Children need new medicines

Children need therapies that are tailored to their needs

Children's growth and development lead to significant pharmacodynamic and pharmacokinetic differences compared to adults, affecting their response to medicines and necessitating different formulations and dosages depending on their body size. However, the lack of research and development in pediatric medicines results in limited treatment options, leading to a reliance on unlicensed or off-label use, potentially exposing children to inappropriate formulations and increased risks of adverse reactions or inadequate treatment.

Numerous studies have revealed that more than half of the medications used to treat children are either off-label (prescribed based on something other than the official use of the medication, such as the route of administration) or unlicensed (i.e., produced by hospitals' own pharmacies).

Children in Europe lack access to sufficiently studied and safe medicines.

The pediatric labeling of medications is being delayed by a lack of data, which leaves caregivers in the dark about the safety and effectiveness of the medications in children.

It has been calculated that it takes more than seven years for adult-approved drugs to be authorized for use in children.

As a result, in clinical practice, typical pediatric pharmacotherapy mostly uses adult-purpose medications for which there is no information available regarding side effects or long-term effects on children. To guarantee that children will have access to new medications for the prevention and treatment of various diseases, medications must be studied in child populations in addition to adult populations.

This development of pediatric medicines has been stimulated in the recent decade, including that in Europe, the Pediatric Regulation was drafted in 2007. This regulatory framework aims to foster the development of more tailored, evidence-based, and safer pharmaceutical products for pediatric use, thereby addressing longstanding therapeutic gaps.

Nevertheless, more can be done to increase the number of medicines available for children. It is clear that for pediatrics, and in particular for rare diseases, collaboration if key to address pediatric unmet needs.