Finding Better Ways to Fill Gaps in Pediatric Health Research

Pediatric health research and development (R&D) has long lagged behind health R&D for adults. Methodological and ethical challenges associated with pediatric research increase the costs of running pediatric trials, and the market for pediatric products is relatively small. The subsequent knowledge gaps and lack of child-specific product development have resulted in high off-label and unlicensed medication prescription rates in children. The past decade has seen increased recognition of this problem, and both the United States and the European Union have implemented legislative measures to stimulate pediatric health research by providing incentives and funding for pediatric studies and by requiring pediatric studies for new drug applications when appropriate. Although these measures have led to an increase in the number of pediatric trials being conducted and to several knowledge gaps being addressed, the trials remain driven largely by market incentives, and evaluations of the measures in the United States and the European Union show that other areas remain neglected. Moreover, the majority of the global pediatric disease burden lies with populations in lowand middle-income countries, for whom the lack of health R&D remains a significant problem.