Researching and developing innovative and therapeutically appropriate medicines for children is an important priority for America’s biopharmaceutical research companies. Developing medicines for children requires specific, tailored approaches to clinical trial recruitment and conduct, study design, and assessing outcomes. By design, this work is complex, iterative in nature and builds on prior scientific advances.
While significant progress has been made over the past few decades, unmet medical needs remain, particularly in scientifically challenging disease areas. The policy frameworks that protect incentives for research and development and account for the nuances of developing medicines for children are essential for spurring continued progress.