In various empiric studies the distribution of off-label and off-license drug use in paediatrics was analysed.

Pandolfini and Bonati produced a meta-analysis based on 30 studies from 1985-2004 concerning off-label drug use in children. They arrive at the conclusion that the prescription rates range from 11%-80% and the off-label use is more common in younger children than in older children. Also more off-label prescriptions are made in hospitals than in private practices.

A study by Mühlbauer and colleagues which was published in 2009 investigates the prescription praxis specifically in Germany. The data from 2002 that were drawn upon by the Gmüder health insurance company prove that out of 1,429,981 pharmaceutical packages that were distributed to children and teenagers (0 to 16 Jahre), 3.2% possessed an off-label and 9.4% no determinable approval status at all. At first these numbers seem low but are put in perspective when the focus is not laid on the number of pharmaceutical packages but is shifted to the active substances. Only 66.1% of the medical active substances were given to the treated patients in a way compliant to the approval. Out of those prescriptions that were not compliant to the approval, 15.7% of all active substances were classified as off-label and 18.2% as being without a determinable approval status.
This study, too, states that especially newborns and infants are affected by the deficit - prescriptions compliant with approval were more frequent from toddler age onward [Mühlbauer 2009].

The addressed deficit for newborns and infants is being focused on in more detail in a study from Austria published in 2009. A survey conducted on the neonatological intensive care unit confirms that during the three months analysed only 49% of all prescribed pharmaceuticals carried an official license. 33% of the pharmaceuticals were used off-label while 18% were implemented off-license [Prandstetter 2009].

With regard to the off-label use, the German National Academy of Sciences Leopoldina points out the importance of creating a new infrastructure which facilitates pharmaceutical research. Furthermore, the Leopoldina frames the continuing research on pharmaceuticals for children and adolescents as an important issue for the future of paediatrics: