Improving risk/benefit estimates in new drug trials

It's all too familiar: researchers announce the discovery of a new drug that eradicates disease in animals. Then, a few years later, the drug bombs in human trials. In the latest issue of the Public Library of Science’s journal PLoS Medicine, ethics experts Jonathan Kimmelman, associate professor at McGill's Biomedical Ethics Unit and Department of Social Studies of Medicine, and Alex John London, associate professor of philosophy at Carnegie Mellon University, argue that this pattern of boom and bust may be related to the way researchers predict outcomes of their work in early stages of drug development.

"We do a fairly good job of predicting the success of interventions that make it to later stages of clinical research," said Dr London, who also directs CMU's Center for Ethics and Policy. "But when it comes to the leap from animal studies to the first trials in humans, there are serious problems," he noted.

Drs Kimmelman and London suggest that the interpretation of preclinical results may suffer from a kind of myopia, in which a narrow focus on the data about the performance of a new drug in pre-clinical studies produces overly optimistic predictions.