Rare Disease Clinical Outcome Assessment Consortium (U01 Clinical Trial Not Allowed) | RFA FD 19 029

Opportunity Information: Apply for RFA FD 19 029

The Rare Disease Clinical Outcome Assessment Consortium (U01 Clinical Trial Not Allowed) is a cooperative agreement funding opportunity from the U.S. Department of Health and Human Services, Food and Drug Administration (FDA), focused on a persistent bottleneck in rare disease drug development: the lack of well-established, fit-for-purpose clinical endpoints and measurement tools that can credibly demonstrate treatment benefit. Under the Orphan Drug Act, a rare disease is one affecting fewer than 200,000 people in the United States, and that small patient population size creates practical and scientific hurdles for developing and validating clinical outcome assessments. The opportunity is built around the FDA perspective, reflected in its draft guidance on common issues in rare disease drug development, that many rare conditions do not have endpoints that are sufficiently tailored, sensitive, and interpretable for regulatory decision-making.

The core idea is to establish a consortium that works in a pre-competitive, data-sharing environment to identify, evaluate, and advance clinical outcome assessments (COAs) that could be used across multiple rare diseases, especially when those diseases share similar clinical features. While rare diseases are individually heterogeneous, many share patterns such as neurodegenerative decline, worsening cognition, and progressive loss of physical abilities. That overlap creates a chance to develop or adapt assessment tools that are not limited to a single ultra-rare condition, but instead can cover a broader spectrum of disorders with comparable functional impacts. The consortium model is meant to bring together stakeholders and existing knowledge so that tools can be assessed more efficiently than if each disease community worked in isolation.

The funding announcement highlights several measurement domains as particularly relevant because they map onto what patients and families often experience as meaningful change in daily life. These domains include cognition, adaptive behavior, and physical function, with examples such as fine motor skills, ambulation, speech, and swallowing. A major emphasis is also placed on assessments that capture the ability to perform activities of daily living, since many rare neurodegenerative or multisystem disorders ultimately impair independence and everyday functioning. In practice, this points toward developing a clearer set of COAs that can demonstrate clinically meaningful benefit in trials, even when trial populations are small and disease progression differs widely between individuals.

Two special considerations are called out because they commonly complicate endpoint selection and interpretation in rare disease studies. First, many rare diseases affect both children and adults, which means outcome assessments may need to work across developmental stages or be appropriately adapted and interpreted for different ages. Second, rare disease trials are frequently multiregional or global due to limited patient availability in any one country, so cross-cultural and cross-linguistic considerations become essential. This includes ensuring that instruments are translated and culturally adapted in a way that preserves meaning, and that the measures perform consistently across regions and settings.

The intended end product is a shared, publicly available resource that describes fit-for-purpose clinical outcome assessments relevant to rare disease drug development. This resource is expected to do more than list tools; it should provide practical, decision-support information such as which populations each assessment is appropriate for, and the strengths and limitations of each tool. By compiling this information in one place and grounding it in shared data and experience, the consortium would help reduce duplicated effort, improve measurement quality, and address unmet needs that currently slow or weaken rare disease development programs.

From an administrative standpoint, the opportunity is identified as RFA-FD-19-029 and uses the U01 cooperative agreement mechanism, meaning the FDA anticipates substantial involvement during the project rather than a hands-off grant. Clinical trials are not allowed under this announcement. The award ceiling listed is $300,000, with an expectation of a single award. Eligible applicants are broad and include various levels of government, public and private institutions of higher education, nonprofit organizations (with or without 501(c)(3) status), tribal entities, individuals, and for-profit organizations (including small businesses). The opportunity was posted June 4, 2019, with an original closing date of July 29, 2019, under CFDA 93.103.

• The Department of Health and Human Services, Food and Drug Administration in the agriculture, consumer protection, food and nutrition sector is offering a public funding opportunity titled "Rare Disease Clinical Outcome Assessment Consortium (U01 Clinical Trial Not Allowed)" 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 Jun 04, 2019.
• Applicants must submit their applications by Jul 29, 2019. (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 $300,000.00 in funding.
• The number of recipients for this funding is limited to 1 candidate(s).
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, 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, Individuals, For profit organizations other than small businesses, Small businesses.

Apply for RFA FD 19 029

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