Paediatric Medicines Development and Authorisation at BfArM

EU Paediatric Regulation Framework

The EU Paediatric Regulation (Regulation (EC) No 1901/2006, as amended) aims to improve the health of children by increasing high-quality research and availability of medicines specifically tested and authorised for paediatric use. In Germany, BfArM implements this regulation for nationally authorised, decentralised, and mutual recognition procedures, while contributing to centralised procedures via EMA. Medicines for children - BfArM

Paediatric Investigation Plan (PIP)

• A PIP is mandatory for new medicinal products (unless waived or deferred) and must outline planned studies to generate paediatric data.
• Applicants submit PIPs to the EMA Paediatric Committee (PDCO); BfArM provides input for procedures where it acts as RMS or national authority.
• PIP compliance is checked at marketing authorisation; non-compliance prevents grant of authorisation (unless justified).
• Waivers apply when paediatric development is not feasible (e.g., disease does not occur in children); deferrals allow adult authorisation first.

Paediatric Use Marketing Authorisation (PUMA)

• A PUMA grants 10 years of data/market exclusivity for off-patent medicines developed specifically for children.
• Requires completion of an agreed PIP and paediatric data submission.
• BfArM processes PUMA applications for national/DCP/MRP routes and supports EMA for centralised PUMAs.
• Incentives encourage development of paediatric formulations (e.g., age-appropriate strengths, liquid forms).

Paediatric Data Requirements

• Clinical trials in children must follow ethical standards and GCP with paediatric-specific considerations.
• Extrapolation from adult data is encouraged when scientifically justified, reducing unnecessary paediatric trials.
• BfArM reviews paediatric data in national assessments and contributes to EU harmonised evaluations.

Practical Support and Guidance

• BfArM offers scientific advice on paediatric development, PIP design, and waiver/deferral requests.
• Applicants can request national paediatric scientific advice before or parallel to EMA PDCO procedures.
• BfArM maintains lists of authorised paediatric indications and formulations available in Germany.

These measures ensure that children benefit from medicines that are appropriately studied, formulated, and labelled, while balancing ethical and scientific needs in paediatric drug development under BfArM oversight. Medicines for children - BfArM