In December 2019, NCATS, the U.S. Food and Drug Administration (FDA), and the Reagan-Udall Foundation held a workshop to discuss research and regulatory challenges of off-patent drug repurposing and formulate actionable solutions to those challenges. Although finding a new therapeutic use for an existing drug seems like a simple way to get more treatments to more patients more quickly, repurposing off-patent drugs generally has little potential for return on the investment made, particularly if generic forms of the drug are already on the market. As a result, despite the potential for repurposed drugs to be brought to market relatively quickly, companies have little financial incentive to invest their resources in finding new therapeutic uses for existing drugs.
One recommendation that emerged from the workshop was to provide a toolbox of information for drug repurposing in general, not just focused on off-patent drugs, as a central location for people who are interested in repurposing research. Workshop participants expressed concern that many researchers may be interested in repurposing research but not know where to start, and a central hub or toolbox could serve as critically needed starting point.
In response to that recommendation, NCATS established the Drug Repurposing Toolbox to provide easy access to guidance, relevant publications, information about repurposing conferences and other resources, including funding and collaboration opportunity announcements.
For questions about the Drug Repurposing Toolbox, please email NewTherapeuticUses@mail.nih.gov.