Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance

Area of Science:

• Pediatric Pharmacology
• Translational Research
• Bioethics

Background:

• A persistent global shortage of pediatric medicines, including advanced therapies, affects children's access to treatment.
• Existing legal frameworks and incentives have not fully resolved challenges in studying and tailoring medications for pediatric populations.
• There is a critical need to advance pediatric research using a translational approach to address unmet medical needs in children.

Purpose of the Study:

• To define translational research within the context of establishing a European Paediatric Research Infrastructure (RI).
• To identify and analyze ethical, legal, and regulatory issues pertinent to children's rights in pediatric medicine development.
• To advocate for a robust governance model for a future European RI focused on pediatric research.

Main Methods:

• Conceptual clarification of translational research in the pediatric context.
• Identification and analysis of ethical, legal, and regulatory challenges.
• Review of existing frameworks and incentives for pediatric drug development.

Main Results:

• Translational research is essential for addressing gaps in pediatric medicine.
• Significant ethical, legal, and regulatory hurdles impede pediatric drug development and access to therapies.
• A dedicated European Paediatric Research Infrastructure is proposed to overcome these challenges.

Conclusions:

• Establishing a European Paediatric Research Infrastructure is crucial for advancing pediatric medicine.
• The proposed RI requires a governance model with independent ethical oversight.
• A multidisciplinary service addressing ethical, legal, and societal issues is vital for children's rights in research.