Rare Disease Cohorts in Heart, Lung, Blood and Sleep Disorders (UG3/UH3 Clinical Trial Not Allowed) | RFA HL 20 014

Frequently Asked Questions (FAQs)

What is the name of this NIH funding opportunity?

The funding opportunity is titled "Rare Disease Cohorts in Heart, Lung, Blood and Sleep Disorders (UG3/UH3 Clinical Trial Not Allowed)" with FOA number RFA-HL-20-014.

What is the main purpose of RFA-HL-20-014?

The opportunity supports the creation of research centers that establish and follow longitudinal patient cohorts for rare diseases affecting the heart, lungs, blood, and sleep (often referred to as HLBS). The goal is to generate rigorous observational data (natural history, outcomes, and subgroup information) that can strengthen the evidence base for improved diagnostics, broader implementation of management guidelines, and better-designed future interventional studies.

What types of studies are supported under this FOA?

This FOA is designed to support well-designed observational cohort studies that follow participants over time. The intent is to build high-quality longitudinal datasets that describe disease progression, outcomes, and clinically meaningful subgroups in rare HLBS disorders.

Are clinical trials allowed under this announcement?

No. Clinical trials are not allowed under this FOA, as indicated by the title and described intent of the program.

What qualifies as a "rare disease" for this program?

The FOA focuses on conditions that affect fewer than 200,000 people in the United States.

Which disease areas does this program focus on?

The focus is on rare diseases affecting the heart, lung, blood, and sleep domains (HLBS).

Why does the FOA emphasize longitudinal cohorts?

Following patients over time allows investigators to define natural history, track outcomes, and understand variation in disease trajectories. For rare diseases, these longitudinal observational data can provide the foundation needed for future diagnostic development and for designing more targeted and feasible interventional studies.

What rare-disease challenges is the FOA designed to address?

The FOA explicitly recognizes realities common in rare disease research, including small sample sizes, geographic dispersion of patients, and clinical heterogeneity. It encourages epidemiologic approaches and practical study designs that can succeed under those constraints.

What does "deep characterization" mean in the context of this FOA?

The FOA emphasizes modern, detailed characterization of patients and disease so researchers can move beyond broad labels and explain why patients who appear to share a diagnosis can have very different clinical courses. This includes efforts to define biologically grounded subtypes and clarify disease mechanisms.

Does the FOA encourage genetic and molecular approaches?

Yes. Applicants are encouraged to incorporate genetics and deep phenotyping to define disease subtypes, clarify mechanisms, and distinguish patients who share similar clinical presentations but differ by underlying mutation, biology, or risk of complications.

What types of data does NIH expect cohorts to collect or integrate?

The FOA highlights integrating multiple data streams, including clinical measures, patient-reported outcomes (PROs), laboratory testing, imaging, environmental exposures, and various "-omics" layers. It also points to using data science methods suitable for complex, multi-dimensional analyses.

What are patient-reported outcomes (PROs), and are they relevant here?

PROs are outcomes reported directly by patients. The FOA specifically includes PROs among the data streams to integrate, reflecting an expectation that cohorts capture meaningful outcomes from the patient perspective as part of the broader observational dataset.

How does this FOA aim to improve future treatment research if it does not allow clinical trials?

By producing rigorous natural history and subgroup data, these cohorts can make future interventional studies (including clinical trials) more targeted, feasible, and informative. The FOA also notes that such datasets can support predictive tools addressing practical questions like which patients are more likely to respond to particular treatments and when intervention might be most beneficial.

Does the FOA support studying more than one rare disease within a single cohort?

Yes. The FOA allows, and in some cases encourages, designing cohorts that include related rare diseases, disorders, conditions, or syndromes together when they share pathogenesis, biochemical or cellular pathways, physiological features, or organ system involvement.

Why would a single cohort include multiple related rare diseases?

The stated rationale is that studying related conditions side-by-side can accelerate discovery by enabling comparisons across related biology and clinical presentations, and by allowing insights from one disorder to inform another.

Does the FOA provide an example of grouping related disorders?

Yes. It gives an example in blood disorders where a cohort could focus on hemoglobin disorders broadly rather than limiting enrollment to a single diagnosis such as sickle cell disease or thalassemia.

Does the FOA encourage remote or decentralized participation methods?

Yes. Telemedicine and other remote participation strategies are explicitly identified as approaches to enroll and retain participants who are geographically isolated, reduce access barriers, and improve representativeness.

What is the funding mechanism used for this opportunity?

The FOA uses the NIH cooperative agreement mechanism, and specifically the UG3/UH3 phased cooperative agreement.

What does it mean that this is a cooperative agreement?

It means awardees should expect substantial NIH program involvement compared with a standard grant, consistent with the cooperative agreement approach described in the FOA summary.

What is the UG3/UH3 phased approach described in the FOA summary?

The UG3/UH3 mechanism generally reflects a milestone-driven approach in which an initial phase supports planning and start-up activities and a subsequent phase supports full implementation. The FOA summary notes this phased structure while emphasizing that clinical trials are not allowed.

Is this opportunity considered discretionary funding?

Yes. The FOA is described as a discretionary NIH opportunity.

What is the activity category for this opportunity?

The activity category is health.

What CFDA program numbers are associated with this FOA?

The CFDA program numbers listed include 93.233 and 93.837 through 93.840.

When was the original closing date for this opportunity?

The original closing date listed in the provided information is March 18, 2020.

Does the provided information state an award ceiling or the expected number of awards?

No. The source text does not provide an award ceiling or an expected number of awards, though it states an intent to fund multiple research centers capable of establishing and maintaining robust cohorts.

Who is eligible to apply?

Eligibility is broad and includes many U.S.-based organizations and governmental entities, including state, county, city, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribally controlled colleges and universities; tribal organizations that are not federally recognized tribal governments; public housing authorities and Indian housing authorities; nonprofits with and without 501(c)(3) status (other than institutions of higher education); for-profit organizations (other than small businesses) and small businesses; and other eligible entities.

Does the FOA specifically mention organizations that serve historically underrepresented groups?

Yes. It highlights inclusion of institutions and organizations that serve groups historically underrepresented in research infrastructure, including Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Alaska Native and Native Hawaiian-serving institutions, and Asian American Native American Pacific Islander-serving institutions (AANAPISIs). It also mentions faith-based or community-based organizations and regional organizations.

Are foreign institutions eligible to apply?

No. Foreign institutions and non-U.S. entities are not eligible to apply under this FOA.

Are non-domestic components of U.S. organizations eligible?

No. Non-domestic components of U.S. organizations are not eligible, based on the provided information.

Are any international elements allowed at all?

Yes. While foreign institutions cannot apply and non-domestic components are not eligible, foreign components are allowed under NIH policy. This means a U.S. applicant may include certain well-justified international elements of the project when permitted by the NIH Grants Policy Statement.

What is the overall outcome NIH is trying to achieve with this program?

The program is described as an infrastructure-and-evidence-building initiative for rare HLBS disorders. It aims to create high-quality longitudinal observational cohorts that define natural history, capture meaningful outcomes (including PROs), reveal biologically grounded subtypes, and produce integrative datasets that make future diagnostic development and clinical trials more targeted, feasible, and informative.