Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01) | RFA FD 19 001

Opportunity Information: Apply for RFA FD 19 001

Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01) is a federal grant opportunity from the U.S. Department of Health and Human Services, Food and Drug Administration (FDA), focused on improving medical product development for rare diseases by funding natural history studies. The central idea is to generate high-quality, practical knowledge about how a rare disease progresses over time in real patients, especially in areas where there are major unanswered questions and where those gaps are slowing down or preventing therapy development. By investing in natural history research that is designed to be efficient and innovative, FDA is aiming for studies that do more than describe a condition in general terms; the expectation is that the work will directly inform the development, testing, and evaluation of drugs, biologics, and other medical products intended to meet patient needs.

The FOA specifically targets rare diseases and conditions with unmet needs, meaning diseases where patients have limited or no effective treatment options and where existing knowledge about disease course, meaningful clinical outcomes, or patient subgroups may be insufficient to support well-designed clinical trials. Natural history studies supported under this program are intended to fill critical knowledge gaps, remove one or more major barriers to progress, and create a broad and significant impact either for one specific rare disease or for multiple rare diseases that share similar underlying biology or pathophysiology. In practical terms, that can include clarifying the typical timeline of disease progression, identifying clinically meaningful endpoints, characterizing variability across patients, understanding prognostic factors, and establishing data that can be used to design or optimize future interventional trials.

A major emphasis of this opportunity is translation into product development. FDA highlights that the resulting data should be useful for informing current or future development programs, including the design of clinical trials. That includes decisions such as which patient populations to enroll, how to stratify patients, what outcomes to measure, when to measure them, and how long studies need to run to detect meaningful change. The intent is to move beyond purely academic description and to produce evidence that can reduce uncertainty for developers and regulators, ultimately supporting the creation of safe and effective medical products for rare disease communities.

Administratively, this funding opportunity is listed as RFA-FD-19-001 and is categorized as a discretionary funding opportunity in the health area (CFDA number 93.103). The funding instrument type is identified as a cooperative agreement, which typically indicates that the funding agency expects to have substantial involvement during the project period compared with a standard grant. The opportunity was created on October 12, 2018, with an original closing date of January 10, 2019, and the announcement anticipated making about five awards. The award ceiling is shown as 0 in the provided record, which generally means the ceiling was not specified in that summary field rather than implying no funds; applicants usually need to consult the full FOA text for detailed budget limits, project periods, and allowable costs.

Eligibility is broad and includes many types of organizations that can carry out rare disease research. Eligible applicants listed include state, county, and local governments; 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 and other tribal organizations; nonprofit organizations both with and without 501(c)(3) status (excluding universities in those categories as noted); for-profit organizations other than small businesses; and small businesses. This wide eligibility reflects the reality that natural history studies may be led by academic medical centers, patient advocacy-linked research groups, hospitals and health systems, specialized research institutes, or industry partners, often involving collaboration across sites to reach enough patients in a rare disease population.

Overall, the grant is designed to strengthen the evidence base needed to develop treatments for rare diseases by supporting well-planned natural history studies that are directly aligned with regulatory and clinical trial planning needs. The expected outcome is a clearer, more actionable understanding of disease trajectories and meaningful measures of change, helping the field move toward trials that are feasible, appropriately powered, and focused on outcomes that matter to patients and can support eventual product approval and adoption.

• The Department of Health and Human Services, Food and Drug Administration in the health sector is offering a public funding opportunity titled "Natural History Studies Addressing Unmet Needs of Rare Diseases: 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 Oct 12, 2018.
• Applicants must submit their applications by Jan 10, 2019. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• 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), 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.

Apply for RFA FD 19 001

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