Guideline on pharmaceutical development of medicines for paediatric use

The EMA Guideline on Pharmaceutical Development of Medicines for Paediatric Use provides scientific recommendations on the pharmaceutical development and quality aspects of medicinal products intended for children from birth to 18 years of age. The guideline addresses key considerations required to ensure that medicines developed for paediatric populations are appropriate for their intended users, including formulation development, dosage form selection, administration routes, pharmaceutical characteristics and quality requirements.

The development of medicines for children requires specific approaches because paediatric patients differ from adults in terms of physiology, ability to swallow medicines, dose requirements, pharmacokinetics and treatment acceptability. The guideline supports researchers and developers in considering these factors early in the development process to ensure that the investigational medicinal product is suitable for paediatric clinical investigation.

This resource is particularly relevant for rare disease clinical trials because a large proportion of rare diseases have paediatric onset and many investigational therapies are developed specifically for children. Rare disease trials frequently involve small and heterogeneous populations, making efficient pharmaceutical development essential. Appropriate formulations, dosing strategies and administration methods can directly influence recruitment feasibility, participant adherence, safety monitoring and overall trial success.

The guidance is also highly relevant for innovative therapies used in rare diseases, including treatments requiring specialised administration approaches or tailored dosing strategies. Ensuring that the investigational product is adapted to the needs of children can be especially important when clinical evidence must be generated in very small populations and when there are limited opportunities to repeat studies.

Although developed within the European regulatory environment, the scientific principles described in this guideline are broadly applicable internationally. The resource provides valuable support for investigators, sponsors and product developers planning paediatric and rare disease clinical trials where pharmaceutical development considerations are central to successful clinical evaluation.