Opportunity Information: Apply for RFA MH 19 200

The NIH funding opportunity titled "Rare Genetic Syndromes as a Window into the Genetic Architecture of Mental Disorders (U01 Clinical Trial Not Allowed)" (RFA-MH-19-200) supports research that uses rare genetic disorders as a powerful lens for understanding how genetics contributes to mental and neuropsychiatric conditions more broadly. The central aim is to clarify the genetic architecture underlying a wide range of neuropsychiatric phenotypes observed in rare syndromes, and to determine how that genetic risk overlaps with the risk for more common, idiopathic psychiatric disorders. In practical terms, the initiative is looking for projects that can move beyond single-gene explanations and instead use genome-wide approaches to explain why people with the same rare syndrome can show very different psychiatric or behavioral outcomes, including variable expressivity (differences in severity or presentation) and incomplete penetrance (some individuals with a risk variant not showing the expected phenotype).

A key expectation is that funded teams will integrate rich phenotypic characterization with high-quality genome-wide data. Projects are intended to comprehensively evaluate how different types of genetic variation contribute to neuropsychiatric outcomes in rare disorders, including the possibility that additional variants elsewhere in the genome modify the clinical presentation of a primary syndrome. Because inconsistent or shallow phenotyping is a major barrier in rare disease research, the FOA places strong emphasis on improving the quality, consistency, and usefulness of phenotype data. Applicants are encouraged to develop or apply phenotyping methods that can be standardized and used across multiple rare genetic disorders, and that can track neuropsychiatric features across developmental time points rather than treating them as static traits. The goal is to create assessment pipelines that allow meaningful cross-disorder comparisons and improve the ability to map genotype-to-phenotype relationships.

The opportunity also explicitly promotes leveraging what already exists. Projects are encouraged to build on established cohorts, biobanks, and collaborative networks that already have infrastructure for reliable clinical data collection and genomic data generation. This reflects an intent to accelerate progress by using proven pipelines and reducing fragmentation in small, hard-to-ascertain populations. Even though this particular FOA is meant for applications that are not multi-site collaborative submissions, awardees are still expected to participate in a broader coordinated effort through a formal network structure.

All funded projects under this FOA, along with those funded under the companion collaborative U01 mechanism (RFA-MH-19-201), will operate under the Mental Health Rare Genetic Disease Network (MHRGDN). Through this network, investigators will harmonize and share clinical and genetic data across studies, helping to ensure that datasets can be combined or compared rather than remaining siloed. A major deliverable is the creation of a widely usable resource for the broader scientific community, including biospecimens and linked phenotypic and genetic datasets. The overarching idea is that the network will not only support individual projects, but will also build an enduring, standardized data and sample resource that enables future discovery and replication.

From an administrative and structural standpoint, this is a discretionary NIH cooperative agreement (U01), meaning NIH will have substantial programmatic involvement compared to a standard research grant. Clinical trials are not allowed under this specific FOA. It is also important that applicants choose the correct submission path: this announcement (RFA-MH-19-200) is for projects that can be completed without requiring two or more collaborating sites under a linked application structure. If the research requires multiple collaborating sites to complete the aims, the applicant should instead use the companion collaborative U01 FOA (RFA-MH-19-201) and submit as a linked set.

Eligibility is broad and includes many types of U.S. organizations and governmental units, such as state, county, and city governments; public and private institutions of higher education; tribal governments and tribal organizations; nonprofits (with or without 501(c)(3) status); for-profit organizations (other than small businesses) and small businesses; as well as other eligible entities. The FOA also highlights inclusion of a wide range of institution types, including Hispanic-serving institutions, HBCUs, tribally controlled colleges and universities, Alaska Native and Native Hawaiian serving institutions, and AANAPISI institutions, along with faith-based or community-based organizations and certain non-U.S. entities. The listed CFDA numbers are 93.242 and 93.865, and the original closing date provided in the source is 2018-08-09.

  • The National Institutes of Health in the health, income security and social services sector is offering a public funding opportunity titled "Rare Genetic Syndromes as a Window into the Genetic Architecture of Mental Disorders (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): 93.242, 93.865.
  • This funding opportunity was created on 2018-06-07.
  • Applicants must submit their applications by 2018-08-09. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
  • 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, Others.
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