ABSTRACT
The assessment of risk versus benet for any candidate biotechnology product is performed by experimentation in the laboratory, in animal models and in man. Specically, this involves understanding the nature of the biological construct or molecule, its purity and potency following manufacture, and the safety and efcacy prole. Nonclinical studies, performed in vitro and in animals, are primary means of measuring potential product risk and much of this testing precedes clinical trials. Results of nonclinical studies serve to better ensure product benet will indeed outweigh risk once it reaches clinical studies and the marketplace. Nonclinical study activities precede clinical research for good reason. It is the user, oftentimes the human subject enrolled in a clinical trial, who bears the burden of risks associated with product use. Thus, the sponsor of a novel biopharmaceutical provides clear experimental evidence that risks are tolerable and the product itself is unlikely to result in disease or death to the human subjects or, upon marketing approval, the public.
