11 Clinical, methodological and patient/parent expert advice in paediatric drug development via conect4children

Introduction Children have the right to new and improved medicines.conect4children (c4c) aims to promote innovative trial design to optimise paediatric development plans while ensuring the voice of young patients and their families is heard. To address this a multidisciplinary advice service was set up Methods A network of Experts, divided into Expert Groups, was set-up via an open call for Experts. In parallel a Patient and Public Involvement (PPI) database was formed to include the expert opinion of paediatric patients and their parents. Advice is given according to the following process: (1) Sponsors contact c4c (2) a scoping interview is held (3) formation of ad-hoc Strategic Feasibility Advice Group (5) an advice meeting is held (6) an advice report is provided. To continuously improve this process, feedback from Experts and requestors on the service was collected Results 24 clinical and innovative methodology Expert Groups, consisting of >300 Experts, diverse in gender, seniority and geographical location were established. The PPI database includes registrations in 4 subgroups. To date (Dec 2021) 30 advice requests were received from academia and industry and 23 have been completed. Clinical, methodology and PPI Experts participated in several of these requests. Sponsors appreciated the diversity of the Expert Groups as well as the quality of the advice which in many cases significantly contributed to shaping the paediatric development strategy. Experts and PPI participants were satisfied with the advice process Conclusions c4c has shown a successful proof of concept for a European, multidisciplinary, advice service for paediatric drug development, tailored to industry and academia. This service presents a new framework for innovative and feasible paediatric trials