Introduction. Despite the relatively high prevalence of rare cancers as a collective group, the low incidence for individual rare and ultra-rare cancers continues to act as an impediment to the development of new therapeutic molecules. One response to this situation is the increased use of the precision medicine paradigm for rare cancers.

Areas covered. Innovative treatment approaches can be explored based on the genotype of individual tumours, thus by-passing the issue of the high genetic heterogeneity of many rare tumour types. Identification of specific molecular targets and pathways allows the selection of candidate drugs aimed at those targets. A number of illustrative examples, in a range of rare cancers, are outlined in this paper. Data from the Repurposing Drugs in Oncology (ReDO) project includes more than 240 candidate drugs with a broad range of identified targets and is supportive of this approach.

Expert opinion. The repurposing of non-cancer drugs extends the reach of precision medicine by widening the search space of drug targets. The repurposing of generic non-cancer medications is therefore a source of innovative therapies in rare cancers and should be considered in all precision medicine trials and initiatives.

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