ArticleThe European respiratory journal2018
Systematic evaluation of the efficacy-effectiveness gap of systemic treatments in metastatic nonsmall cell lung cancer.
Article in The European respiratory journal, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
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Who cites it
34 citing papers in PubMed.
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- 'Is it my last Christmas?' Using real-world data as a prompt to reflect on goal-concordant advanced lung cancer care-a retrospective, longitudinal study.BJC reports · 2025Article
- A Sensitivity Analysis Framework Using the Proxy Pattern-Mixture Model for Generalization of Experimental Results.Statistics in medicine · 2025Article
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- ICTIS: A Novel Scoring System to Assess the Inclusivity of Advanced NSCLC Immunotherapy Trials.JTO clinical and research reports · 2025Article
- Real-world treatment patterns and clinical outcomes with tucatinib-based therapy in patients with HER2-positive metastatic breast cancer: analyses of two nationwide administrative health claims databases.International journal of clinical oncology · 2025Article
- Cost-Effectiveness of Pembrolizumab Monotherapy for High Programmed Death Ligand 1 Advanced or Metastatic Non-small Cell Lung Cancer Depends on Long-Term Survivors.Clinical drug investigation · 2025Article
- Health Care Contact Days and Outcomes in Clinical Trials vs Routine Care Among Patients With Non-Small Cell Lung Cancer.JAMA network open · 2025Article
- Recent Survival Gains in Stage IV NSCLC by Sociodemographic Strata.JTO clinical and research reports · 2025Article
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- Real-world evidence for pembrolizumab in non-small cell lung cancer: a nationwide cohort study.British journal of cancer · 2025Article
- Review
- Exploring the Cost Effectiveness of a Whole-Genome Sequencing-Based Biomarker for Treatment Selection in Patients with Advanced Lung Cancer Ineligible for Targeted Therapy.PharmacoEconomics · 2024Article
- The potential for deprescribing in a palliative oncology patient population: a cross-sectional study.European journal of hospital pharmacy : science and practice · 2023Article
- Effectiveness of first-line anticancer treatment may predict treatment response in further lines in stage III/IV patients with non-small cell lung cancer.Journal of cancer research and clinical oncology · 2023Article
- Analysis of the characteristics and the degree of pragmatism exhibited by pragmatic-labelled trials of antineoplastic treatments.BMC medical research methodology · 2023Review
- Development and validation of a decision model for the evaluation of novel lung cancer treatments in the Netherlands.Scientific reports · 2023Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The divergence between clinical trial results and real-world outcomes is largely unknown for many cancer types. The present study aims overall to assess the efficacy-effectiveness gap (difference between outcomes in clinical trials and the real world) in systemic treatment for metastatic nonsmall cell lung cancer (NSCLC).All patients diagnosed with stage IV NSCLC between 2008 and 2014 within a network of seven Dutch large teaching hospitals (Santeon) were studied. For every patient, an efficacy-effectiveness (EE) factor was calculated by dividing individual patients' overall survival (OS) by the pooled median OS assessed from clinical trials with the respective treatment.From 2989 diagnosed patients, 1214 (41%) started with first-line treatment. For all studied regimens, real-world OS was shorter than OS reported in clinical trials. Overall, the EE factor was 0.77 (95% CI 0.70-0.85; p<0.001). Real-world patients completed their treatment plan less often and proceeded less frequently to further lines of treatment. These parameters together with Eastern Cooperative Oncology Group performance status explained 35% of the variation in EE factor.Survival of patients with metastatic NSCLC treated with chemotherapy or targeted therapy in real-world practice is nearly one-quarter shorter than for patients included in trials. Patients' performance status, earlier discontinuation and fewer subsequent lines of treatment partly explained this difference.
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