Drug development for pediatric cancers has been slower than for adult cancers, and regulatory authorities in the United States and the European Union have introduced legislation to accelerate pediatric drug development.

To comply with the new regulations, sponsors need to consider the need for studies in a pediatric population, for each drug being developed.

Compared with adult studies, the specific needs of a pediatric population may require changes in the assessment of preclinical rationale and translational relevance, formulation development, pharmacokinetics/pharmacodynamics, clinical endpoints, and clinical trial design.