POLYMER-MODIFIED PROTEINS - PRECLINICAL DEVELOPMENT AND REGULATORY STRATEGIES

A valuable way for drug developers to anticipate the preclinical requirements for novel drugs, such as polymer-modified proteins, is to take the perspective of the regulatory scientist. This manuscript will describe the preclinical review process for investigational new drugs at the U.S. Food and Drug Administration (FDA) using a hypothetical polymer-modified protein as an example. The validity of this approach is that understanding the audience, namely the regulatory scientist, helps target the proposal, namely the Investigational New Drug Application (IND). By understanding how regulatory scientists review preclinical information for new drugs, developers can anticipate the critical regulatory issues about safety and efficacy. The developer can then narrow the choices for preclinical studies that will successfully support the risk/benefit assessment of the IND. This manuscript suggests that the preclinical development of polymer-modified proteins should be patterned after classical synthetic drugs and the focus of the FDA regulatory review will be primarily on safety, not efficacy.