WHO presents strategy for transforming paediatric drug system
Despite advances in medical technology and innovation, children — particularly in low- and middle-income countries — still face barriers in accessing safe, effective and age-appropriate medicines.
Major challenges:
- Development and launch of pediatric medicines takes up to 25 years
- Only 30% of medicines on the WHO Essential Medicines List are available in child-friendly forms
- Medicines are often given to children in crushed or broken "adult" forms, which can lead to dosing errors
- Only 10% of registered clinical trials from 2000 to 2023 included children
- 38% - 50% of medicines are used in children off-label, without evidence of efficacy and safety
To change this, WHO is developing the Global Accelerator for Paediatric Formulations (GAP-f) is a platform that brings together regulators, researchers, manufacturers, clinicians, doctors and patient organizations. The goal is to reduce the time it takes to develop, register and introduce pediatric drugs and ensure their availability in all countries.
The new “30 by 2030” strategy plans to:
- Prioritize 10 diseases with a high childhood burden
- Accelerate the promotion of 10 key drugs
- Create a sustainable access system in 10 countries
In conditions of limited data, especially for small groups of patients, GAP-f emphasizes the importance of real-world experience (RWE) data. They are needed to:
- assess the safety and efficacy of new drugs
- support regulatory decisions
- update clinical guidelines
- monitor the effects of early access and adaptation of therapy
WHO recommendations for national regulators:
- Use mutual recognition and regulatory cooperation mechanisms (e.g. Paediatric Regulatory Network)
- Apply extrapolation of data from adults to children (in accordance with ICH E11A)
- Develop infrastructure for research in pediatrics
- Integrate RWE into regulatory processes and post-registration surveillance
These steps are particularly important to expand access to therapy in children with rare diseases, where traditional research approaches are often not possible.
