Preparedness of medicines’ clinical trials in paediatrics

This document provides practical recommendations to improve the preparedness of paediatric clinical trials before protocol finalisation and study initiation. It complements regulatory guidance by focusing on the operational, methodological and patient-centred elements that contribute to successful trial design and implementation. The recommendations are highly relevant for rare disease clinical trials, where limited patient populations, complex study logistics and the need for international collaboration require careful planning from the earliest stages of protocol development.

The guidance encourages researchers to consider feasibility, recruitment strategies, eligibility criteria, endpoint selection, participant burden, study procedures, age-appropriate assessments, and long-term follow-up during protocol development. It also highlights the importance of engaging patients, families, healthcare professionals, research networks and other stakeholders early in the design process to improve study relevance, acceptability and recruitment. Operational aspects such as site selection, trial logistics, training, communication, and contingency planning are addressed to enhance trial readiness and minimise avoidable delays.

For rare disease researchers, this resource supports the development of scientifically robust, feasible and participant-centred protocols by promoting early trial preparedness, multidisciplinary collaboration, risk assessment, and proactive planning. It is particularly useful when designing paediatric studies that require international multicentre collaboration, innovative trial designs, and strategies to optimise recruitment and retention while minimising the burden on children and their families.