Comparison of the treatment results in patients with inoperable non-small cell lung cancer in clinical trials and in standard clinical practice using the pseudorandomization method

Rationale: Non-small cell lung cancer (NSCLC) is an aggressive disease with median survival of 12–14 months in inoperable patients in the pre-im-munotherapy era. Nowadays, under treatment with checkpoint inhibitors median survival is 19– 22 months. However, only a proportion of patients are sensitive to immune therapy. In this regard, inclusion into clinical trials remains a priority option for patients from medical perspective. Aim: To compare the results in NSCLC patients treated in accordance with the current clinical guidelines and in international clinical trials using the propensity score matching. Materials and methods: The study included data from 344 patients with histologically verified unresectable advanced NSCLC without activat-ing mutations, who received the 1st line systemic medical therapy at various combinations (single agent chemotherapy, platinum doublet-based therapy, chemoimmunotherapy, single agent immunotherapy) within the compulsory health in-surance (CHI), and from 90 patients, who received therapy in clinical trials. A direct comparison of long-term treatment results was carried out with the log-rank method. To exclude any influence of individual factors on survival rates, an univariate regression analysis and pseudorandomization ac-counting for these factors were carried out. Results: The direct comparison of the treatment results showed a higher progression-free survival rate in the patients treated according to clinical trial protocols, than in those treated under CHI (13.3 [95% confidence interval (CI) 8.1–18.5] months vs 6.4 [95% CI 5.9–6.9] months). Pseudorandomization of patients based on a combination of statistically significant parameters from the CHI and clinical trial groups showed a significantly longer time to progression in the trial group (13.3 [95% CI 8.3–18.3] vs 6.3 [95% CI 4.8–7.7] months). Conclusion: Participation in clinical trials is per se a factor that can significantly impact the longer duration of the treatment effect. This indicates the necessity of the most active use of this tool in clinical practice. © 2022, Moscow Regional Research and Clinical Institute. All rights reserved.