Abstract
BACKGROUND: Although randomized, controlled trials (RCTs) are seen as the gold standard for evidence in clinical medicine, a number of considerations are increasing the use of real-world data (RWD) to generate evidence. A series of methodological challenges must be overcome in order for such real-world evidence (RWE) to gain acceptance. In diabetes, RWE faces some particular issues that have limited its development. As the natural history of diabetes progresses, patients' disease changes over time and treatments will be modified as a result. This evolving disease and treatment pattern requires application of methods that account for such changes over time. Research developing RWE in diabetes and other conditions has sometimes been subject to important biases, and researchers should be aware of, and take steps to mitigate potential for bias in order to enhance the evidence produced. RESULTS: We review a RWE study that replicated and extended evidence provided by a RCT regarding the effects of weekly exenatide relative to basal insulin (glargine or detemir) to illustrate a potential application of RWE. This study observed a 0.7% decrease in HbA1C for weekly exenatide relative to a 0.5% decrease in HbA1C for the comparator along with a 2 kg weight loss for weekly exenatide relative to a 0.25 kg weight gain, effects that were close to those from the RCT. Further, the RWE study was able to extend results to patient populations that were not well represented in the RCT. CONCLUSION: Despite numerous challenges, RWE can be used to complement evidence from RCTs.