About New Therapeutic Uses

Therapeutic development is a costly, complex and time-consuming process. The average length of time from target discovery to approval of a new drug is about 14 years. The failure rate during this process exceeds 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.

NCATS designed its New Therapeutic Uses program to align with one of its primary goals: to foster approaches that improve the translational research pipeline and ultimately accelerate the pace at which discoveries are turned into new preventions, treatments and cures for human diseases.

Drug Development Collaboratory

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The Drug Development Collaboratory enables a partnership with the NCATS Therapeutic Development Branch to conduct IND-enabling studies and provides clinical trial funding from the NCATS Drug Development Partnership program once an IND is cleared.

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 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.

New Therapeutic Uses Funding and Projects Information

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


Christine Colvis, Ph.D. (link sends e-mail)