January 10, 2023
RFA-TR-22-030 - Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases (R03 Clinical Trial Not Allowed)
National Center for Advancing Translational Sciences (NCATS)
The purpose of this notice is to clarify the requirements for responsive applications to the FOA RFA-TR-22-030: Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases (R03 Clinical Trial Not Allowed).
Section I. Funding Opportunity Description
Objectives and Scope
Currently reads:
This FOA is intended to jumpstart research on understudied proteins that are associated with rare diseases and provide applicants with funding to perform basic biochemical and/or biological work to further the characterization of understudied proteins associated with rare disease. The proposed project must focus on one or more of the eligible proteins listed above. Preliminary data are not required, and appropriate justification for the proposed approach can be provided through literature citations, data from other sources, or from investigator-generated data if any are available. The project should address critical barriers to understanding the role of understudied proteins in fundamental physiology, in disease processes, and/or as novel therapeutic agents.
The following will be considered non-responsive under this FOA and returned to the applicant without review:
- Projects that meet the NIH definition of a clinical trial;
- Projects where the majority of the proposed work focuses on proteins outside of those featured on the above lists;
- Applications that propose clinical drug development studies for understudied protein(s).
Modified to read:
This FOA is intended to jumpstart research on understudied proteins that are associated with rare diseases and provide applicants with funding to perform basic biochemical and/or biological work to further the characterization of understudied proteins associated with rare disease. The proposed project must focus on one or more of the eligible proteins listed above. Preliminary data are not required, and appropriate justification for the proposed approach can be provided through literature citations, data from other sources, or from investigator-generated data if any are available. The project should address critical barriers to understanding the role of understudied proteins in fundamental physiology, in disease processes, and/or as novel therapeutic agents.
Rare Disease Identification-The proposed project must also be focused within the primary context of a rare disease. A list of rare diseases can be found in the NCATS GARD database https://rarediseases.info.nih.gov/ or the NORD database https://rarediseases.org/for-patients-and-families/information-resources/rare-disease-information/. If the disease is not listed in either of these databases, a rare disease may be identified by one or more references confirming that the prevalence of the disease/condition is 200,000 or fewer patients in the U.S.
The following will be considered non-responsive under this FOA and returned to the applicant without review:
- Projects that meet the NIH definition of a clinical trial;
- Projects where the majority of the proposed work focuses on proteins outside of those featured on the above lists;
- Applications that propose clinical drug development studies for understudied protein(s);
- Applications proposing studies of diseases/conditions other than rare diseases (200,000 or fewer patients in the U.S.).
.
All other aspects of this FOA remain unchanged.
Karlie Sharma, Ph.D.
National Center for Advancing Translational Sciences (NCATS)
Telephone: 240-381-3118
Email:[email protected]
and Human Services (HHS)
