Development of Medicines for Paediatric and Rare Diseases

The value of licensed and appropriately labeled medicines for children and adults is increasingly a focus of public awareness. This is, for a large part, the result of US and EU legislation on paediatric and orphan diseases. However, paediatric clinical practice has changed less than expected, and the initial enthusiasm has been replaced by a more realistic assessment. Breakthrough therapeutic innovations have been introduced in the last decades, e.g. for cystic fibrosis, metabolic diseases or hormonal defects. However, none of these were triggered by paediatric legislation. The aim of this conference and its related Special Issue is to provide an update on both state-of-the-art methodology and operational challenges in research & development of paediatric and rare diseases. It aims at re-evaluating what is needed for more progress in the development of treatments for both paediatric and rare diseases. We welcome original research, review, opinion papers, editorials, or short communications on the following topics: paediatric legislation, commercial drug development, development of orphan and paediatric drugs, paediatric clinical research, developmental pharmacology, neonatal pharmacology, paediatric pharmacology, neonatal medicine, neonatal infectious diseases, pharmacokinetics, pharmacodynamics, and neonatal clinical trials.

Dr. Klaus Rose
Guest Editor

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