Abstract

With the Paediatric Regulation in action for ten years it is becoming apparent that drug studies in children are far from what was expected. Drug studies are the restricted domain of drug companies, for drug development reasons and pharmacovigilance afterwards. Academics are only advisers to companies and executioners of submitted studies to FDA and EMA. Companies only have to prove that their drug has an effect even statistical significance of efficacy is not mandatory for acquiring market authorisation. This leads to small numbered studies that fail to elucidate specifics of children. Reasons are companies without proper knowledge of academic incentives, academics without regulatory knowledge, patient organisations not sufficiently involved, regulators with insufficient information and power to require proper studies.

Advantages of large collaborations is development of disease related data bases, data bases of genetics of diseases, for whom a certain drug most likely should be selected to treat, larger more adequate numbers are possible to include in studies.

Current industry cohorts for single drugs in children lack possibility to relate to national cancer registries to compare incidence cases, changes to newer drugs makes these data cohorts vulnerable for meaningful assessments. Long term follow up for adverse events in comprehensive academic databases is more appropriate, drug industry should by contract have full access to their drug related data for temporarily submission to regulatory agencies FDA and EMA. Transition into adult data bases for the same disease is of clear advantage. A European signature to these databases is mandatory.

For rare diseases development of European data bases is mandatory for development of treatment strategies, to benefit most of money issues, to distribute scientific burden between industry and academia with an optimal patient, industry, academia relationship, to generate numbers, give more academic ideas a fair chance, to establish criteria for what is a meaningful improvement, to get European academics interested in drug research in children.