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Discovering New Therapeutics Uses for Existing Molecules

Frequently Asked Questions About RFA-TR-17-002 and RFA-TR-17-003

Application Information

Pediatric Indications

Confidential Disclosure and Collaborative Research Agreements

Application Review

Budget/Funding


Application Information

Should there be an introduction page with a response to the written feedback from the X02?

No. The full application is not considered a resubmission; therefore, an introduction should not be included.

Having a small amount of the asset would enable investigators to quickly generate preliminary data for a UG3/UH3 application prior to the receipt date. Can investigators execute an agreement now to facilitate transfer of materials for preliminary data generation?

No. Pharmaceutical partners should not be asked to provide an asset before the application receipt date. To be fair to all applicants, no application is expected to contain preliminary data on the asset with the proposed new use.

Should applicants include a support letter from the pharmaceutical partner?

Yes. A letter indicating that applicants will have access to the asset (if the application is funded) is required. Applications that do not include such a letter will not be accepted for review.

The time frame for grants may not be optimal for certain disease conditions that are age-dependent and chronically progressive (such as neurodegenerative disorders), in which case both the pre-clinical and clinical studies (e.g., Phase I clinical trial) could take longer. What flexibility do applicants have to propose more time for the application?

As with any funding opportunity, the solicitation will not be optimal for all projects. For adult indications, the UG3 must be completed within 12 months, and the UH3 must be completed within 36 months. For pediatric indications, the UG3 must be completed within 24 months, and the UH3 must be completed within 36 months. The clinical outcomes measured in the application must conform to these timelines.

Should steering committee members be named in the application?

No. The committee will not be identified until after an award is made. Applicants should NOT name or contact potential steering committee members; only describe their role, experience and function.

Can the formulation of an asset be changed?

No. Only the formulations listed for specific assets provided by the pharmaceutical companies are available. The only exception is for pediatric indications, which may need to be formulated as a solution/suspension for oral administration (ages 6 to 11) or a small tablet/capsule (ages 12 to 18). Palatability issues also may have to be addressed for pediatric administration.

Pediatric Indications

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.

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 Investigational New Drug 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).

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

Yes. In general, pediatric populations to be considered for this funding opportunity announcement (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).

The assets that 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 one of the companion UG3/UH3 FOA focusing on adult populations.

Confidential Disclosure and Collaborative Research Agreements

Will NIH accept a UG3/UH3 application if a confidential disclosure agreement (CDA) and collaborative research agreement (CRA) have not been executed?

No. 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.

NCATS indicates that the collaborative research agreement (CRA) must be in place with the pharmaceutical partner for the full UG3/UH3 application to be accepted. If more than one institution is involved, do all institutions need to have a CRA in place with the pharmaceutical partner?

The pharmaceutical partners’ requirements vary in terms of institutions with which they will execute a CRA when multiple institutions are involved.

Would a CRA with one institution and/or lead investigator be permitted if there are multiple principal investigators or co-investigators from different institutions?

Ultimately, the agreements have to provide the investigative team with access to the asset and associated data. Documentation of that access must be provided as part of the UG3/UH3 application.

Application Review

What are the submission requirements for UG3/UH3 application acceptance?

An application must meet the following criteria to be accepted for review:

  • Use only one asset.
  • Use the same asset or mechanism of action as described in the X02 pre-application.
  • Be the same therapeutic use as proposed in the X02 pre-application.
  • Provide a letter of support from the pharmaceutical company partner documenting access to the asset and associated data needed for conducting the proposed pre-clinical studies and for filing an Investigational New Drug application for conducting the proposed clinical trials.

Budget/Funding

Will the asset be provided by the pharmaceutical partner free of charge, or does this need to be negotiated and accounted for during budgeting?

The asset and placebo will be provided by the company for the UG3 and UH3 studies at no cost to NIH or the applicant.

How will the UG3/UH3 grant applications be selected for funding?

The following will be considered in making funding decisions:

  • Scientific and technical merit of the proposed project as determined by scientific peer review
  • Availability of funds
  • Relevance of the proposed project to program priorities
Last updated: 11-08-2017
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