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New Therapeutic Uses

We aim to identify new therapeutic uses for existing drugs or compounds to speed the translation of scientific discoveries into potential treatments.

About New Therapeutic Uses

Therapeutic development is a costly, complex and time-consuming process. The average time from target discovery to approval of a new drug is about 14 years. The failure rate during this process is more than 95 percent, and the cost per successful drug can be $1 billion or more. The high therapeutic development failure rate means there are many existing therapeutic candidates that could be repurposed for use in a new disease indication.

Repurposing Off-Patent Drugs Workshop/Drug Repurposing Toolbox

In December 2019, NCATS, the U.S. Food and Drug Administration (FDA) and the Reagan-Udall Foundation held a workshop to discuss research on and regulatory challenges of off-patent drug repurposing and create practical 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, 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 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 only off-patent drugs, as a central location for people who are interested in repurposing research. In response, NCATS created 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.

New Therapeutic Uses Funding and Project Information

Past and currently awarded projects can be found for the following initiatives via NIH Research Portfolio Online Reporting Tools (RePORT). Search by the Notice of Funding Opportunity of interest.