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Discovering New Therapeutic Uses for Existing Molecules

Researcher using a pipette and a microscope

Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) is a collaborative program designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance therapeutics development. This innovative program matches researchers with a selection of pharmaceutical industry agents to test ideas for new therapeutic uses, with the ultimate goal of identifying promising new treatments for patients.

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 $2 billion or more. The high therapeutic development failure rate means there are many existing, partially developed therapeutic candidates that could be repurposed for use in a new disease indication. Bringing together the best agents from pharmaceutical companies with the best new ideas from academic researchers could produce new treatments much more quickly than starting from scratch.

Launched in May 2012, NCATS' New Therapeutic Uses program helps re-engineer the research pipeline using an innovative strategy to identify new uses for agents that have undergone significant research and development by industry, including safety testing in humans. By using agents that already have cleared several key steps in the development process, scientists nationwide have a strong starting point to contribute their unique expertise and accelerate the pace of therapeutics development.

Funding Information

In May 2014, NCATS issued the following four funding announcements:

  • PAR-14-213: Pre-application for the NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (X02)
  • PAR-14-212: Limited Competition for NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (UH2/UH3)
  • PAR-14-211: Limited Competition for NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (UH3)
  • PAR-14-210: Limited Competition for NIH-Industry Program: Discovering Pediatric New Therapeutic Uses for Existing Molecules (UH2/UH3)

In addition to NCATS, multiple NIH Institutes and Centers and the Food and Drug Administration's Office of Orphan Products Development are contributing funding for these announcements.

This new round of funding opportunities builds on the pilot phase of the program. In June 2013 in response to the initial set of funding announcements, NIH awarded $12.7 million to nine academic research groups for projects to explore new treatments for patients in eight disease areas, including two rare diseases.

Library of Agents

The New Therapeutic Uses Industry-Provided Agents tables feature agents that have undergone significant pre-clinical, safety and dosing testing and are ready for additional testing in humans. In 2014, NCATS collaborated with AstraZeneca, Janssen Research & Development, L.L.C., Pfizer Inc. and Sanofi to make 26 agents available. For the first time, the pharmaceutical companies have identified agents for the program that are suitable for exploring pediatric indications.

Template Agreements

Diagram of identifying new therapies for patients through (1) pharma, which create drugs, provide agents, sign agreements with researchers and form alliances with NIH/NCATS; (2) researchers, who provide new therapeutic use ideas, access patient populations, conduct clinical trials, sign agreements with pharma and receive funding from NIH/NCATS; and (3) NIH/NCATS, which posts agent information, develops agreement templates, crowdsources ideas, provides funding to researchers and forms alliances with pharma.

Click for a larger view.

To facilitate and speed academia-industry collaborations, NIH developed template agreements designed to streamline the legal and administrative process for research collaboration by multiple organizations. These agreements help facilitate complex negotiations among all parties involved in the program, enabling the research to begin faster. In fact, one third of the project investigators in the New Therapeutic Uses pilot phase were testing compounds in humans for new uses within three months of receipt of funding, including potential treatments for schizophrenia and Alzheimer's disease.

Related Links


Christine Colvis, 301-451-3903

New Funding Opportunities

NCATS announced new funding opportunities for its New Therapeutic Uses program. Apply now.

Technical Assistance Webinar

NCATS held a technical assistance webinar for applicants to the New Therapeutic Uses program on Thursday, May 29, 2014. Download the slides.

For More Information

Funding Information

Industry-Provided Agents

Template Agreements

Fact Sheet

FAQ About New Therapeutic Uses

2013 Project Awards

Program Videos

Program Audio Clips

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