Intensive care medicine

Surgery

Clinical trial

Lung cancer

Systemic therapy

Internal medicine

Oncology

Medicine

Gastroenterology

Breast cancer

Disease

Cancer

Lung Cancer

Background: The aim of this study is to assess the difference between outcomes in clinical trials and in real-world (efficacy-effectiveness gap) of systemic treatments for ED SCLC. Methods: All patients diagnosed with ED SCLC between 2008 and 2014 in four Dutch large teaching hospitals (Santeon network) were studied. For every patient, an efficacy-effectiveness factor (EE-factor) was calculated by dividing individual patients' overall survival (OS) by the pooled median OS assessed from clinical trials with the respective treatment. Results: From 501 diagnosed patients, 366 (73%) started first-line treatment. Table 1 outlines the real-world OS, reference OS from clinical trials, and EE-factor for the most used first-line regimens (n=358). Overall, the median EE-factor was 0.80 (p&lt;0.001 from 1.00). Poor performance status (ECOG≥2) was an independent predictor for a lower EE-factor. The EE-gap was 15% in patients with ECOG0-1 and 37% in patients with ECOG≥2 (EE-factor 0.63). The proportion of patients with ECOG≥2 in real-world was different from those in clinical trials (25% versus 16%, p&lt;0.001). Conclusion: Overall survival of patients with ED SCLC treated with systemic therapy in real-world practice is 20% shorter than for patients included in trials. Performance status partly explained this gap.

Systematic evaluation of the efficacy-effectiveness gap of systemic treatments in extensive disease small cell lung cancer (ED SCLC)