Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

The need for juvenile animal studies - a critical review ***

Dr. Liane Söllner (Abschlußjahr: 2011)

Language: English

With the introduction of specific paediatric legislation in the United States (US) and the European Union (EU) requiring the development of medicines for children the interest in juvenile animal studies (JAS) increased, but also the discussion about the value of such studies and thus the necessity to perform them.

Regulatory guidance with regard to JAS is available from the ICH M3 (R2) guideline. In addition, more specific guidance documents dealing with the conduct of JAS have been issued by the US Food and Drug Administration (FDA) as well as the European Medicines Agency (EMA).

This thesis summarises and discusses the regulatory requirements regarding the need for JAS, their design and timing. Based on the experience with such studies currently available in the literature it tries to answer the question for the benefit of JAS for the human use.
The following conclusions are drawn: The relevant guidance documents as well as several authors support a case-by-case approach regarding the need for and design of JAS. JAS should be considered, especially in cases when organ systems undergoing postnatal development, like the kidney, the skeleton or the CNS, and so on, have been identified as target organs for toxic effects from available human data or previous animal data, as developing organ systems in general are more sensitive to toxicity than mature organs. The relevance of the findings from JAS for the human use will essentially depend on an appropriate study design. In this regard the choice of species as well as the age of the juvenile animal at the start of study are of particular importance. As the available data from JAS show, the requirements of the relevant guidance documents are usually considered when designing such studies. Unfortunately, so far, there is not enough experience to finally answer the question for the usefulness of JAS not least because of the lack of understanding of the animal models used in JAS which makes the interpretation of the results from JAS and extrapolation of the data to the paediatric population difficult. Further data will be necessary to ultimately clarify the need for such studies.

Pages: 44