Unlicensed and off-label drug use in children

Many commercially available drugs are only licensed for use in adults and for many drugs the available formulations are unsuitable for pediatric use.' This reality leads to use of drugs which are unlicensed or off -label for use in children. There are several reasons for this highly unsatisfactory situation. Firstly, children represent only a small market in an absolute sense but also relatively as most children are healthy. Hence, from a commercial perspective it may not be profitable to invest in pediatric drug research. Secondly, fear is growing for unforeseen adverse effects and long-term toxicity which may be difficult to study. Therefore, the current requirements for licensing of a drug may have become a barrier to make proper drugs available to a vulnerable group of patients. Thirdly, there are ethical problems to conduct drug studies in children. In Europe the problems in pediatric drug use and research receive relatively little interest and there is a substantial Jack of funding of pediatric drug research. N. a result, pediatric drug trials are relatively scarce and often include only a limited number of patients. Despite the well-known thalidomide disaster, which happened already forty years ago and caused an epidemic of congenital malformations, very little has changed regarding the labeling of drugs in infants and children. Although the disaster has stimulated legislation of drug licensing, children ironically remain 'therapeutic orphans'. This is difficult to accept because many diseases in adults can also occur in children while pharmacokinetics and pharmacodynamics often differ. Prescription of drugs should be proven to be equally safe in children as in adnlts, and efficacy of the product should be thoroughly assessed for all probable users. Exposure of infants and children to drugs which are not proven to be safe and efficacious during growth and development, cannot always be prevented. Hence, the health implications of this 'therapeutic orphanhood' may be larger than is sometimes assumed. The scope of this thesis was to assess the extent of the problem of unlicensed and offlabel drug use in children both in clinical care as well as in general practice. Although it is just a first step on a lengthy road, we hope that results of our studies may revive awareness of the problem and stimulate adequate regulatory actions.

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