Therapeutic development is a costly, complex and time-consuming process. The average length of time from target discovery to approval of a new drug is about 14 years. The failure rate during this process exceeds 95 percent, and the cost per successful drug can be $1 billion or more. The high therapeutic development failure rate means there are many existing therapeutic candidates that could be repurposed for use in a new disease indication.
NCATS designed its New Therapeutic Uses program to align with one of its primary goals: to foster approaches that improve the translational research pipeline and ultimately accelerate the pace at which discoveries are turned into new preventions, treatments and cures for human diseases.
NCATS is particularly interested in projects that repurpose existing drugs or biologics (existing therapeutics) that have already begun or completed a Phase I clinical trial.
- Disease Sequelae: The purpose of this urgent funding opportunity announcement is to invite applications to repurpose existing therapeutic agents to treat Coronavirus Disease 2019 (COVID-19) sequelae and associated complications that result from Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) infections. The therapeutic agent must not require additional regulatory studies for the new indication prior to starting a clinical trial.
- Therapeutic/Indication Pairing: The hypothesis for proposed studies must be developed using innovative processes to identify the therapeutic/indication pair. Examples include the following:
- Testing a publicly posted therapeutic candidate for use to treat COVID-19. Examples include clinical candidate therapeutics in documents publicly posted by the World Health Organization (types/classes of candidate therapeutics) and (candidates for clinical evaluation).
- Testing a candidate therapy to treat COVID-19 that was already identified with a publicly available computational approach.
- Testing of existing therapeutic candidates that work on mechanistic targets shared among other viruses that may be relevant to SARS-CoV-2.
- Learn more from NOT-TR-20-012: Notice of Special Interest (NOSI): Repurposing Existing Therapeutics to Address the 2019 Novel Coronavirus Disease (COVID-19).
- Additional NIH opportunities for SARS-CoV-2/COVID-19: Applicants who wish to test anti-virals or immune modulators against SARS-CoV-2/COVID-19 can find additional opportunities on the Coronavirus Disease 2019 (COVID-19): Information for NIH Applicants and Recipients of NIH Funding page. Please contact the scientific contact listed in a specific funding opportunity announcement to learn more.
NCATS has a 3-way partnership with pharmaceutical companies and the biomedical research community to advance therapeutics development. The focus is on matching researchers with a selection of pharmaceutical assets to help the scientists test ideas for new therapeutic uses.
Through this initiative, NCATS is re-engineering the research pipeline, using innovative strategies to identify new uses for existing therapies that have undergone significant research and development by industry, including safety testing in humans. Coordinating the access that scientists nationwide have to existing therapies that have already cleared several key steps in the development process can accelerate the pace of therapeutics development and get more treatments to more patients more quickly.
During the pilot phase of this initiative, NCATS tested the utility of its newly created template agreements, which proved to be effective in facilitating negotiations, enabling the research to begin more quickly. The template agreements reduced the time required to establish collaborations between industry and academia to as few as four months from the more typical nine months to one year.
Participating NIH-Industry partners varies, depending on whether companies have assets that meet the criteria for inclusion in the program for a specific funding opportunity. NCATS partners include the following: AstraZeneca, MedImmune (subsidiary of AstraZeneca), AbbVie, Bristol-Myers Squibb, Eli Lilly and Company, GlaxoSmithKline, Janssen Pharmaceutical Research & Development, L.L.C, Mereo BioPharma, Pfizer, and Sanofi.
Through its NIH-Industry Partnerships initiative, NCATS supports and advances research on a wide range of common and neglected diseases.
NCATS has released a set of funding opportunities to explore a potential new use of an existing investigational therapy, Food and Drug Administration-approved drug, or licensed biologic. Through the Therapeutic/Indication Pairing Strategies initiative, NCATS will support preclinical studies, clinical feasibility studies or proof-of-concept clinical trials to test the utility of an independent crowdsourcing effort or computational algorithm to predict new uses of a drug or biologic.
These studies will serve as an opportunity to demonstrate the usefulness of a drug-indication pairing method. One key goal is to identify therapeutic/indication pairing strategies that prove successful in the clinic and can be adopted by the scientific community to improve the data usability and efficiency of predicting new indications for existing therapeutics.
This new initiative represents an expansion of NCATS' New Therapeutic Uses program and is intended to complement ongoing efforts.