Background
Real-World Data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. Real-World Evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWD can be analyzed to produce RWE on the usage and/or benefits and risks of a medication or a medical product. RWD and RWE offer the possibility to bring about novel insights on patients’ clinical care, regulatory decision-making, and health technology assessment. Detailed information on data sources, data standards, data reliability, how to generate evidence, and regulatory considerations can be found on FDA’s document titled Framework for FDA’s Real-World Evidence Program (U.S. FDA, 2018). RWE has the potential to supplement information derived from clinical trials, providing accessible data in a shorter time and at lower costs. This is especially relevant to pediatrics, where the evidence base to guide medication use in children may be insufficient. Studies using RWD offer the opportunity to assess long-term effects and the safety of medications that are used to treat pediatrics population (Lasky et al., 2020).
