RFA-DD-19-001 FINAL.pdf

Centers for Disease Control

National Center on Birth Defects and Developmental Disabilities Extramural Research Program

Office

Research Approaches to Improve the Care and Outcomes of People Living with Spina Bifida

RFA-DD-19-001

Application Due Date: 02/13/2019

Research Approaches to Improve the Care and Outcomes of People Living with Spina Bifida

RFA-DD-19-001

TABLE OF CONTENTS

Part 1. Overview Information Key Dates Required Application Instructions Executive Summary

Part 2. Full Text 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 1. Overview Information Participating Organization(s) Centers for Disease Control Components of Participating Organizations National Center on Birth Defects and Developmental Disabilities Extramural Research Program Office (NCBDDD ERPO) National Center on Birth Defects and Developmental Disabilities (NCBDDD) Notice of Funding Opportunity (NOFO) Title Research Approaches to Improve the Care and Outcomes of People Living with Spina Bifida Activity Code U01

Notice of Funding Opportunity Type New Agency Notice of Funding Opportunity Number

RFA-DD-19-001

Assistance Listings (CFDA) Number(s) 93.315 Category of Funding Activity:

Health NOFO Purpose The purpose of this funding opportunity is to identify health care and clinic practices that are associated with the best outcomes for people living with spina bifida (SB) and to communicate and encourage adoption of best practices by SB clinics including providing needed educational/informational resources for the SB patient and provider communities, health care professionals, families, and educators.

Component A: Expand the work of the Spina Bifida Collaborative Care Network (CCN) to identify and encourage rapid adoption of best practices based on available evidence for care for persons with SB.

Component B: The National Spina Bifida Patient Registry (NSBPR) will collect and improve data quality of longitudinal data on children and adults with SB in order to identify health care and clinic practices that are associated with the best outcomes for people living with SB.

Component C: Implement and evaluate the Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol). This iterative protocol aims to manage the urinary system in infants and young children so that their initial normal kidney function is preserved as much as possible.

Key Dates Publication Date: To receive notification of any changes to RFA-DD-19-001, return to the synopsis page of this announcement at www.grants.gov and click on the https://www.grants.gov

"Send Me Change Notification Emails" link. An email address is needed for this service.

Letter of Intent Due Date: 01/14/2019

Application Due Date: 02/13/2019

On-time submission requires that electronic applications be error-free and made available to CDC for processing from the NIH eRA system on or before the deadline date. Applications must be submitted to and validated successfully by Grants.gov no later than 5:00 PM U.S.

Eastern Time. Applications must be submitted using the Application Submission System & Interface for Submission Tracking (ASSIST) module which is a web-based service used for the preparation and submission of grant applications to CDC through Grants.gov. ASSIST provides the ability for applicants to prepare their applications online, and offers the applicant additional capabilities including the ability to preview the application image, validate the application against required business rules, and prepopulate data from an applicant organization's records, therefore identifying issues earlier in the application submission process.

Note: HHS/CDC grant submission procedures do not provide a grace period beyond the application due date time 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).

Scientific Merit Review: 04/10/2019

Secondary Review: 05/20/2019

Estimated Start Date: 09/01/2019

Expiration Date: 02/14/2019 Due Dates for E.O. 12372: Executive Order 12372 does not apply to this program.

Required Application Instructions

**ELECTRONIC APPLICATION SUBMISSION VIA ASSIST IS PREFERRED**

It is recommended that applicants use ASSIST for the electronic preparation and submission of applications through Grants.gov to CDC. ASSIST is an alternative method to prepare and submit applications, and provides many features to facilitate the application submission process which improves data quality (e.g., pre-population of organization data, pre-submission validation of business rules, and preview of the application image used for review). Use of the Grants.gov downloadable Adobe application packages and submission process will still be supported.

It is critical that applicants follow the instructions in the SF 424 (R&R) Application Guide except where instructed to do otherwise in this NOFO. Conformance to all requirements (both http://grants.nih.gov/grants/funding/424/SF424_RR_Guide_General_VerC.pdf in the Application Guide and the NOFO) 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.

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 (See Part 2, Section IV, 7. Page Limitations).

Executive Summary Purpose. The purpose of this funding opportunity is to identify health care and clinic practices that are associated with the best outcomes for people living with spina bifida (SB) and to communicate and encourage adoption of best practices by SB clinics including providing needed educational/informational resources for the SB patient and provider communities, health care professionals, families, and educators.

Mechanism of Support. Cooperative Agreement Funds Available and Anticipated Number of Awards.

Component A: It is anticipated that one award will be made to a single recipient for up to $600,000 in FY 2019.

o Component B: It is anticipated that up to fourteen awards will be made to up to fourteen recipients for up to $68,000 each in FY 2019.

o Component C: It is anticipated that up to nine awards will be made to up to nine recipients for up to $20,500 each in FY 2019.

Awards issued under this NOFO are contingent upon availability of funds and a sufficient number of meritorious applications. Because the nature and scope of the proposed research will vary from application to application, it is also anticipated that the size and duration of each award may also vary. The total amount awarded and the number of awards will depend upon the number, quality, duration and cost of the applications received.

Budget and Project Period. The estimated total funding for all components (direct and indirect) for the first budget period, 9/1/2019 - 8/31/2020, will be $1,736,5000. The estimated total funding for all components (direct and indirect) for the entire Project Period, 9/1/2019 - 8/31/2024, will be $10,025,000.

Application Research Strategy Length: Page limits for the Research Strategy are clearly specified in Section IV. Application and Submission Information of this announcement.

Eligible Institutions/Organizations. Institutions/organizations listed in Section III, 1.A.

are eligible to apply.

Eligible Project Directors/Principal Investigators (PDs/PIs). Individuals with the skills, knowledge, and resources necessary to carry out the proposed research are invited http://www.cdc.gov/od/pgo/funding/grants/additional_req.shtm#SectionIV http://era.nih.gov/commons/index.cfm#SectionIII1A to work with their institution/organization to develop an application for support. NOTE:

CDC does not make awards to individuals directly. Individuals from underrepresented racial and ethnic groups as well as individuals with disabilities are always encouraged to apply.

Number of PDs/PIs. Applications may include more than one PI; however, the first PI listed on the application will be the “contact PI” for all correspondence. Additional PIs are permitted, but would be referred to as Co-PIs.

Number of Applications.Only one application per institution (normally identified by having a unique DUNS number) is allowed for each component.

Application Type. New Special Date(s). CDC will conduct one conference call for prospective applicants on

December 13, 2018 from 2:00 pm - 3:30 pm EST. The conference call number is 1-855- 644-0229 and Conference ID: 5069266. This session will provide information about the NOFO and will answer questions pertinent to preparing applications in response to this

NOFO.

Application Materials. See Section IV.1 for application materials. Please note that Form D is to be used when downloading the application package. http://grants.nih.gov/grants /how-to-apply-application-guide/forms-d/general-forms-d.pdf%20

Hearing Impaired. Telecommunications for the hearing impaired are available at:

TTY: 1-888-232-6348.

Part 2. Full Text Section I. Funding Opportunity Description

Statutory Authority This program is authorized under Sections 301 and 317C of the Public Health Service Act, (42 USC Sections 241 and 247b-4), as amended.

1. Background and Purpose Background An estimated 160,000 people in the United States are currently living with spina bifida (SB), making it the most common permanently disabling birth defect. The care of persons with SB is complex, involving different organ systems and, correspondingly, different clinical specialists.

Many of those affected by SB receive specialty care in one of the estimated 96 SB multidisciplinary clinics in the U.S.

In 2005 and 2006, a number of organizations working with the Spina Bifida Association (SBA) undertook an assessment of current care in programs/clinics that serve people with SB. The objectives of the assessment were to:

obtain a clearer overall description of SB clinics in the U.S., gain understanding of clinic operations and services, and elicit information related to strengthening the quality of care provided in clinics.

This assessment identified variation across clinic programs and notable gaps between what http://grants.nih.gov/grants/ElectronicReceipt/submit_app.htm#SectionIV1 http://grants.nih.gov/grants/how-to-apply-application-guide/forms-d/general-forms-d.pdf%20 http://grants.nih.gov/grants/how-to-apply-application-guide/forms-d/general-forms-d.pdf%20 programs considered essential services and the services that they were actually delivering. As a result of this assessment, the Professional Advisory Council of the SBA endorsed the concept of building a Collaborative Care Network (CCN) and the establishment of the National Spina Bifida Patient Registry so that care would become more uniform across clinics and best practices would be identified and disseminated. In 2008, the National Spina Bifida Patient Registry (NSBPR) was established as a cooperative agreement with nine SB clinics across the US. Since that time, CDC has continued cooperative agreements with clinics to collect, analyze and publish findings from longitudinal data, including clinical interventions and health outcomes for persons with some of the more severe types of SB including myelomeningocele.

As of July 2018, 14 funded clinics and 10 self-funded clinics participate in the NSBPR, and 15 manuscripts [Thibadeau (2013), Sawin (2015), Schechter (2015), Kim (2015), Dicianno (2015), Freeman (2017), Routh (2017), Routh (2018), Routh (2016), Liu (2018), Wiener (2017), Wiener (2018), Kim (2018), Kim (2018), Alibi (2018)] have been published using NSBPR data. Nearly 9,000 persons with spina bifida are currently enrolled in the NSBPR, with approximately 20% recording two visits and over 50% recording 3-8 visits. The project has been overseen by a Coordinating Committee (CC) comprised of Principal Investigators at the participating clinics, and CDC staff and scientists; data use is overseen by the Committee on Science and Publications (CSP) comprised of investigators at the participating clinics and CDC staff and scientists. (CSP Guidance available upon request ) In 2014, nine NSBPR clinics were funded to establish and implement the “Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida” (UMPIRE Protocol). The UMPIRE Protocol was established to manage and evaluate the urinary system in infants and young children so as to preserve initial normal kidney function.

In 2014, the CCN was established. Its structure includes a network of both NSBPR and non- NSBPR SB clinics (healthcare providers), SBA chapters (persons with SB and families), and other care providers for persons with SB, and it is governed by a board of directors that includes representatives from these entities as well as SBA staff and medical directors. The CCN provides input for NSBPR research priorities and supports the adoption of best practices identified via the NSBPR and the UMPIRE Protocol. As part of this mission, the CCN recently revised and published the Spina Bifida Health Care Guidelines for adults and children with SB

[SBA, 2018].

Future Needs Over the years, the NSBPR and the UMPIRE protocol have made strides towards identifying best practices for people living with spina bifida, but much work is still needed. Most of the NSBPR publications so far have been based on descriptive data. While these descriptions of what to expect have provided important contributions, longitudinal data are necessary to identify healthcare practices that are associated with the best outcomes, and more years of NSBPR and UMPIRE protocol data collection are necessary to compile these longitudinal data.

Another need is to explore/describe the clinic attributes not currently collected by the NSBPR that are associated with good health outcomes.

Although the CCN has established mechanisms for connecting persons with spina bifida and clinicians caring for persons with spina bifida to emerging information about SB care, communication of new findings and the guidance of research priorities are ongoing needs. The CCN is a necessary conduit for continued connection to the communities utilizing updated information and providing feedback to the NSBPR and UMPIRE Protocol researchers.

In summary, the foundation has been laid and much good work has been produced from NSBPR, the UMPIRE protocol, and the CCN, but more longitudinal data and communication of research findings are needed to identify and share the best ways to care for persons with spina bifida.

Purpose The purpose of this funding opportunity is to identify health care and clinic practices that are associated with the best outcomes for people living with spina bifida (SB) and to communicate and encourage adoption of best practices by SB clinics including providing needed educational/informational resources for the SB patient and provider communities, health care professionals, families, and educators.

Healthy People 2020 and other National Strategic Priorities The National Center on Birth Defects and Developmental Disabilities (NCBDDD) of CDC, within HHS, is committed to achieving the health promotion and disease prevention objectives of "Healthy People 2020" and to measuring program performance as stipulated by the Government Performance and Review Act (GPRA). This NOFO addresses “Healthy People 2020” priority area(s):

1. Improve access to comprehensive, quality health care services.

o AHS-6: Reduce the proportion of individuals who are unable to obtain or delay in obtaining necessary medical care, dental care, or prescription medicines

2. Promote the health and well-being of people with disabilities.

o DH-4: (Developmental) Reduce the proportion of people with disabilities who report delays in receiving primary and periodic preventive care due to specific barriers

Public Health Impact Individuals affected by SB can expect to live full lives both in terms of quality and duration.

This NOFO is intended to provide health care professionals with a big-picture view of the health status of patients with SB aggregated across clinics, to support them in making informed decisions about implementing available interventions that yield the best outcomes in the many areas of health and living affected by SB. When the NSBPR began in 2008, there was no organized communication among the health care professionals involved with SB clinics in the US. The NSBPR and CCN now connect many of the SB clinics throughout the US, and the NSBPR currently collects information on SB care and outcomes at 24 clinics. The projects proposed in this NOFO will continue to identify health care and clinic practices that are associated with the best outcomes for people living with spina bifida (SB) and to communicate and encourage adoption of best practices by SB clinics including providing needed educational/informational resources for the SB patient and provider communities, health care professionals, families, and educators.

Relevant Work CDC has a long history of support for research about SB, rare disorders and health outcomes;

over this time, approaches and tools have been developed and refined that have benefited SB research efforts. SB clinic researchers worked together with technical support form CDC to develop the following tools:

Forms for clinics to use when abstracting SB patient data (Available upon request) Committee of Science and Publication Guidelines for Data Use (Available upon request) A five year protocol “Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida” (UMPIRE Protocol) (Available upon request) Forms for abstracting SB patient data about urologic health (Available upon request)

In addition, one main focus of CDC’s SB work has been disseminating research via peer-reviewed publications. To date, 15 manuscripts [Thibadeau (2013), Sawin (2015), Schechter (2015), Kim (2015), Dicianno (2015), Freeman (2017), Routh (2017), Routh (2018), Routh (2016), Liu (2018), Wiener (2017), Wiener (2018), Kim (2018), Kim (2018), Alibi (2018)] have been published in peer reviewed journals using and summarizing NSBPR and UMPIRE Protocol data.

2. Approach There are three components to this NOFO with separate, but synergistic approaches, each contributing uniquely to the overall purpose to identify health care and clinic practices that are associated with the best outcomes for people living with spina bifida (SB), and to communicate and encourage adoption of best practices by SB clinics including providing needed educational/informational resources for the SB patient and provider communities:

Component A. Collaborative Care Network (CCN) The CCN approach is to bridge the gap between SB research and practice through the following: enhance and expand communication to the target populations and promote the adoption of best practices; identify, communicate and support exploration of research gaps/priorities by collecting and providing NSBPR researchers with information about SB patient and clinician research priorities and research gaps – and supporting exploration efforts when gaps are identified (e.g., little knowledge on sudden and unexplained deaths in patients with SB); and by determining and promoting attributes of NSBPR clinics that are associated with the best patient outcomes. Applicants for Component A should be able to assess crosscutting SB research needs and priorities that span NSBPR and non-NSBPR clinics that serve SB patients, and broadly promote the adoption of best practices.

Component B. National Spina Bifida Patient Registry (NSBPR) The approach of the NSBPR is to collect high-quality longitudinal data on children and adults with SB who receive care in specialized SB clinics. The longitudinal aspect of data collection is critical for the analytic value of the NSBPR. In addition, initial data analyses have indicated that there are some opportunities for data quality checks and improvement on data collection. This NOFO provides a new focus on expanding longitudinal data collection and incorporating data quality measures in standard activities. Eligible clinics are those with a census of 200 eligible patients, committed to approaching all diagnosed patients for inclusion in the NSBPR and to developing proposals or a research plan in one of the areas for which data are being collected.

Applicants for Component B can be new clinics or clinics that have previously participated in the NSBPR.

Component C: The “Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida” (UMPIRE Protocol) The approach of this component is to implement and evaluate the UMPIRE Protocol (UMPIRE Protocol available upon request). This iterative protocol aims to manage the urinary system in infants and young children with myelomeningocele so that their initial normal kidney function is preserved as much as possible as they age. As with component B, the longitudinal aspect of data collection is critical in that many of the important outcomes will be measurable in persons who have been enrolled in the protocol for more than five years.

Objectives/Outcomes The three components will employ individual but connected objectives to achieve outcomes that utilize the above approaches.

Component A. Collaborative Care Network (CCN) Objectives/Outcomes:

Enhance Communication to Target Populations and Promote the Adoption of Best Practices among Health Care Providers

Maintain and update the CCN Communication list that includes SB healthcare clinics in the US, independent SB care providers, SBA chapters, and families/adults with SB

Create and implement a plan for promoting and disseminating SB health care guidance [SBA, 2018] for children and adults with SB, health care providers in SB clinics, and providers outside of the clinic structure; plan will include updating educational materials to reflect updated guidance

Widely disseminate new findings from NSBPR data Create an editorial/visual brand for the Network that reflects its public health purpose to bridge the gap between SB research and practice; this effort should include communication elements and potentially an evaluation of the current naming convention

Identify, Promote and Support Exploration of Research Gaps/Priorities

Identify and promote research priorities for the NSBPR according to the needs of the affected population and, the gaps identified in the healthcare guidelines [SBA, 2018]

Support continued exploration when knowledge gaps are identified, including supporting efforts to learn more about recently reported cases of sudden and unexplained death in patients with SB

Use Outcomes Data to Identify Best Clinic Practices

Develop and implement a plan to identify attributes of SB NSBPR clinics with the best outcomes at a given patient age (e.g. bowel and bladder continence, no skin breakdown, community ambulation as identified by analyses of NSBPR data) o The data collection plan will collect systems-level and qualitative information to complement the quantitative NSBPR data. The plan and tools should be developed in consultation with systems-level and qualitative research experts and should consider the US Cystic Fibrosis Foundation’s clinical practice benchmarking project for research design and tool examples [Boyle, 2014] o The investigator is responsible for defining the scope, methods, and design for the data collection, but the awardee may request assistance from CDC o The plan to identify attributes of best performing clinics is encouraged, but not required, to consider an exploration of the use of care coordination in the clinic o Promote the adoption of clinical characteristics associated with the best outcomes

Component B. National Spina Bifida Patient Registry (NSBPR) Collect longitudinal data on persons with SB Objectives/Outcomes:

Collect longitudinal data on persons with SB

For currently enrolled NSBPR participants, collect data at annual visits according to protocol and submit data via the SB Electronic Medical Record (EMR)

Approach all eligible participants (NSBPR eligible SB diagnoses include myelomeningocele, meningocele, lipomyelomeningocele, fatty/thickened filum, split cord malformation, and terminal myelocystocele), and, for those whose consent is obtained, collect data according to protocol, and submit data via the SB Electronic Medical Record (EMR). Note: previous NSBPR data collection forms are available upon request

Each year, collect minimal non-identifiable data (e.g. sex, race/ethnicity, insurance status) on eligible patients who did not enroll in the NSBPR to test for potential differences with the eligible patients who enrolled

Participate in quarterly NSBPR Coordinating Committee (CC) meetings via phone and up to two in-person meeting yearly, and other meetings and committees as appropriate.

Conduct data quality checks and report findings from these checks per guidance from the NSBPR CC and the NSBPR Committee for Science and Publication

Data coordinator participates in abstractor training (no travel) per guidance from the NSBPR CC and Committee for Science and Publications

During the 5-year study period, conduct at least one data linkage with the National Death Index to identify deaths among the participants who are lost to follow up, per guidance from the NSBPR CC and with technical assistance from CDC

Participate in analysis of NSBPR data, sharing data via presentations and publications, evaluating the work of the project, and/or participating with other awardees on committees and work groups

Clinics can participate in other data collection efforts including:

Examining the uptake of the soon-to-be-released SB care guidance by using the NSBPR to evaluate practice and potentially outcomes pre- and post-guidance release o Identify a small number of clinical care metrics in the NSBPR that can be used to measure quality of care provided to SB patients Collecting and entering additional information on NSBPR participants who are lost to follow up Linking existing medical administrative datasets and NSBPR to enhance information on

NSBPR participants Collecting additional information from clinics about facility, personnel and specialist costs, for cost and cost-effectiveness analysis of operating a spina bifida clinic Participating and reporting outcomes in SB Electronic Medical Record (EMR) of the intervention to prevent skin breakdown Participating in optional modules for additional data collection and submitting via the

SB Electronic Medical Record (EMR) the following and other topics as identified (optional modules may become part of SB EMR as determined by CDC in collaboration with the NSBPR CC and NSBPR Committee for Science and Publication):

o Sleep disordered breathing o Quality of life o Neurosurgical repair of myelomeningocele o Developmental and learning assessments for participants o Other topics as identified

Component C. Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol) Objectives/Outcomes:

Implement and Evaluate the UMPIRE Protocol

For currently enrolled UMPIRE Protocol participants, collect data according to protocol and submit data via the SB Electronic Medical Record (EMR)

Create a plan for approaching all infants with myelomeningocele SB who are 3 months old or younger and will get their care at a participating site for at least 6 months and enrolling those for whom parental consent is obtained in the Urologic Management to Preserve Renal Function Protocol for Young Children with Spina Bifida (developed in the previous funding cycle)

Each year, collect minimal non-identifiable data (e.g. sex, race/ethnicity, insurance status) on eligible patients who did not enroll in the UMPIRE Protocol to test for potential differences with the eligible patients who enrolled

Clinics will implement the UMPIRE Protocol among UMPIRE Protocol Participants, and if the participant clinic deviates from the protocol (which is allowed), submit a description of the deviation and the reason for it to through the SB EMR.

UMPIRE Protocol PIs will participate in quarterly project calls to discuss analysis and revision of the current protocol for newborn to age 5 years and extending the protocol for years 6-10 years for returning participants (when applicable)

UMPIRE Protocol PIs will attend at least one in-person meeting annually PIs will provide input on how UMPIRE Protocol data are to be analyzed and what the analysis results mean to inform evaluation and potential revision of protocol Participate in data quality checks per guidance from the UMPIRE Protocol CC

Support standardization of documenting urology test results

Continue to promote consistency in measures across sites and in the interpretation of urology test results and classification of renal function

Clinics can participate in other data collection efforts including:

Link existing medical administrative datasets to enhance information on UMPIRE Protocol participants

Complete cost-effectiveness analysis of UMPIRE Protocol administration

Target Population Component A. Collaborative Care Network (CCN) The target population for the CCN is SB providers, clinics, and SBA chapters in the US, as well as children and adults who are living with SB and their families.

Component B. National Spina Bifida Patient Registry (NSBPR) The target population for the NSBPR is children and adults with an eligible SB diagnosis who attend any of the SB clinics participating in the NSBPR.

Component C. Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol) The target population for this component is infants/young children with myelomeningocele who attend an SB clinic participating in the implementation of the protocol.

Collaboration/Partnerships The applicant is expected to maintain existing partnerships and establish new partnerships, as needed, to accomplish the goals of the NOFO. These partnerships include clinicians and clinician groups who treat patients with SB and seek to disseminate information relevant to individuals with SB and their families. Applicants who are awarded funds under this cooperative agreement are expected to collaborate with other awardees and CDC under this award.

Evaluation/Performance Measurement Component A. Collaborative Care Network (CCN) The applicant is is expected to collaborate with CDC to develop and implement an evaluation plan throughout the duration of the cooperative agreement. This plan will evaluate the main project elements:

Enhance communication to target populations and promote the adoption of best practices among health care providers

Identify, communicate and support exploration of research gaps/priorities

Use outcomes data to identify best clinic practices

The applicant should state in their application that they would participate as active members of the committee in planning the evaluation component and in all evaluation activities specified in the plan.

Component B. National Spina Bifida Patient Registry (NSBPR) The applicant is expected to collaborate with the NSBPR CC and CDC in data quality evaluation throughout the project period. This collaboration includes participating in data quality activities determined by the NSBPR CC and CSP in collaboration with CDC, which requires (1) responding to monthly data quality queries; (2) participating in periodic assessments of data quality as determined by project PIs and CDC; and (3) participating in data quality improvement training exercises. The applicant should state in their application that they will actively participate in planning and implementing the evaluation of data quality and in all activities specified in the plan.

Component C. Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol) The applicant is expected to collaborate with the UMPIRE Protocol CC and CDC in data quality evaluation throughout the duration of the cooperative agreement. This collaboration includes participating in data quality improvement exercises as determined by the UMPIRE Protocol PIs and CDC, which requires (1) responding to quarterly data quality queries and updating database accordingly; and 2) participating in periodic assessments of data quality as determined by project PIs and CDC. The applicant should state in their application that they would actively participate with CDC in planning the evaluation and conducting all activities specified in the plan.

Translation Plan Component A. Collaborative Care Network (CCN) At a minimum, summaries of NSBPR findings should be shared with the sources of information; i.e., SB clinics, SBA chapters, adults with SB and relevant health care professionals, using the World Wide Web, social media, and meetings of participating partner organizations. This sharing is expected to include findings published from the other two components (the NSBPR and the UMPIRE Protocol) as well as healthcare guidelines [SBA, 2018]

Component B. National Spina Bifida Patient Registry (NSBPR) Findings should be shared with study subjects and specialized researchers via professional meetings, publications in peer-reviewed journals, and with SB clinical centers directly via the

SB CCN.

Component C. Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol) Findings should be shared with SB clinics via the CCN, the corresponding professional societies, especially the Society of Pediatric Urology; and published in peer-reviewed journals.

OMB/PRA is not expected to apply; If an award under this NOFO changes the aim or scope that was previously reviewed, an updated Project Description form and OMB-PRA determination will be resubmitted.

References:

Guidelines for the Care of People with Spina Bifida (SBA), October 2018: http://spina bifidaassociation.org/wp-content/uploads/2018/10/GFPWSB-Update-102518.pdf

Thibadeau, J. K., Ward, E. A., Soe, M. M., Liu, T., Swanson, M., Sawin, K. J., Schechter, M. S. (2013). Testing the Feasibility of a National Spina Bifida Patient Registry. Birth defects research. Part A, Clinical and molecular teratology, 97(1), 36-

41. doi:10.1002/bdra.23094

Sawin, K. J., Liu, T., Ward, E., Thibadeau, J., Schechter, M. S., Soe, M. M., & Walker, W. (2015). The National Spina Bifida Patient Registry: Profile of a Large Cohort of Participants from the First 10 Clinics. The Journal of pediatrics, 166(2), 444-450.e441.

doi:10.1016/j.jpeds.2014.09.039

Schechter, M. S., Liu, T., Soe, M., Swanson, M., Ward, E., & Thibadeau, J. (2015).

Sociodemographic Attributes and Spina Bifida Outcomes. Pediatrics, 135(4), e957-e964. doi:10.1542/peds.2014-25762014.

Kim, S., Ward, E., Dicianno, B. E., Clayton, G. H., Sawin, K. J., Beierwaltes, P.

National Spina Bifida Patient, R. (2015). Factors Associated With Pressure Ulcers in Individuals With Spina Bifida. Archives of physical medicine and rehabilitation, 96(8), 1435-1441.e1431. doi:10.1016/j.apmr.2015.02.029

Dicianno, B. E., Karmarkar, A., Houtrow, A., Crytzer, T. M., Cushanick, K. M., McCoy, A., Collins, D. M. (2015). Factors Associated with Mobility Outcomes in a National Spina Bifida Patient Registry. American journal of physical medicine & rehabilitation, 94(12), 1015-1025. doi:10.1097/PHM.0000000000000404

Freeman, K., Castillo, H., Castillo, J., Liu, T., Schechter, M., Wiener, J., Brei, T. (2017).

Variation in bowel and bladder continence across US spina bifida programs: A descriptive study. Journal of Pediatric Rehabilitation Medicine, 10(3-4), 231-241.

Routh, J., Joseph, D., Liu, T., Schechter, M., Thibadeau, J., Chad Wallis, M.,Wiener, J.

(2017). Variation in surgical management of neurogenic bowel among centers participating in National Spina Bifida Patient Registry. Journal of Pediatric Rehabilitation Medicine, 10(3-4), 303-312.

Routh, J., Joseph, D., Liu, T., Schechter, M., Thibadeau, J., Wallis, M. C., Wiener, J.

(2018). Bladder Reconstruction Rates Differ among Centers Participating in National Spina Bifida Patient Registry. The Journal of urology, 199(1), 268-273.

Routh, J. C., Cheng, E. Y., Austin, J. C., Baum, M. A., Gargollo, P. C., Grady, R. W., Joseph, D. B. (2016). Design and Methodological Considerations of the Centers for http://spinabifidaassociation.org/wp-content/uploads/2018/10/GFPWSB-Update-102518.pdf http://spinabifidaassociation.org/wp-content/uploads/2018/10/GFPWSB-Update-102518.pdf

Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida. The Journal of urology, 196(6), 1728-1734.

doi:10.1016/j.juro.2016.07.081

Liu, T., Ouyang, L., Thibadeau, J., Wiener, J. S., Routh, J. C., Castillo, H., Valdez, R.

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Section II. Award Information Funding Instrument Type: 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:

New - An application that is submitted for funding for the first time. Includes multiple submission attempts within the same round.

Estimated Total Funding: $1,736,500 The estimated total funding for all components (direct and indirect costs) for the first budget period, 9/1/2019 - 8/31/2020, is $1,736,500.

Component A: The estimated total funding for all components (direct and indirect costs) for the first budget period is up to $600,000 in FY 2019. Award Floor amount is $0 and Award Ceiling amount is $800,000.

Component B: The estimated total funding for all components (direct and indirect costs) for the first budget period is up to $68,000 each in FY 2019. Award Floor amount is $0 and Award Ceiling amount is $70,000.

Component C: The estimated total funding for all components (direct and indirect costs) for the first budget period is up to $20,500 each in FY 2019. Award Floor amount is $0 and Award Ceiling amount is $25,000.

The estimated total funding for all components (direct and indirect) for the entire project period, 9/1/2019 - 8/31/2024, is $10,025,000.

Anticipated Number of Awards: 24 Component A: It is anticipated that one award will be made to a single recipient.

Component B: It is anticipated that up to fourteen awards will be made to up to fourteen recipients.

Component C: It is anticipated that up to nine awards will be made to up to nine recipients.

Awards issued under this NOFO are contingent upon the availability of funds and a sufficient number of meritorious applications.

Because the nature and scope of the proposed research will vary from application to application, it is also anticipated that the size and duration of each award may also vary. The total amount awarded and the number of awards will depend upon the number, quality, duration and cost of the applications that qualify.

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

Award Ceiling: $800,000 Per Budget Period Award Floor: $0 Per Budget Period Total Period of Performance Length: 5 year(s)

Throughout the Period of Performance, CDC's commitment to continuation of awards will depend on the availability of funds, evidence of satisfactory progress by the recipient (as documented in required reports), and CDC’s 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 (http://www.hhs.gov/ sites/default/files/grants/grants/policies-regulations/hhsgps107.pdf) will apply to the applications submitted and awards made in response to this NOFO.

Section III. Eligibility Information

1. Eligible Applicants Eligibility Category: State governments

County governments City or township governments Special district governments Independent school districts Public and State controlled institutions of higher education Native American tribal governments (Federally recognized) Public housing authorities/Indian housing authorities Native American tribal organizations (other than Federally recognized tribal governments) Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Nonprofits without 501(c)(3) status with the IRS, other than institutions of higher education Private institutions of higher education For profit organizations other than small businesses Small businesses 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:

2. Foreign Organizations Foreign Organizations are not eligible to apply.

Foreign components of U.S. Organizations are not eligible to apply.

For this announcement, applicants may not include collaborators or consultants from foreign http://www.hhs.gov/sites/default/files/grants/grants/policies-regulations/hhsgps107.pdf institutions. All applicable federal laws and policies apply.

3. Special Eligibility Requirements

For an applicant to be considered they must be responsive to the information here.

Component A. Collaborative Care Network (CCN)

Applicant must demonstrate existing relationship and collaboration with SBA chapters and clinics across the US as shown by activities such as joint meetings, collaboration on projects, committee participation

Component B. National Spina Bifida Patient Registry (NSBPR)

Applicant must have documented evidence of treatment of a minimum of 200 patients in 2017 or 2018 with NSBPR eligible diagnoses (myelomeningocele, meningocele, lipomyelomeningocele, fatty/thickened filum, split cord malformation, and terminal myelocystocele) as evidenced by the provision of a 2017 or 2018 patient census for the SB clinic that includes age, gender, race/ethnicity, and diagnosis o Note: more than one clinic (e.g. smaller clinics, pediatric/adult clinics) can join together as one NOFO applicant

Applicant must demonstrate access to patients with SB and commitment to the collection of the data elements in the NSBPR as demonstrated by a letter of commitment from a representative of the participating clinic(s) to participate as described

Component C. Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol)

Applicant must see at least four infants up to 3 months old with spina bifida annually in the clinic as demonstrated by the provision of a clinic census that lists infants up to 3 months old treated at birth in the past 3 years, averaging 4 each year with a diagnosis of myelomeningocele

Applicant must include a pediatric urologist as part of the research team

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

4. Justification for Less than Maximum Competition N/A

5. Responsiveness If your application is incomplete or non-responsive to these requirements, it will not enter into the review process. Applications will be deemed unresponsive if the proposed budget is greater than the ceiling amounts of this NOFO.

Component A applicants must provide the following:

Documentation of existing relationship with five or more SBA chapters and clinics across the US as evidenced by meeting minutes, letters of support, and reports of collaborative projects coordinated by the applicant

Evidence of relationships with five or more health care professionals across the US that represent SB clinics and SB practices demonstrated by meeting minutes from an established functioning committee and/or letters of support from committee participants.

Component B applicants must provide the following:

Documentation of number of patients by diagnosis in 2017 or 2018 indicating that at least 200 patients attended the clinic with one of the following diagnoses:

myelomeningocele, meningocele, lipomyelomeningocele, fatty/thickened filum, split cord malformation, or terminal myelocystocele.

o Note: more than one clinic (e.g. smaller clinics, pediatric/adult clinics) can join together as one NOFO applicant

Documentation of access to patients with SB and to the collection of the data elements in the NSBPR as demonstrated by letters of commitment from a representative of the participating clinic(s) to participate as described

Component C applicants must provide the following:

Evidence of treating at least four infants up to three months old with spina bifida annually in the clinic as demonstrated by the provision of a clinic census that lists infants up to three months old treated at birth in the past 3 years, averaging 4 each year with a diagnosis of myelomeningocele

Applicant must include a pediatric urologist as part of the research team

For an applicant to be considered they must be responsive to the information here. If your application is incomplete or non-responsive to the special eligibility requirements listed in this section, it will not enter into the review process.

Evidence of these Special Eligibility Requirements should be placed in Appendix A of the application.

6. 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.

(Foreign entities only): Special Instructions for acquiring a Commercial and Governmental Entity (NCAGE) Code: https:// eportal.nspa.nato. int/ AC135Public/ https://eportal.nspa.nato.int/AC135Public/Docs/US%20Instructions%20for%20NSPA%20NCAGE.pdf

Docs/ US%20Instructions%2 0for%20NSPA%20NCAGE.pdf System for Award Management (SAM) – must maintain current registration in SAM

(the replacement system for the Central Contractor Registration) to be renewed annually, https://www.sam.gov/portal/SAM/.

Grants.gov eRA Commons

All applicant organizations must register with Grants.gov. Please visit www.Grants.gov at least 30 days prior to submitting your application to familiarize yourself with the registration and submission processes. The “one-time” registration process will take three to five days to complete. However, it is best to start the registration process at least two weeks prior to application submission.

All Program Directors/Principal Investigators (PD/PIs) must also work with their institutional officials to register with the eRA Commons or ensure their existing Principle Investigator (PD/PI) 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 eRA Commons registration process at least four (4) weeks prior to the application due date. ASSIST requires that applicant users have active eRA Commons account in order to prepare an application. It also requires that the applicant organization's Signing Official have an active eRA Commons Signing Official account in order to initiate the submission process.