Developing a Regulatory Strategy for Monoclonal Antibodies

Medical research is rapidly moving forward in the development of new diagnostics and therapeutics for life-threatening diseases. Among the most novel approaches to diseases such as cancer, septic shock, and multiple sclerosis, are the cell-targeting agents, monoclonal antibodies (MABs). Although many biotechnology and pharmaceutical firms are investing significant financial and human resources toward the development of these unique molecules, few monoclonal antibodies have achieved marketing approval in the United States or worldwide. The reasons for the limited success in obtaining approvals include lack of familiarity, both by sponsors and regulatory agencies, with monoclonal antibodies as therapeutics; the unique aspects of developing monoclonal antibodies; and safety and clinical trial design issues. To overcome these limitations to successful achievement of rapid approvals, it is imperative that pharmaceutical sponsors formulate regulatory strategies early in the development process that will address these issues influencing product approvability. The goal of such a strategy would be to identify key issues that must be addressed to demonstrate safety and efficacy and provide recommendations to facilitate an effective development plan. Drawing upon the regulatory histories of several monoclonal antibodies which have been reviewed by the Food and Drug Administration (FDA), this paper identifies regulatory issues that are important in the development and marketing approval of monoclonal antibodies in the United States, and makes recommendations for developing a regulatory strategy.