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National Center for Advancing Translational Sciences (NCATS) Discovering New Therapeutic Uses for Existing Molecules

NAMHC Concept Clearance


Linda Brady, Ph.D.
Director, Division of Neuroscience and Basic Behavioral Science (DNBBS)


On December 23, 2011, Congress created a new center at the National Institutes of Health (NIH), the National Center for Advancing Translational Sciences (NCATS, Public Law 112-74, amending the Public Health Service Act, 42 U.S.C. § 287). One aspect of the NCATS mission is to focus on developing innovative new methods and tools to reduce or eliminate barriers to drug and diagnostic development. By developing new methods that can be adopted across the entire medical product development sector, NCATS seeks to enhance others’ ability to bring safe and effective products to patients.


The NCATS New Therapeutic Uses Program will fund research to identify new therapeutic uses of proprietary investigational drugs and biologics (Agents), made available by participating pharmaceutical partners, to improve healthcare and benefit to patients across a broad range of human diseases in areas of unmet medical need. The Program intends to match candidate Agents and associated data from participating pharmaceutical companies with the best ideas from the biomedical science community for new therapeutic uses.


Drug rescue and repurposing have been broadly accepted as valuable approaches to speed the development of new drugs and diagnostics. 1 At this time of unprecedented challenge and opportunity in translational science, NIH has an important role to play in accelerating and advancing therapeutics development. 2 In April 2011, NIH convened an NIH-Industry Roundtable that included a group of senior leaders and experts from the pharmaceutical industry, government, academia, and the non-profit sector to explore opportunities to foster new NIH-industry partnerships that facilitate drug rescue and repurposing. Some of the challenges that were identified include: resource implications (the time and resources for a pharmaceutical company to maintain, update, and organize their compound libraries for drug rescue and repurposing); patent considerations (off-patent agents or agents whose patents are close to expiring may not be attractive to industry because the financial return and market incentives for the product may be limited); and, transactional hurdles related to developing, negotiating, and implementing appropriate legal agreements among the parties, including addressing concerns such as intellectual property rights and liability. The Roundtable participants agreed that more can and should be done to increase engagement and partnerships in rescue and repurposing and to enhance the success of these efforts. In response to one of the recommendations of the meeting, NCATS plans to develop the New Therapeutic Uses Program.

NCATS proposes to initiate the New Therapeutic Uses Program through a limited pilot initiative which, if successful, will be expanded in terms of number of compounds, areas of interest and number of projects, subject to availability of Agents and funding. A key feature of the Program will be to incorporate pre-negotiated template agreements (“Partnership Agreements”) as a means to implement public-private partnerships for drug rescue. Successful investigators will have an opportunity to validate novel human mechanisms of disease using Agents that have been made available by pharmaceutical companies for use in small interventional (Phase IIa) studies. The resulting clinical data package from a successful research project should provide sufficient confidence that the Agent is safe and has efficacy in the disease population. It is anticipated that pre-negotiated Partnership Agreements will define license options to develop further and commercialize Agents for new indications, including requisite Phase III clinical trials, ultimately providing a novel therapeutic intervention for that disease whenever feasible.

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1 Drug rescue refers to research involving discontinued drugs or candidate drugs that are not currently in development, whereas repurposing refers to research on approved drugs for new indications.

2 Collins F.S., Opportunities for research and NIH. Science 327: 36-37, 2010.