TY - JOUR
T1 - Systematic evaluation of the efficacy-effectiveness gap of systemic treatments in metastatic nonsmall cell lung cancer
AU - Santeon NSCLC Study Grp
AU - Cramer-van der Welle, Christine M.
AU - Peters, Bas J. M.
AU - Schramel, Franz M. N. H.
AU - Klungel, Olaf H.
AU - Groen, Harry J. M.
AU - van de Garde, Ewoudt M. W.
AU - Polman, A. J.
AU - van den Borne, B. E. E. M.
AU - van Putten, J. W. G.
AU - Smit, A. A. J.
AU - Termeer, A.
N1 - Copyright ©ERS 2018.
PY - 2018/12/1
Y1 - 2018/12/1
N2 - 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; pSurvival 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.
AB - 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; pSurvival 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.
KW - PHASE-III TRIAL
KW - GEMCITABINE PLUS CARBOPLATIN
KW - COOPERATIVE-ONCOLOGY-GROUP
KW - CISPLATIN VS. GEMCITABINE
KW - 1ST-LINE TREATMENT
KW - OPEN-LABEL
KW - PERFORMANCE STATUS
KW - STAGE IIIB
KW - MAINTENANCE BEVACIZUMAB
KW - MULTICENTER TRIAL
U2 - 10.1183/13993003.01100-2018
DO - 10.1183/13993003.01100-2018
M3 - Article
C2 - 30487206
SN - 0903-1936
VL - 52
JO - European Respiratory Journal
JF - European Respiratory Journal
IS - 6
M1 - 1801100
ER -