Opportunity Information: Apply for RFA FD 16 043
Apply for RFA FD 16 043
- The HHS-FDA in the health sector is offering a public funding opportunity titled "Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01)" and is now available to receive applicants.
- Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.103.
- This funding opportunity was created on Apr 15, 2016 and posted on Apr 15, 2016.
- Applicants must submit their applications by Oct 15, 2018. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
- Each selected applicant is eligible to receive up to $400,000.00 in funding.
- The number of recipients for this funding is limited to 5 candidate(s).
- Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For profit organizations other than small businesses, Small businesses.
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Opportunity Summary:
Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) is an HHS-FDA funding opportunity (CFDA 93.103; opportunity number RFA-FD-16-043) aimed at building the kinds of real-world, disease-specific evidence that can directly move rare disease therapies, vaccines, diagnostics, and other medical products toward approval. The program focuses on natural history studies, meaning structured research that documents how a rare disease behaves over time in the absence of (or alongside) standard care. FDA is using these grants to fill common evidence gaps in rare disease development, such as unclear progression patterns, small and heterogeneous patient populations, and a lack of validated endpoints that regulators and sponsors can rely on in clinical trials.
At its core, the grant supports studies that (1) characterize the natural course of rare diseases or conditions, (2) identify clinically meaningful subgroups based on genotype and/or phenotype, and (3) develop or validate tools needed to measure disease and treatment effects. Those tools can include clinical outcome assessments (for example, functional scales, patient-reported outcomes, or performance-based measures), biomarkers, and companion diagnostics. In practical terms, FDA is looking for projects that produce usable data for designing trials, selecting eligible participants, stratifying by disease subtype, choosing endpoints, and interpreting clinical meaningfulness. The intended payoff is a smoother path to clinical development for products targeting diseases where there is no available therapy, or where a new product could reasonably be expected to outperform existing options.
A key requirement is that the application clearly establishes the condition as an orphan disease in the United States. In the Background and Significance section, applicants must document that the estimated U.S. prevalence is under 200,000 people. For vaccines or diagnostics, the threshold is framed in terms of use: the product should be administered to fewer than 200,000 people per year in the U.S. The application also needs to make a direct, credible case for how the proposed natural history work will either support eventual product approval or generate essential development data (for example, by validating endpoints that could be used in pivotal trials or by defining progression benchmarks that enable efficient study design). FDA notes that it may request additional details during review, such as further justification of prevalence estimates and the rationale behind population calculations.
The award mechanism is listed as a cooperative agreement, which typically signals more active involvement from the funding agency than a standard grant. While the title references an R01-style research project grant, the listing specifies a cooperative agreement as the funding instrument type, implying that FDA may expect ongoing coordination, milestone-based progress, or other forms of programmatic engagement. The opportunity sits in the health funding activity category and is managed by HHS-FDA.
In terms of who can apply, eligibility is broad and includes many types of organizations that might have the patient access, clinical expertise, or research infrastructure required for rare disease natural history work. Eligible applicants include state, county, and local governments; special districts; independent school districts; public and private institutions of higher education; federally recognized tribal governments and certain tribal organizations; public housing authorities/Indian housing authorities; nonprofit organizations (both 501(c)(3) and non-501(c)(3), excluding institutions of higher education in those categories); for-profit organizations other than small businesses; and small businesses. This wide eligibility reflects the reality that rare disease expertise and patient cohorts may be distributed across academic centers, hospitals, foundations, registries, companies, and community-based research networks.
Financially, the opportunity lists an award ceiling of $400,000 and an expected number of five awards. The posting and creation date are April 15, 2016, and the original and current closing date shown is October 15, 2018. Even though the deadline in the source data is in the past, the program description is still useful as a blueprint for what FDA’s Orphan Products natural history funding has prioritized: studies that do more than describe a condition in general terms and instead generate regulatory-relevant evidence that can materially de-risk and accelerate rare disease product development.
Frequently Asked Questions (FAQs)
What is the name of this grant opportunity?
The opportunity is titled Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01).
Which agency is offering this funding?
This is a U.S. Department of Health and Human Services (HHS) funding opportunity managed by the Food and Drug Administration (FDA).
What is the purpose of this grant program?
The program is designed to support natural history studies that build disease-specific, real-world evidence to help move rare disease therapies, vaccines, diagnostics, and other medical products toward FDA approval. FDA is using these awards to address common evidence gaps that slow rare disease product development.
What does FDA mean by a "natural history study" in this program?
In this program, a natural history study refers to structured research that documents how a rare disease behaves over time, either in the absence of treatment or alongside standard care. The aim is to generate data that can be used for designing and interpreting clinical studies for product development.
What kinds of evidence gaps is FDA trying to fill through these grants?
FDA highlights several recurring gaps in rare disease development that these studies can help address, including:
- Unclear disease progression patterns
- Small and heterogeneous patient populations
- Lack of validated endpoints that can be relied on in clinical trials
What are the main project goals FDA is looking for?
The grant supports studies that aim to:
- Characterize the natural course of rare diseases or conditions
- Identify clinically meaningful subgroups based on genotype and/or phenotype
- Develop or validate tools needed to measure disease and treatment effects
What types of tools can be developed or validated under this opportunity?
Examples of tools mentioned include:
- Clinical outcome assessments (such as functional scales, patient-reported outcomes, or performance-based measures)
- Biomarkers
- Companion diagnostics
How is this research expected to help rare disease product development?
FDA is looking for projects that produce usable data for regulatory-relevant decisions, such as:
- Designing clinical trials
- Selecting eligible participants
- Stratifying by disease subtype
- Choosing endpoints
- Interpreting clinical meaningfulness of observed changes
Does the project need to be connected to eventual FDA product approval?
Yes. The application needs to make a direct and credible case that the proposed natural history work will either support eventual product approval or generate essential development data (for example, validating endpoints for pivotal trials or defining progression benchmarks that enable efficient study design).
Are there specific types of rare disease situations FDA is prioritizing?
The described intended payoff is to make clinical development smoother for products targeting diseases where no therapy is available, or where a new product could reasonably be expected to outperform existing options.
What is a key eligibility requirement related to the disease itself?
A key requirement is that the application clearly establishes the condition as an orphan disease in the United States.
How must applicants document that a disease is an orphan disease?
In the Background and Significance section, applicants must document that the estimated U.S. prevalence is under 200,000 people.
How is the orphan threshold described for vaccines or diagnostics?
For vaccines or diagnostics, the threshold is framed in terms of use: the product should be administered to fewer than 200,000 people per year in the United States.
Can FDA request additional information about prevalence estimates?
Yes. FDA notes it may request additional details during review, such as further justification of prevalence estimates and the rationale behind population calculations.
What is the funding mechanism for this opportunity?
The award mechanism is listed as a cooperative agreement.
What does it mean that this is a cooperative agreement?
Based on the description provided, a cooperative agreement typically signals more active involvement from the funding agency than a standard grant. The listing implies FDA may expect ongoing coordination, milestone-based progress, or other forms of programmatic engagement.
The title mentions an R01. Is this a standard R01 grant?
The title references an R01-style research project grant, but the listing specifies a cooperative agreement as the funding instrument type. This suggests it may not operate like a standard research grant with minimal agency involvement.
What is the CFDA number and opportunity number?
The CFDA number is 93.103 and the opportunity number is RFA-FD-16-043.
What funding activity category does this fall under?
The opportunity is listed under the health funding activity category.
Who is eligible to apply for this grant?
Eligibility is broad. Eligible applicants include:
- State governments
- County governments
- City or township governments
- Special district governments
- Independent school districts
- Public and private institutions of higher education
- Federally recognized tribal governments
- Certain tribal organizations
- Public housing authorities/Indian housing authorities
- Nonprofit organizations (501(c)(3) and non-501(c)(3), excluding institutions of higher education in those nonprofit categories)
- For-profit organizations other than small businesses
- Small businesses
Why is eligibility so broad for this program?
The description indicates that rare disease expertise and patient cohorts can be distributed across academic centers, hospitals, foundations, registries, companies, and community-based research networks, so the eligibility reflects that reality.
What is the maximum award amount?
The opportunity lists an award ceiling of $400,000.
How many awards does FDA expect to make?
The opportunity lists an expected number of five awards.
What are the posting and closing dates shown for this opportunity?
The posting/creation date shown is April 15, 2016. The original and current closing date shown is October 15, 2018.
Is the deadline still open?
Based on the dates provided, the closing date shown (October 15, 2018) is in the past.
If the deadline is in the past, why is this opportunity description still useful?
The description is still useful as a blueprint for what FDA's Orphan Products natural history funding has prioritized: natural history work that goes beyond general description and instead generates regulatory-relevant evidence that can de-risk and accelerate rare disease product development.
What makes a strong project fit for this funding description?
Based on the information provided, strong-fit projects are those that produce practical, usable outputs for product development, such as defining progression benchmarks, identifying meaningful subgroups, and developing or validating endpoints, biomarkers, or diagnostics that could be relied on in clinical trials.
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