Clinical Studies in Infants (Pediatric Pharmacology)

Treatment of children with effective and safe medicines is crucial to improve their outcome. Despite this relevance, it is still common practice in children to administer medicines outside their market authorization. Even if authorized, pediatric medicines may not be age-appropriate for a broad range of therapeutic areas. This has been recognized as very unsatisfactory by all stakeholders involved and makes clinical pharmacological studies in children an obvious need. However, clinical trials of medicines in children come with their specific burdens. These burdens can be qualified as either related to the specific aspects of pediatric pharmacokinetics (PK) and pharmacodynamics (PD) or relate to the logistics of clinical trials of medicines in children. This is followed by a stakeholder’s analysis, discussing specific aspects related to parents and their children (International Children’s Advisory Network, iCAN), recruitment challenges, and research capacity building. We hereby tried to focus on recent evolutions, including initiatives to further develop this research capacity (Institute for Advanced Clinical Trials, iACT for children; Innovative Medicines Initiative, IMI2). Perhaps progress is slower than anticipated, but pediatric medicines research is evolving, and we should keep this momentum. A further structured collaboration between the different stakeholders involved (the society, parents and children, sponsors, regulatory authorities) at the international level is crucial to use the available, but limited, resources as effective as possible to further improve pharmaceutical care in children.</p