ICH Clinical investigation of medicinal products in the paediatric population E11 (R1)

This guideline provides internationally harmonised recommendations for the design and conduct of clinical studies involving children and is particularly relevant for rare diseases, many of which have paediatric onset. It supports protocol development by addressing key considerations such as age-appropriate study design, selection of clinically meaningful endpoints, dose selection and optimisation, pharmacokinetic and pharmacodynamic assessments, safety monitoring, and formulation development suitable for children. The guideline also discusses the use of extrapolation, modelling and simulation, and innovative approaches to reduce the number of children required for clinical studies while maintaining scientific validity.

The guidance emphasises ethical principles specific to paediatric research, including minimising risks and burden, ensuring age-appropriate assent and informed consent processes, and designing studies that are feasible for children and their families. It also encourages the use of existing knowledge and international collaboration to optimise study design, which is particularly important in rare diseases where patient populations are small. Researchers developing paediatric rare disease trial protocols can use this guideline to ensure that their studies are scientifically robust, ethically appropriate, and aligned with international regulatory expectations.