FDA Perspective on the Use of Transgenic Animals in the Manufacture of Therapeutic Products Intended for Human Use: Safety Concerns

Abstract

Many novel therapeutic products have been successfully introduced into clinical trials over the past five years. Transgenic animal technology has often complimented both the discovery and testing of many of these new products. Transgenic technology is now being exploited to develop genetically modified animals as “factories” (“pharm” animals) to produce therapeutic products for human use. FDA’s challenge is to ensure public safety while at the same time not inhibit the development of a new field. Throughout the history of biologies the development and implementation of regulations has been a dynamic process, demanded by scientific and technological developments. While general regulatory principles apply, optimal safety evaluation programs for new areas of research must also invoke a consideration of the fundamental biology of the product, the intended population and the proposed clinical application. The regulatoru concerns common to the characterization of all biologies include: demonstration of safety, efficacy, identity, purity, and potency; quality control of the product and intermediates; and reproducibility or consistency of lots.