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Discovering New Therapeutics Uses for Existing Molecules

RFA-TR-16-015 Frequently Asked Questions

NCATS is seeking applications for rigorous, pre-clinical research projects that are based on repurposing existing drugs or biologics (therapeutics).

Pre-clinical studies funded through this initiative will demonstrate the utility of an independent crowdsourcing effort or of a computational algorithm to predict new therapeutic uses of an existing drug or biologic. The goal of an individual project must be to explore the potential new use of an existing investigational therapeutic or one already approved by the Food and Drug Administration (FDA) to treat another disease.

Applications are due November 14, 2016, by 5:00 PM local time of applicant organization.

General Information

Project Approaches

Budget/Funding

Application Review


General Information

How does NCATS define "published or publicly available method" for identifying a therapeutic/indication pair or combination therapy?

Investigators may submit ideas to test new therapeutic uses on projects for which the hypothesis originates from use of a published or publicly available computational strategy or a published or publicly available crowdsourcing site. A published method is generally one that is in a peer-reviewed publication. A publicly available method could be available via a website or a commercially available product. The publicly available strategy does not need to be free, but it should be available to investigators that would like to use it.

How does NCATS define "computational algorithm"?

NCATS is interested in applications that have an identified therapeutic/indication pair for repurposing, using an existing computational algorithm. A computational algorithm is the business end of bioinformatics. A computational algorithm will mine existing data and in this case identify candidate therapeutic/indication pairs for experimental investigation. NCATS is particularly interested in demonstrating the value of computational algorithms for repurposing due to the potential uptake of successful approaches by the broader research community to subsequently identify additional therapeutic/indication pairs. NCATS is not interested in applications that seek to develop or test a new computational algorithm. NCATS seeks use cases that test methods with the potential to improve prediction of successful therapeutic/indication pairs, which can be demonstrated fully at the clinical testing stage.

How does NCATS define "crowdsourcing"?

Crowdsourcing occurs when an investigational drug is publicly posted for investigators to propose ideas for new therapeutic uses. Generally, crowdsourcing is an approach used for investigational therapeutics, not therapeutics approved by the FDA, since approved drugs already are known to the public.

An example of crowdsourcing is how NCATS matches researchers with a selection of pharmaceutical industry assets to test ideas for new therapeutic uses, with the ultimate goal of identifying promising new treatments for patients. Another example of a crowdsourcing approach is AstraZeneca's Open Innovation program or any independent website that lists investigational drugs available for repurposing.

What is an example of a successful crowdsourcing approach?

NCATS has demonstrated that public posting of industry assets to crowdsource ideas for new therapeutic uses from the academic community is an effective way to launch new collaborations. NCATS serves as a matchmaker between academic experts and pharmaceutical partners, enabling successful projects like one at Yale University that demonstrated a compound originally developed as a cancer therapy could be used to treat Alzheimer's disease. The Yale scientists gave the compound to mice that model Alzheimer's disease. After four to six weeks, the mice showed reversal of Alzheimer's symptoms such as spatial learning impairments and memory loss. The drug already was tested for safety in humans and passed key steps in the development process before the project began. By repurposing an existing drug, investigators began testing the drug in humans within three months; it typically would take a decade from the discovery of a promising compound to a drug ready for clinical trials.

How does NCATS define "rigorous" pre-clinical studies?

Rigor ensures robust and unbiased experimental design, methodology, analysis, interpretation and reporting of results. When a result can be reproduced by multiple scientists, it validates the original results and readiness to progress to the next phase of research. Reproducibility is especially important for clinical trials involving humans; these trials are built on studies that have demonstrated a particular effect or outcome. Learn more about rigor and reproducibility.

In October 2015, NIH published guidance about rigor and reproducibility for grant applications due on or after Jan. 25, 2016.

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Project Approaches

Are any research areas of higher interest to NCATS than others?

NCATS is "disease agnostic." Rather than focusing on a single disease or biological system, NCATS focuses on improving the translational science process. Therefore, NCATS is interested in funding applications that demonstrate an approach to identifying the therapeutic/indication pair that, if successful, could be applied to other diseases and improve the efficiency of predicting new repurposing targets. This funding opportunity supports projects that scientifically have supported rationales with measurable, objective, quantifiable effects of the drug or biologic (therapeutic).

NCATS will consider funding projects that address diseases that have no available treatment or an inadequate treatment, with strong scientific rationale for the new therapeutic use. Use cases that repurpose a drug or biologic originally developed or approved for a completely different disease indication are of high interest. For example, a computational prediction that a drug originally developed to treat melanoma may be effective in treating rheumatoid arthritis would be of greater interest than if that same drug may be effective in a different solid tumor cancer.

Can applicants request funding to conduct a virtual screen to identify the therapeutic/indication pair?

No. Applicants must identify a therapeutic/indication pair in the application.

Literature indicates that the proposed drug or biologic of interest would be effective in treating a disease different than that for which it was developed. Can one submit an application to conduct the pre-clinical studies to test this?

Although repurposing projects that are solely the result of traditional experimental methods and literature searches are important, they would not demonstrate the utility of a method for repurposing that ultimately could be broadly disseminated and, therefore, would not be responsive to this funding opportunity. However, if a drug is available through independent crowdsourcing, it may be eligible.

Are there any restrictions on the type of drugs or biologics that can be repurposed?

Applicants may propose to test an experimental or an FDA-approved drug or biologic. Additionally, the drug or biologic should have been tested in a Phase I clinical trial.

Applicants can propose testing a drug or biologic combination for which the combination therapy was identified through an innovative process. However, this funding mechanism can support only proof-of-concept testing, rather than all the required pre-clinical testing to determine toxicity as a combination product.

Applications for testing the effectiveness of a dietary supplement are not of interest.

Why is that important to start with therapeutics that have been in Phase I clinical trials?

Starting with investigational or FDA-approved drugs and biologics that already have passed key steps in the therapeutics development process speeds the pace of development. These compounds already have advanced to clinical studies with established pharmacokinetics and a safety profile, which enable further clinical investigation for other potential therapeutic uses.

Can applicants access assets from companies that participated in prior NCATS crowdsourcing initiatives?

Potentially. Companies that participated in the Discovering New Therapeutic Uses for Existing Molecules program are not obligated to provide access to assets from that program. However, if the company is willing to consider making the asset available, NCATS can connect interested applicants with representatives from the company. To find out more, refer to the tables on the Industry-Provided Assets page.

Refer to the FOA section "Letters of Support" for confirmation of drug access assurance.

Does this opportunity support reformulation of existing drugs?

Because investigational therapies explored through this funding opportunity must already have been in a Phase I clinical trial, NCATS expects that applicants already will have determined a drug or biologic's toxicity through Investigational New Drug-enabling pre-clinical studies. Changes in formulation that require additional Phase I studies would not be supported through this opportunity. If applicants have existing proof that the FDA would accept the available pre-clinical and Phase I data despite the formulation change, NCATS would consider the proposed therapeutic Phase II-ready.

Is the inclusion of proprietary information in an application considered disclosure that would disqualify my company from applying for patents?

Refer to the NIH Small Business Innovation Research program website for more information about intellectual property and applications.

Do I need to submit a methods manuscript if the method used to identify the new therapeutic/indication pair is publicly available?

If the method used to identify the new therapeutic/indication pair is publicly available, you only need to provide a citation or website for the published or publicly available method. If the method has not yet been published and will be by the time an award is made, provide the manuscript that has been submitted for publication. 

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Budget/Funding

How does the consortium budget affect the direct cost limit?

Pursuant to the NIH Grants Policy Statement, the direct cost limit excludes the consortium indirect costs. Questions about consortium budget should be directed to the grants management specialist identified as the point of contact in the FOA.

Can asset providers offer supplemental funding and resources if needed to complete the project?

Yes. NCATS encourages multiparty collaborations to maximize the use of federal funds; however, such additional support is not required and will not be used as a factor during the application review.

Can applicants include cost for manufacturing the active pharmaceutical ingredient?

Yes. Application budgets are limited to $200,000 in direct costs per year in year 1 and 2.

How many awards does NCATS anticipate making? How much money is available?

NCATS anticipates funding up to 10 awards, for a total level of support of $3.5 million in fiscal year 2017. The actual number of applications funded depends on the quality of applications received, budgets required for individual applications, and programmatic priorities.

The instructions ask for details on the UH3 portion of the grant in the specific aims and research strategy, which to my understanding, is just one year of clinical trial planning ($100,000 direct costs). However, the budget asks for a listing of resources for the entire clinical trial, which clearly would go far beyond $100,000 and multiple years (far beyond the UH3 phase).

The UH3 portion of the award is intended for planning the clinical trial. The information listed in the budget section was provided in error. Applicants should provide a budget and budget justification that includes the resources needed to accomplish the activities proposed for clinical trial planning.

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Application Review

Will reviewers assess the novelty of the method to identify the therapeutic/indication pair?

Reviewers will assess the proposed project according to the review criteria as stated in the FOA.

Last updated: 11-08-2017
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