FAQ About New Therapeutic Uses
- Which disease areas will be addressed via the pilot phase of the New Therapeutic Uses awards?
- How is NIH administering and funding this pilot phase of New Therapeutic Uses?
- Why is the NIH Common Fund paying for these awards, rather than NCATS’ Cures Acceleration Network (CAN)?
- When will these pilot phase New Therapeutic Uses research projects be completed?
- How many companies are collaborating in the New Therapeutic Uses pilot program?
- How is this model different from previous public-private partnerships for research that support the discovery and development of new medicines?
- Why is this program a good use of taxpayer dollars?
- How will the program speed therapeutic development and save dollars?
- What criteria are used for the UH2 to move to the UH3 stage within this pilot program time frame of three years?
- What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines?
- How will the program be evaluated for success?
- Are these agents approved by the Food and Drug Administration for clinical use?
- Where can investigators find out more about the molecules that were part of the New Therapeutic Uses pilot program?
- Why were these molecules selected for New Therapeutic Uses?
- Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project?
- What are future application plans for the program and when will that funding opportunity occur?
- Will NCATS expand this program if the pilot is deemed successful?
The eight disease areas represented are alcoholism, Alzheimer’s disease, calcific aortic valve stenosis, nicotine dependence, peripheral artery disease, schizophrenia and two rare diseases, Duchenne muscular dystrophy and the lung disease lymphangioleiomyomatosis (LAM).
NCATS is administering the program and the NIH Common Fund is providing the funds. In fiscal year 2013, NIH awarded $12.7 million to fund two- to three-year cooperative research grants.
Why is the NIH Common Fund paying for these awards, rather than NCATS’ Cures Acceleration Network (CAN)?
NCATS had anticipated a greater CAN appropriation than was received. Recognizing the value and importance of the program, the NIH Director provided funds from the Common Fund to support the pilot program.
The pilot phase of this program has enabled funding for milestone-driven projects for researchers to conduct pre-clinical validation, clinical feasibility studies or proof-of-concept clinical trials. The projects will be supported for up to three years.
Eight companies provided a total of 58 molecules for this current pilot program.
How is this model different from previous public-private partnerships for research that support the discovery and development of new medicines?
Some prior public-private partnerships enabled agreements between a single company and a single investigator or biomedical research institution. A key feature of this program is the involvement of multiple pharmaceutical companies and the potential for any U.S. researcher to participate. The program provides model template agreements between the NIH and the pharmaceutical company, and between the company and the biomedical research partner. The template agreements streamline and limit the amount of negotiation that is required before a project can begin. This program could serve as a model for similar collaborations among government, biomedical research organizations and industry.
Effective treatments for thousands of diseases still are needed. By working collaboratively to address challenges in medicine development in this new, innovative way, there is a greater chance that the NIH can more quickly advance the science required to enable new treatments for people who need them.
The average time to develop a drug is 13 to 15 years. These molecules already have undergone significant pre-clinical and Phase I testing and are ready for additional testing in humans. With these promising compounds available to the entire research community, investigators with great ideas have the opportunity to test hypotheses for new uses of the compounds. This will avoid duplication of research and reduce the time and money required to determine if these well-developed compounds can be used to treat a variety of important medical conditions.
In addition, by providing template agreements for the company and biomedical research partner, this program helps reduce the amount of time it takes for each of the partners to negotiate the terms. Traditionally, these negotiations are required on a case-by-case basis for each project, which takes significant effort and time for both parties, and can result in failure to launch the project.
What criteria are used for the UH2 to move to the UH3 stage within this pilot program time frame of three years?
Each of the funded applications has award terms and conditions that outline the specific goals and criteria for success. If the milestones are not met, the applicant will not advance to the next stage. All applicants were required to have project-specific quantifiable criteria that define success. The studies will be guided by a Steering Committee that includes NIH staff and the pharmaceutical partners during development of the investigational drug.
What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines?
The NIH will support studies through Phase IIa trials as defined in the funding announcements: RFA-TR-12-004, RFA-TR-12-005, and PAR-12-203. The pharmaceutical company collaborator will have the first option to license the academic research partners’ new intellectual property (IP) arising out of the research. In cases where the pharmaceutical company collaborator owns active patents on a molecule, this means they will decide whether to advance the molecule through further clinical studies to commercialize the new indication or to enable another company to do so. In cases where there are no longer active patents covering a molecule, if the pharmaceutical company passes on its commercialization option, the biomedical research partner is free to find another commercial collaborator. Commercial options and licenses also are covered in the collaborative research agreements on the Template Agreements page.
The success of the pilot will be evaluated based on achieving any one of the following factors:
- Level of interest from the scientific community
- Results in fundamental new information and scientific insight about disease
- Advances molecular compounds for new therapeutic uses
- Informs the science of translation and drug development from the data and tools produced by the initiative
- Facilitates a larger number of pharmaceutical-academia collaborations at a faster rate than traditional public-private partnerships
- Use of template agreements to speed negotiation time
- Leads to the development of new therapeutics
None of the agents used in these studies are FDA-approved drugs. However, before any agents will be used in clinical studies, each investigator will file an investigator-sponsored Investigational New Drug (IND) application with the FDA in order to conduct the proposed clinical trials.
Where can investigators find out more about the molecules that were part of the New Therapeutic Uses pilot program?
To find out more, visit the New Therapeutic Uses pilot program molecule directory.
The molecules selected for the pilot program have advanced to clinical studies but were deprioritized for business reasons or are no longer being pursued for their original therapeutic indication. They have a safety profile, which allows for further clinical investigation for other potential therapeutic uses.
Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project?
NIH is currently evaluating the pilot program in order to plan for the next funding opportunity, scheduled for fiscal year 2015.
NCATS has received a tremendous amount of positive feedback on the program and is hoping to receive sufficient congressional appropriations to harness the momentum that is currently behind this concept.The Center is exploring the possibility of expanding the pilot program to include other companies as well as additional agents. NCATS and the Office of Strategic Coordination have published a Request for Information (RFI) that seeks input on the administration of the Discovering New Therapeutic Uses for Existing Molecules program. NCATS is interested in collecting feedback from the biomedical research community, pharmaceutical companies and other members of the public about the program and its application processes. View details in the RFI (NOT-RM-13-021).
New Therapeutic Uses
In this video, Kathryn Wagner, M.D., Ph.D., director of the Center for Genetic Muscle Disorders at Kennedy Krieger Institute in Baltimore, discusses a Discovering New Therapeutic Uses for Existing Molecules project.
Frequently Asked Questions
- About the UH2/UH3 and UH3 Limited Competition Applications (Expired 12/18/12)
- About the X02 Pre-Application PAR-12-203 (Expired 8/15/12)
Learn More About New Therapeutic Uses
In May 2012, NIH unveiled its Discovering New Therapeutic Uses for Existing Molecules program. Learn more about this program.