Recommendations and the way forward

Abstract

Considerable investment has been made by both pharmaceutical and biotechnology companies in pharmaceutical products of biotechnology. Since the introduction of the first ‘biotech’ product in 1982, there has been an increasing percentage of biotechnologi-cally-derived new active substances (BNASs) marketed, as a proportion of all new molecular entities. Because of the nature of these biotech products, it is often not possible to follow the conventional safety testing programme used for chemicals, and they are evaluated using a case-by-case approach. Although the general principles for designing internationally acceptable preclinical safety evaluation programmes have been agreed under the auspices of ICH (the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use), the guideline ‘Preclinical safety evaluation of biotechnology-derived pharmaceuticals’ emphasises the importance of maintaining a flexible approach (Appendix I). However, because of the relatively small number of biotech products which have been marketed, individual companies and regulatory authorities may have little experience with these compounds, and utilising a case-by-case approach has, in the past, led to some inconsistency in the information on non-clinical safety testing requested by regulatory authorities in Europe, the USA and Japan. One of the main objectives of this CMR International workshop was therefore to recommend which studies should be considered and which may be unnecessary for safety evaluation for specific product classes.