Randomized controlled clinical trials (RCT) are the gold standard for establishing the safety and efficacy of new diabetes treatments. But real-world observations can add important information that RCTs may not provide.
“Randomized clinical trials provide evidence about efficacy and safety of medications, but real-world evidence can provide information about the effectiveness of a medication beyond the strict confines of a tightly controlled and monitored trial population,” said Lawrence Blonde, MD, Director of the Diabetes Clinical Research Institute, Frank Riddick Diabetes Institute in the Department of Endocrinology at Ochsner Medical Center. “Real-world studies provide information about the effectiveness of medications in the broader population receiving real care in real clinical practices.”
Dr. Blonde is one of five experts who will review the latest real-world diabetes findings and discuss the advantages and limitations of real-world evidence during a Scientific Sessions symposium on Friday, June 22. The two-hour symposium is titled Real-World Evidence in Diabetes.
Symposium presenters include Kamlesh Khunti, FRCGP, FRCP, MD, PhD, FMedSci, Head of Department and Professor of Primary Care Diabetes and Vascular Medicine at the University of Leicester; Stewart B. Harris, MD, MPH, FCFP, FACPM, Professor and the Canadian Diabetes Association Chair in Diabetes Management, and the Ian McWhinney Chair of Family Medicine in the Schulich School of Medicine & Dentistry at the University of Western Ontario; and Timothy Bailey, MD, FACE, FACP, Director of Advanced Metabolic Care + Research.
Real-world studies generally include a greater mix and variety of patients than RCTs, Dr. Blonde noted. There are likely to be more older adults in real-world studies, as well as greater ethnic and racial diversity. Due to the real-world setting, these patients present a more varied mix of patient histories, comorbidities, adherence patterns, and other variables. Real-world studies can also address questions that may be impractical to explore in the RCT setting.
One real-world study, for example, found that switching to glargine-300 (Gla-300) compared to switching to other basal insulins [primarily glargine-100 and insulin detemir] resulted in a similar change in A1C at baseline, but that the incidence of hypoglycemia and adjusted event rates were significantly lower with Gla-300, as was the utilization of healthcare resources related to hypoglycemia [Zhou, F.L., et al., 2018].
However, not everyone understands the limitations of collecting and analyzing real-world data and how those limitations can be addressed, Dr. Blonde said.
“Real-world studies frequently involve nonrandomized patient groups,” he noted. “As a result, there can be more risk for confounding factors that could influence study results. For example, the reasons for the choice of different therapies are frequently not available, which can result in selection bias. While many of these confounding factors can be easier to control in an RCT, a number of statistical methods can be used to try to address these limitations in real-world studies, and will be discussed in this session.
“Real-world studies, for now, are not felt to produce as high a level of evidence as RCTs,” Dr. Blonde continued. “But combining randomized controlled trials with real-world evidence can provide complementary information that can be valuable to regulatory authorities, payors, and particularly clinicians seeing patients in everyday practice. This session hopes to help attendees to better use the information that comes from real-world studies in their patient care and research activities.”