PhD defence: Real-world treatment outcomes in metastatic lung cancer From efficacy-effectiveness gap evaluation to informed clinical decis

In clinical trials, new treatments are tested to provide evidence of clinical efficacy. Physicians use these results to advice patients on what type of treatment to choose. However, results in clinical trials may differ from outcomes in real-world practice. Our studies provide evidence for a gap between efficacy of chemotherapy and immunotherapy in patients with metastatic lung cancer as reported in clinical trials and effectiveness in real-world practice in the Santeon hospitals. Patients treated in real-world practice live on average one-quarter shorter than clinical trial participants. One explanation for this gap is that patients participating in clinical trials often have a better performance status, while in daily practice many different patients are given systemic treatment.

As a result of these studies, we developed a web-based tool in which the outcomes of various treatments for patients with metastatic lung cancer of all Santeon hospitals between 2008 and 2014 are listed. Thoracic oncologists select characteristics of the patient, such as age and condition, and tumour characteristics. The web-based tool shows outcomes of the different treatment options from comparable patients. Such a tool based on real-world practice is unique and could enable patients’ informed decision-making. In our pilot study, both patients and thoracic oncologists reported an added value of the tool in clinical practice. Future effort is needed to further develop and implement the tool in daily care for patients with lung cancer.