Using human experience to identify drug repurposing opportunities: theory and practice

Retrospective evidence drawn from real-world experience of a medicine's use outside its labelled indication is one of a number of techniques used in drug repurposing (DRP). Relying as it does on large numbers of real incidences of human experience, rather than individual case reports with limited statistical support, preclinical experiments with poor translatability or in silico associations, which are early-stage hypotheses, it represents the best validated form of DRP. Cancer is the most frequent of such DRP examples (e.g. aspirin in pancreatic cancer, hazard ratio = 0.25). This approach can be combined with pathway analysis to provide first-in-class treatments for complex diseases. Alternatively, it can be combined with prospective preclinical studies to uncover a validated mechanism for a new indication, after which a repurposed molecule is chemically optimized.