Guiding future paediatric drug studies based on existing pharmacokinetic and efficacy data. Cardiovascular drugs as a proof of concept.

We demonstrated that for the majority of off-label cardiovascular drugs, only low quality efficacy studies were available. However, high quality PK studies were frequently available. Combining these PK data with extrapolation of efficacy data from adults may aid to close the current information gap and prioritize the drugs for which clinical studies and safety data are urgently needed.