The RACE for Children Act provides hope we can accelerate therapies to children, adolescents, and young adults with cancer. As precision medicine increasingly influences paediatric cancer care, we must consider which drugs to prioritise, how to develop them through global collaboration and novel trial designs, and how to measure success. Melissa Hansen, Joanna L. Perkins and Harris Dalrymple at PRA Health Sciences discuss the need to re-evaluate pediatric cancer endpoints.