- How is this model different from other public-private partnerships for research that support the discovery and development of new medicines?
- How is the program speeding therapeutic development and saving money?
- What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines?
- How does NCATS define Phase I and Phase II clinical trials?
- Have all the assets been shelved by the company?
- What are the asset selection criteria?
- Are these assets approved by the Food and Drug Administration (FDA) for clinical use?
- Is it possible to get access to assets from previous years?
- 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 Assets) from participating pharmaceutical companies.
A novel drug can take 10 to 15 years and more than $2 billion to develop, and failure rates occur in about 95 percent of human studies. 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 assets 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 assets 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 happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines?
NIH will support studies through Phase II clinical trials. 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 asset, the company will decide whether to advance the asset 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 asset 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.
Phase I clinical studies are conducted in the target patient population to evaluate safety, determine a safe dose range, and identify side effects prior to conducting a Phase II clinical trial. Phase II clinical trials are conducted in a larger patient population, typically 150 subjects or less for trials in adults. The purpose of the Phase II clinical study is to provide a preliminary signal of efficacy.
Applicants proposing to test a pediatric indication should refer to RFA-TR-17-003 and FDA guidance for pediatric drug products for more information.
Some assets 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.
Assets 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 assets used in these studies are FDA-approved drugs. However, before any assets 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.
Through this program, applicants will not have access to the assets unless an award is made. The publicly posted assets may or may not still be available after the associated funding opportunity expires. Applicants to NIH-Industry Partnerships Initiative funding opportunities should only propose using assets that are posted for the current grant opportunity. Only those applicants that provide evidence that they will have access to the pharmaceutical partner asset of interest will be eligible to apply.
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?
NCATS has released the following funding opportunity announcements:
- RFA-TR-17-001: Pre-application for the NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (X02)
- RFA-TR-17-002: Limited Competition for NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (UG3/UH3)
- RFA-TR-17-003: Limited Competition for NIH-Industry Program: Discovering Pediatric New Therapeutic Uses for Existing Molecules (UG3/UH3)