Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

•Survival and response rates remain most prominent endpoints in paediatric ALL.•Intermediate clinical endpoints / molecular biomarkers for efficacy are being studied.•Mid / long-term safety endpoints including secondary malignancies are being studied.•Current treatment success is driving new paradigms to optimise drug development.•Regulatory tools in place support meaningful drug development in paediatric ALL.