Since its enactment in 2003, the Pediatric Research Equity Act (PREA) has aimed to ensure that new drugs are adequately tested in children before entering the market. A recent analysis examines the implementation of PREA for drugs approved between 2015 and 2021, shedding light on the prevalence and nature of pediatric testing during this period.
PREA's Impact on Pediatric Drug Development
PREA mandates preapproval pediatric testing for new drugs, intending to prevent children from being “therapeutic afterthoughts.” However, the law includes exemptions for drugs indicated for rare diseases, allows deferred trials for drugs otherwise ready for use in adults, and permits trial waivers in impractical circumstances, such as diseases not affecting children. Congress eliminated the rare disease exemption for certain adult cancer drugs submitted after 2020.
The study underscores the importance of ongoing evaluation of PREA's effectiveness in ensuring that children benefit from evidence-based medicine tailored to their unique physiological needs. Further research and policy adjustments may be necessary to optimize pediatric drug development and address unmet medical needs in this vulnerable population.
