The doctor treating a minor is obliged to inform the parents or their representatives if he is using a drug "off-label". Even if he believes that he has adequate information about possible risks for the individual underage patient, it is only possible in exceptional cases for him to obtain the necessary "informed consent" for studies on efficacy, safety and dose finding.
These days, both the public and doctors expect that manufacturers will only include recommendations in their summaries of product characteristics for which there are clear supporting data. It would be desirable for the summary of product characteristics to refer to published scientific data or to provide the data if requested. As no placebo-controlled studies on pregnant women or children have been performed for most available drugs, manufacturers are obliged to state that they cannot be used in this context.
The doctor retains responsibility for the treatment he provides, whatever his legitimation or knowledge. This may have serious consequences, both after treatment or if no treatment is provided. This problem cannot be solved by the manufacturer either. Even the valuable efforts of Dr. Seyberth (1, 2) and the German Society for Pediatrics and Adolescent Medicine (3) remain wishful thinking, as long as there are no evidence-based data available. Any legal regulation would be of little or no use. We must therefore search for new approaches for "drug studies" and drug approval for the unborn, neonates, babies, infants and schoolchildren.
Footnotes
Conflict of interest statement
The author declares that no conflict of interest exists according to the guidelines of the International Committee of Medical Journal Editors.
References
- 1.Seyberth HW. Arzneimittel in der Pädiatrie: ein Paradigmenwechsel bahnt sich an. Dtsch Arztebl. 2008;105(27):A 1497–A 1499. [Google Scholar]
- 2.Seyberth HW. Problems of Prescription Drug Use in Children. Dtsch Arztebl Int. 2009;106(3):23–24. doi: 10.3238/arztebl.2009.0023. [DOI] [PMC free article] [PubMed] [Google Scholar]
- 3.Richter-Kuhlmann E. Arzneimittel für Kinder - Häufig am Rande der Legalität. Dtsch Arztebl. 2008;105(6):227–228. [Google Scholar]
- 4.Mühlbauer B, Janhsen K, Pichler J, Schoettler P. Off-Label Use of Prescription Drugs in Childhood and Adolescence—an Analysis of Prescription Patterns in Germany. Dtsch Arztebl Int. 2009;106(3):25–31. doi: 10.3238/arztebl.2009.0025. [DOI] [PMC free article] [PubMed] [Google Scholar]