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

Abstract

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.

The Clinical Significance of Routine Risk Categorization in Metastatic Renal Cell Carcinoma and its Impact on Treatment Decision-Making: A Systematic Review

“…The efficacy of the mRCC treatments in clinical trials appeared to be similar to the reported effectiveness in observational studies. This is consistent with other analyses, which suggest a role for real-world data as a complementary source of evidence to RCTs to aid clinical decision-making [24]. The exception may be the ORR for sunitinib reported by Di Giorgi et al [25], which appeared to be substantially higher than that reported in clinical trials.…”

Patient selection and risk factors in the changing treatment landscape of metastatic renal cell carcinoma

“…In this respect, there may also be an increase in the number of real-world data analyses published. Available studies suggest that data from randomized controlled trials are generally consistent with those of real-world data, which may provide complimentary evidence to inform treatment selection [65][66][67][68].…”

What is the Role of Real-World Data in Metastatic Renal Cell Cancer?

“…Patients with mRCC seen in everyday clinical practice are more likely to have poor-risk disease and impaired performance status compared with phase III trial participants; this may have an impact on the variation in post-trial reported survival outcomes [18]. Reassuringly, meta-analyses have shown that overall and progression-free survival with TKI therapy in real-world datasets and clinical trials are remarkably similar [22,23].…”