Non-clinical efficacy-related studies for human medicines: An overview and retrospective analysis of data for a group of approved medicines

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The lack of efficacy is a major cause of medicine's development failure at the clinical phase, which may lead to question, among other aspects, the translation of the non-clinical data into humans. The objectives of the work here presented were (i) to get an overview (based on public assessment reports) of the nature of the non-clinical efficacy-related studies presented to the regulatory authorities at the marketing authorization application's stage for a group of approved anticancer human medicines (15 in total) and (ii) to conduct a retrospective analysis of such studies in terms of any identified insufficiencies and consistency with the current regulatory non-clinical guidelines. Each medicine has been tested in a number of in vitro assays and animal studies, which, all together, are judged to be capable of providing information on the activity of the active substance and demonstrating an anti-tumour effect, as well as to be generally consistent with the available, although limited detailed, guidance. In spite of this, some aspects were identified which could have a potential impact on the translation on non-clinical data into humans, namely, apparent insufficiencies in terms of animal model/human bridging data/knowledge and in vivo data on pharmacokinetics/pharmacodynamics relationships.HighlightsOverview of nature of non-clinical efficacy-related studies for group of medicines.Anticancer monoclonal antibodies and FDA and/or EMA's public assessment reports.General consistency with little detailed regulatory guidelines.Aspects with potential impact on data translation into humans identified.Apparent insufficient animal-human bridging data and PK/PD investigation.

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