OBJECTIVES: Randomized controlled trials (RCTs) are the gold-standard data source for National Institute for Care and Health Excellence (NICE) appraisals; however, it is possible that their results might not be generalizable to patients seen in clinical practice. This research aimed to evaluate the extent of real-world evidence (RWE) use in NICE submissions for oncology in the United Kingdom (UK).
METHODS: All publicly available appraisal documents from the UK National Institute for Health and Care Excellence (NICE) published between October 1, 2019 and October 31, 2022 were reviewed. Appraisals in oncology reporting the use of RWE to demonstrate treatment effects were identified and key data were extracted.
RESULTS: Nearly half of the appraisals (46/102) utilized RWE for assessing drug effectiveness. The data sources varied but primarily included data from the Cancer Drugs Fund and other clinical databases (eg, Flatiron), registry data, and compassionate use programs. RWE was primarily utilized for assessing comparative treatment effects, such as matching-adjusted indirect comparisons and naïve/unadjusted indirect treatment comparisons or utilizing external/historical/pseudo/simulated control arms. RWE also supported modeling by allowing extrapolation of survival data, model validation for projected survival data, and providing inputs for various scenario analyses. However, the Evidence Review Group critiqued the use of RWE and highlighted the small sample size/lack of generalizability, high risk of bias, and key population differences compared to the trial population (ie, differences in potential treatment effect modifiers not adequately accounted for in comparisons).
CONCLUSIONS: There is a significant utilization of RWE in NICE appraisals either alongside RCTs or as a distinct evidence source. NICE is currently working on developing its RWE Framework, which will provide detailed guidance regarding the conduct of real-world studies. Routine use of RWE may help with filling evidence gaps and accelerate patients’ access to innovative technologies.
