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Research Letter
July 12, 2021

Summary of Literature on Pediatric Real-world Evidence and Effectiveness

Author Affiliations
  • 1Office of Pediatric Therapeutics, Office of the Commissioner, Food and Drug Administration, Silver Spring, Maryland
  • 2Office of Medical Policy, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland
  • 3Office of the Commissioner, Food and Drug Administration, Silver Spring, Maryland
JAMA Pediatr. Published online July 12, 2021. doi:10.1001/jamapediatrics.2021.2149

The US Food and Drug Administration (FDA) has long used clinical real-world data (RWD) and clinical real-world evidence (RWE) to supplement information from traditional randomized clinical trials on the safety of medications in children and adults, but RWD and RWE are not commonly used to demonstrate effectiveness, especially in pediatrics. With the widespread use of electronic health care data, there may be new opportunities for RWE to assess the effectiveness of medical products that are on the market. The 21st Century Cures Act of 2016 provides specific milestones for the FDA to achieve in evaluating potential uses of clinical RWE to support regulatory decision-making. As part of that effort, the FDA published its Framework for FDA’s Real-World Evidence Program in 2018 (https://www.fda.gov/media/120060/download). The initial question within the domain of clinical pediatric RWE is: what are the descriptive characteristics of existing clinical pediatric RWE effectiveness studies? This review of clinical pediatric RWE studies assessed studies from ClinicalTrials.gov and other published drug studies that focused on therapeutic effectiveness from 5 subspecialties (ie, psychiatry, cardiology, rheumatology, pulmonology, and oncology), which, based in part on analogous adult studies, could have promising clinical RWE application and could differ from one another in their approach to using clinical RWE to evaluate effectiveness.

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