FAQ About Therapeutics Discovery
- How many companies are collaborating in the Therapeutics Discovery 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 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?
- Where can investigators find out more about the molecules that are part of the Therapeutics Discovery pilot program?
- Why were these molecules selected for Therapeutics Discovery?
There are eight companies that have 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 have developed 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 will 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 hundreds of diseases still are needed. By working with industry, there is a greater chance that the NIH can advance the science required to enable new treatments for people who need them. NCATS is working collaboratively to address challenges in medicine development in a new, innovative way, for the benefit of patients and their families. This is a good use of the public's investment in biomedical research.
The average time to develop a drug is 13 to 15 years. These molecules have already undergone significant pre-clinical and Phase I testing and are ready for additional testing in humans. With these promising molecules made available through the Therapeutics Discovery program to the entire research community, investigators can test biological hypotheses about new uses of the compounds in unexplored disease areas. This will avoid duplication of research and reduce the time and money required to determine if these brand new molecules can be used to treat a variety of important medical conditions.
In addition, by providing template agreements between the company and biomedical research partner, this program will significantly 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 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 X02 pre-application. The pharmaceutical company collaborator will have the first option to license the biomedical 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.
The success of the pilot will be evaluated based on achieving any one of the following factors:
- Results in fundamental new information and scientific insight about disease.
- Advances a molecule 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 partners at a faster rate than traditional public-private partnerships.
Where can investigators find out more about the molecules that are part of the Therapeutics Discovery pilot program?
The Therapeutics Discovery pilot program molecule directory is available on the National Center for Advancing Translational Sciences’ (NCATS) website.
The molecules selected for the pilot program have advanced to clinical studies but were not pursued for their original therapeutic indication. They have a safety profile, which means that further clinical investigation could be possible for other potential therapeutic uses.
Frequently Asked Questions
- About the UH2/UH3 and UH3 Limited Competition Applications (Expired December 18, 2012)
- About the X02 Pre-Application PAR-12-203 (Expired August 15, 2012)
Learn More About Therapeutics Discovery
In May 2012, NIH unveiled its Discovering New Therapeutic Uses for Existing Molecules program. Learn more about this program.