Sara Y. Tartof, PhD, MPH, Kaiser Permanente Bernard J. Tyson School of Medicine (KPSOM) Assistant Professor of Health Systems Science, has coauthored a study showing that while randomized controlled trials (RCTs) are the gold standard for treatment evaluation, real-world evidence can provide valuable insights.
The study found that, after a randomized controlled trial investigating the effect on 28-day mortality in severe COVID-19 pneumonia of tocilzumab (a rheumatoid arthritis treatment) failed to yield conclusive results, researchers created a real-world external comparator arm mirroring the RCT participants. An external comparator arm is a cohort of patients from real-world data, such as registries, natural history studies, or medical records, and the data it provided was used to supplement RCT findings.
The comparative study revealed that patients receiving tocilizumab had a similar risk of death to those in the external comparator arm, confirming the RCT results. This approach showcases the potential of using external comparator arms to complement RCT data and validate primary RCT analyses, offering a broader perspective on treatment effectiveness.