Opportunity Information: Apply for RFA HD 10 019

  • The National Institutes of Health in the health income security and social services sector is offering a public funding opportunity titled "Natural History of Disorders Identifiable by Newborn Screening (R01)" and is now available to receive applicants.
  • Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.865 Child Health and Human Development Extramural Research.
  • This funding opportunity was created on May 7, 2010 and posted on May 7, 2010.
  • Applicants must submit their applications by Aug 3, 2010. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
  • The funding agency has allocated a total of $2,000,000.00 to eligible and selected applicants.
  • Each selected applicant is eligible to receive up to $1,000,000.00 in funding.
  • The number of recipients for this funding is limited to 2 candidate(s).
  • Eligible applicants include: Others (see text field entitled Additional Information on Eligibility for clarification) Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education City or township governments Independent school districts Public and State controlled institutions of higher education Native American tribal governments (Federally recognized) Small businesses Public housing authorities/Indian housing authorities 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 State governments Special district governments County governments Native American tribal organizations (other than Federally recognized tribal governments).
  • Other Eligible Applicants include the following Alaska Native and Native Hawaiian Serving Institutions Eligible Agencies of the Federal Government Faith based or Community based Organizations Hispanic serving Institutions Historically Black Colleges and Universities (HBCUs) Indian/Native American Tribal Governments (Other than Federally Recognized) Non domestic (non U.S.) Entities (Foreign Organizations) Regional Organizations Tribally Controlled Colleges and Universities (TCCUs) U.S. Territory or Possession.
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Opportunity Summary:

The Natural History of Disorders Identifiable by Newborn Screening (R01) funding opportunity (RFA-HD-10-019) is a National Institutes of Health (NIH) grant announcement issued by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Its central goal is to support research that builds a clear, comprehensive picture of the natural history of conditions that are already detected through newborn screening (NBS), as well as disorders that could plausibly benefit from being identified early through NBS. In practical terms, NICHD is looking for projects that can map out how these disorders unfold over time, including when symptoms first appear, how complications develop, the typical sequence of clinical changes, and how variable the course can be from person to person. The intent is to generate high-quality, longitudinal knowledge that becomes a foundational resource for clinicians, researchers, screening programs, and families.

A major emphasis of the opportunity is improving understanding of clinical variability and prediction. For some disorders, NICHD highlights the potential value of genotype-phenotype correlations, where a persons genetic findings (or specific variants) could help forecast disease severity, timing of onset, or likely complications. For other disorders, the FOA points to the importance of identifying additional modifiers that shape outcomes, including genetic modifiers, epigenetic influences, and environmental factors. By pulling these pieces together, the supported research is expected to clarify why two children with the same screened condition might experience very different clinical paths and what factors might explain or predict those differences.

NICHD frames the payoff of this natural history work in several concrete ways. First, better natural history data can strengthen diagnostic accuracy and reduce uncertainty after a positive screen, especially for conditions with broad clinical spectra or borderline biochemical findings. Second, it can improve understanding of genetic and clinical heterogeneity, meaning the full range of how the disorder can present and progress. Third, it can reveal or refine hypotheses about underlying disease mechanisms linked to the primary biological defect, supporting further basic and translational research. Fourth, a clearer picture of when and how complications emerge can guide prevention strategies, clinical monitoring, management decisions, and treatment timing. Finally, the FOA emphasizes family impact: strong natural history evidence can help providers give families more useful anticipatory guidance, realistic expectations, and supportive information tailored to the likely clinical course.

The mechanism of support is the NIH Research Project Grant (R01), which is the standard investigator-initiated research grant mechanism used for substantial, hypothesis-driven or data-generating projects. The announcement anticipates funding multi-state consortia of screening programs that can collectively provide robust natural history information for one or more disorders. This consortium orientation signals an interest in larger-scale, coordinated efforts that can capture enough participants, diversity, and follow-up time to make the findings broadly meaningful rather than limited to a single clinic or state program.

In terms of funding scope, NICHD planned to commit about $2,000,000 total in FY2011 to make up to two awards under this FOA, contingent on the availability of funds and the scientific merit of applications received. Applicants could propose projects lasting up to five years and request up to $1,000,000 in total costs per year, reflecting the expectation that meaningful natural history studies may require multi-site infrastructure, data coordination, long-term follow-up, and collaboration across screening programs and clinical centers.

Eligibility is broad and includes many types of domestic and non-domestic organizations. Eligible applicants listed include public and private institutions of higher education, nonprofit organizations (with or without 501(c)(3) status), for-profit organizations (including small businesses), state and local governments, tribal governments and tribal organizations, public housing authorities, independent school districts, and special district governments. The FOA also specifies additional eligible applicants such as historically Black colleges and universities (HBCUs), Hispanic-serving institutions, tribally controlled colleges and universities (TCCUs), Alaska Native and Native Hawaiian-serving institutions, faith-based or community-based organizations, regional organizations, U.S. territories or possessions, federal agencies, and foreign organizations. There is no cost sharing or matching requirement.

Key administrative details include: the opportunity was posted and created on May 7, 2010; the application closing date was August 3, 2010; and it was archived on September 3, 2010. The CFDA number associated with the program is 93.865 (Child Health and Human Development Extramural Research). The full announcement was available through the NIH grants website, and general assistance was offered through NIH Grants Information (phone 301-435-0714) and the NIH Office of Extramural Research (OER) webmaster for technical access issues.

Frequently Asked Questions (FAQs)

What is the name of this funding opportunity?

The funding opportunity is titled "The Natural History of Disorders Identifiable by Newborn Screening (R01)" and is associated with FOA number RFA-HD-10-019.

Which agency and NIH institute issued this announcement?

This is a National Institutes of Health (NIH) grant announcement issued by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

What is the central goal of this FOA?

The central goal is to support research that builds a clear, comprehensive picture of the natural history of conditions already detected through newborn screening (NBS), as well as disorders that could plausibly benefit from early identification through NBS.

What does NICHD mean by "natural history" in this context?

Natural history refers to how a disorder unfolds over time, including when symptoms first appear, how complications develop, the typical sequence of clinical changes, and how variable the course can be across individuals.

What kinds of disorders are in scope for the research?

The scope includes (1) conditions already detected through newborn screening, and (2) disorders that could plausibly benefit from being identified early through newborn screening.

What types of research questions does NICHD emphasize?

NICHD emphasizes longitudinal research that can clarify clinical variability, improve prediction of outcomes, and map disease trajectories over time, including onset, progression, and complication patterns.

Why is clinical variability a major emphasis?

The FOA highlights that children with the same screened condition may experience very different clinical paths. Understanding and predicting that variability is a key objective of the natural history work.

Does the FOA mention genotype-phenotype correlations?

Yes. For some disorders, the FOA highlights the potential value of genotype-phenotype correlations, where genetic findings (including specific variants) may help forecast disease severity, timing of onset, or likely complications.

Are factors beyond genotype discussed as contributors to outcomes?

Yes. The FOA points to the importance of identifying additional modifiers that shape outcomes, including genetic modifiers, epigenetic influences, and environmental factors.

How can better natural history data help after a positive newborn screen?

The FOA states that improved natural history data can strengthen diagnostic accuracy and reduce uncertainty after a positive screen, particularly for conditions with broad clinical spectra or borderline biochemical findings.

What is meant by genetic and clinical heterogeneity in this FOA?

Genetic and clinical heterogeneity refers to the full range of how a disorder can present and progress, including differences in genetic causes and differences in clinical manifestations over time.

How does NICHD expect natural history studies to support disease mechanism research?

The FOA indicates that a refined picture of natural history can reveal or sharpen hypotheses about underlying disease mechanisms linked to the primary biological defect, supporting further basic and translational research.

How might this research influence clinical monitoring and management?

By clarifying when and how complications emerge, the FOA suggests the research can guide prevention strategies, clinical monitoring, management decisions, and treatment timing.

What family-facing benefits does the FOA describe?

The FOA emphasizes that strong natural history evidence can help providers offer families more useful anticipatory guidance, more realistic expectations, and supportive information tailored to the likely clinical course.

What grant mechanism is used for this opportunity?

The mechanism of support is the NIH Research Project Grant (R01).

What does using an R01 mechanism imply about the type of project?

The FOA characterizes the R01 as NIH's standard investigator-initiated research grant mechanism used for substantial, hypothesis-driven or data-generating projects.

Does the FOA encourage single-site projects or multi-site efforts?

The FOA anticipates funding multi-state consortia of screening programs, signaling an interest in larger-scale, coordinated efforts rather than projects limited to a single clinic or single state program.

Why is a consortium approach emphasized?

The FOA indicates that multi-state consortia can provide more robust natural history information by capturing enough participants, diversity, and follow-up time to make findings broadly meaningful.

How many awards did NICHD plan to make, and what was the total planned commitment?

NICHD planned to commit about $2,000,000 total in FY2011 to make up to two awards, contingent on the availability of funds and the scientific merit of applications received.

What is the maximum project period allowed?

Applicants could propose projects lasting up to five years.

What is the maximum annual budget that could be requested?

Applicants could request up to $1,000,000 in total costs per year.

Why does the FOA allow up to $1,000,000 per year?

The FOA reflects the expectation that meaningful natural history studies may require multi-site infrastructure, data coordination, long-term follow-up, and collaboration across screening programs and clinical centers.

Is cost sharing or matching required?

No. The FOA states there is no cost sharing or matching requirement.

What types of organizations are eligible to apply?

Eligibility is broad and includes many types of domestic and non-domestic organizations, including public and private institutions of higher education, nonprofit organizations (with or without 501(c)(3) status), for-profit organizations (including small businesses), and multiple levels of government entities.

Are state and local governments eligible?

Yes. State and local governments are listed as eligible applicants.

Are tribal governments and tribal organizations eligible?

Yes. Tribal governments and tribal organizations are listed as eligible applicants.

Are for-profit organizations eligible?

Yes. For-profit organizations, including small businesses, are listed as eligible applicants.

Are foreign (non-U.S.) organizations eligible?

Yes. The FOA lists foreign organizations among eligible applicants.

Are specific institution types (HBCUs, HSIs, TCCUs) mentioned as eligible?

Yes. The FOA explicitly lists historically Black colleges and universities (HBCUs), Hispanic-serving institutions, and tribally controlled colleges and universities (TCCUs), among other institution types.

Are community-based or faith-based organizations eligible?

Yes. The FOA lists faith-based or community-based organizations as eligible applicants.

Are U.S. territories or possessions included in eligibility?

Yes. The FOA includes U.S. territories or possessions among eligible applicants.

Are federal agencies eligible to apply?

Yes. Federal agencies are included in the list of eligible applicants.

When was this opportunity posted and created?

The opportunity was posted and created on May 7, 2010.

What was the application closing date?

The application closing date was August 3, 2010.

What does it mean that the opportunity is archived?

The FOA was archived on September 3, 2010, indicating it is no longer an active, open announcement.

What is the CFDA number for this program?

The CFDA number associated with the program is 93.865 (Child Health and Human Development Extramural Research).

Where was the full announcement available?

The full announcement was available through the NIH grants website.

Who can be contacted for general assistance?

General assistance was offered through NIH Grants Information at 301-435-0714.

Who can be contacted for technical access issues?

Technical access issues were directed to the NIH Office of Extramural Research (OER) webmaster.

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