Challenges in prescribing drugs for children with cancer

SummaryPaediatric oncology has achieved high cure rates despite the limited availability of drugs that have been specifically studied for use in children with cancer. Efficacy of these drugs has received more attention than their safety, but permanent side-effects in growing children need to be considered. An absence of pharmacokinetic data, dose-defining studies, schedules defined by age, and appropriate formulations can lead to underdosing or overdosing in specific age groups, resulting in a potential lack of benefit, development of resistance, and increased adverse drug reactions. These major clinical concerns have promoted initiatives in Europe since 2003 regarding the need for a Paediatric Regulation, aimed at improving the risk–benefit ratio of such drugs in children and providing the legal framework to overcome the limitations of the past. However, to undertake the appropriate studies of these drugs in this setting, financial support is essential. Europe is now showing its commitment to overcome the present difficulties of drug prescribing for children with cancer by introducing measures that will encourage new public–private partnerships. All those involved, including researchers, paediatric oncologists, learned societies, regulatory agencies, national agencies, and pharmaceutical companies, need to become more familiar with the opportunities opened up by the new regulation, which is aimed at providing an increased cooperation between researchers and drug developers for the benefit of children.