Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Pharmaceutical development of medicines for paediatric use - requirements, challenges and implications

Dr. Axel Dienemann (Abschlußjahr: 2012)

Summary

The WHO guidance document “Development of paediatric medicines: points to consider in formulation” and the EMA draft Guideline on Pharmaceutical Development of Medicines for Paediatric Use, address the need for improving the availability of paediatric medicines by providing guidance for pharmaceutical development. The WHO guidance is the first internationally endorsed guidance in this respect and is intended to inform regulatory authorities and manufacturers on issues and areas of special interest related to pharmaceutical development of paediatric medicines and has a focus on special needs in developing countries. The EMA Guideline is presently only available as a draft version and supplements the legally binding Note for Guidance on Pharmaceutical Development related to specific paediatric aspects for discussion and justification and inclusion in Module 3.2.P of regulatory submissions in the ICH CTD format.

The present thesis summarizes the history of regulatory guidance related to paediatric pharmaceutical development, presents the EMA draft Guideline, compares it to the WHO guidance and discusses the impact on pharmaceutical development taking into account recent literature.

The guidance documents by WHO and EMA continue the risk to benefit approach into pharmaceutical development of paediatric formulations. Both guidances highlight the importance of a solid strategy covering adequate risk minimisation throughout pharmaceutical development, discuss items for concern and set standards based on current knowledge. Key issues are selection of appropriate dosage forms, safe excipients, palatability and appropriate administration devices. Further progress in science and work by recently established networks (US PFI, EuPFI, WHO PRMN) will spark advancements in paediatric formulation.