gms | German Medical Science

10. Jahrestagung der GAA Gesellschaft für Arzneimittelforschung und Arzneimittelepidemiologie

16. bis 17.10.2003, Bonn

Off-lable drug use and clinical investigations in children

Off-label-use von Arzneimitteln im Kindesalter

Meeting Abstract

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  • corresponding author Bernd Mühlbauer - Institut für Klinische Pharmakologie, Zentralkrankenhaus Sankt-Jürgen-Strasse, 28205 Bremen, Germany , Tel. +49 (0)421 497 3562

Gesellschaft für Arzneimittelanwendungsforschung u. Arzneimittelepidemiologie (GAA) e.V.. 10. Jahrestagung der Gesellschaft für Arzneimittelforschung und Arzneimittelepidemiologie (GAA) e.V.. Bonn, 16.-17.10.2003. Düsseldorf, Köln: German Medical Science; 2003. Doc03gaa11

The electronic version of this article is the complete one and can be found online at:

Published: October 16, 2003

© 2003 Mühlbauer.
This is an Open Access article distributed under the terms of the Creative Commons Attribution License ( You are free: to Share – to copy, distribute and transmit the work, provided the original author and source are credited.



The dilemma is well known: a significant part of pharmaceutical preparations used in daily practice in children has never been approved for this clinical situation by the drug authorities, mainly due to the lack of systematic investigations. Differences in both pharmacokinetics and -dynamics prohibit to directly transfer pharmacological data obtained in adult subjects to the pediatric patient, e.g. by dose adaptation according to body weight or surface. Apart from the uncomfortable legal situation that many drugs in pediatrics are used off-lable, this implies an uncalculable risk of untowards effects specific for children. On the other hand, drugs of clinical value may be withheld from children because of the fear of untoward effects. Among the reasons for this unsatisfactory situation are legal and ethical reservations to perform clinical trials in patients unable to express their informed consent. Observational investigations might well contribute to the pediatric database. However, their informational impact with respect to clinical efficacy is limited due to the lack of valid control groups or series. In addition, observational investigations employing off-lable drug preparations are compromised by an unclear legal situation. At present, amendments of the drug legislation in the countries of the European Community are being developped, on the basis of the EU Directive 2001/20 EG, which will define a more differentiated legal basis for clinical trials in children. The presentation will adress pharmacological specialties of child age and will give examples of both valuable and dangerous drugs in this patient population, as well as a brief description of the present and future legal basis for clinical research in children.