CDC-RFA-DD-24-0064 revised March 15 .pdf

Centers for Disease Control and Prevention

NATIONAL CENTER ON BIRTH DEFECTS AND DEVELOPMENTAL DISABILITIES

Surveillance of Muscular Dystrophies

CDC-RFA-DD-24-0064

05/08/2024

Amendments 3/15/24:

• A – Funding Opportunity Description: 2 - CDC Project Description: a – Approach: iii –

Strategies and Activities: 1 – Collaborations: a. With other CDC programs and CDC-funded organizations: Sentence added on ability to share data with CDC and project sites.

• A – Funding Opportunity Description: 2 - CDC Project Description: a – Approach: iii –

Strategies and Activities: 1 – Collaborations: b. With organizations not funded by CDC:

Sentence added on access to multiple sources to enable creation of population-based cohort

• A – Funding Opportunity Description: 2 - CDC Project Description: a – Approach: iii –

Strategies and Activities: 2 – Population(s) of Focus: Verbiage on population requirements moved from eligibility section.

• A – Funding Opportunity Description: 2 - CDC Project Description: c. Organizational

Capacity of Recipients to Implement the Approach: Modified key capacity considerations for Component A to include applicants describing experience conducting surveillance.

• C – Eligibility Information: 2 Additional Information on Eligibility: Deleted Component

A Eligibility Criteria.

• E – Review and Selection Process: 1. Review and Selection Process: Applications will be reviewed in three phases: b. Phase II Review: i – Approach: Component A: Added and modified verbiage to include description of the surveillance population, creation of population-based cohort, and ability to share data with CDC and project sites.

• E – Review and Selection Process: 1. Review and Selection Process: Applications will be reviewed in three phases: c. Phase III Review: Deleted verbiage on factors that may affect funding decision.

• G. Agency Contacts: Grants Management Office Information: Changed contact information for Grants Management Specialist.

Table of Contents A. Funding Opportunity Description

B. Award Information

C. Eligibility Information

D. Application and Submission Information

E. Review and Selection Process

F. Award Administration Information

G. Agency Contacts

H. Other Information

I. Glossary

Part I. Overview

Applicants must go to the synopsis page of this announcement at www.grants.gov and click on the "Subscribe" button link to ensure they receive notifications of any changes to CDC-RFA- DD-24-0064. Applicants also must provide an e-mail address to www.grants.gov to receive notifications of changes.

A. Federal Agency Name:

Centers for Disease Control and Prevention (CDC)

B. Notice of Funding Opportunity (NOFO) Title:

Surveillance of Muscular Dystrophies

C. Announcement Type: New - Type 1:

This announcement is only for non-research activities supported by CDC. If research is proposed, the application will not be considered. For purposes of this NOFO, research is defined as set forth in 45 CFR 75.2 and, for further clarity, as set forth in 42 CFR 52.2 (see eCFR :: 45 CFR 75.2 -- Definitions and https://www.gpo.gov/fdsys/pkg/CFR-2007-title42-vol1/pdf/CFR-

2007-title42-vol1-sec52-2.pdf. In addition, for purposes of research involving human subjects and available exceptions for public health activities, please see 45 CFR 46.102(l) (https://www.ecfr.gov/current/title-45/subtitle-A/subchapter-A/part-46/subpart-A/section-

46.102#p-46.102(l)).

D. Agency Notice of Funding Opportunity Number:

CDC-RFA-DD-24-0064

E. Assistance Listings Number:

93.073

F. Dates:

1. Due Date for Letter of Intent (LOI):

04/08/2024

Recommended but not Required

A letter of intent is recommended, but not required. The purpose of an LOI is to allow CDC program staff to estimate the number of and plan for the review of submitted applications.

LOI must be sent via email to:

Kandi Givner

CDC, NCBDDD

Email address: xla7@cdc.gov

2. Due Date for Applications:

11:59 p.m. U.S. Eastern Standard Time, at www.grants.gov.

3. Due Date for Informational Conference Call

An informational call for potential applicants will take place on March 28, 2024, from 2:00-3:00 PM Eastern Daylight Time (EDT). The call will be held via Zoom:

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G. Executive Summary:

Summary Paragraph

The purpose of this NOFO is to conduct longitudinal, population-based surveillance of select muscular dystrophies (MD) [Duchenne MD (DMD), Becker MD (BMD), myotonic dystrophy (DM), facioscapulohumeral muscular dystrophy (FSHD), limb-girdle MD (LGMD), congenital MD (CMD), Emery-Dreifuss MD (EDMD), and distal MD] to describe key health outcomes and health inequities, with the goal of improving the health of individuals living with muscular dystrophies. This NOFO has three components to achieve the purpose of the program:

Component A is the core surveillance component and will fund longitudinal data collection, analysis, and dissemination of results. Component B will fund one applicant to conduct abstractor training and activities that improve quality of the surveillance data. Component C is an optional component in which recipients will either 1) develop and evaluate novel approaches to case finding or longitudinal data collection (e.g., through machine learning algorithms, automated data extraction) or 2) describe the use of MD-specific ICD-10-CM codes by source.

The goals of Components B and C are to improve accuracy and efficiency in case finding and data collection to improve data quality and timeliness. Surveillance data will be analyzed and results disseminated to determine prevalence, mortality/survival, care utilization, differences in access to care and new treatments, disease progression, comorbidities, and factors that lead to better outcomes.

a. NOFO Type:

CA (Cooperative Agreement)

b. Approximate Number of Awards

5-7 (Component A); 1 (Component B); 2 (Component C)

c. Total Period of Performance Funding:

$15,200,000

d. Average One Year Award Amount:

$450,000

Component A: $450-500,000 per year

Component B: $100,000 per year

Component C: $100,000 per year

e. Total Period of Performance Length:

4 year(s)

f. Estimated Award Date:

September 01, 2024

g. Cost Sharing and / or Matching Requirements:

No

Part II. Full Text

A. Funding Opportunity Description

1. Background

a. Overview

Muscular dystrophies (MDs) are a group of rare inherited disorders characterized by progressive and irreversible muscle weakness and wasting. They vary by age of onset, muscle groups affected, genes involved, severity, and progression of disease. Although MDs have been studied in numerous clinic-based groups, population-based surveillance is needed to estimate prevalence, morbidity, and mortality, and to describe the variability and disparities in access to care and treatments received among individuals with MD. Epidemiological data also support the establishment and evaluation of policies and health care services and enable assessment of health equity and outcomes among people affected by MD.

CDC’s surveillance for muscular dystrophies started in 2002 after passage of the MD CARE Act of 2001, which authorized CDC to award grants and cooperative agreements to public or nonprofit entities for a national surveillance program for muscular dystrophies. Initially, surveillance focused on the most common muscular dystrophies, Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). During this first phase of MD surveillance, the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) was developed. The network collected, analyzed and published MD surveillance data, leading to an improved understanding of DMD and BMD. Topics covered in publications thus far include prevalence, survival/mortality, delays in diagnosis, disparities in diagnosis and care received, and association of treatments on disease progression (Sahay, Paramsothy, Thomas, Mann, Butterfield). Surveillance of MDs later expanded, under the MD STARnet surveillance efforts, to include surveillance of myotonic dystrophy (DM), facioscapulohumeral MD (FSHD), limb-girdle MD (LGMD), congenital MD (CMD), Emory-Dreifuss MD (EDMD), distal MD, and oculopharyngeal MD (OPMD)—and described the sociodemographic and key clinical attributes of these populations (Do, Wallace). Currently, several analyses are underway to describe prevalence, diagnosis, survival, and health services and treatments received in these populations.

Additional years of surveillance data are needed to understand changes that have occurred in the prevalence and health of these populations especially considering recent advances in diagnosis and treatment.

The approach of this NOFO will be to conduct longitudinal, population-based surveillance of MDs, building on the methods and lessons learned from previous surveillance of MDs. This NOFO will support the development and implementation of surveillance of MDs across the lifespan, collection and analysis of surveillance data, and dissemination of results. This NOFO has three components: Component A is the core surveillance component and will fund data collection, analysis, and dissemination. Component B will fund one applicant to conduct abstractor training and activities that improve quality of the surveillance data. Component C is an optional component in which recipients will either 1) develop and evaluate novel approaches to case finding or longitudinal data collection (e.g., through machine learning algorithms, automated data extraction) or 2) describe the use of MD-specific ICD-10-CM codes by source.

b. Statutory Authorities

Sections 317C and 317Q of the Public Health Service Act (42 U.S.C. Sections 247b-4 and 247 b- 18).

c. Healthy People 2030

N/A

d. Other National Public Health Priorities and Strategies

The strategies and activities of this NOFO are consistent with the missions of the National Center on Birth Defects and Developmental Disabilities (NCBDDD) and the Division of Birth Defects and Infant Disorders (DBDID):

NCBDDD mission:

• Improving the health of people living with disabilities

• Helping children live to the fullest by understanding developmental disabilities.

DBDID mission:

• Identify and address preventable causes of birth defects, infant disorders, and related conditions.

• Promote and improve the health of people living with birth defects, infant disorders, and related conditions.

e. Relevant Work

Since 2002, MD STARnet has improved the understanding of the impact of MD on affected individuals and their families through collection, analysis, and dissemination of population-based data to stakeholder audiences. The current NOFO will build upon the work done by MD STARnet by using MD STARnet methodology to conduct surveillance for individuals with MD.

More information about MD STARnet can be found at: Muscular Dystrophy Research and Tracking | CDC.

2. CDC Project Description

a. Approach

Bold indicates period of performance outcome.CDC-RFA-DD-24-0064 Logic Model:

Surveillance of Muscular Dystrophies

Bold indicates period of performance outcome

Strategies and

Activities Short-term Outcomes Intermediate

Outcomes Long-Term

Outcomes

Component A Component A Component A Components A, B, & C

Surveillance Capacity

Conduct longitudinal, population-based data collection on individuals with muscular dystrophy

Access clinical and administrative data sources for case identification and data collection

Data Management and Reporting

Use standardized database provided by CDC for data collection and submit individual-level data with limited identifiers to CDC for pooling

Routinely monitor data quality and improve quality as needed

Dissemination:

Collaborate with CDC and other

Increased awareness and understanding among providers, patients, caregivers about:

• disease prevalence

• mortality/survival

• care utilization

• access to care and new treatments

• disease progression and comorbidities

• factors that lead to better outcomes

Improved ability of policymakers to make data-informed decisions

Improved ability to develop effective secondary prevention strategies to reduce the public health impact of MDs

Improvements in clinical care for individuals with

MD

Improved health for individuals with MDs

Prolonged survival for individuals with MDs

Improved quality of life for individuals with MDs and their families grantees in the analysis of surveillance data and dissemination of findings to stakeholder audiences

Component B Components A, B, and C Components A, B and C

Abstractor training and data quality improvement

Train, certify, and conduct ongoing training of abstractors

Plan and implement activities that improve quality of surveillance data

Improved accuracy and efficiency in case finding and data collection

Improved data quality and availability of data for action

Component C Component C

Efficiencies in Data Collection

Develop, implement, and evaluate novel approaches to case-finding and data collection

Develop and implement study to evaluate the use of MD-specific ICD-10 codes by source

Improved understanding of the accuracy of ICD codes in identifying individuals with MD

i. Purpose

The purpose of this NOFO is to conduct longitudinal, population-based surveillance of select muscular dystrophies [Duchenne MD (DMD), Becker MD (BMD), myotonic dystrophy (DM), facioscapulohumeral muscular dystrophy (FSHD), limb-girdle MD (LGMD), congenital MD (CMD), Emery-Dreifuss MD (EDMD), and distal MD] to describe key health outcomes and health inequities, with the goal of identifying opportunities to improve the health of all individuals living with muscular dystrophies.

ii. Outcomes

Recipients are expected to demonstrate measurable progress toward addressing the following short and intermediate outcomes in the logic model:

Component A

• Increased awareness and understanding among providers, patients, caregivers about:

o disease prevalence o mortality/survival o care utilization o access to care and new treatments o disease progression and comorbidities o factors that lead to better outcomes

Components A, B, and C

• Improved accuracy and efficiency in case finding and data collection

• Improved data quality and availability of data for action

Component C

• Improved understanding of the accuracy of ICD codes in identifying individuals with MD

iii. Strategies and Activities

The strategies and activities of this NOFO include those that are listed in the Strategies and Activities column in the logic model above and are restated here with additional detail. Each recipient will conduct all activities within their component and is expected to collaborate and coordinate across NOFO recipients as part of a multidisciplinary collaborative. The purpose of Component B is to provide support to Component A. The purpose of optional Component C is to develop efficient methods of case ascertainment and data collection that could be applied across NOFO recipient sites.

Component A

Surveillance Capacity

• Conduct longitudinal, population-based data collection on individuals with muscular dystrophies.

o Build on surveillance methods developed as part of MD STARnet, to identify individuals with MDs (DMD, BMD, DM, FSHD, LGMD, CMD, EDMD, and Distal MD). Eligibility criteria include:

Health encounter on or after January 1, 2015

Residency in the surveillance area at any time after January 1, 2015

Diagnosis of eligible MD o Collect data from medical records including:

Demographics (e.g., birth month/year, death month/year, insurance, employment status, educational attainment)

Diagnosis (signs and symptoms, screening and diagnostic tests)

Key clinical assessments, interventions, medications, and procedures related to MD care (e.g., neuromuscular, cardiac, pulmonary, endocrine, neurobehavioral) o Build new and maintain existing relationships with state agencies and institutions to conduct and enhance case ascertainment and data collection activities (e.g., public health authority to conduct surveillance for MDs, access to administrative data, access to data from birth defects and other surveillance systems).

o Engage in data modernization activities including testing novel approaches to data collection developed by CDC or recipient of Component C.

o Utilize MD surveillance protocols previously developed as part of MD STARnet to develop a site-specific surveillance manual or standard operating procedure and update as needed.

• Access clinical and administrative data sources for case identification and data collection o Access multiple sources, such as neuromuscular, specialty care and other outpatient clinics, hospitals; administrative data (e.g., vital records, Medicaid/Medicare, hospital discharge); or other sources available to funding recipients to generate an accurate and complete population-based cohort for the surveillance area.

o Use clinic sources to determine other healthcare sources where individuals with MD receive specialty care (e.g., cardiac and pulmonary care) and collect data at these sources.

o Recipients whose geographic areas share a physical border should collaborate to obtain medical record data of individuals who receive care in the other’s surveillance area.

o Use address tracing services to obtain address histories on eligible cases.

Data Management and Reporting

• Use standardized database and applications provided by CDC for data collection and submit individual-level data with limited identifiers, including but not limited to date of birth (month/year), date of death (month/year), and clinic visits/procedures (month/year) to CDC.

o Abstract data from medical and vital records into standardized database.

o Provide data to CDC on community indicators (e.g., socio-economic status) by linking to census data.

o Provide individual-level data to CDC. Pooled analytic data sets for each MD that contain limited identifiers will be provided to all recipients.

• Routinely monitor data quality and improve quality as needed.

o Participate in initial and ongoing abstractor training and data quality activities.

Applicant should plan for abstractors to travel to Component B recipient site for initial, focused, comprehensive training and annual trainings in conjunction with Principal Investigator meetings.

o Clean local data and perform data quality checks according to protocols.

Dissemination

• Collaborate with CDC and other grantees in the analysis of surveillance data and dissemination of findings to stakeholder audiences.

o Collaborate with CDC and other grantees in identifying knowledge gaps and prioritizing analyses.

o Propose and describe at least 3 project ideas that are expected to be developed into manuscripts.

o Present project results at least 3 times at national conferences, professional societies or other meetings that include target audiences (i.e., clinicians, industry representatives, patient advocacy groups, families and individuals with MD, regional communities, researchers, and/or public health professionals).

Presentation of results could include webinars, oral or poster presentations at conferences or other methods designed to reach target audiences.

o Conduct secondary analysis for at least 1 other research project led by another recipient or CDC.

o Participate in the development of analytic variables.

o Participate in analyses, abstracts, and manuscripts led by other recipient or CDC investigators that require epidemiologic, clinical, analytical, and other expertise available in that site. Build and maintain partnerships with the regional and local MD community (e.g., patient advocacy groups, clinicians who treat patients with MD) to inform community about surveillance results. Collaborate with CDC and national MD partners to inform community about surveillance results.

Component B

Abstractor training and data quality improvement

• Train, certify and provide ongoing training to abstractors in the standardized collection of data from medical records.

• Conduct data quality activities (e.g., inter-rater reliability exercises) to improve data quality of data collection from medical records.

• Develop, modify, and update training materials and manuals.

• Communicate regularly with abstractors through leading monthly abstractor calls and providing assistance to recipient sites as needed. Provide technical assistance as needed through participation in committees, workgroups, and Principal Investigator meetings.

Component C

Efficiencies in Data Collection

• Develop, implement, and evaluate novel approaches to case-finding and data collection.

o Develop data extraction tool (e.g., machine learning algorithms, natural language processing, automated data extraction/integration) to reduce abstractor effort and time to abstract cases.

o Pilot data extraction using clinic sources within site(s) and compare with current method of data abstraction.

o Develop documentation that clearly describes the functionality of the data extraction tool, with specifics on use of machine learning and natural language processing.

• Develop and implement study to evaluate the use of MD-specific ICD-10 codes by source.

o Development a plan to collect MD ICD-10 codes for eligible individuals by source where cases were ascertained.

o Collect data to determine the use of MD-specific ICD-10 codes by source.

o Analyze data and publish findings.

1. Collaborations

a. With other CDC programs and CDC-funded organizations:

Each recipient will conduct all required activities indicated for their respective component and is expected to collaborate and coordinate with other recipients and CDC. Applicants must describe how they plan to work with other recipients and CDC to achieve the outcomes of the NOFO.

Anticipated collaborations include implementation of data sharing, pooling of data across recipient sites, coordination of methods for data collection, determining case status, participating in abstractor training and data quality activities, engaging in data modernization activities, evaluating progress, analyzing data and disseminating results, and participating on committees and workgroups. Applicants will also collaborate with CDC and national patient advocacy groups to disseminate results from analysis of MD surveillance data.

Applicant must be able to share data with limited identifiers with CDC for inclusion in pooled analytic data sets that will be accessed by CDC and other project sites. A Data Use Agreement (DUA) is generally not needed between a NOFO recipient and CDC, since the NOFO itself serves that function. If a DUA is needed with CDC and/or other NOFO recipients, the recipient must document that within the proposal. If the NOFO recipient currently has DUAs in place they plan to extend or renew, those should be noted and a copy of the DUA provided. NOFO recipients will need to collaborate with CDC to develop and sign new DUAs within the first 6 months of the award, using CDC’s DUA template.

b. With organizations not funded by CDC:

Recipients are expected to maintain existing partnerships and establish new partnerships as needed. Recipients will maintain or build partnerships with their state health departments or other state agencies to maintain or establish public health authority for MD surveillance, facilitate access to administrative data, and to communicate information gathered from surveillance that can be useful to states in tracking their patient populations and informing changes in policy and services. Recipient must be able to access multiple sources within their site that enables creation of a population-based cohort of individuals with MD. Formal agreements already in place that are related to conducting surveillance and accessing records should be included with the application. Applicants should provide MOU/MOAs, DUAs, or other documentation to indicate the applicant has access to source records. Applicant must name the file as appropriate (e.g., MOU/MOA, DUA) and upload as a PDF, Word, or Excel file format at www.grants.gov. Renewal or development of new MOUs/MOAs or DUAs are expected to be finalized within 6 months of the award date. Recipients should also maintain and establish partnerships with local and regional patient advocacy groups and clinicians who treat individuals with MD to assist in disseminating results from surveillance data.

2. Population(s) of Focus

The target population for this NOFO includes all individuals with one of the eight eligible types of MD residing in the surveillance area. Applicants will identify a geographic area (e.g., a state or area within a state) with a population of at least 3 million people. Applicant must show evidence that their proposed site has a population of at least 3 million people and provide the distribution of this population by race and ethnicity. Evidence can be in the form of tables or figures based on census estimates within the last 5 years showing total population for the proposed study area with the distribution by race and ethnicity. Eligible individuals with DMD, BMD, DM, FSHD, LGMD, CMD, EDMD, or Distal MD must have a health encounter on or after January 1, 2015 and reside in the surveillance area at any time after January 1, 2015.

This NOFO, including funding and eligibility, is not limited based on, and does not discriminate on the basis of race, color, national origin, disability, age, sex (including gender identity, sexual orientation, and pregnancy) or other constitutionally protected statuses.

a. Health Disparities

The goal of health equity is for everyone to have a fair and just opportunity to attain their highest level of health. Achieving this requires focused and ongoing societal efforts to address historical and contemporary injustices; overcome economic, social, and other obstacles to health and healthcare; and eliminate preventable health disparities.

Broadly defined, social determinants of health are non-medical factors that influence health outcomes. They are the conditions in which people are born, grow, work, live, and age, and the wider set of forces and systems shaping the conditions of daily life. These forces (e.g., racism, climate) and systems include economic policies and systems, development agendas, social norms, social policies, and political systems. See content below and in other sections (e.g., Approach, Collaborations, Populations of Focus) for information on how this specific NOFO affects social determinants of health.

A health disparity is a preventable difference in the burden of disease, injury, violence, or opportunities to achieve optimal health that are experienced by populations that have been socially, economically, geographically, and environmentally disadvantaged. Health disparities are inextricably linked to a complex blend of social determinants that influence which populations are most disproportionately affected by these diseases and conditions.

This NOFO will provide data on the prevalence, diagnosis, and clinical care of individuals with MD among sub-populations (e.g., sex, race/ethnicity, urban vs. rural areas). Understanding any differences by these factors will help inform policies that address access to care for all individuals with MD.

iv. Funding Strategy

This NOFO includes three components. Component A includes the core surveillance and dissemination activities. Component B includes enhanced activities of abstractor training and data quality improvement. Component C is an optional component; awards will be subject to availability of funding. Component A is required for all applicants. Component B is not required for all applicants, however there are enhanced activities under Component B and one applicant should be awarded Component B. Component C is optional. Component A applicants may apply for both Components B and C or can apply for either Component B or C. The annual award range for Component A is $450,000-$500,000. The annual funding ceiling for Components B and C is $100,000. Funding for Component C is anticipated to be for 2-3 years of the project period.

b. Evaluation and Performance Measurement

i. CDC Evaluation and Performance Strategy

The evaluation and performance measurement strategy will be used to assess the performance of the overall project. Measures are subject to change. Evaluation and performance measures must be tracked by recipients and reported to CDC as requested. Measures are meant to be collective across recipients unless otherwise indicated. Applicants must include an evaluation plan that aligns with the strategies and activities in the logic model, includes performance measures, describes personnel assigned to activities, includes a timeline, describes anticipated outcomes, and describes use of evaluation results for program improvement.

CDC and recipients will use findings from these measures to: 1) ensure program implementation and continuous system improvement and 2) demonstrate achievement of activities and short-term outcomes. Information will lead to improvements in implementation of current and future longitudinal surveillance activities and demonstrate progress toward reaching program goals.

CDC has provided example process and outcome measures that can be used by recipients to describe how they will monitor and report performance measurement data.

Process measures

Component A:

Surveillance capacity

• Current MOAs/MOUs, DUAs, and other agreements that enable access to sources and ability to conduct population-based surveillance provided in the application, as needed

• Updated or new MOA/MOUs, DUAs, and other agreements provided within 6 months of award, as needed

• IRB protocols approved or exempted from review with 6 months of award.

• Site-specific surveillance manual or standard operating procedure based on MD STARnet protocol is developed within 6 months of award and updated as needed

• Number of staff hired and trained within 6 months of award

• Number of clinic sources contacted and case lists obtained

• Address tracing service identified and purchased

Data Management and Reporting

• Standardized database received and installed

• Training on use of database completed

• Number of cases abstracted by source

• Number of cases assigned case definition

• Timely response to CDC in performing periodic data quality checks

• Link birth, death, and census data prior to data submission

• Number of data sets sent to CDC

Dissemination

• Number of recipient-led analyses completed

• Number of recipient-led presentations to target audiences

• Number of recipient-led manuscripts submitted for journal peer-review

• Number of projects involved as co-author for projects led by other recipients

Component B:

Abstractor training and data quality improvement

• Number of abstractors trained and certified

• Number of data quality activities conducted

• Over 90% agreement (or other metric) for data fields in inter-rater reliability exercises

• Number of training materials and manuals developed or modified

Component C:

Efficiencies in Data Collection

• Study methods or tool developed

• Number of abstractions completed by tool

• Number of discrepant results comparing abstractions from tool and current methods

• Number of MD ICD codes by source for each case

• Manuscript published describing use of new MD ICD codes across sources or development of data collection tool

Outcome measures

Components A and C:

• Reduced time in abstracting cases when implementing data collection tool

• Number and description of conferences/meetings and number of attendees where results are presented

• Altmetrics scores of publications

Data Management Plan:

All recipients will conduct data collection for core surveillance activities. At periodic intervals, recipients will submit standardized data to CDC. Once the data are received by CDC, it is subject to applicable federal law, including for purposes of data sharing. CDC will pool data with limited identifiers (i.e., dates) from all sites into pooled analytic datasets which will be furnished to all recipients. A subset of this dataset may be requested by outside investigators in the form of a public-use dataset containing no personally identifying information and constructed to minimize the possibility of identification of individuals. CDC will coordinate with recipients on which data elements to make publicly available, but it is a requirement of all federally funded programs to comply with the CDC Data Management and Sharing Policy

(http://www.cdc.gov/maso/Policy/ReleasingData.pdf) to furnish data for public use, so all recipients are required to make these data available in an established manner.

For Component A, applicants must include a draft data management plan that includes:

• A description of the data to be collected or generated

• The standards to be used for the collected or generated data

• Mechanisms for, or limitations to, providing access to the data, including a description for the provisions for the protection of privacy, confidentiality, security, and intellectual property, or other rights

• Statement of the use of data standards that ensure all documentation describes the method of collection and what the data represent

• Plans for archiving and long-term preservation of the data, or explaining why long-term preservation and access are not justified

• Other additional requirements based on the program

During the project period, CDC will provide guidance and technical assistance to recipients for updating or revising DMP, as needed.

Since Component B does not involve the generation or collection of public health data, a Data Management Plan is not required. For projects proposed under Component C, a DMP will be required if the project involves the generation or collection of public health data.

ii. Applicant Evaluation and Performance Measurement Plan

Applicants must provide an evaluation and performance measurement plan that demonstrates how the recipient will fulfill the requirements described in the CDC Evaluation and Performance Measurement and Project Description sections of this NOFO. At a minimum, the plan must describe:

• How applicant will collect the performance measures, respond to the evaluation questions, and use evaluation findings for continuous program quality improvement, including, as applicable to the award, how findings will contribute to reducing or eliminating health disparities and inequities.

• How key program partners will participate in the evaluation and performance measurement planning processes.

• Available data sources, feasibility of collecting appropriate evaluation and performance data, and other relevant data information (e.g., performance measures proposed by the applicant).

• How evaluation findings will be disseminated to communities and populations of interest in a manner that is suitable to their needs.

• Plans for updating the Data Management Plan (DMP) as new pertinent information becomes available. If applicable, throughout the lifecycle of the project. Updates to DMP should be provided in annual progress reports. The DMP should provide a description of the data that will be produced using these NOFO funds; access to data;

data standards ensuring released data have documentation describing methods of collection, what the data represent, and data limitations; and archival and long-term data preservation plans. For more information about CDC’s policy on the DMP, see https://www.cdc.gov/grants/additional-requirements/ar-25.html.

Where the applicant chooses to, or is expected to, take on specific evaluation studies, the applicant should be directed to:

• Describe the type of evaluations (i.e., process, outcome, or both).

• Describe key evaluation questions to be addressed by these evaluations.

• Describe other information (e.g., measures, data sources).

Recipients will be required to submit a more detailed Evaluation and Performance Measurement plan, including a DMP, if applicable, within the first 6 months of award, as described in the Reporting Section of this NOFO.

d. Work Plan

Applicants are required to provide a work plan that provides both a high-level overview of the entire four-year period of performance and a detailed description of the first year of the award.

The work plan must demonstrate how the outcomes, strategies, activities, timelines, and staffing/collaborations work together. Information on performance measures should be included in the work plan.

The proposed Project Period Timeline:

• Year 1: Update/finalize MOUs/DUAs and protocols, finalize research questions and variables, pilot database, begin data collection for FSHD

• Year 2: Conduct data collection for FSHD and DMD/BMD

• Year 3: Conduct data collection for LGMD, CMD, EDMD, and distal MD and DM

• Year 4: Finish data collection for DM

For each component, the First-Year Work Plan should include:

• Program strategies

• Program activities

• Performance Measures

• Timeframe

• Persons responsible

• Activity completion date

A sample workplan that includes the first strategy and activities as an example is below:

Strategy Activity Performance

Measure Person

Responsible Timeframe Activity

Completion

Date

Surveillance Capacity

Conduct longitudinal data collection on individuals with muscular dystrophy

Access clinical and administrative data sources for case identification and data collection

For each component, the Four-Year Overview of the Project Work Plan should include intended strategies, activities, outputs, and outcomes for the entire four-year period of performance.

c. Organizational Capacity of Recipients to Implement the Approach

All applicants must demonstrate their existing or potential capacity to successfully execute all proposed strategies and activities to meet the project requirements of each funding component to which they are applying. Applicants should indicate their understanding and capacity to share data with CDC as described in the NOFO. Applicants should address infrastructure (the applicant organization’s physical space, equipment, and capacity to work remotely), partnership development, evaluation and performance monitoring, financial reporting, budget management and administration, personnel management (including developing staffing plans, and developing and training workforce), and expertise and experience related to all component-specific project focus areas. Furthermore, applicants must be fully capable of managing the required procurement efforts, including the ability to write and award contracts in accordance with 45 CFR Part 75.

Key capacity considerations for Component A to address in the application:

• Applicants must describe their experience conducting surveillance for MD or rare diseases/birth defects and provide documentation of current operational capacity to conduct multiple source surveillance using methodology similar to that used in MD STARnet. Each applicant must provide evidence (e.g., MOU/MOA, DUA, or letter of support) from the state government or governing body (e.g., health system, university) that they would have access to health records, if funded, and be able to submit individual-level data with limited identifiers to CDC. Public health authority to conduct surveillance for MD is strongly encouraged.

• Applicants must describe their resources and staffing levels. The exact personnel and percentage of time allotted will vary by site. However, there are basic project staff requirements, including having actively involved principal investigator(s), project coordinator, epidemiologist, record abstractors (at least one full-time equivalent), clinician reviewer, data analysts, and data management/programmer support.

• Applicants must demonstrate that they have adequate technical and facility resources to meet the project’s goals, including informational technology (IT) infrastructure such as the capacity to maintain a database application (e.g., SQL Server) on a local network server.

Key capacity considerations for Component B to address in the application:

• Applicants must describe their experience in conducting abstractor training and data quality improvement activities. They must describe resources and staffing roles and responsibilities.

Key capacity considerations for Component C to address in the application:

• Applicants must describe their experience related to the content area of their proposed project (e.g., development of machine learning algorithms/data extraction tools). They must also describe resources and staffing roles and responsibilities.

e. CDC Monitoring and Accountability Approach

Monitoring activities include routine and ongoing communication between CDC and recipients, site visits, and recipient reporting (including work plans, performance, and financial reporting).

Consistent with applicable grants regulations and policies, CDC expects the following to be included in post-award monitoring for grants and cooperative agreements:

• Tracking recipient progress in achieving the desired outcomes.

• Ensuring the adequacy of recipient systems that underlie and generate data reports.

• Creating an environment that fosters integrity in program performance and results.

Monitoring may also include the following activities deemed necessary to monitor the award:

• Ensuring that work plans are feasible based on the budget and consistent with the intent of the award.

• Ensuring that recipients are performing at a sufficient level to achieve outcomes within stated timeframes.

• Working with recipients on adjusting the work plan based on achievement of outcomes, evaluation results and changing budgets.

• Monitoring performance measures (both programmatic and financial) to assure satisfactory performance levels.

Monitoring and reporting activities that assist grants management staff (e.g., grants management officers and specialists, and project officers) in the identification, notification, and management of high-risk recipients.

CDC and recipients will collaborate on development of measures for performance monitoring.

Emphasis will be placed on process measures for monthly reporting and on outcome measures for yearly reporting. CDC will provide feedback on process measures during site-specific monthly calls and on aggregated (all-site) outcome measures during coordinating committee calls and PI meetings.

f. CDC Program Support to Recipients

CDC staff will be substantially involved beyond site visits and regular performance and financial monitoring during the period of performance of this cooperative agreement. Substantial involvement means that the recipient can expect federal programmatic partnerships in carrying out the efforts under the award. The CDC program will work in partnership with the recipient to ensure the success of the cooperative agreement by providing:

i. Technical Assistance

• Assist recipients with surveillance activities including surveillance protocols, the operationalization of case definitions, and providing data dictionaries and other resources

• Provide technical consultation regarding surveillance methods including data collection and abstraction, quality assurance, evaluation, case review, analyses, and reporting

• Hold monthly calls and periodic site visits as needed with recipients to monitor performance and provide technical assistance

• Share data collection systems and provide technical support to qualified data management/programmer staff

• Provide tools and guidance for extracting data with limited identifiers for submission to

CDC

• Pool data from all sites and distribute datasets to recipients

• Assist in the analyses, interpretation, and reporting of findings in the literature

• Provide updated information on relevant CDC, US Department of Health and Human Services (HHS), and other policies and regulations that affect the programs

ii. Information Sharing among Recipients:

• Facilitate implementation and updating of data sharing guidelines.

• Maintain a platform (i.e. SharePoint) which allows documents and other key project materials to be shared among sites.

• Coordinate and facilitate discussion on committee conference calls (e.g., coordinating, data sharing, data management); develop agendas and provide meeting minutes; follow-up with sites on action items from calls.

• Organize and lead an annual in-person (Atlanta) or virtual Principal Investigator meeting.

B. Award Information

1. Funding Instrument Type:

CA (Cooperative Agreement)

CDC's substantial involvement in this program appears in the CDC Program Support to Recipients Section.

2. Award Mechanism:

U01

3. Fiscal Year:

Estimated Total Funding:

$15,200,000

4. Approximate Total Fiscal Year Funding:

$3,800,000

Component A: $2,250,000 – $3,500,000 per year

Component B – $100,000 per year

Component C – fund if available: $200,000 per year

This amount is subject to the availability of funds.

5. Approximate Period of Performance Funding:

$15,200,000

Component A: $9,000,000 – $14,000,000

Component B – $400,000

Component C – fund if available: $800,000

6. Total Period of Performance Length:

4 year(s)

7. Expected Number of Awards:

5-7 (Component A); 1 (Component B); 2 (Component C)

8. Approximate Average Award:

$380,000

Per Budget Period

Component A: $450-500,000 per year

Component B: $100,000 per year

Component C: $100,000 per year

9. Award Ceiling:

$700,000

Per Budget Period

Component A: $500,000 per year

Component B: $100,000 per year

Component C: $100,000 per year

10. Award Floor:

$250,000

Per Budget Period

Component A: $250,000

Component B: $50,000

Component C: $50,000

11. Estimated Award Date:

September 01, 2024

12. Budget Period Length:

12 month(s)

Throughout the period of performance, CDC will continue the award based on the availability of funds, the 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.

The total number of years for which federal support has been approved (period of performance) will be shown in the “Notice of Award.” This information does not constitute a commitment by the federal government to fund the entire period. The total period of performance comprises the initial competitive segment and any subsequent non-competitive continuation award(s).

13. Direct Assistance

Direct Assistance (DA) is not available through this NOFO.

If you are successful and receive a Notice of Award, in accepting the award, you agree that the award and any activities thereunder are subject to all provisions of 45 CFR Part 75, currently in effect or implemented during the period of the award, other Department regulations and policies in effect at the time of the award, and applicable statutory provisions.

C. Eligibility Information

1. Eligible Applicants

Eligibility Category:

00 (State governments)

01 (County governments)

02 (City or township governments)

04 (Special district governments)

05 (Independent school districts)

06 (Public and State controlled institutions of higher education)

07 (Native American tribal governments (Federally recognized))

08 (Public housing authorities/Indian housing authorities)

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

12 (Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education)

13 (Nonprofits without 501(c)(3) status with the IRS, other than institutions of higher education)

20 (Private institutions of higher education)

22 (For profit organizations other than small businesses)

23 (Small businesses)

25 (Others (see text field entitled "Additional Information on Eligibility" for clarification))

99 (Unrestricted (i.e., open to any type of entity above), subject to any clarification in text field entitled "Additional Information on Eligibility")

Additional Eligibility Category:

Government Organizations:

State (includes the District of Columbia)

Local governments or their bona fide agents

Territorial governments or their bona fide agents in the Commonwealth of Puerto Rico, the Virgin Islands, the Commonwealth of the Northern Marianna Islands, American Samoa, Guam, the Federated States of Micronesia, the Republic of the Marshall Islands, and the Republic of Palau

State controlled institutions of higher education

American Indian or Alaska Native tribal governments (federally recognized or state-recognized)

American Indian or Alaska native tribally designated organizations

Other:

Private colleges and universities

Community-based organizations

Faith-based organizations

2. Additional Information on Eligibility

For Component B or C:

• Applicants must demonstrate that they have applied for Component A. Evidence can include a copy of the face page of the application for Component A.

3. Justification for Less than Maximum Competition

N/A

4. Cost Sharing or Matching

Cost Sharing / Matching Requirement:

No

5. Maintenance of Effort

Maintenance of effort is not required for this program.

D. Application and Submission Information

1. Required Registrations

An organization must be registered at the three following locations before it can submit an application for funding at www.grants.gov.

PLEASE NOTE: Effective April 4, 2022, applicants must have a Unique Entity Identifier

(UEI) at the time of application submission (SF-424, field 8c). The UEI is generated as part of

SAM.gov registration. Current SAM.gov registrants have already been assigned their UEI and can view it in SAM.gov and Grants.gov. Additional information is available on the GSA website, SAM.gov, and Grants.gov- Finding the UEI.

a. Unique Entity Identifier (UEI):

All applicant organizations must obtain a Unique Entity Identifier (UEI) number associated with your organization’s physical location prior to submitting an application. A UEI number is a unique twelve-digit identification number assigned through SAM.gov registration. Some organizations may have multiple UEI numbers.