[Comment] Randomised trials in children with rhabdomyosarcoma: time for a change?

In The Lancet Oncology, Abha Gupta and colleagues1 report the results of a randomised, open-label, phase 3 trial (ARST1431) from the Children’s Oncology Group (COG), which investigated, for the first time, combining cytotoxic chemotherapy with a drug targeting specific molecular alterations as front-line therapy for patients with rhabdomyosarcoma. Rhabdomyosarcoma can be considered a rare disease, with approximately 450 children diagnosed each year in both Europe and the USA.