Clinical trials for children with cancer in Europe - still a long way from harmonisation: a report from SIOP Europe.

Clinical trials for children with cancer have been particularly hard hit by the introduction of the EU Clinical Trials Directive in 2004. Largely investigator-led and lacking in commercial sponsorship, they have struggled to find the resources necessary to comply with the complex bureaucracy. These rare diseases require multinational participation to permit appropriately powered clinical trials to be undertaken. Differences in interpretation of the Directive by national regulatory authorities have had a disproportionate effect on trials in children, highlighted by differences in what is deemed an 'investigational medicinal product' when paediatric use of an old drug is outside its licensed indication. Insurance costs have increased a 100-fold with no increase in actual risk between consecutive trials from the same study group. Issues raised at the recent conference held to reappraise the operation of the Directive are summarised to emphasise the particular issues for trials in children with cancer.