Harnessing real-world evidence in paediatric randomised controlled trials

Randomised controlled trials (RCTs) provide the highest level of evidence by rigorously evaluating treatments to ensure clinical decisions are grounded in robust data that improve patient outcomes. However, in paediatrics, certain factors can make their conduct more difficult, including recruitment challenges and ethical complexities. There is growing interest in using real-world evidence through routinely collected data (RCD) to improve the design, delivery and applicability of paediatric RCTs across diverse healthcare settings worldwide. This editorial is part of a series on RCD-enabled studies and their analysis. Other articles will explore the use of real-world evidence in observational research and examine specific analytical methods, including interrupted time series and difference in differences.