Systematic evaluation of the efficacy-effectiveness gap of systemic treatments in metastatic NSCLC
- Christine M. Cramer – van der Welle1,
- Bas J.M. Peters2,
- Franz M.N.H. Schramel3,
- Olaf H. Klungel4,
- Harry J.M. Groen5 and
- Ewoudt M.W. van de Garde2,4
- for the Santeon NSCLC Study Group*
- 1Santeon Hospital Group, Utrecht, The Netherlands
- 2Department of Clinical Pharmacy, St. Antonius Hospital, Utrecht/Nieuwegein, The Netherlands
- 3Department of Pulmonary Diseases, St Antonius Hospital, Utrecht/Nieuwegein, The Netherlands
- 4Division of Pharmacoepidemiology and Clinical Pharmacology, Department of Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands
- 5University of Groningen and University Medical Center Groningen, Department of Pulmonary Diseases, Groningen, the Netherlands
- CM Cramer – van der Welle, Santeon, Herculesplein 38, 3584 AA Utrecht, the Netherlands. E-mail: c.van.der.welle{at}antoniusziekenhuis.nl
* The Santeon NSCLC Study Group (collaborators) are: A.J. Polman, Medisch Spectrum Twente, Enschede, The Netherlands; B.E.E.M. van den Borne, Catharina Hospital, Eindhoven, The Netherlands; J.W.G. van Putten, Martini Hospital, Groningen, The Netherlands; F.M.N.H. Schramel, St. Antonius Hospital, Nieuwegein, The Netherlands; A.A.J. Smit, OLVG, Amsterdam, The Netherlands; A. Termeer, Canisius Wilhelmina Hospital, Nijmegen, The Netherlands
Abstract
The divergence between clinical trial results and real-world outcomes is largely unknown for many cancer types. The present study aims to overall assess the efficacy-effectiveness gap (difference between outcomes in clinical trials and real-world) in systemic treatment for metastatic non-small 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 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.
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 ECOG 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 less subsequent lines of treatment partly explained this difference.
Footnotes
This manuscript has recently been accepted for publication in the European Respiratory Journal. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Please open or download the PDF to view this article.
Conflict of interest: Dr. Schramel has nothing to disclose.
Conflict of interest: Dr. Klungel reports grants from GSK, outside the submitted work.
Conflict of interest: Dr. Groen reports other from BMS, other from Roche, other from Novartis, grants from Boehringer-Ingelheim, other from Merck, other from Pfizer, outside the submitted work.
Conflict of interest: Dr. van de Garde reports grants from Dutch Cancer Society, grants from Roche Netherlands BV, during the conduct of the study.
Conflict of interest: Dr. Cramer - van der Welle has nothing to disclose.
Conflict of interest: Dr. Peters has nothing to disclose.
This is a PDF-only article. Please click on the PDF link above to read it.
- Copyright ©ERS 2018