In recent years, the costs of new drug development have skyrocketed. At the same time, each year fewer new molecular entities are approved, and recently several approved drugs have been withdrawn from the market for safety reasons. Finding ways to select successful drug candidates earlier in development could save millions or even billions of dollars, reduce the costs of drugs on the market, and increase the number of new drugs with improved safety profiles that are available to patients.

Traditional methods for assessing a drug’s safety prior to approval are limited in their ability to detect rare safety problems, and these methods are unlikely to identify rare safety problems before a drug is marketed. There is, however, an emerging safety science that seeks to change this paradigm by attempting to understand a drug’s safety or toxicity earlier in its development. This emerging science is focused in two areas: (1) the use of basic science to understand the mechanisms underlying toxicity and to predict when a particular compound will have safety issues; and (2) the use of new analytical tools for mining large data sets to identify signals that indicate safety problems and even to derive insights regarding a drug’s mechanism of toxicity.

In April 2007, the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation convened a workshop entitled “Emerging Safety Science” to examine the use of emerging safety science technologies in drug development and to consider their application in the drug review process. This publication summarizes the presentations and discussions during that workshop.