The safety, efficacy and regulatory triangle in drug development: Impact for animal models and the use of animals

Knowledge Source Identification
Knowledge source name The safety, efficacy and regulatory triangle in drug development: Impact for animal models and the use of animals
Owner/Developer European Journal of Pharmacology
Country Netherlands
Languages English
URL http://www.sciencedirect.com/science/article/pii/S0014299915002551
Description In this manuscript we review the connections in the triangle between nonclinical efficacy/safety studies and regulatory aspects, with focus on in vivo testing of drugs. These connections differ for different drugs (chemistry-based low molecular weight compounds, recombinant proteins, cell therapy or gene therapy products). Regarding animal models and their translational value we focus on regulatory aspects and indications where scientific outcomes warrant changes, reduction or replacement, like for, e.g., biosimilar evaluation and safety testing of monoclonal antibodies. On the other hand, we present applications where translational value has been clearly demonstrated, e.g., immunosuppressives in transplantation. Especially for drugs of more recent date like recombinant proteins, cell therapy products and gene therapy products, a regulatory approach that allows the possibility to conduct combined efficacy/safety testing in validated animal models should strengthen scientific outcomes and improve translational value, while reducing the numbers of animals necessary.
Knowledge Source Category
Category Publication
Sub categories Review / Research article
Knowledge Dissemination and Sharing
Dissemination channel Website
Targeted audience (specified/objective analysis) Researchers, Scientists, Industry
Users access Restricted access, Fee-based access
Knowledge Characterization
3Rs relevance Replacement, Reduction, Refinement
Purpose Documentation and information, Regulatory testing, Toxicological and safety evaluation, Efficacy testing
Technology/Tools Animal testing (in vivo)