Lack of compliance of European Public Assessment Reports to guidelines for paediatric drug development before the introduction of paediatric investigation plans

BackgroundAccording to the International Conference on Harmonisation (ICH) and Food and Drug Administration (FDA) guidelines for paediatric clinical trials, bridging procedures can be used if disease progression, exposure–response relationships, and clinical endpoints are similar in adults and children. In these circumstances, confirmatory efficacy trials are not necessary; the evaluation of pharmacokinetics and safety ought to be sufficient for drug approval.PurposeThe aim of this study was to assess whether the clinical trials and strategy for market approval authorisation (MAAs) in paediatric indications reflect the guidelines for bridging of adult data.MethodsA total of 95 European Public Assessment Reports (EPARs) published between 1995 and 2007 were reviewed. From every report, data extraction was performed according to the phase of development, scope of analysis, number of dose levels, dosage form, and demographics of the subjects enrolled in the trial. Data analysis consisted of an initial groupin...