Augmenting the randomized controlled trial with real-world data to aid clinical decision making in metastatic renal cell carcinoma: a systematic review and meta-analysis

Aim: To evaluate how efficacy outcomes from real-world data (RWD) can support those from randomized controlled trials (RCTs), in the context of first-line tyrosine kinase inhibitor treatment of metastatic renal cell carcinoma. Patients & methods: PubMed, Ovid, MEDLINE and EMBASE were searched for RCTs and RWD studies with >= 50 adult patients per arm published in 2000-2017. Outcome measures were median progression-free survival, median overall survival and objective response rate. Results: A total of 13 RCTs and 22 RWD studies met eligibility criteria; 31, 28 and 25 studies, respectively, reported median progression-free survival, median overall survival and objective response rate. Summary outcome measures were similar in RWD and RCTs. Conclusion: RWD validates efficacy-based outcomes from RCTs and may provide supportive evidence to inform clinical decisions. Lay abstract: Clinical trials are an established way of gathering evidence to show that new drugs are effective. There are concerns that clinical trials do not fully represent those patients seen in everyday clinical practice. 'Real-world' studies look at the effect of drugs under normal conditions (everyday clinical practice). This study compared the results from 13 clinical trials and 22 real-world studies for a group of drugs called tyrosine kinase inhibitors in patients with metastatic renal cell carcinoma, a type of kidney cancer that has spread to other parts of the body. Overall, results from real-world studies were similar to those from clinical trials. This suggests that results from real-world studies may help doctors decide on the most effective treatment.