European Medicines Agency Evaluation of Medicines for Human Use

The Note for Guidance ICH S1B Testing for Carcinogenicity of Pharmaceuticals (CPMP/ICH/299/95) offers the option to use short or medium-term in vivo rodent test systems, such as transgenic and knockout animal models, in place of a second 2-year rodent bioassay. Ideally, such studies should supplement the long-term carcinogenicity study and provide additional information that is not readily available from the long-term assay. In the spring of 2002, the ILSI/HESI Alternatives to Carcinogenicity Testing Project comprising 21 selected substances was finalised. From the regulatory point of view, the most crucial factor in considering the use of transgenic/knockout mice is how well the now available experience supports regulatory acceptance. For this reason, an ad hoc-group consisting of four assessors from EU regulatory authorities has reviewed the data generated by the ILSI/HESI project.