Assessing the Benefit-Risk for New Drugs

Abstract

The U.S. Food and Drug Administration (FDA) regulates which drugs can be marketed in the U.S. as well as their label requirements. A congressionally authorized advisory committee process allows the FDA to receive input from outside experts on important regulatory decisions (1). Further, the advisory committee process enhances transparency by allowing the public, including health care professionals, to understand the issues being considered. The Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) “reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of endocrine and metabolic disorders, and makes appropriate recommendations to the Commissioner of Food and Drugs” (2). Operationally, the FDA’s Division of Metabolism and Endocrinology Products (DMEP) is responsible for the review of most drugs used for indications relevant to endocrinologists and coordinates the EMDAC’s meetings. In recent years, the EMDAC has been asked to review a number of controversial drugs, particularly those for the treatment of diabetes and obesity. Often the assessment of these drugs requires integration of preclinical and clinical data to formulate a benefit-risk assessment. The assessment of benefit-risk for drugs is increasingly recognized to be a challenging task, and formal tools have been proposed to make this process more effective (3–7). In contrast with the DMEP and other FDA staff, most EMDAC members will have limited experience in these types of integrative, complex evaluations. Further, EMDAC members will spend considerably less time evaluating the available data in comparison with DMEP staff. Thus, it seems important to compare the recommendations made by EMDAC with the subsequent marketing and label decisions to assess concordance between the evaluative approaches used by EMDAC and that of DMEP (DMEP will be used as shorthand for the full FDA’s involvement in regulatory decisions). At most of the EMDAC …