Rare Disease Cohorts in Heart, Lung, Blood and Sleep Disorders (UG3/UH3 Clinical Trial Not Allowed)

Q1: Will study of a cohort that includes rare or uncommon subtypes of a common disease be eligible?

A1: No. However, study of a cohort that includes rare subtypes of an established rare disease will be eligible.

Q2: Are cohorts of participants with specific, rare genetic variants eligible for this FOA?

A2: No, studies of rare variants in and of themselves are not eligible. The rare variants would need to be established to have a prevalence of <200,000 in the US with a measurable phenotypic outcome.

Q3: Is animal work allowed?

A3: Yes. Although the intent of this RFA is to support human cohort(s) studies and clinically-oriented research, animal work that helps better understand the rare disease is permitted. Animal work cannot be major part of the proposed research.

Q4: Are non-human primate disease-model cohorts eligible for this FOA?

A4: No. Only human cohorts are eligible for this FOA.

Q5: Are biospecimens required to be deposited into BioLINCC at the end of the study period?

A5: This is encouraged, but not required. The "Guide to Building Biospecimen Collections for Study and Future Research Use" includes information on the planning, collection, processing, storage and tracking of biospecimens in clinical studies. Applicants and awardees are expected to collect, process and store biospecimens according to these guidelines.

Q6: Is it ok to utilize existing samples from subjects with the rare disease under study in addition to samples obtained from subjects enrolled in the cohort funded by this RFA?

A6: Yes. Applicants and awardees are expected to consult the "Guide to Building Biospecimen Collections for Study and Future Research Use" for planning the collection, processing, storage and tracking of biospecimens funded by this RFA. Applications will be evaluated for the strengths and weaknesses of the applicant's plan for collecting, processing, analyzing, managing and tracking, and storing study biospecimens.

Q7: If control subjects (e.g. participants without the rare disease) are enrolled to serve as a source of comparison to those enrolled in the cohort with the rare disease, can funds from this RFA support the control cohort?

A7: Yes. Establishment of control cohorts is not required but may be considered if justified and necessary to answer question(s) related to the rare disease under study.

Q8: Is my proposed cohort study eligible for this RFA?

A8: The NCATS GARD website may be a helpful resource as a means to verify eligibility if needed. Pre-submission discussion with a scientific NHLBI contact (listed at the end of the FOA) is recommended. It is also recommended that investigators provide evidence in the application that the disease under study meets the definition of a rare disease (see the RFA).

Q9: Can this FOA support cohort research in foreign countries?

A9: The intent is for this RFA to support rare disease cohort studies in which most participants are enrolled at US sites. However, enrollment of participants from foreign sites is permitted if justified to answer study-related questions.

Q10: Are MPI applications allowed for this RFA.

A10: Yes

Q11: Can an investigator from a foreign institution be included in the MPI team?

A11: Yes, if justified to answer study-related questions. Application has to be from a US institution. Foreign PI’s institution can be a subcontract.