Background Advanced non-small-cell lung cancer (NSCLC) harbours many genetic aberrations that can be targeted with systemic treatments. Whole-genome sequencing (WGS) can simultaneously detect these (and possibly new) molecular targets. However, the exact added clinical value of WGS is unknown. Objective The objective of this study was to determine the early cost effectiveness of using WGS in diagnostic strategies compared with currently used molecular diagnostics for patients with inoperable stage IIIB,C/IV non-squamous NSCLC from a Dutch healthcare perspective. Methods A decision tree represented the diagnostic pathway, and a cohort state transition model represented disease progression. Three diagnostic strategies were modelled: standard of care (SoC) alone, WGS as a diagnostic test, and SoC followed by WGS. Treatment effectiveness was based on a systematic review. Probabilistic cost-effectiveness analyses were performed, and threshold analyses (using €80,000 per quality-adjusted life-year [QALY]) was used to explore the early cost effectiveness of WGS. Results WGS as a diagnostic test resulted in more QALYs (0.002) and costs (€1534 [incremental net monetary benefit -€1349]), and SoC followed by WGS resulted in fewer QALYs (-0.002) and more costs (€1059 [-€1194]) compared with SoC alone. WGS as a diagnostic test was only cost effective if it was priced at €2000 per patient and identified 2.7% more actionable patients than SoC alone. Treating these additional identified patients with new treatments costing >€4069 per month decreased the probability of cost effectiveness. Conclusions Our analysis suggests that providing WGS as a diagnostic test is cost effective compared with SoC followed by WGS and SoC alone if costs for WGS decrease and additional patients with actionable targets are identified. This costeffectiveness model can be used to incorporate new findings iteratively and to support ongoing decision making regarding the use of WGS in this rapidly evolving field.

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“…The cost-effectiveness and wider public benefits of WGS versus SOC for advanced NSCLC are being assessed, as a blueprint for tumor-overarching modeling. First, a systematic review was performed on the long-term treatment effects of targeted therapies and immunotherapies in patients with metastatic NSCLC [43].…”

Section: Wp4: Cost-effectiveness Of Wgs Compared To Socmentioning

confidence: 99%

“…Outcomes of the cost-effectiveness analysis were expected costs, effects (quality adjusted life year), and incremental cost-effectiveness ratio (ICER). Input was based on literature, including the systematic review [43] and extensive expert opinions. The aim of the cost-effectiveness model was to estimate the ranges where WGS is potentially cost-effective compared to SOC.…”

Section: Wp4: Cost-effectiveness Of Wgs Compared To Socmentioning

confidence: 99%

Early technology assessment of using whole genome sequencing in personalized oncology

Simons

Ven

Coupé

et al. 2021

Expert Review of Pharmacoeconomics & Outcomes Research

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Introduction: Personalized medicine-based treatments in advanced cancer hold the promise to offer substantial health benefits to genetic subgroups, but require efficient biomarker-based patient stratification to match the right treatment and may be expensive. Standard molecular diagnostics are currently very heterogeneous, and tests are often performed sequentially. The alternative to whole genome sequencing (WGS) i.e. simultaneously testing for all relevant DNA-based biomarkers thereby allowing immediate selection of the most optimal therapy, is more costly than current techniques. In the current implementation stage, it is important to explore the added value and cost-effectiveness of using WGS on a patient level and to assess optimal introduction of WGS on the level of the healthcare system. Areas covered: First, an overview of current worldwide initiatives concerning the use of WGS in clinical practice for cancer diagnostics is given. Second, a comprehensive, early health technology assessment (HTA) approach of evaluating WGS in the Netherlands is described, relating to the following aspects: diagnostic value, WGS-based treatment decisions, assessment of long-term health benefits and harms, early cost-effectiveness modeling, nation-wide organization, and Ethical, Legal and Societal Implications. Expert opinion:This study provides evidence to guide further development and implementation of WGS in clinical practice and the healthcare system.

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Observed versus modelled lifetime overall survival of targeted therapies and immunotherapies for advanced non-small cell lung cancer patients – A systematic review

Cited by 9 publications

References 60 publications

Modelling of Lung Cancer Therapy; Modelled And Real-World Impact of Targeted Therapy And Immunotherapy In The Netherlands

Modelling of Lung Cancer Therapy; Modelled And Real-World Impact of Targeted Therapy And Immunotherapy In The Netherlands

Early Cost Effectiveness of Whole-Genome Sequencing as a Clinical Diagnostic Test for Patients with Inoperable Stage IIIB,C/IV Non-squamous Non-small-Cell Lung Cancer

Early technology assessment of using whole genome sequencing in personalized oncology

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