FAQ About New Therapeutic Uses
- How is this model different from other 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 is the program speeding therapeutic development and saving money?
- What criteria are used for the UH2 to move to the UH3 stage within the program’s time frame of one year for adults or two years for pediatrics?
- What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines?
- What is the purpose of the Industry-Provided Agents tables?
- Have all the agents been shelved by the company?
- What are the agent selection criteria?
- Are these agents approved by the Food and Drug Administration (FDA) for clinical use?
- Where can investigators find out more about the agents that are part of the current program?
- Will researchers be able to use the original agents from the pilot program for projects proposed in response to the current funding opportunity announcements (FOAs)?
- Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project?
- What are the current and future application plans for the program, and when will those funding opportunities occur?
How is this model different from other public-private partnerships for research that support the discovery and development of new medicines?
Some other 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 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. The program also provides open access to a limited amount of confidential information about partially developed therapeutic candidates (referred to as agents) from participating pharmaceutical companies.
Effective treatments for thousands of diseases still are needed. By working collaboratively to address challenges in medicine development in this new, innovative way, NIH has a greater chance of more quickly advancing the science required to enable new treatments for people who need them.
The average length of time from discovery of a therapeutic target to approval of a new drug currently is about 14 years. More than 95 percent of these projects fail, and the cost per successful drug can be $2 billion or more. This failure rate means that many existing therapeutic candidates could be repositioned for a new use and advanced to clinical trials more quickly than starting from scratch. Existing candidates already have undergone significant pre-clinical and Phase I safety testing and are ready for additional testing in humans. With the promising agents available to the entire research community through this program, investigators with great ideas have the opportunity to test hypotheses for new uses. This approach avoids research duplication and reduces the time and money required to determine if these well-developed agents can be used to treat a variety of important medical conditions.
In addition, use of NCATS’ template agreements has shortened the amount of time it takes for each of the partners to negotiate the terms for research collaborations. Specifically, the time to establish collaborations between industry and academia for this program is only about three months, whereas typically it can take nine months to one year. These delays can result in a failure to launch the project when the science moves forward at a faster pace than the legal negotiations.
What criteria are used for the UH2 to move to the UH3 stage within the program’s time frame of one year for adults or two years for pediatrics?
Each of the funded applications will have award terms and conditions outlining goals and criteria for success that will be specific for the project. If the milestones are not met, the applicant will not advance to the next stage. 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?
NIH will support studies through Phase IIa clinical trials as defined in the X02, UH2/UH3 and UH3 funding announcements for the current program. The pharmaceutical company collaborator will have the first option to license the academic research partners’ new intellectual property arising out of the research. In cases where the pharmaceutical company collaborator owns active patents on an agent, they will decide whether to advance the agent 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 an agent and 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.
NCATS facilitates collaborations between academia and pharmaceutical companies by making limited information about proprietary drug candidates known to crowdsource new ideas. The Industry-Provided Agents tables contain links to additional information for each of the agents. This publicly available information is intended to provide sufficient data for a prospective applicant to submit an X02 pre-application.
Some agents are proprietary drug candidates that failed to show efficacy for the original indication or were deprioritized for business reasons and no longer are being pursued for their original therapeutic indication. Others are under active investigation for specific indications.
Agents selected for the program have advanced to clinical studies, and they have a safety profile, which allows further clinical investigation for other potential therapeutic uses. The mechanism of action for each compound is known, and pharmacokinetics is suitable for exploring the mechanism for a new indication.
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 application with the FDA to conduct the proposed clinical trials.
To find out more, visit the tables on the Industry-Provided Agents page.
Will researchers be able to use the original agents from the pilot program for projects proposed in response to the current funding opportunity announcements (FOAs)?
Generally, researchers may not use agents from the pilot program for current FOAs. However, researchers may use them for pediatric indications because pediatric uses were not solicited under the pilot program. The pharmaceutical companies have indicated whether a particular agent from the pilot would be considered for use in children. These agents are included under this FOA.
Additionally, proposed studies for disease populations for which there is no adult equivalent will be considered. Indications in pediatric populations that also have an adult population may be considered if a strong scientific rationale provides justification for the Phase IIa trials in the pediatric population even though an adult patient population exists. Applicants exploring therapies for diseases that occur in children and adults should consider applying in response to one of the companion UH2/UH3 or UH3 FOAs focusing on adult populations.
Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project?
What are the current and future application plans for the program, and when will those funding opportunities occur?
NIH is using information learned from the pilot program to inform decisions about its current and future funding opportunities. NCATS seeks applications for the following funding opportunities:
- 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)
New Therapeutic Uses
In this video, Stephen M. Strittmatter, M.D., Ph.D., Vincent Coates Professor of Neurology at Yale University School of Medicine in New Haven, Conn., discusses a Discovering New Therapeutic Uses for Existing Molecules project.