Despite major progress over the last 50 years (80% of young people with malignancy are disease free at 5 years after diagnosis), cancer remains the most common cause of death by disease beyond the age of one.
Recently, there have been major breakthroughs in understanding cancer and developing many innovative effective therapies for adults. Over the last 20 years, the landscape of drug development for children and adolescents has significantly changed, as result of regulatory initiatives in the US (Best Pharmaceutical Act, 2002; Pediatric Research Equity Act, 2003) and in Europe (The European Paediatric Medicine Regulation, 2006). These regulations have been successful in several paediatric specialties providing better medicines for children. However, in paediatric oncology, needs are far from being addressed with too many developments of relevant oncology drugs in children being waived or significantly delayed.
Therefore, there is a need to accelerate the process of evaluating innovative therapies and introducing them in standard cancer care in children and adolescents.