FAQ About TRND
- How does TRND support projects?
- Once a project is accepted into TRND, what type of agreement will TRND use to establish collaborations?
- If my project is not accepted into TRND, what type of feedback can I expect?
- What mechanism does TRND use to fund the project?
- Are non-U.S. entities eligible to apply to TRND?
- Do I need an NIH intramural collaborator to apply to TRND?
- Does TRND accept gene therapy projects?
- Would TRND consider a natural product for IND-enabling studies?
- Would TRND take on a natural product for lead optimization?
- I need Good Laboratory Practices (GLP) manufacturing of my compound. Can TRND do that?
- I am concerned that an application to TRND may have consequences for potential patent application filing. Is it possible for TRND program staff to sign a confidentiality agreement to review my application?
- Is my project advanced enough for a TRND application?
- Can you tell me if my disease of interest is considered “rare” or “neglected” for the purposes of applying to TRND?
- May I also apply to TRND if I have already received funding from another NIH program or plan to submit a separate NIH grant proposal
- My research is already a collaboration between my academic lab, that of a colleague and a foundation. Are we “too big” to apply to TRND?
The Therapeutics for Rare and Neglected Diseases (TRND) program supports scientific research projects by providing access to specialized expertise and resources. TRND is not a grant-funded program. It provides selected researchers opportunities to collaborate with TRND experts and use its scientific capabilities and resources in an effort to move promising small molecules and biologics into clinical testing.
Successful applicants can partner with TRND staff to develop and execute a milestone-driven drug development program. TRND provides drug development operational support such as medicinal chemistry, animal pharmacology, or Investigational New Drug enabling studies to advance the drug program. Collaborators provide drug starting points for the project and the ongoing rare or neglected disease expertise. TRND resources for selected projects depend upon the projects’ stage and needs. When developing a TRND proposal, potential costs for the project may be included, but is not required.
Once a project is accepted into TRND, what type of agreement will TRND use to establish collaborations?
It depends. The NIH has several mechanisms that can be used to initiate a collaborative partnership through the TRND program, such as the Cooperative Research and Development Agreement (CRADA) and a Research Collaboration Agreement. The type of mechanism used depends upon the project scope and the status and type of intellectual property used in the TRND collaboration. The decision on what agreement to use will be made in consultation with the applying organization. A TRND technology transfer representative can discuss which NIH mechanism is most appropriate based upon individual needs.
During the application process, TRND receives assessments of all submitted data packages that are within program scope. At the close of the review process, TRND will return feedback reports to all applicants, including specific strengths and weaknesses identified by the reviewers. To address additional questions about the feedback report, TRND staff can arrange for and participated in a teleconference.
TRND is an opportunity to collaborate; hence, it is not a grant mechanism. A collaboration agreement (e.g., NIH Collaborative Research and Development Agreement, Memorandum of Understanding) will be established between TRND and successful applicants, NIH intramural funds will be used to support the costs of the project.
No. Anyone with a qualified drug development program that has at least advanced to the lead candidate stage may apply to collaborate with TRND. Extramural researchers do not need to be associated with NIH Intramural investigators. If a project is selected, the applicant investigators will work with the TRND investigators.
Currently, TRND cannot accept gene therapy projects.
Potentially. The biomass and the isolation procedures would have to be well established so that kilogram quantities would be readily available. This means difficult-to-harvest or endangered sources of the natural product would not be likely to be ranked highly.
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 group contains expertise in realizing a developmental candidate from a natural product lead, semi-synthetically.
No. TRND projects are based on a collaborative model for therapeutic development programs. TRND does not work on specific tasks in the therapeutic pipeline, but rather works as a full collaborator in the process. NIH has several other programs that are task-based: Bridging Interventional Development Gaps (formerly NIH Rapid Access to Intervention Development [RAID]); NCI Experimental Therapeutics (NExT); and NIH Blueprint for Neuroscience Research Grand Challenge. A more complete list of NIH programs in therapeutic development is below.
Other NIH Programs in Therapeutic Development: NIH has a significant investment in therapeutic development. Some of the programs can be found below:
- Molecular Libraries Program
- Optimization of Small Molecule Probes for the Nervous System Program:
- NCATS Clinical and Translational Science Awards
- NCATS BrIDGs Program
- NCI Experimental Therapeutics Program
- NIH Bench-to-Bedside Program
- NIH Blueprint for Neuroscience Research Neurotherapeutics Grand Challenge
- NINDS Cooperative Program in Translational Research Funding Opportunities
- NHLBI Science Moving towArds Research Translation and Therapy Program
- NHLBI Gene Therapy Resource Program
- NHLBI Production Assistance for Cellular Therapies
I am concerned that an application to TRND may have consequences for potential patent application filing. Is it possible for TRND program staff to sign a confidentiality agreement to review my application?
No. Please be assured that applications are submitted for evaluation purposes only and are reviewed in a closed private (non-public) session by reviewers who are under strict confidentiality agreements with the TRND program. Therefore, information contained in applications would normally not be considered as a public disclosure and should not affect the applicant’s ability to apply for patents. Filing patent applications prior to submitting an application to the TRND program is something an applicant should consider in conjunction with his or her legal counsel. However, please note that the applicant’s submitted lay abstract would be published upon acceptance to the TRND program, so the abstract should be drafted carefully so as not to be considered as an enabling disclosure.
It depends. Projects must be at least at the stage of a validated lead series in order to be considered for TRND. “Validated lead series” means: lead optimization stage including clear structure-activity relationships (SAR) in at least two structurally distinct chemical series, or a well-defined biological lead; 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 (ADME) properties.
Projects requiring earlier-stage resources, including assay development, high-throughput screening, and initial medicinal chemistry optimization of screening hits, are not appropriate for the TRND solicitation. However, there are other NIH resources that may be more appropriate, including the Molecular Libraries Program and the NCI Chemical Biology Consortium/NExT program.
Can you tell me if my disease of interest is considered “rare” or “neglected” for the purposes of applying to TRND?
The TRND program relies on the applicants to know their disease area fields and to make their own analysis of whether or not their disease is rare and neglected. In most cases, this can be done with a literature and textbook analysis. In general, we consider rare diseases as defined by the Orphan Drug Act (i.e., affecting fewer than 200,000 patients in the U.S.), and neglected tropical diseases as listed by the World Health Organization. Additionally, you may find it helpful to contact the NCATS Office of Rare Diseases Research, the nonprofit National Organization for Rare Disorders or any patient advocacy nonprofits that work in your specific disease area of interest.
May I also apply to TRND if I have already received funding from another NIH program or plan to submit a separate NIH grant proposal?
Yes. It may make sense for you to apply to TRND to collaborate on an aspect of your project, particularly considering TRND’s primary expertise and focus on pre-clinical drug development. As part of the selection process, TRND consults with a trans-NIH advisory group to solicit input from additional NIH Institutes and Centers regarding potential program synergies. This would not necessarily constitute an inappropriate “double-dipping.”
My research is already a collaboration between my academic lab, that of a colleague and a foundation. Are we “too big” to apply to TRND?
No. Multiple parties can jointly write a proposal in a 3-way/4-way collaboration, such as an academic and a foundation, or a pharmaceutical company and multiple academic researchers. The strengths and resources of these types of proposals can improve the strength of the program. Just be certain to follow the TRND application instructions governing institutional intellectual property and provision of key investigator biosketches.