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Discovering New Therapeutics Uses for Existing Molecules

Frequently Asked Questions About the X02 Pre-Application

In collaboration with AstraZeneca and Janssen Research & Development, LLC, NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for patients, using existing experimental drugs or biologics. NCATS plans to commit an estimated $6 million to fund six to 10 awards to support partnerships between the pharmaceutical companies and the biomedical research community by making a selection of industry assets available to test ideas for new therapeutic uses. NIH’s National Institute of Dental and Craniofacial Research also is participating in these new opportunities.

Interested researchers are asked to submit a letter of intent by March 17, 2017, and pre-applications are due April 17, 2017.

Industry-Provided Assets

Application Information

Pediatric Indications

Applicant Eligibility

NIH Intramural Research Program (IRP)

Budget/Funding


Industry-Provided Assets

How does NCATS solicit assets for inclusion in the New Therapeutic Uses program?

NCATS invites prospective pharmaceutical companies to partner with NCATS to explore new indications for drug candidates (assets) across a broad range of human diseases. Requirements for pharmaceutical partner participation and asset selection criteria follow:

  • Number of assets: At least three compounds will be contributed for the solicitation.
  • Asset characteristics
    • Mechanism of action is known.
    • Pharmacokinetics are suitable to explore the mechanism in a new indication.
    • Phase I clinical trial has been completed – safety profile is understood.
    • Assets currently in clinical development can be included.
  • Major company responsibilities include:
    • Provide asset information to be posted on the NCATS website.
    • Provide pre-clinical and clinical supply for studies (both drug and placebo).
    • Provide regulatory documents (i.e., cross reference letter or study reports) to enable a funded investigator to file an Investigational New Drug (IND) application in the U.S. in time to meet project timeline and milestones.
    • Use template agreements that are negotiated with NCATS. See samples here: http://www.ncats.nih.gov/ntu/assets/agreements.

Please email NewTherapeuticUses@mail.nih.gov if you work for a company that wishes to partner with NCATS.

Is this funding opportunity limited to the assets provided through the New Therapeutic Uses program, or can investigators propose other assets? If investigators have an asset they believe may have a new use but is not listed in the industry-provided assets tables for this program, can they apply?

This funding opportunity is limited to those assets provided by pharmaceutical company collaborators for the New Therapeutic Uses program through a Memorandum of Understanding with NIH. The program will not provide support for assets not listed in the tables on the Industry-Provided Assets page. NCATS encourages inquiries concerning this funding opportunity and welcomes the opportunity to answer questions from potential applicants. Institute or Center contacts can assist in directing investigators to other funding opportunity announcements (FOAs) to propose new therapeutic uses of other assets that might be of interest in specific disease areas.

Will chemical structures for the proposed assets be available?

Applicants who have meritorious X02 applications and are given the opportunity to submit a UG3/UH3 application will have access to any chemical structures and additional data after they sign a confidential disclosure agreement with the pharmaceutical partner.

There is more than one asset with the same target/mechanism of action on tables found on the Industry-Provided Assets page. Do potential grantees need to choose one in particular when submitting an X02 application?

No. In some cases, there will be sufficient information in the one-page summary charts for applicants to choose the most appropriate asset for the proposed study. In cases where the information provided on the NCATS website is not sufficient for an applicant to choose, he or she may simply identify the target/mechanism of action in the X02 application. X02 applicants who are notified of the opportunity to submit UG3/UH3 applications will receive company contact information to execute a confidential disclosure agreement with asset providers and obtain more information on the assets. To be responsive, the UG3/UH3 application must identify a single asset.

Could investigators have access to all listed assets under this announcement?

No. This program will not support screening the collection of assets to identify new uses.

Can an investigator submit an application requesting the collection of assets for pre-clinical studies, including screening?

No. This program does not support screening of the assets. The primary focus of applications should be on clinical trials (Phases I and II). If proposed, pre-clinical studies should be justified and tied to go/no-go decisions to test the asset in the patient population.

Could investigators apply separately for each asset on the list?

Applicants may submit more than one application, provided that each application is scientifically distinct. However, applicants should focus on one asset to develop biological evidence for the proposed new use. The initiative focuses on Phase II clinical trials with pre-clinical studies proposed if necessary to support the potential new use. Phase II clinical trials provide data on the relationship of dosing and response for the particular intended use (including trials on the impact of dose ranging on safety, biomarkers and proof of concept).

Application Information

Should applicants contact the pharmaceutical company before submitting an X02?

No. Applicants should not contact the pharmaceutical companies before submitting the X02. Applicants whose X02 pre-applications are identified as being highly meritorious and relevant to NIH program priorities will be notified of the opportunity to submit UG3/UH3 applications. The notification will indicate the appropriate pharmaceutical company contact. However, applicants should work with their institutions in advance to discuss the conditions in the collaborative research agreement for the selected asset prior to submitting the X02 pre-application.

What if two or more applicants submit X02 applications on the same asset for the same therapeutic indication?

If two or more X02 applications are identified as being highly meritorious, these applicants will be notified of the opportunity to submit UG3/UH3 applications.

Are proposed new uses for this program limited to stand-alone interventions?

No. The program supports clinical studies/trials to develop new uses of the assets as stand-alone interventions or as add-ons to current treatments if there is no evidence of drug-to-drug interactions with the standard-of-care treatment.

Are applicants required to use the template confidential disclosure agreements (CDAs) and collaborative research agreements (CRAs) posted on the NCATS website?

In the X02 pre-application, applicants must include a letter of support from the appropriate institutional official, confirming the institution’s willingness to engage in the necessary negotiations with the pharmaceutical company. One of the barriers encountered in moving forward projects that involve the private sector and the academic sector or other collaborators is the time it takes to execute a CRA or equivalent document. In recognition of this barrier, template agreements have been developed to streamline interactions among the parties for the program. Applicants should use the agreements.

X02 applicants should consider their willingness and the willingness of their institution to agree to the conditions in the appropriate CRA for the selected asset prior to submitting a pre-application. Investigators should work with the appropriate office within their organization to finalize the terms and conditions of the CDA and CRA for the selected asset prior to submission of a UG3/UH3 application.

Use of the template agreements is not required. However, UG3/UH3 applications submitted without evidence of access to and the ability to work with the assets, such as evidence that a CRA or equivalent document has been executed, will be deemed incomplete and returned to the applicant without review.

Pediatric Indications

Are there any limitations on the use of assets in pediatric populations?

Yes. In general, pediatric populations to be considered for this FOA refer to disease populations for which there is no adult equivalent and thus no adult population in which the drug could be tested prior to testing it in children. However, trials in pediatric or juvenile populations for indications that also have an adult population (e.g., type 2 diabetes, autism, osteoarthritis) may be considered if there is a strong scientific rationale that justifies why Phase II trials in the pediatric population are required even though an adult patient population exists (e.g., the target in the pediatric population may differ from that in the adult or treatment of children may reduce progression or severity of the disease).

Assets that the pharmaceutical companies will consider for use in pediatric populations are listed in the Table of Assets for Pediatric Indications. Applicants must click on the asset code number in the first column of the table to obtain more detailed asset information. To determine the type(s) of pediatric diseases the pharmaceutical company will consider (e.g., only trials in pediatric populations for which there is no adult population; trials for diseases/conditions that have a pediatric and adult population, if the trials in a pediatric population are scientifically justified), open the asset of interest and refer to the “Additional Characteristics” row. Applicants exploring therapies for diseases that occur in children and adults should consider applying in response to the companion UG3/UH3 FOA focusing on adult populations.

What types of studies are acceptable for pediatric indications?

Applicants should refer to the Table of Assets for Pediatric Indications to determine if the asset will be considered by the pharmaceutical company for pediatric use. After clicking on the asset of choice in the first column of the table, refer to the "Additional Characteristics" row of the more detailed asset information chart. This row provides information on the types of pediatric indications that the pharmaceutical company will consider (e.g., only trials in pediatric populations for which there is no adult population; trials for diseases/conditions that have a pediatric and adult population, if the trials in a pediatric population are scientifically justified).

Applicants must provide NIH with documentation of access to the asset and associated data needed for conducting the proposed pre-clinical studies and pediatric clinical trials and for filing an investigator-sponsored IND application to conduct the proposed clinical trials in a UG3/UH3 application (e.g., letter from the pharmaceutical company providing access to the asset for the indicated use).

Applicant Eligibility

Can foreign applicants or companies apply to the program?

No. Foreign investigators and institutions are not eligible to apply. However, they may participate as subcontractors of an awarded U.S. institution or investigator. Foreign components, as defined in the NIH Grants Policy Statement, are allowed.

Can investigators from small businesses and for-profit organizations apply for funding?

Yes. Small businesses and for-profit organizations with more than 51 percent U.S. ownership are eligible to apply.

Can an academic researcher and a biotech company collaborate to apply for the New Therapeutic Uses program?

Yes. NIH encourages scientific collaboration to leverage additional expertise and resources.

NIH Intramural Research Program (IRP)

Are NIH IRP investigators eligible to apply for these funding opportunities?

Yes. NIH IRP investigators can apply as program director(s)/principal investigator(s) (PDs/PIs), as multiple PDs/PIs in conjunction with extramural investigators, or as collaborators with extramural academic or biotechnology company investigators, pending the availability of their respective Institute's or Center's intramural funds to support the project. IRP investigators and laboratories cannot request extramural funds. An official letter from the IRP applicant’s scientific director that indicates approval of the IRP scientist's role as PD/PI or as collaborator in the project must be included as a letter of support in the submission of the X02 pre-application.

In what ways can an IRP investigator collaborate with an extramural investigator?

IRP investigators can collaborate with extramural investigators in a variety of ways:

  • The IRP and extramural investigators can submit an application as multiple PDs/PIs; however, if the applicant institution is an NIH Institute or Center, funds may not be requested for an extramural component/collaborator.
  • The IRP investigator can be a collaborator on an application submitted by an extramural investigator. However, the requests by NIH intramural scientists will be limited to the incremental costs required for participation.
  • The IRP investigator can be the PD/PI on a consortium of the extramural investigator’s application.

Does the IRP program director/principal investigator need to be a tenured/tenure-track investigator?

Yes. Tenure-track investigators can apply if they will be at NIH for the full duration of the project; they should have approximately four years left at NIH (or five years if a pediatric trial will be proposed) at the time of application because the submission, review and award process can take up to one year. Postdoctoral fellows, staff scientists and others are not eligible to apply.

If a potential grantee has a dual appointment with NIH and an extramural research organization, which organization should be listed as the applicant organization?

Choice of applicant institution depends on a variety of factors, including but not limited to the duration of the appointments, the specific terms of each appointment, the availability of funds at the NIH laboratory, the available resources of each organization and many other individual factors.

Budget/Funding

What is the total budget limit for each application?

For the X02 pre-application phase, no budget should be proposed. No direct awards will result from the X02 pre-application.

Last updated: 11-08-2017
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