Background: Real World Evidence (RWE) complements the data of randomised clinical trials, that are based on selected, homogeneous patient populations and a limited study duration, with long-term data from clinical practice. As there has been no evaluation of immunotherapy in everyday care in Austria to date, a retrospective pilot study was conducted in six Austrian hospitals on the efficacy and safety of anti-PD-1/PDL1 monotherapy in patients with NSCLC using routine data.

Method: A matched-pair analysis to compare overall survival and progression-free survival of patients receiving immunotherapy with those receiving chemotherapy from the Tyrol Lung Cancer Project ("Tyrol Study") was conducted to describe the characteristics of NSCLC patients treated with immunotherapy in everyday hospital care; to analyse overall survival (OS), progression-free survival (PFS), objective response rate (ORR) and disease control rate (DCR) as well as safety endpoints (grade 3 and 4 adverse events) and to compare patient characteristics and outcomes from everyday care with the results of the pivotal studies.

Results: Especially for second-line therapy with immunotherapy, a higher objective response rate and a slightly longer median overall survival could be observed compared to the clinical trials, as well as a comparable median progression-free survival. In contrast, results from pivotal clinical trials for first-line pembrolizumab monotherapy could not be confirmed.

Conclusion: In summary, the present retrospective pilot study shows clinically relevant results regarding the use of immunotherapy in daily care practice and underlines the need for real world evidence to demonstrate the efficacy and safety of new therapies. Larger (prospective) real world studies are needed to contribute to a better understanding of the efficacy of immunotherapy in daily practice and to complement the results from randomised controlled trials to ensure the care of patients who benefit most from immunotherapy.