CDC-RFA-DD19-1903.pdf

Centers for Disease Control

National Center on Birth Defects and Developmental Disabilities

Characterizing the Complications Associated with Therapeutic Blood Transfusions for

Hemoglobinopathies

CDC-RFA-DD19-1903

Application Due Date: 05/03/2019

Characterizing the Complications Associated with Therapeutic Blood Transfusions for

Hemoglobinopathies

CDC-RFA-DD19-1903

TABLE OF CONTENTS

Part I. Overview Information A. Federal Agency Name B. Funding Opportunity Title C. Announcement Type D. Agency Funding Opportunity Number E. Assistance Listings (CFDA) Number F. Dates G. Executive Summary

Part II. Full Text 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 Information Applicants must go to the synopsis page of this announcement at www.grants.gov and click on the "Send Me Change Notifications Emails" link to ensure they receive notifications of any changes to CDC-RFA-DD19-1903. 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) / Agency for Toxic Substances and Disease Registry (ATSDR) B. Notice of Funding Opportunity (NOFO) Title:

Characterizing the Complications Associated with Therapeutic Blood Transfusions for Hemoglobinopathies 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 this purpose, research is defined at https ://www.gpo.gov/fdsys/pkg/CFR-2007-title42-vol1/pdf/CFR-2007-title42-vol1-sec52-2.pdf.

Guidance on how CDC interprets the definition of research in the context of public health can be found at https://www.hhs.gov/ohrp/regulations-and-policy/regulations/45-cfr-46/index.html (See section 45 CFR 46.102(d)).

D. Agency Notice of Funding Opportunity Number:

CDC-RFA-DD19-1903

E. Assistance Listings (CFDA) Number:

93.080 F. Dates:

1. Due Date for Letter of Intent (LOI): 04/05/2019

2. Due Date for Applications: 05/03/2019, 11:59 p.m. U.S. Eastern

Standard Time, at www.grants.gov.

A Letter of Intent is requested by not required.

3. Date for Informational Conference Call:

03/14/19 Scheduled for 2:00 - 3:00 pm, Eastern Standard Time.

Conference Number: 1-877-721-9741 Conference I.D.: 8946896# Questions may be submitted to ibx5@cdc.gov as soon as the NOFO is released.

G. Executive Summary:

1. Summary Paragraph:

This funding opportunity is comprised of two components – thalassemia and sickle cell disease.

All applicants must apply for the Thalassemia Component in order to be considered for the Sickle Cell Disease Component via a single application that clearly specifies the activities within each component. Only those successful applicants under the Thalassemia Component https://www.grants.gov https://www.grants.gov https://www.gpo.gov/fdsys/pkg/CFR-2007-title42-vol1/pdf/CFR-2007-title42-vol1-sec52-2.pdf https://www.gpo.gov/fdsys/pkg/CFR-2007-title42-vol1/pdf/CFR-2007-title42-vol1-sec52-2.pdf https://www.hhs.gov/ohrp/regulations-and-policy/regulations/45-cfr-46/index.html https://www.grants.gov will be eligible for additional Sickle Cell Disease Component funding. Applicants must propose a comprehensive work plan that addresses transfusions and their related complications in alignment with the broad strategies indicated in the NOFO.

Thalassemia Component:

the development and validation of methods for identifying individuals with thalassemia;

the development and dissemination of information for patients, families, blood banks, and health care providers about the interpretation of laboratory results indicating (1) a potential and (2) a confirmed thalassemia diagnosis and management of transfusions for thalassemia; and the collection of biological specimens from individuals with thalassemia.

Sickle Cell Disease Component:

the development and dissemination of information for patients, families, blood banks, and health care providers about the interpretation of laboratory results indicating (1) a potential and (2) a confirmed sickle cell disease diagnosis and management of transfusions for sickle cell disease and the collection of biological specimens from individuals with sickle cell disease.

a. Eligible Applicants: Open Competition

b. NOFO Type: Cooperative Agreement

c. Approximate Number of Awards: 2 The awards may be a mixture of Thalassemia Component only and Thalassemia & Sickle Cell Disease Components. Final number of funded award recipients will be determined by the objective review scoring and available funding.

d. Total Period of Performance Funding: $3,750,000

e. Average One Year Award Amount: $375,000

f. Total Period of Performance Length: 5

g. Estimated Award Date: 09/30/2019

h. Cost Sharing and / or Matching Requirements: N Cost sharing or matching funds are not required for this program. Although no statutory matching requirement for this NOFO exists, leveraging other resources and related ongoing efforts to promote sustainability is strongly encouraged.

Part II. Full Text A. Funding Opportunity Description

Part II. Full Text

1. Background

a. Overview Red blood cell (RBC) transfusions are an important part of medical care for many patients with thalassemia or sickle cell disease. Some patients never need a transfusion, others may have infrequent or intermittent needs, and still others may need chronic transfusions every few weeks. Determining which patients need to receive transfusions, when and how often they need to receive them, and what steps minimize complications from the transfusions may lead to improved health outcomes.

In the U.S., individuals with thalassemia and/or their health care providers may be unaware of their diagnosis until overt clinical signs begin to manifest. The initiation of transfusion may be delayed until complications appear. If the diagnosis is never made, patients continue living without the benefits that transfusions could provide. Iron chelation therapy is indicated for all transfused patients when iron measures indicate concentrations that exceed threshold levels.

Red blood cell alloimmunization occurs when the immune system is exposed to foreign antigens on red blood cells that are different from its own. Frequent blood transfusions may also expose individuals to infectious agents that may be transmitted through blood. By improving the identification of people who have these conditions and may benefit from appropriately administered transfusions, the understanding of treatment for them, and the standardization of information that is available about them, therapeutic transfusions may be administered in ways that maximize their benefits and minimize their adverse effects.

This NOFO aims to improve access to, coordination of, and continuity of health care for individuals with thalassemia or sickle cell disease which will lead to a decreased occurrence of transfusion-related complications, improved quality and increased length of life. These health care improvements will result from a multi-faceted approach that includes (1) identification of patients, families, communities, and providers who will benefit from increased knowledge about therapeutic transfusions and their potential complications, (2) development and dissemination of materials that will increase understanding of the diagnosis and management of these blood disorders, and (3) development of a biospecimen repository from a sentinel population of transfused patients in which to assess new or emerging pathogens.

This NOFO builds upon findings from the work completed as part of CDC-RFA-DD14-1406.

In particular, (1) the difficulty in identifying any thalassemia patient in the U.S. and their health care provider prior to medical complications manifesting, other than those born in states where the condition is a part of the mandatory newborn screening panel, (2) the lack of standardized practices across, and sometimes within, clinical care settings and blood banks for transfusions of patients with thalassemia or sickle cell disease, (3) the need for dissemination of evidence- or consensus-based guidelines about best practices for transfusions, (4) the room for improvement in increasing blood donation from communities most affected by thalassemia or sickle cell disease, and (5) the occurrence of new or emerging pathogens that may arise in the blood supply and pose a serious potential risk to patients with thalassemia or sickle cell disease who receive multiple therapeutic transfusions.

b. Statutory Authorities Public Health Service Act Sections 301(a) and 317C (42 U.S.C. Sections 241, 241(a), 243, and

247b-4), as amended.

c. Healthy People 2020 BDBS-17: Increase the proportion of persons who donate blood.

BDBS-18: Reduce the proportion of persons who develop adverse events resulting from the use of blood and blood products.

https://www.healthypeople.gov/2020/topics-objectives/topic/blood-disorders-and-blood-safety /objectives

d. Other National Public Health Priorities and Strategies N/A

e. Relevant Work This NOFO builds on the accomplishments and outcomes achieved in CDC-RFA-DD14-1406 (https://www.cdc.gov/ncbddd/hemoglobinopathies/blood-transfusions.html).

2. CDC Project Description

a. Approach

Bold indicates period of performance outcome.

Strategies and Activities

Short-term Outcomes Intermediate Outcomes Long-Term Outcomes

Develop and validate a method for identifying previously undiagnosed individuals with thalassemia Develop new processes, or improve upon existing processes, for the dissemination of information pertaining to thalassemia/sickle cell disease Develop a toolkit of educational materials about the interpretation of laboratory results that indicate (1) a

Improved understanding of the prevalence of thalassemia in the United States Increased community awareness of the need for blood donation by donors from communities who are most impacted by thalassemia/sickle cell disease Increased understanding of the interpretation of laboratory results that indicate (1) a potential and (2) a

Improved understanding of the natural history of disease for thalassemia Increased proportion of persons who donate blood Increased standardization of transfusion management for individuals with thalassemia/sickle cell disease Decreased occurrence of transfusion-related complications for

Improved access to, coordination of, and continuity of health care for individuals with thalassemia/sickle cell disease Improved quality of life for individuals with thalassemia/sickle cell disease https://www.healthypeople.gov/2020/topics-objectives/topic/blood-disorders-and-blood-safety/objectives https://www.healthypeople.gov/2020/topics-objectives/topic/blood-disorders-and-blood-safety/objectives https://www.cdc.gov/ncbddd/hemoglobinopathies/blood-transfusions.html potential and (2) a confirmed thalassemia/sickle cell disease diagnosis Develop a toolkit of educational materials about evidence- or consensus-based management of transfusions for thalassemia/sickle cell disease Collect biological specimens from individuals with thalassemia/sickle cell disease confirmed thalassemia/sickle cell disease diagnosis Increased understanding of evidence- or consensus-based guidelines on management of transfusions for thalassemia/sickle cell disease Improved understanding of how infectious agents play a role in blood transfusion complications in thalassemia/sickle cell disease individuals with thalassemia/sickle cell disease

i. Purpose Individuals with thalassemia or sickle cell disease may require therapeutic blood transfusions.

These blood transfusions, in turn, may cause additional complications. This NOFO will support a multifaceted approach towards the development and validation of methods for reducing these complications. The implementation and evaluation of these strategies will lead to a decreased occurrence of transfusion-related complications and improve the quality of life for individuals with thalassemia/sickle cell disease.

ii. Outcomes This NOFO has two components: (1) a Thalassemia Component that is mandatory for all applicants and (2) a Sickle Cell Disease Component that is optional.

The following short-term outcomes will be achieved by the end of the period of performance:

Thalassemia Component:

Improved understanding of the prevalence of thalassemia in the United States, including those individuals who do not currently receive chronic transfusion therapy

Increased community awareness of the need for blood donation by donors from communities who are most impacted by thalassemia

Increased understanding among patients, families, and health care providers of the interpretation of laboratory results (newborn screening, confirmatory diagnosis, etc.) that indicate (1) a potential and (2) a confirmed thalassemia diagnosis and the implications that this result will have on the need for therapeutic transfusion management Increased understanding among patients, families, health care providers, and blood banks of evidence- or consensus-based guidelines on management of transfusions for thalassemia

Improved understanding among patients, families, health care providers, and blood banks of how infectious agents play a role in blood transfusion complications in thalassemia

Sickle Cell Disease Component:

Increased community awareness of the need for blood donation by donors from communities who are most impacted by sickle cell disease

Increased understanding among patients, families, and health care providers of the interpretation of laboratory results (newborn screening, confirmatory diagnosis, etc.) that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis and the implications that this result will have on the need for therapeutic transfusion management

Increased understanding among patients, families, health care providers, and blood banks of evidence- or consensus-based guidelines on management of transfusions for sickle cell disease

Improved understanding among patients, families, health care providers, and blood banks of how infectious agents play a role in blood transfusion complications in sickle cell disease

These short-term outcomes will lead to the following intermediate outcomes by the end of the period of performance:

Thalassemia Component:

Improved understanding of the natural history of disease for thalassemia Increased proportion of persons who donate blood from communities who are most impacted by thalassemia Increased standardization of transfusion management for individuals with thalassemia by health care providers and blood banks

Sickle Cell Disease Component:

Increased proportion of persons who donate blood from communities who are most impacted by sickle cell disease

Increased standardization of transfusion management for individuals with sickle cell disease by health care providers and blood banks

iii. Strategies and Activities The Thalassemia Component and Sickle Cell Disease Component strategies are listed below.

Applicants who choose to apply for both components are encouraged to implement strategies in ways that may benefit both communities, when applicable, so that work on these strategies is mutually reinforcing. Complementary strategies should be addressed in a way that benefits both the thalassemia and sickle cell disease communities.

Thalassemia Component:

Develop and validate a method for identifying previously undiagnosed individuals with thalassemia

Develop new processes, or improve upon existing processes, for the dissemination of information pertaining to thalassemia

Develop a toolkit of educational materials about the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed thalassemia diagnosis

Develop a toolkit of educational materials about evidence- or consensus-based management of transfusions for thalassemia

Collect biological specimens from individuals with thalassemia

Sickle Cell Disease Component:

Develop new processes, or improve upon existing processes, for the dissemination of information pertaining to sickle cell disease

Develop a toolkit of educational materials about the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis

Develop a toolkit of educational materials about evidence- or consensus-based management of transfusions for sickle cell disease

Collect biological specimens from individuals with sickle cell disease

In addition to the above activities, all recipients will be expected to participate in collaborative recipient calls and meetings, site visits by CDC staff at recipient location, as needed, and yearly reverse site visits in Atlanta. All materials developed as part of this project will be share with CDC for use consistent with our federal purposes.

1. Collaborations

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

Applicants are encouraged to collaborate with other related CDC programs or CDC-funded organizations that have a role in achieving the NOFO outcomes. Collaborations may center on shared data; partner organizations; communication approaches/messages; and/or patient identification methodology. Related CDC programs or CDC-funded organizations include, but are not limited to Thalassemia Component:

Organization(s) funded under DD14-1405

Sickle Cell Disease Component:

Organization(s) funded under OT18-1802 or participating in the Sickle Cell Data

Collection (SCDC) program

Letters of support are strongly encouraged when the applicant will be working with one of these organizations. These letters should provide detailed information about the nature of the collaborative effort and how the collaboration will help the applicant achieve the objectives of this NOFO. Letters of support should be saved using the name of the agency/organization providing the letter and uploaded as a PDF to www.grants.gov.

b. With organizations not funded by CDC:

Collaborations with a variety of public and private organizations are encouraged to leverage resources and maximize reach and impact for the strategies selected. Applicants should establish or continue strategic partnerships that support the NOFO goals with professional organizations; not-for-profit organizations; community-based organizations; community advocates and members; and other stakeholders that may have a vested interest in improving the health of individuals with thalassemia or sickle cell disease.

Letters of support are strongly encouraged when the applicant will be working with one or more of these organizations. For each organization named as a collaborator, the letter should provide detailed information about the nature of the collaborative effort and how the collaboration will help the applicant achieve the objectives of this NOFO. Letters of support should be saved using the name of the agency/organization providing the letter and uploaded as a PDF to www.grants.gov.

2. Target Populations The target populations are:

Thalassemia Component:

Individuals with thalassemia who do not currently have a laboratory-confirmed diagnosis

Individuals with thalassemia who receive chronic or intermittent transfusions to manage the complications of their disease or who are likely to receive transfusions at a later date

Families and caregivers of individuals with thalassemia Members of populations most commonly affected by thalassemia Health care providers of individuals with thalassemia Blood bank administrators and transfusion medicine providers

Sickle Cell Disease Component:

Individuals with sickle cell disease who receive chronic or intermittent transfusions to manage the complications of their disease or who are likely to receive transfusions at a later date

Families and caregivers of individuals with sickle cell disease Members of populations most commonly affected by sickle cell disease Health care providers of individuals with sickle cell disease Blood bank administrators and transfusion medicine providers

a. Health Disparities In the United States, both thalassemia and sickle cell disease disproportionately affect individuals of particular ancestry. Moreover, some consider the condition itself, especially sickle cell disease, to be an indicator of a health disparity. This NOFO will address the challenges and health inequities of these two chronic genetic diseases that disproportionately burden certain communities.

iv. Funding Strategy There are two funding components for this funding opportunity. Applicants must apply for the Thalassemia Component. Additionally applicants can choose to apply for the Sickle Cell Disease Component via a single application that clearly identifies the two components to receive additional funds for these sickle cell disease activities.

Applicants will be placed in a rank order list based on their score on the Thalassemia Component. If any of the applicants who are selected to be funded for the Thalassemia Component also submit an application for the Sickle Cell Disease Component, those scoring highest in the Sickle Cell Disease Component will be selected to be funded for both components.

Applicants applying for both the Thalassemia and Sickle Cell Disease Components:

Must submit a single application covering both components Must clearly label all activities as “thalassemia only,” “sickle cell disease only,” or

“combined” in the Work Plan Must provide two separate budgets, one for each component Must provide an additional letter of support from each collaborator named in the application

Each component will be reviewed and scored separately, with a different score assigned to each component.

b. Evaluation and Performance Measurement

i. CDC Evaluation and Performance Measurement Strategy Evaluation and Performance Measurement help demonstrate program accomplishments and determine whether the NOFO strategies and activities have been implemented as expected.

Evaluation and Performance Measurement also tracks if the intended outcomes have been achieved during the period of performance. This information is integral to help CDC keep NOFO recipients on track with implementation, to demonstrate the effectiveness of the NOFO on reaching outcomes, and to suggest ways to improve the program.

Evaluation and Performance Measurement will be approached through assessing process and outcome measures. Throughout the period of performance, CDC will work individually and collectively with recipients to track the implementation of recipient strategies and activities and assess progress in achieving NOFO outcomes. Process measures will inform CDC to what extent has the recipient implemented the activities as intended. Outcome measures will inform

CDC if the recipient achieved the period of performance outcomes.

The evaluation will be guided by the following evaluation questions. Evaluation questions are pulled from the activities, short-, and long-term outcomes. Both the process and outcome evaluations will seek to answer the following overarching evaluation questions:

1. Approach: To what extent have recipients' implementation approaches been successful in achieving the desired outcomes?

2. Effectiveness:

1. To what extent has the NOFO increased understanding of the prevalence of thalassemia?

2. To what extent has implementation of strategies led to increased dissemination of educational information related to the target audiences?

3. What factors or components were associated with effective implementation of strategies?

4. What strategies implemented by recipients can be replicated and/or scaled?

3. Impact: To what extent has the intervention contributed to a measurable change in health, behavior, or environment among target audiences?

In order to answer the evaluation questions above, NOFO recipients will provide quarterly reports on the process measures listed below and annual reports on all other process and outcome measures. These reports will be presented during the all-recipient calls or reverse site visits and submitted electronically immediately following the call/site visit. The data collected by CDC for performance measurement and evaluation are directly related to the implementation of the strategies and/or the desired outcomes indicated in the logic model. The data collected for this NOFO for performance measurement do not include any personally identifiable information. Data being collected are strictly related to the implementation of the NOFO strategies and shall be used for assessing and reporting progress and for other pertinent program improvement actions.

Short-term measures for each strategy and intermediate-term measures reported by all recipients are described in the table below. The table aligns with the logic model and shows the alignment between the overarching focus areas, specific strategies, outcomes and performance measures.

Process Measures – Thalassemia Component

Activity Measure

Develop and validate a method for identifying previously undiagnosed individuals with thalassemia

Quarterly report on the development and validation of the method to identify previously undiagnosed individuals with thalassemia

Develop new processes, or improve upon existing processes, for the dissemination of information pertaining to thalassemia

Quarterly report documenting the development or improvement of a process for the dissemination of information pertaining to thalassemia

Quarterly report on the development of the toolkit of educational materials

# of toolkits delivered to stakeholders [patients, families, health care providers]

Develop a toolkit of educational materials about the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed thalassemia diagnosis

# of people reached by educational materials

Quarterly report on the development of the toolkit of educational materials

# of toolkits delivered to stakeholders [patients, families, health care providers, blood banks]

Develop a toolkit of educational materials about evidence- or consensus-based management of transfusions for thalassemia

# of people reached by educational materials

Quarterly report on the development and implementation of methods for collecting specimens and shipping to CDC

Collect biological specimens from individuals with thalassemia

# of biological specimens collected from individuals with thalassemia and shipped to CDC

Process Measures – Sickle Cell Disease Component

Activity Measure

Develop new processes, or improve upon existing processes, for the dissemination of information pertaining to sickle cell disease

Quarterly report documenting the development or improvement of a process for the dissemination of information pertaining to sickle cell disease

Quarterly report on the development of the toolkit of educational materials

# of toolkits delivered to stakeholders [patients, families, health care providers]

Develop a toolkit of educational materials about the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis

# of people reached by educational materials

Quarterly report on the development of the toolkit of educational materials

# of toolkits delivered to stakeholders [patients, families, health care providers]

Develop a toolkit of educational materials about evidence- or consensus-based management of transfusions for sickle cell disease

# of people reached by educational materials

Collect biological specimens from individuals with sickle cell disease

Quarterly report on the development and implementation of methods for collecting specimens and shipping to CDC

# of biological specimens collected from individuals with sickle cell disease and shipped to CDC

Short Term Outcome Evaluation Measures – Thalassemia Component

Outcome Measure

Improved understanding of the prevalence of thalassemia in the United States, including those individuals who do not currently receive chronic transfusion therapy

[baseline #; XX% increase over baseline] of individuals who are identified with thalassemia that

(1) receive chronic transfusion therapy and (2) do not currently receive chronic transfusion therapy

Increased community awareness of the need for blood donation by donors from communities who are most impacted by thalassemia

[baseline #; XX% increase over baseline] of community members who are aware of the need for blood donation by donors from communities who are most impacted by thalassemia

Increased understanding of the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed thalassemia diagnosis and the implications that this result will have on the need for therapeutic transfusion management by patients, families and caregivers, and health care providers

[baseline #; XX% increase over baseline] of patients who understand the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed thalassemia diagnosis and the implications that this result will have on the need for therapeutic transfusion management

[baseline #; XX% increase over baseline] of family members and/or caregivers who understand the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed thalassemia diagnosis and the implications that this result will have on the need for therapeutic transfusion management

[baseline #; XX% increase over baseline] of health care providers who understand the interpretation of laboratory results that indicate (1) a potential and

(2) a confirmed thalassemia diagnosis and the implications that this result will have on the need for therapeutic transfusion management

[baseline #; XX% increase over baseline] of patients who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for thalassemia

[baseline #; XX% increase over baseline] of family members and/or caregivers who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for thalassemia

Increased understanding of evidence- or consensus-based guidelines on management of transfusions for thalassemia by patients, families, health care providers, and blood banks care providers who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for thalassemia care providers at blood banks who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for thalassemia

Improved understanding of how infectious agents play a role in blood transfusion complications in thalassemia

Biological specimens from a minimum of 25 unique individuals with thalassemia who are chronically or intermittently transfused will be collected and transferred to CDC on an annual basis, along with requested clinical data (minimum of 125 patients during the 5 year period of performance)

Short Term Outcome Evaluation Measures – Sickle Cell Disease Component

Outcome Measure

Increased community awareness of the need for blood donation by donors from communities who are most impacted by sickle cell disease

[baseline #; XX% increase over baseline] of community members who are aware of the need for blood donation by donors from communities who are most impacted by sickle cell disease

[baseline #; XX% increase over baseline] of patients who understand the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis and the implications that this result will have on the need for therapeutic transfusion management

Increased understanding of the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis and the implications that this result will have on the need for therapeutic transfusion management by patients, families and caregivers, and health care providers

[baseline #; XX% increase over baseline] of family members and/or caregivers who understand the interpretation of laboratory results that indicate (1) a potential and (2) a confirmed sickle cell disease diagnosis and the implications that this result will have on the need for therapeutic transfusion management care providers who understand the interpretation of laboratory results that indicate (1) a potential and

(2) a confirmed sickle cell disease diagnosis and the implications that this result will have on the need for therapeutic transfusion management

[baseline #; XX% increase over baseline] of patients who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for sickle cell disease

Increased understanding of evidence- or consensus-based guidelines on management of transfusions for sickle cell disease by patients, families, health care providers, and blood banks

[baseline #; XX% increase over baseline] of family members and/or caregivers who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for sickle cell disease care providers who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for sickle cell disease care providers at blood banks who are aware of and understand evidence- or consensus-based guidelines on management of transfusions for sickle cell disease

Improved understanding of how infectious agents play a role in blood transfusion complications in sickle cell disease

Biological specimens from a minimum of 50 unique individuals with sickle cell disease who are chronically or intermittently transfused will be collected and transferred to CDC on an annual basis, along with requested clinical data (minimum of 250 patients during the 5 year period of performance)

These short-term outcomes and measures above will lead to the following intermediate outcomes by the end of the period of performance:

Intermediate Outcome Evaluation Measures – Thalassemia Component

Outcome Measure

Improved understanding of the natural history of disease for thalassemia

Submission of at least one scientific article per year on new findings regarding the natural history of disease for thalassemia will be submitted for peer-review in years 3, 4, and 5 of the period of performance

Increased number of persons who donate blood from communities who are most impacted by thalassemia

[baseline #; XX% increase over baseline] of persons who donate blood from communities who are most impacted by thalassemia

[baseline #; XX% increase over baseline] of health care providers who adhere to evidence- or consensus-based guidelines on management of transfusions for thalassemia

Increased standardization of transfusion management for individuals with thalassemia by health care providers and blood banks [baseline #; XX% increase over baseline] of blood banks who adhere to evidence- or consensus-based guidelines on management of transfusions for thalassemia

Intermediate Outcome Evaluation Measures – Sickle Cell Disease Component

Outcome Measure

Increased number of persons who donate blood from communities who are most impacted by sickle cell disease

[baseline #; XX% increase over baseline] of persons who donate blood from communities who are most impacted by sickle cell disease

Increased standardization of transfusion management for individuals with sickle cell disease by health care providers and blood banks care providers who adhere to evidence- or consensus-based guidelines on management of transfusions for sickle cell disease [baseline #; XX% increase over baseline] of blood banks who adhere to evidence- or consensus-based guidelines on management of transfusions for sickle cell disease

CDC will work with recipients on operationalizing and further defining each performance measure, and guidance will be provided prior to the first year of reporting. Quarterly reports on the process measures, as listed above, and annual reports on all other process and outcome measures will be provided annually to CDC, and CDC will manage and analyze the data to assess recipient program improvements, respond to broader technical assistance needs, and report to stakeholders.

All evaluation findings produced by the recipients will contribute to: (1) continuous improvement of quality and effectiveness of recipient program strategies, (2) the evidence base of Thalassemia/Sickle Cell Disease, (3) documentation and sharing of lessons learned to support replication and scaling of these program strategies, and/or (4) future funding opportunities supported by CDC.

CDC will review the work plan and evaluation and performance management plan, meet with the recipient virtually during conference calls every month, and host an annual reverse site visit in Atlanta to discuss progress towards the project outcomes.

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.

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)

Plans for updating the Data Management Plan (DMP), if applicable, for accuracy throughout the lifecycle of the project. 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/additionalrequirements/ar-25.html.

Where the applicant chooses to, or is expected to, take on specific evaluation studies, they 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.

CDC will review and approve the recipient's monitoring and evaluation plan to ensure that it is appropriate for the activities to be undertaken as part of the agreement and for compliance with the monitoring and evaluation guidance established by CDC or other guidance otherwise applicable to this cooperative agreement.

c. Organizational Capacity of Recipients to Implement the Approach Applicants must describe their organizational capacity to carry out the strategies included in the Component(s). CDC anticipates that all applicants will be able to demonstrate capacity to carry out the activities outlined over the period of performance. Successful applicants will demonstrate the necessary skills and relevant experience and capacity to successfully implement the strategies and activities listed above. Demonstrated readiness to implement the evidence-based strategies includes the ability of applicants to describe the following:

Evidence that they have the organizational capacity to facilitate the program strategies and meet the period of performance outcomes by describing their program staffing structure, the role of each proposed staff member, their relevant skills, and the percent time expected of each staff for each major activity proposed in the work plan.

Established partnerships with groups/organizations relevant to the strategies selected, which may include patient and/or community organizations, newborn screening programs, blood banks, professional health care associations, and groups with experience in health education or health promotion.

Prior experience working on the strategies selected.

https://www.cdc.gov/grants/additionalrequirements/ar-25.html

Experience and capacity to conduct program evaluation, both process and outcome, and implement the evaluation plan.

Sufficient leadership for program planning and development including the identification, hiring, or reassignment and supervision of staff, contractors, and/or consultants that have the knowledge and skills to implement the program and are sufficient in number.

Proven ability or demonstrate sufficient capacity to develop and execute contracts for program services in an expedient manner and capacity to monitor contracts once implemented.

Established organizational policies and procedures to ensure sufficient fiscal and budgetary oversight and capacity to provide required reports.

Ability to participate in project-related meetings and site visits.

Evidence that they have the capacity to either collect biological specimens from individuals with thalassemia or sickle cell disease or work with partner organizations to facilitate the collection of biological specimens and their shipment to CDC.

Applicants must provide CVs/resumes for key program personnel, name this file "CVs/Resumes", and upload it at www.grants.gov.

d. Work Plan Applicants must submit a detailed work plan for Year 1 of the award and provide a general summary of work plan activities for Years 2-5 in narrative form. If the applicant is applying for both the Thalassemia and Sickle cell disease components, a single work plan should be used for both components, with clear labeling of which table applies to which component. Additionally, the work plan should demonstrate how any activities that address both component’s strategies will be implemented, so that work on these strategies may be mutually reinforcing. Cost sharing of positions to support multiple activities across components is encouraged.

The work plan should describe how the applicant plans to implement all of the required strategies and activities to achieve NOFO outcomes. A sample work plan format is presented below to show how a traditional work plan aligns with the logic model and narrative. In this format, the table would be completed for each period of performance outcome. If a particular activity leads to multiple outcomes, it should be described under each outcome measure. Applicants are not required to use the example work plan format; however, all work plans must contain all of the elements shown in the example (outcome(s), outcome measure(s), etc).

Example Work Plan Table Period of Performance Outcome: (from Logic Model) Outcome Measure: (from Evaluation and Performance Measurement)

Strategies and Activities

Process Measure (from Evaluation and Performance Measurement)

Responsible Position/Party

Population of focus

Scope Setting(s) Completion Date

1.

2.

3.

4.

5.

A separate table should be included in the work plan for each of the outcomes that will be completed in Year 1 of the award. If a particular activity leads to multiple outcomes, it should be described under each outcome measure.

Applicants must name this file Project Narrative_name of applicant and upload it as a PDF file.

CDC will provide feedback and technical assistance to award recipients to finalize the work plan activities post-award.

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 will analyze performance measurement data to review progress and identify technical assistance needs for all NOFO strategies on an annual basis. The proposed work plan and performance measures will be reviewed annually by the project officer and evaluation staff and may need to be modified to better reflect program activities as outlined in the NOFO. Findings from the annual analysis of performance measure data will be used to identify areas of program improvement, broader technical assistance needs, and to report to stakeholders. CDC will review the work plan to track implementation of their surveillance system as outlined in recipient strategies and activities (process evaluation) and determine progress on achieving the period of performance outcomes (outcome evaluation). CDC will provide routine feedback to recipients throughout the period of performance using phone calls, emails, and site visits. These findings may be presented during site visits and recipient meetings.

All evaluation findings produced by CDC and recipients, where appropriate, will contribute to:

(1) continuous improvement of quality and effectiveness of program strategies; (2) the evidence base; (3) documentation and sharing of lessons learned to support replication and scaling of these program strategies; and/or (4) future funding opportunities supported by CDC.

f. CDC Program Support to Recipients (THIS SECTION APPLIES ONLY TO

COOPERATIVE AGREEMENTS)

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

Supporting recipients in implementing cooperative agreement requirements and meeting program outcomes

Providing technical assistance to revise annual work plans Assisting recipients in advancing program activities to achieve project outcomes Providing scientific subject matter expertise and resources in support of the selected strategies Collaborating with recipients to develop and implement evaluation plans that align with

CDC evaluation activities Providing technical assistance on recipients' evaluation and performance measurement plans Providing technical assistance to define and operationalize performance measures Using webinars and social media for recipients and CDC to communicate and share tools and resources Participating in relevant meetings, committees, conference calls, and working groups related to the cooperative agreement requirements to achieve outcomes Coordinating communication and program linkages with other CDC programs and

Federal agencies, such as the Health Resources and Services Administration (HRSA) and the National Institutes of Health (NIH)

Translating and disseminating lessons learned through publications, meetings, and other means on promising and best practices to expand the evidence base

Hosting a reverse site visit during each year of the period of performance.

Encouraging information sharing between recipients: CDC will host quarterly all-recipient calls to allow for information and resource sharing, as well as capacity building and opportunity for collaboration between recipients

Ensuring that recipients have access to expertise found throughout NCBDDD/DBD. For example, health scientists, epidemiologists, statisticians, policy analysts, communication specialists, and evaluators to provide technical assistance to recipients. Technical assistance teams will also work in collaboration with other programs across NCBDDD/DBD to identify specific actions that improve efficiency and greater public health impact.

Collaborating with recipients to explore appropriate flexibilities needed to meet public health outcomes and goals. Flexibility in cooperative agreements includes recipient’s ability to propose alternative methods to achieve the outcomes and goals of the cooperative agreement that align with recipient’s opportunities for success, infrastructure, partner and stakeholder buy-in, demographics, and burden. This includes bringing together resources from multiple cooperative agreements to jointly advance the goals of each and expanding the dialogue to bring in other CDC and recipient staff to reach a win/win solution.

Creating greater efficiencies and consistency across NCBDDD/DBD programs for recipients. Examples of how programs work together to achieve this include but are not limited to:

o Joint site visits that maximize the ability to do collaborative problem solving, offer insights and ideas to strengthen or augment recipient approaches, and increase understanding of recipient’s context to accomplish improved health outcomes for thalassemia/sickle cell disease.

o Jointly developed resources and tools to ensure consistent messages and to meet recipient technical assistance needs.

Providing packaging materials and technical laboratory assistance to support the collection, processing, and shipment of biological specimens to CDC for the creation of a biorepository that can be monitored for new and emerging pathogens that may pose a serious potential risk to these…