Evaluation of publicly-sponsored lung cancer trials in US: Are experimental treatments better than the control ones?

Abstract

7157 Background: Lung cancer is one of the leading causes of cancer deaths in the US. A considerable number of these patients are treated in randomized clinical trials (RCTs). However, how often experimental lung treatments are superior to control treatments is not known. To accurately assess this, three factors have to be taken into consideration: publication rate, methodological quality of trials and the choice of the comparator intervention. Methods: All phase 3 RCTs that were completed to date by 3 National Cancer Institute cooperative groups (ECOG, NCCTG and RTOG) were eligible for the analysis. We identified 50 RCTs enrolling 11,631 lung cancer patients. The methodological quality of the trials was assessed for possible effects of bias and random error on the outcomes of the trials. The possible impact of the choice of a comparator intervention was also assessed. To evaluate the outcomes, we extracted data on survival (OS), disease free survival (DFS), response rate (RR) and treatment-related mortality (TRM). In addition, the final investigators’ preference about experimental or control interventions was used to assess whether experimental treatments were better than controls. Results: In terms of OS, DFS and RR experimental treatments were as likely as control treatments to be successful—Hazard ratio [HR]= 0.96 (99%CI 0.92–1.01)], [HR = 0.98 (99%CI 0.98–1.17)] and [RR = 1.11 (99%CI 0.84–1.46)], respectively. TRM was worse with experimental treatments [HR = 1.61 (99% CI 1.10–2.36)]. Investigators’ preferences for experimental vs. control treatments were 30% and 70%, respectively (p < 0.001). The quality of trials was high. We did not find any evidence that the methodological quality of trials/choice of comparator influenced the results. Conclusions: We found that there is no clear pattern that predicts which treatment will be better. In aggregate, there is about an equal chance for experimental and standard treatments to result in successful outcomes or that the outcomes may not differ between two types of the treatments, although TRM is slightly higher with experimental arms. This is a welcome finding because if one intervention (experimental or control) was consistently better, maintaining randomization will be difficult. No significant financial relationships to disclose.