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Therapeutics for Rare and Neglected Diseases

TRND Frequently Asked Questions


How does TRND support projects?

TRND is not a grant-based funding program. TRND is an intramural NIH research program that enables collaborative partnerships with external investigators. Collaborating partners do not receive funds from the TRND program.

External partners collaborate with TRND scientists to develop and execute a milestone-driven drug development program. TRND provides project management and drug development support, such as medicinal chemistry, animal pharmacology or Investigational New Drug (IND) application-enabling studies to advance the project, using internal TRND funds. Collaborators provide drug starting points for the project and ongoing expertise in the rare or neglected disease. The specific resources provided to each collaborative partnership depend on the project’s stage and development needs.

What type of agreement does TRND use to establish collaborations?

It depends on the project. NIH has several available mechanisms to start a collaborative partnership through the TRND program, such as the Cooperative Research and Development Agreement or a Research Collaboration Agreement. The type of mechanism used depends on the project scope and the status and type of intellectual property involved in the TRND collaboration. The decision on what agreement to use is made in consultation with the collaborating partner. Learn more about available standard model agreements.

Are non-U.S. entities eligible to collaborate with TRND?

Yes.

Does an investigator need an existing NIH intramural collaborator to work with TRND?

No. Anyone with a qualified drug development program that has at least advanced to the lead candidate stage may propose a collaboration with TRND. Extramural researchers do not need to have pre-existing collaborative relationships with any NIH intramural investigators.

What types of therapeutics are of interest to TRND?

TRND is interested in developing a range of different therapeutics, including proteins, peptides, antibodies, oligonucleotides, gene- or cell-based therapies, and small molecule drugs. Currently, TRND does not support development of vaccines, devices, diagnostics or medical procedures.

Would TRND consider a natural product for IND-enabling studies?

Potentially. The biomass and the isolation procedures must be well established so that kilogram quantities are readily available, which means difficult-to-harvest or endangered sources of the natural product would not be appropriate candidates for TRND collaboration.

Would TRND take on a natural product for lead optimization?

Potentially. If the biomass required for the natural product starting material is plentiful and the process to isolate the natural product could yield multi-gram quantities, TRND would consider a natural product lead molecule. The TRND chemistry team offers expertise in realizing a developmental candidate from a natural product lead, semi-synthetically.

If only Good Laboratory Practices (GLP) manufacturing of the compound is needed, will TRND do that?

No. TRND is not a task-based service center resource. Rather, the program serves as a research collaborator in the pre-clinical development process. However, NCATS’ Bridging Interventional Development Gaps program may consider collaborations with more limited IND-enabling needs. Investigators may wish to explore NIH small business research funding opportunities or consult with extramural program staff at other NIH Institutes and Centers to discuss other funding options.

If a potential collaborator is concerned that a proposal to TRND may have consequences for potential patent application filing, could TRND staff sign a confidentiality agreement to receive the proposal?

No. TRND program staff and any external experts who may provide feedback about the proposal work under strict confidentiality agreements. Therefore, information contained in a TRND proposal is normally not considered a public disclosure and should not affect a collaborator’s ability to apply for patents. Filing patent applications prior to submitting a proposal to TRND is something an interested investigator should consider in conjunction with his or her legal counsel.

Is the proposed project advanced enough for TRND?

It depends. Projects must be at least at the stage of a validated small molecule lead or lead series — or related stage in biologics development — to be considered for TRND. “Validated” means:

  • Lead optimization stage, including clear structure-activity relationships in at least two structurally distinct chemical series or a well-defined biological lead (e.g., an affinity-matured and humanized antibody);
  • Reproducible activity in primary and orthogonal assays;
  • Efficacy in an accepted animal model (or when not available, cellular model) of the disease; and
  • Initial indications of favorable absorption, distribution, metabolism and excretion properties.

Projects requiring earlier-stage resources, including assay development, high-throughput screening, initial medicinal chemistry optimization of screening hits, gene-therapy vector codon optimization, and affinity maturation or humanization of a therapeutic antibody, are not appropriate for TRND. However, other collaborative research programs within the NCATS Division of Pre-Clinical Innovation could support projects at these earlier stages.

Is the proposed disease of interest considered “rare” or “neglected” for the purposes of collaborating with TRND?

The TRND program relies on external investigators to know their disease areas and to make their own determination of whether their disease is rare or neglected. In most cases, this can be done with a literature analysis. In general, TRND considers rare diseases as defined by the FDA Office of Orphan Products Development and the Orphan Drug Act (i.e., affecting fewer than 200,000 patients in the U.S.) and neglected tropical diseases as described by the World Health Organization. Additionally, investigators can contact NCATS’ Genetic and Rare Diseases Information Center, the nonprofit National Organization for Rare Disorders or any patient advocacy nonprofit organizations that work in specific disease areas of interest.

May an investigator collaborate with TRND if he or she already has received funding from another NIH program or plans to submit a separate NIH grant proposal?

Yes. It may make sense to seek collaboration with TRND to advance certain aspects of the proposed project, particularly considering TRND’s primary expertise and focus on pre-clinical drug development. However, TRND would not support development tasks already supported through other NIH grant or contract mechanisms. When considering new proposed collaborations, TRND seeks feedback from other NIH Institutes and Centers regarding potential program synergies and overlap.

If the proposed project already is a collaboration among multiple parties, is it “too big” for TRND?

No. Multiple parties can jointly submit a proposal for collaboration with TRND, such as an academic investigator and a foundation or a pharmaceutical company and multiple academic researchers. The strengths and resources inherent in such proposals can improve the overall quality of the proposed partnership. Follow the TRND proposal instructions governing institutional intellectual property and provision of key investigator biosketches.