Systematic Evaluation of Drug–Disease Relationships to Identify Leads for Novel Drug Uses

Drug repositioning refers to the discovery of alternative uses for drugs—uses that are different from that for which the drugs were originally intended. One challenge in this effort lies in choosing the indication for which a drug of interest could be prospectively tested. We systematically evaluated a drug treatment–based view of diseases in order to address this challenge. Suggestions for novel drug uses were generated using a “guilt by association” approach. When compared with a control group of drug uses, the suggested novel drug uses generated by this approach were significantly enriched with respect to previous and ongoing clinical trials.