Usage of unpublished paediatric data

Area of Science:

• Pharmaceutical Science
• Pediatric Medicine
• Regulatory Affairs

Background:

• The European Paediatric Regulation (EC No 1901/2006) aims to improve pediatric medicines through increased appropriate drug options, enhanced information, and ethical research.
• Article 45 mandates pharmaceutical companies to submit existing pediatric study data for regulatory review and product information updates.

Purpose of the Study:

• To assess the impact and outcomes of the European Paediatric Regulation on pediatric medicine development and information dissemination.
• To evaluate the review process of submitted pediatric study reports and their contribution to updating product information.

Main Methods:

• Identification and review of approximately 19,000 study reports for a thousand active substances.
• Assessment of data by member states' competent authorities in collaboration with the European Medicines Agency (EMA).
• Analysis of outcomes, including new pediatric indications and updates to product information.

Main Results:

• Over 7 years, 262 active substances (including 62 centrally approved and nearly 200 nationally approved medicines) have been assessed.
• The review has resulted in 16 new pediatric indications, addressing unmet needs, particularly in younger age groups.
• Public accessibility of reviewed data through an EMA database and the European Clinical Trials Register.

Conclusions:

• The European Paediatric Regulation is effectively increasing pediatric medicine information and addressing unmet needs.
• Publicly accessible data enhances awareness for researchers and healthcare professionals, preventing redundant pediatric trials.
• The regulation successfully stimulates high-quality ethical research with children and improves drug utilization in pediatric populations.