27th Annual EuroMeeting

Despite obvious phenotypic differences, increasing evidence demonstrates that adult and paediatric diseases may share similar molecular determinants of disease occurrence and progression. Pursuing a disease-centred, rather than drug-centred approach, allows a change in emphasis from adult indications with paediatric counterparts to diseases in children with greatest unmet need based on the mechanism-of-action and therapeutic target of a molecule. While the current era of targeted therapies and personalized medicine has created the opportunity to change the paradigm of paediatric drug development, clinical trials in rare paediatric conditions remains a major challenge. Accurate projection of recruitment rates and successful accrual of patients in paediatric trials is often difficult due to the limited number of cases, the absence of historical data of paediatric patients participating in clinical trials, and logistical challenges of conducting a global clinical trial in rare paediatric conditions. Leveraging academic/industry partnerships and clinical trial networks are an efficient way to share scientific and clinical information across geographic regions, conduct comprehensive feasibilities to inform initial enrolment projections, and provide ongoing feasibility to mitigate clinical trial conduct challenges.