dd11-004 foa final 12.13.10.doc

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Department of Health and Human Services

Part 1. Overview Information

For other OpDivs add the relevant components (centers/offices) at the end of the list, following the same format.

National Center on Birth Defects and Developmental Disabilities (NCBDDD)

U01 Research Project Cooperative Agreements

Announcement Type

New Keep relevant text; enter previous FOA Number if a reissuance

Delete the non-relevant choices (RFA/PA. Fill in Fiscal Year (FY). For a CIO RFA, insert the RFA number for NNN. Leave the FOA number as NNN and it will be assigned by PGO staff.

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Insert appropriate number for CFDA

http://www.grants.gov/search/category.do e.g.:

Health

Please limit this field to a brief description of ¼ to ½ page in length. Brevity is appreciated. THIS SHOULD BE THE FIRST BULLET FROM THE OLD EXECUTIVE SUMMARY

Key Dates Publication Date

December 13, 2010PGO will add this information when posted. This field is not available for CIO edit.

The Letter of Intent due date must be at least one month (assume 30 days in one month) before application due date, and generally at least one month after posting date. If not applicable, replace “Month Day, Year” with “Not Applicable”

On-time submission requires that electronic applications be error-free and made available to CDC for processing from eRA Commons on or before the deadline date. Applications must be submitted to and validated successfully by Grants.gov/eRA Commons no later than 5:00 PM Eastern Time. Note: HHS/CDC grant submission procedures do not provide a period of time beyond the application due date to correct any error or warning notices of noncompliance with application instructions that are identified by Grants.gov or eRA systems (i.e., error correction window).

Date is expected to be three months (minimum two months) after publication of the announcement (assume 30 days in one month).

Due no later than 60 days after the application receipt date.

Or:

Executive Order 12372 does not apply to this program.

Rarely applicable See THIS IS ONLY USED WHEN STATE HEALTH DEPARTMENTS ARE INVOLVED http://www.cdc.gov/od/pgo/funding/grants/additional_req.shtm#ar7 for details.

Required Application Instructions It is critical that applicants follow the instructions in the SF 424 (R&R) Application Guide except where instructed to do otherwise (in this FOA or in a Notice from the NIH Guide for Grants and Contracts). Conformance to all requirements (both in the Application Guide and the FOA) is required and strictly enforced. Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions.

Insert as applicable and include the page limits:

Note: The Research Strategy component of the Research Plan is limited to 25 pages.

Applications that do not comply with these instructions may be delayed or not accepted for review.

Telecommunications for the Hearing Impaired: TTY 1-888-232-6348 “Apply Electronically” Text and link inserted here by PGO.

Table of Contents

Part 1. Overview Information Part 2. Full Text of the Announcement Section I. Funding Opportunity Description Section II. Award Information Section III. Eligibility Information Section IV. Application and Submission Information Section V. Application Review Information Section VI. Award Administration Information Section VII. Agency Contacts Section VIII. Other Information Part 2. Full Text of Announcement Section I. Funding Opportunity Description

Recommended length: 1-5 pages. Present the purpose of the funding opportunity in paragraph form, addressing all of the items below. Please keep in mind that readers will appreciate clarity and brevity. The following headers may be useful in preparing this section:

Purpose/Research objectives (describe the nature and need of the research opportunity)

Specific Areas of Research Interest (include examples of research topics. In such cases, add a statement indicating that appropriate topics “include but are not limited to those listed below.”

This section may also address scientific knowledge to be achieved through research supported by the special program and types of research and experimental approaches that are being sought to achieve the objectives.

In this section Use the following headings and add text below The Add CIO(s) Here NCBDDD of CDC within HHS is committed to achieving the health promotion and disease prevention objectives of "Healthy People 2010." This RFA addresses “Healthy People 2010” priority area(s) of Maternal, Infant, and Child Health and is in alignment with the NCBDDD priority to reduce racial and ethnic disparities among people with birth defects, disabilities and blood disorders. For more information go to www.healthypeople.gov. Add Priority Area(s) Here. In addition, this FOA addresses a number of high impact public health areas, including the CDC Health Protection Goal for Healthy People in Every Stage of Life, whereby all people, as especially those at greater risk of health disparities, will achieve their optimal lifespan with the best possible quality of health in every stage of life. For more information, see http://www.cdc.gov/osi/goals/people/index.html The FOA supports activities in keeping with congressional language related to muscular dystrophy, spina bifida, and fragile-x. CDC is encouraged to monitor the changing conditions over time and track health outcomes for people with muscular dystrophy; improve the quality of life of people with spina bifida; prevent and reduce the occurrence of and suffering from fragile x; and to identify ongoing needs and effective treatments for people with fragile x by increasing epidemiological research and surveillance.

Nature of the Research Opportunity –This is the purpose of the FOA This funding opportunity announcement (FOA) solicits applications to advance the understanding of disabilities resulting from rare disorders (for this FOA, CDC considers spina bifida, muscular dystrophy, and fragile x syndrome as prototypes of rare disorders). The goals of this program are to pilot test methods using linked administrative datasets to make population-based estimates for youth with rare disorders including:

1. Current prevalence estimate,

2. Well-being in terms of health, education or work, and social participation,

3. Use of services, and, for these persons,

4. Observed associations between services and well-being, as well as

5. Changes in access to health care and other services associated with transition from adolescence to adulthood.

“Well-being” is considered in the context of three key domains: 1) health, 2) education or work, and 3) social participation, for example community involvement and relationships. The short-term goal of this program is to develop and pilot test methods for identifying a defined population of young adults (ages 15-24- years) with rare disorders. For this program, spina bifida, muscular dystrophy, and fragile x syndrome will be considered as prototypes of rare disorders. Future references to “selected rare disorders” apply to these three rare disorders. Similarly, references to “service use” or “use of services” should be interpreted broadly as the specific services used and the source of payment for these services. In this FOA, “states” includes state health departments, their designees and other state agencies.

This FOA will address the validity of state-specific prevalence estimates generated by linking administrative datasets. It will compare these estimates to determine if they are consistent with estimates from other known sources and the published literature. The methods developed by this program should be capable of being easily modified to reflect the circumstances associated with a wide range of rare disorders. The methods need to be feasible for many states to implement with their currently available resources. The methods should use electronic administrative datasets that contain sufficient personal identifiers for linking to other datasets and are accurate, complete, inexpensive, and readily available in many states. After successfully implementing these methods, states will have methodologically comparable data that can be used to identify differences among states, as well as opportunities for improvement in well-being.

In this Announcement, Objective 1 - Cross-sectional Pilot Study, is an analysis of recent cross-sectional data to describe the well-being of and services used by persons with selected rare disorders. Objective 2 – Longitudinal Pilot Study is the retrospective, longitudinal observation of the well-being of and services used by persons with selected rare disorders. Both objectives must be addressed in the application.

Background - This is the story so far Rare disorders comprise a heterogeneous group of 5,000 to 8,000 diseases1, affecting approximately 25 million individuals in North America2. According to a 1984 federal law3, “ the term, rare disease or condition means any disease or condition which (a) affects less than 200,000 persons in the U. S.… 4. Most rare disorders have identified genetic origins and are often chronic, progressive, degenerative, life-threatening, and/or disabling for the affected individual. Although individually rare, the cumulative public health impact is substantial, with 6-8%5 of the US population experiencing a rare disorder during their lifetimes. These disorders are challenging for the individual, family, and society, often leading to considerable morbidity and mortality; disproportionate use of health care services; and significant social, emotional, and financial difficulties.

Lifelong support for individuals and families who are affected by rare disorders include:

1. Multidisciplinary health care services,

2. Education services

3. Rehabilitation services

4. Enhanced day care services,

5. Respite centers,

6. Emergency services,

7. Vocational training.

Thus, states need to evaluate the estimated prevalence, well-being, and service use of individuals who have rare disorders. For these officials, this information is essential for allocating and targeting resources to help individuals with rare disorders attain optimal well-being.

A triumph of medical advances is the survival to adulthood of many children affected by rare disorders. Because, until recently, so few children survived to adulthood, pediatric health providers were almost exclusively the only clinicians trained to care for affected persons. Providers for young adults with these rare disorders lacked training in their care. Now that many children with rare disorders survive to adulthood, an understanding of their transition from pediatric to adult care is needed. Further, systems for special education services and transition planning have been available for many decades. However, improvements are still needed in population outcomes in transition to higher education and work, relative independence, and active social and civic participation by young adults with rare disorders. The success of this transition can be measured by the well-being of young adults. The relationship of outcomes with services needs to be explored and depicted. Thus, this FOA focuses on the transition from adolescence (ages 15-19 years) to young adults (ages 20-24 years).

The World Health Organization (WHO) defines health as “a state of complete physical, mental and social well-being and not merely the absence of disease or infirmity”. Given the intractable nature of most rare disorders, affected persons rarely can attain health comparable to that of unaffected persons. Attaining the maximally optimal health for affected persons, however, is a reasonable public health goal.

Measuring well-being through administrative datasets may be challenging. Linking these administrative datasets may permit identification of new measures of well-being and service use. It is anticipated that linkage can yield new age-specific quantitative and qualitative measures in key domains such as health, education or work, and social participation, community involvement and relationships. These pilot programs seek to develop and refine methods that, when applied in the future, will enable many states to conduct population-based monitoring of the well-being and service use of persons with selected rare disorders.

Scientific Knowledge to be Achieved through this Funding Opportunity

Big goals/vision Because rare disorders occur infrequently, seldom will data gathered from a part of a state yield sufficient number of persons with rare disorders to permit inferences about their well-being and service use. Data for the entire state population are needed to gather sufficient data to draw inferences. It is anticipated that methods developed from these pilot states will enable other states, in the future to:

1) Implement population-based cross-sectional surveillance and a longitudinal study of rare disorders to;

a) Estimate the prevalence of rare disorders in the state’s population;

b) Estimate the well-being of persons with rare disorders (health, education/work; social participation);

c) Estimate the use of services for persons with rare disorders;

d) Examine associations between access and use of services and well-being;

e) Monitor changes in the well-being or access/use of services associated with transition from adolescence to adulthood;

f) Identify services that are associated with improved well-being;

g) Compare their findings with those from other states.

Research Objectives and Approach Key steps to achieve purpose - details/framework of what we want them to explain to us in Other Submission Requirements/how they will accomplish this Objective 1– Cross-sectional Pilot Study Approach This FOA will provide support to develop and evaluate a cross-sectional model based on linked administrative data sets that states can use to describe the well-being and service use of persons with rare disorders (spina bifida, muscular dystrophy and fragile x). With these data, states should be able to estimate the prevalence of rare disorders among adolescents and young adults, their current status in terms of health, education or work and social participation, none of which have been described previously at the state level.

When the word “data” is mentioned in the following activities, it refers to the most recent calendar year that has relevant data for state residents. When “cohort” is mentioned, it refers to the group of young state residents, aged 15-24-years, who have a specific rare disorder. This program includes three cohorts: spina bifida, muscular dystrophy, and fragile X syndrome. Applicants should describe their cross-sectional approaches for these three prototype rare disorders. When the text describes a “multi‑dimensional” measure, it refers to measures that include constellations of health; education/employment and/or social services; and health conditions. Candidate datasets for linking include Medicaid or other outpatient claim files, hospital discharge data, emergency department data, special education records, other educational records, early intervention records, , child protective services records, drivers’ licenses, and for assessing work status, voter registration records and state tax records.

1) Estimate the prevalence of each rare disorders and distribution of co-morbidities among young adults.

2) Estimate the prevalence of well-being of young adults with each of the rare disorders. Some examples of measures that address well-being include:

a) Health status as indicated by hospitalizations, emergency department visits, comorbid conditions including obesity;

b) Living situation and/or social participation in school or work;

c) The amount and or quality of services received; and

d) The differences in well-being by race, ethnicity, geography and other attributes.

3) Describe services received by young adults with rare disorders (including, but not limited to, health services):

a) The transition from pediatric to adult health care often involves difficulty in finding a health care provider who is knowledgeable about a specific rare disorder6. Finding an provider with knowledge on rare disorders may reflect the success of the young adult in making this transition;

b) The services received by young adults with a rare disorder have not been examined by racial, ethnic or geographical attributes;

4) Describe the associations between current service use and current well-being;

a) Well-being may vary by the types and combination of services used.

b) The availability and use of services may vary by racial, ethnic, geographical or other attributes.

5) Identify changes in use of health care or other services associated with transition from adolescence ( ages 15-19 years) to adulthood (ages 20-24 years).

6) Identify limitations or gaps in knowledge that may need to be supplemented or filled through direct data collection. Currently, limited information is available to describe the following for persons with rare disorders:

a) Health care needs;

b) Presence of co-morbidities;

c) Preventive as well as therapeutic service use among these young adults;

d) Participation in school or work;

e) Social or civic participation.

Objective 2 – Longitudinal Pilot Study Approach

The FOA also provides support for a pilot test to develop, implement. and evaluate a retrospective, longitudinal study using linked administrative datasets to investigate factors that may have influenced the current well-being and service use of young adults with rare disorders. Analyzing the data produced by these methods may suggest to state officials changes in services (or in the quality or quantity of services) that could be associated with the subsequently improved well-being for these young adults. These methods also will yield data that can be compared across states.

Analyzing retrospective longitudinally linked data (for at least the past 5 years and longer, if possible) for the state’s young adult residents with rare disorders will enable a state to utilize the cohort identified for the cross-sectional study as the starting point for the following activities:

1) Describe patterns of outcomes and well-being for individuals with rare disorders;

2) Describe patterns of use of services for individuals with rare disorders;

3) From the literature, identify co-morbidities that have been reported among persons with each rare disorder; using each cohort, describe the distribution of co-morbidities and note patterns; assess accuracy, completeness and possible biases of ascertaining co-morbidities;

4) Assess the impact of the types and patterns of services received during adolescence on current well-being and health;

5) Assess the utility of administrative datasets for creating complete longitudinal records and gain a better understanding of the specific challenges faced by young adults with rare disorders and their proximal and distal well-being and service use;

6) Evaluate the strengths and weaknesses of the methods and propose revisions;

7) Propose methods to supplement information limitations and fill in information gaps (e.g., individual surveys of sample populations; linkage to state and national datasets, such as Medicaid, etc.).

References

1. (http://rarediseases.info.nih.gov/Resources/Rare_Diseases_Information.aspx) (accessed August 31, 2010)

2. Schieppati A, Henter J-I, Daina E, Aperia A. Why rare diseases are an important medical and social issue. The Lancet. 2008/6/20/ 2008;371(9629):2039-2041.

3. (Orphan Drug Act as amended in 1984 by P.L. 98-551 to add a numeric prevalence threshold to the definition of rare diseases.)

4. http://rarediseases.info.nih.gov/files/Newsletter%20Final%20May242010.pdf (accessed August 24, 2010)

5. European Commission. Rare diseases. http://ec.europa.eu/health/ph_threats/non-com/rare_diseases-en.htm (accessed August 28, 2010)

6. McDonnell WM, Kocolas I, Roosevelt AT, et al. 2010. Pediatric emergency department use by adults with chronic pediatric disorders. Archives of Pediatrics and Adolescent Medicine 164(6):572-576.

Section II. Award Information

Cooperative Agreement: A support mechanism used when there will be substantial Federal scientific or programmatic involvement. Substantial involvement means that, after award, scientific or program staff will assist, guide, coordinate, or participate in project activities.

Application Types Allowed

Choose one of the following:.

New

Funds Available and Anticipated Number of Awards

Indicate the total funds available for the entire program. Sometimes this area is complicated if multiple types of awards are awarded Up to 2 awards (must include Objectives 1 and 2) will be funded at approximately $400,000 for the first 12 months.

Awards issued under this FOA are contingent on the availability of funds and submission of a sufficient number of meritorious applications.

INSERT maximum $ amount; amount that you will not exceed OR INSERT "None." This ceiling is for the first 12-month budget period. Indicate if this is direct cost only or total cost, which would include indirect costs. none INSERT any minimum $ amount, if you will not make an award smaller than a floor amount OR INSERT "None."

If project period is more than five years, approval is required.

Throughout the project period, CDC's commitment to continuation of awards will be conditional on the availability of funds, evidence of satisfactory progress by the recipient (as documented in required reports), and the determination that continued funding is in the best interest of the Federal government.

HHS/CDC grants policies as described in the HHS Grants Policy Statement will apply to the applications submitted and awards made in response to this FOA.

Section III. Eligibility Information

1. Eligible Applicants Eligible Organizations

Open competitionAre you doing open or limited competition? Justification letter is required if limited competition. Delete organizations that are not eligible to apply.

Higher Education Institutions:

· Public/State Controlled Institutions of Higher Education

· Private Institutions of Higher Education

The following types of Higher Education Institutions are always encouraged to apply for CDC support as Public or Private Institutions of Higher Education:

· Hispanic-serving Institutions

· Historically Black Colleges and Universities (HBCUs)

· Tribally Controlled Colleges and Universities (TCCUs)

· Alaska Native and Native Hawaiian Serving Institutions

Nonprofits Other Than Institutions of Higher Education

· Nonprofits (Other than Institutions of Higher Education)

For Profit Organizations

· Small Businesses

· For-Profit Organizations (Other than Small Businesses)

Governments

· State Governments

· County Governments

· City or Township Governments

· Special District Governments

· Indian/Native American Tribal Governments (Federally Recognized)

· Indian/Native American Tribal Governments (Other than Federally Recognized)

· Eligible Agencies of the Federal Government

· U.S. Territory or Possession

Other

· Independent School Districts

· Public Housing Authorities/Indian Housing Authorities

· Native American tribal organizations (other than Federally recognized tribal governments)

· Faith-based or Community-based Organizations

· Regional Organizations

· Bona Fide Agents

A Bona Fide Agent is an agency/organization identified by the state as eligible to submit an application under the state eligibility in lieu of a state application. If applying as a bona fide agent of a state or local government, a legal, binding agreement from the state or local government as documentation of the status is required. Attach with "Other Attachment Forms" when submitting via www.grants.gov.

If foreign organizations are eligible to apply, then delete the following sentence Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply. Foreign (non-U.S.) components of U.S. Organizations are not if foreign components are not allowed, delete “not” allowed.

Add additional eligibility requirements here for institutions THIS WAS SECTION III, 1.A - WHERE WE COULD LIMIT ELIGIBILITY Required Registrations Applicant organizations must complete the following registrations as described in the SF 424 (R&R) Application Guide to be eligible to apply for or receive an award. Applicants must have a valid Dun and Bradstreet Universal Numbering System (DUNS) number in order to begin each of the following registrations.

· Central Contractor Registration (CCR) – must be renewed annually

· Grants.gov

· eRA Commons

All Program Directors/Principal Investigators (PD/PIs) must also work with their institutional officials to register with the eRA Commons or ensure their existing eRA Commons account is affiliated with the eRA Commons account of the applicant organization.

All registrations must be successfully completed and active before the application due date. Applicant organizations are strongly encouraged to start the registration process at least four (4) weeks prior to the application due date.

Eligible Individuals (Project Director/Principal Investigator) in Organizations/Institutions Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Project Director/Principal Investigator (PD/PI) is invited to work with his/her organization to develop an application for support. Individuals from underrepresented racial and ethnic groups as well as individuals with disabilities are always encouraged to apply for HHS/CDC support.

For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF 424 (R&R) Application Guide.

Add additional eligibility requirements here for individuals.

2. Cost Sharing

This FOA does not require cost sharing as defined in the HHS Grants Policy Statement.

THIS WAS SECTION III, 2

3. Additional Information on Eligibility Number of Applications

Only one application per institution (normally identified by having a unique DUNS Number) is allowed. Applicant organization may submit more than one application, provided that each application is scientifically distinct. I AM CHANGING THE DEFAULT TO ONE APPLICATION ONLY. IF MULTIPE AWARDS (ENHANCED ACTIVITIES) THIS WILL NEED FURTHER MODIFICATION.

If the sentence above is not true, , CIO must insert a comment to change the sentence above to “Only one application per institution (normally identified by having a unique DUNS number) is allowed.”

HHS/CDC will not accept any application in response to this FOA that is essentially the same as one currently pending initial peer review unless the applicant withdraws the pending application. HHS/CDC will not accept any application that is essentially the same as one already reviewed.

Eligible applicants must show that they can:

1) Document a study population of at least 30,000 live births per year based on US census data;

2) Have access to at least 3 electronic datasets that:

a) Contain personally identifying data that can be used to link individual persons;

b) Are widely available in most states at no cost or at low cost;

c) Can be used to identify 3 cohorts of at least 20 persons each with spina bifida, muscular dystrophy, and fragile x syndrome to ascertain their co-morbidities and service use over time (Note – States are expected to include all persons identified who have muscular dystrophy, spina bifida or fragile-x. If 20 people with fragile x can not be identified, states may include a sample of persons with intellectual disability can be used to supplement the Fragile-X population.);

d) Are representative of the populations of interest and contain data for at least each of the preceding 5 years;

e) Can be accessed after approval from no more than 3 entities, and

f) Are expected to produce state-level estimates for at least the three selected disorders.

3) Provide evidence of capacity and demonstrated experience with linking data for individuals from large datasets and analyzing linked data;

4) Demonstrate interest by state public health practitioners (professionals in who would use these linked data to conduct public health activities) in the form of letters of support. If proposal is submitted by a group of states demonstrable experience working together. If a proposal includes plans to produce state-level estimates for greater than one state, then letters of support from each state are required that reflect the ongoing relationship and commitment to support the public health goals of this FOA.

Add Information Here related to eligibility and number of applications.

THIS WAS PREVIOUSLY “Other-Special Eligibility cRITERIA”

For an applicant to even get considered they must be responsive to the information here. Our FOA responsiveness document is developed from these criteria.

Use words like: evidence of, access to, etc.

THIS LINE MUST BE INCLUDED IF YOU HAVE SPECIAL ELIGIBILITY REQUIREMENTS HERE

If your application is incomplete or non-responsive to the special requirements listed in this section, it will not enter into the review process.

Section IV. Application and Submission Information

1. Obtaining an Application Package

Applicants must download the SF424 (R&R) application package associated with this funding opportunity from Grants.gov.

2. Content and Form of Application Submission

It is critical that applicants follow the instructions in the SF424 (R&R) Application Guide, except where instructed in this funding opportunity announcement to do otherwise. Conformance to the requirements in the Application Guide is required and strictly enforced. Applications that are out of compliance with these instructions may be delayed or not accepted for review.

Letter of Intent The only acceptable modifications to this section are those related to the staff contact information. Current policy does not allow any changes to the type of information requested in the Letter of Intent. If Letter of Intent is not applicable for this FOA, delete the heading above and the section below.

Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows IC staff to estimate the potential review workload and plan the review.

By the date listed in Part 1. Overview Information, prospective applicants are asked to submit a letter of intent that includes the following information:

Descriptive title of proposed research Name, address, and telephone number of the PD(s)/PI(s) Names of other key personnel Participating institutions Number and title of this funding opportunity

The letter of intent should be sent to:

Michael A. Brown MPHADD YOUR NAME Extramural Research Program Office

National Center for Chronic Disease Prevention and Health Promotion

Centers for Disease Control and Prevention

U.S. Department of Health and Human Services

4770 Buford Hwy, N.E., M/S K-92

Atlanta, GA 30341

Telephone: (770) 488-5118 ADD YOUR PHONE INFO Fax: (770) 488-8046

Email: mab5@cdc.gov

YOUR E-MAIL ADDRESS

FedEx address

Koger Center-Davidson Building

2858 Woodcock Blvd.

Atlanta, GA 30341 Required and Optional Components A complete application has many components, both required and optional. The forms package associated with this FOA in Grants.gov includes all applicable components for this FOA, required and optional.

Page Limitations

All page limitations described in this individual FOA must be followed.

For this FOA, the Research Strategy section is limited to 25 pages.

, insert text for page limits for the Research Strategy section of the application.

Use additional text to describe other exceptions to the page limits described in the Application Guide.

Research Plan Components All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions:

Add text in the space below (before Appendix) to supply any additional instructions related to the Research Plan, but only include information that is different from application guide.

THIS WAS PREVIOUSLY “OTHER SUBMISSION REQUIREMENTS.”

This is where you identify special programmatic, performance, or reporting requirements, etc. This is really where you tell them what you want them to do, and for them to respond to in their application.

Use action words like: a description of, demonstrate, describe, document, experience, identify, information on, plans, procedures, provide, report, etc.

Applicants’ research plan should address activities that will be conducted over the entire 3-year project period. Applicants should provide documentation that includes:

1) Describe long-term successful experience in protecting confidential data that include personal identifiers.

2) Give details about the applicant’s capacity and demonstrated experience in linking data for an individual from multiple large datasets and in analyzing linked data.

3) Provide evidence of statistical ability to design, conduct and interpret analyses, especially analyses involving repeated measures for the same individual.

4) Describe research capacity as demonstrated by staff expertise, including senior staff with a track record of successful research and publication in peer-reviewed journals. Provide evidence of research experience related to disability, birth defects or rare disorders; and health services.

5) Describe knowledge of and experience with the procedures and data used by state health and education officials to assess the status of a group and its need for services.

6) Describe any relationships with professional groups, local parent groups or advocacy groups and how they will be involved in this project. If involved, then letters of support should be provided.

7) Demonstrate ability to consider public health actions as resulting from analysis of linked administrative data.

8) Describe a clear management and staffing plan.

9) Describe of the project’s timeline with specifics for each year.

10) Demonstrate access to very recent datasets that permit accurately identifying in a defined population all persons ages 15-24 years with spina bifida, muscular dystrophy, and fragile X syndrome and, if needed, persons with intellectual disability.

11) Description of the availability in other states of the same or comparable datasets used in the awardee’s methods and confirm that the awardee’s methods can be used in another state. For example, indicate how many states have electronic records for Medicaid data, hospital discharge data, emergency department data, special education records etc.

12) Provide a budget that includes funds for three trips a year to Atlanta for project activities. The budget may include funds for travel to one professional U.S. national meeting per year (e.g., American Public Health Association, Society for Epidemiologic Research, etc.) to present findings from this project.

13) Develop case definitions for persons with each of the three selected rare disorders;

a) Using the definitions, operationalize, implement, and evaluate methods to identify all persons in a defined population with each of the selected rare disorders;

14) Develop conceptual and operational definitions for well-being of persons with a selected rare disorder that includes all three domains: health; education/work; social participation, community involvement, relationships; Describe advantages and disadvantages of operational definition.

15) Develop operational definitions for medical, educational, rehabilitation or social services used by persons with a selected rare disorder:

a) Describe advantages and disadvantages of operational definitions; Identify 3 cohorts of persons with spina bifida, muscular dystrophy, and fragile x syndrome in a state and ascertain their co-morbidities and service use over time; other conditions can be included;

b) Include a minimum of 20 persons for each disorder. (Note –All persons identified who have muscular dystrophy, spina bifida or fragile-x are expected to be included. If 20 people with fragile x cannot be identified, a sample of persons with intellectual disability can be used to supplement the Fragile-X population.).

16) Describe key variables from each of the datasets that will be used in analysis.

17) Describe your approach to data linkage, variables you will use for linkage and how you will evaluate the accuracy of the linkage. Specify the level of acceptable accuracy for the linkage.

18) From the literature, identify co-morbidities that have been reported among persons with each rare disorder; using each cohort. Identify the 3 most important/impactful co-morbid conditions for each of the 3 cohorts (co‑morbid conditions may be age- or sex-specific).

19) For each rare disorder, analyze and interpret the linked data for:

a) Estimated prevalence, especially as compared to prevalence estimates in published literature;

b) Well-being and service use for the entire population as well as by race, ethnicity and geographic region; and

c) Distributions of co-morbidities and distribution of individuals using combinations of services.

20) Provide a plan to manage study data that demonstrates the ability to maintain and improve the ability to track individuals over time.

21) Discuss approaches to compensate for missing data and interpret the impact of missing data on the results. Missing data refer to missing values for variables in the data for an individual as well as partial or complete absence of data for an individual for a period of time.

22) Describe the pattern of in and out migration of persons with rare disorders and how this can subsequently impact on successful follow up in the longitudinal study component.

23) Provide a brief and general description of at least two manuscripts that will be written for peer-reviewed journals during the second or third year of the award, including:

a) Collaboration,

b) Analytic approach, and

c) Target audience.

24) Describe methods used to identify persons with rare disorders; and describe advantages and disadvantages of identification method, considering:

a) Ease of implementation, and replication in another state,

b) Accuracy of linkage;

c) Accuracy of diagnosis of rare disorder;

d) Possible biases (e.g., does the identification method systematically fail to include affected persons with certain attributes?);

e) If appropriate, propose changes to the methods that could improve them and;

f) Timeliness of data.

25) Develop a plan for a prototype report that could be given to state officials in the state where the program is conducted to identify new services that may benefit the health of persons with rare disorders or extant services for which methods are needed to improve their availability and delivery.

26) Develop and apply methods for analyzing data to identify associations between patterns of services and outcomes of persons with rare disorders, and associations between access to health care and other services and transition from adolescence to adulthood.

Appendix

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3. Submission Dates and Times

Part I. Overview Information contains information about Key Dates. Applicants are encouraged to submit in advance of the deadline to ensure they have time to make any application corrections that might be necessary for successful submission.

Organizations must submit applications via Grants.gov, the online portal to find and apply for grants across all Federal agencies. Applicants must then complete the submission process by tracking the status of the application in the eRA Commons, NIH’s electronic system for grants administration.

Applicants are responsible for viewing their application in the eRA Commons to ensure accurate and successful submission.

Information on the submission process is provided in the SF424 (R&R) Application Guide.

Note: HHS/CDC grant submission procedures do not provide a period of time beyond the grant application due date to correct any error or warning notices of noncompliance with application instructions that are identified by Grants.gov or eRA systems (i.e. error correction window).

The application package is not complete until it has passed the Grants.gov/eRA Commons validation process. This process and email notifications of receipt and validation or rejection may take two (2) business days. Applicants are strongly encouraged to allocate additional time prior to the submission deadline to submit their applications and to correct errors identified in the validation process. Applicants are encouraged also to check the status of their application submission to determine if the application packages are complete and error-free. Applicants who encounter system errors when submitting their applications must attempt to resolve them by contacting the Grants.gov Contact Center (1-800-518-4726; support@grants.gov). If the system errors cannot be resolved, applicants must contact PGO TIMS for guidance at least 3 calendar days before the deadline date.

4. Intergovernmental Review (E.O. 12372) This initiative is not subject to intergovernmental review.

Or:

Your application is subject to Intergovernmental Review of Federal Programs, as governed by Executive Order 12372. This order sets up a system for state and local review of proposed federal assistance applications. You should contact your state single point of contact (SPOC) as early as possible to alert the SPOC to prospective applications, and to receive instructions on your state’s process. Click on the following link to get the current SPOC list: http://www.whitehouse.gov/omb/grants_spoc/

5. Funding Restrictions

All HHS/CDC awards are subject to the terms and conditions, cost principles, and other considerations described in the HHS Grants Policy Statement.

Pre-award costs are allowable only as described in the HHS Grants Policy Statement.

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6. Other Submission Requirements and Information

Applications must be submitted electronically following the instructions described in the SF 424 (R&R) Application Guide. Paper applications will not be accepted.

Applicants must complete all required registrations before the application due date. Section III. Eligibility Information contains information about registration.

For assistance with your electronic application or for more information on the electronic submission process, visit Applying Electronically.

Important reminders:

All PD/PIs must include their eRA Commons ID in the Credential field of the Senior/Key Person Profile Component of the SF 424(R&R) Application Package. Failure to register in the Commons and to include a valid PD/PI Commons ID in the credential field will prevent the successful submission of an electronic application to CDC.

The applicant organization must ensure that the DUNS number it provides on the application is the same number used in the organization’s profile in the eRA Commons and for the Central Contractor Registration (CCR). Additional information may be found in the SF424 (R&R) Application Guide.

See more tips for avoiding common errors.

Upon receipt, applications will be evaluated for completeness by the CDC Procurement and Grants Office (PGO) and responsiveness by PGO and the Center, Institute or Office of the CDC. Applications that are incomplete and/or nonresponsive will not be reviewed.

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Add any other Submission Requirements and Information; include heading (and description. Do not include information about the CONTENT and FORM of the application, which should instead be placed above in Section IV.2. Content and Form.

Section V. Application Review Information

1. Criteria

Only the review criteria described below will be considered in the review process. As part of the CDC mission, all applications submitted to the CDC in support of public health research are evaluated for scientific and technical merit through the CDC peer review system.

Overall Impact

Reviewers will provide an overall impact/priority score to reflect their assessment of the likelihood for the project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the project proposed).

Overall Impact is not editable.

Scored Review Criteria Reviewers will consider each of the review criteria below in the determination of scientific merit, and give a separate score for each. An application does not need to be strong in all categories to be judged likely to have major scientific impact. For example, a project that by its nature is not innovative may be essential to advance a field. Here is what the peer reviewers are going to be evaluating the application on. Add more here if the generic are not adequate. These would come from other submission requirements.

Significance

Does the project address an important problem or a critical barrier to progress in the field? If the aims of the project are achieved, how will scientific knowledge, technical capability, and/or clinical practice be improved? How will successful completion of the aims change the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field?

Additional Questions may be added.

Did the applicant explain the public health burden that the program will address? To what extent does the applicant demonstrate understanding of the need for this program and the planned activities? Did the applicant adequately describe a clear, concise understanding of the requirements, objectives, and purpose of this FOA?

Investigator(s)

Are the PD/PIs, collaborators, and other researchers well suited to the project? Have they demonstrated an ongoing record of accomplishments that have advanced their field(s)? If the project is collaborative or multi-PD/PI, do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project?

Does the investigator have experience in linking data for an individual from…