02-13632. Surveillance and Epidemiologic Research of Duchenne and Becker Muscular Dystrophy and Other Single Gene Disorders; Notice of Availability of Funds

A. Purpose

The Centers for Disease Control and Prevention (CDC), announces the availability of fiscal year (FY) 2002 funds for a cooperative agreement program on surveillance and epidemiologic research of (1) Duchenne and Becker Muscular Dystrophy (DBMD) and other single gene disorders, and (2) genetic and other disorders identified through tandem mass spectrometry and other newborn screening technologies. This program addresses the “Healthy People 2010” priority areas for Disability and Secondary Conditions.

The purpose of the program is to: (1) Develop and/or expand surveillance systems to characterize the epidemiology of DBMD and other single gene disorders and their complications; and (2) develop long-term follow-up tracking of children identified through tandem mass spectrometry and other newborn screening technologies.

This program consists of two types of projects (Type I and Type II):

Type I: Surveillance and Tracking of DBMD and Other Single Gene Disorders Without Universal Screening Programs

The purpose of Type I funding is to determine the prevalence of DBMD and other single gene disorders that are not detected through newborn screening programs and that therefore require active case finding activities. Long-term population-based follow-up research activities will be planned to describe history of treated and/or untreated cases, and to determine factors that affect the outcome of the condition among three populations: (a) Those who access care at specialty clinics (e.g., Muscular Dystrophy Association (MDA) or other muscular dystrophy clinics), (b) those who receive their care elsewhere, and (c) those who are not receiving care or are undiagnosed.

Type II: Surveillance and Tracking of Conditions With Universal Screening Programs

The purpose of Type II funding is to determine the prevalence of single gene or other conditions identified using existing or potential newborn screening programs, and to conduct population-based long-term follow-up research to describe the history of treated and/or untreated cases. Potential newborn screening programs include, but are not limited to, newborn blood spot screening using tandem mass spectrometry.

Quantifiable outcomes of the cooperative agreement will be measured against the following Government Performance Results Act performance goal: To find causes and risk factors for birth defects and developmental disabilities in order to develop prevention strategies.

B. Authority and Catalog of Federal Domestic Assistance Number

This program is authorized under Sections 301, 311 and 317(C) of the Public Health Service Act [42 U.S.C. 241, 243, and 247b-4 as amended]. The Catalog of Federal Domestic Assistance number is 93.184.

C. Eligible Applicants

Assistance will be provided only to the health departments of States or their bona fide agents including the District of Columbia, the Commonwealth of Puerto Rico, the Virgin Islands, the Commonwealth of the Northern Mariana Islands, American Samoa, Guam, the Federated States of Micronesia, the Republic of the Marshall Islands, the Republic of Palau, and federally recognized Indian tribal governments.

To be eligible, applicants must document a study population of at least 30,000 live births per year within a State, a contiguous area of a State (such as the catchment of a local health agency), or a contiguous area comprising a combination of States, based on U.S. Census Data. This information should be placed directly behind the face page of the application. Applications that fail to submit the evidence requested above will be considered non-responsive and returned without review.

If an applicant chooses to apply for both a Type I and a Type II award, a separate complete application must be submitted for each type.

Note:

Title 2 of the United States Code, Section 1611 states that an organization described in section 501(c)(4) of the Internal Revenue Code that engages in lobbying activities is not eligible to receive Federal funds constituting an award, grant, or loan.

D. Availability of Funds

Approximately $1,600,000 is available in FY 2002 funding to fund up to five awards. It is expected that up to three Type I awards will be made, ranging from $350,000 to $450,000, and that up to two Type II awards will be made, ranging from $100,000 to $150,000. It is expected that the award will begin on or about September 1, 2002, and will be made for a 12-month budget period within a three year project period. Funding estimates may change.

Continuation awards within the project period will be made on the basis of satisfactory programmatic progress and the availability of funds.

Funding preference will include: (1) Geographic balance, (2) Type I programs that target DBMD, and (3) Type II programs that target fatty acid oxidation and/or organic acid disorders.

E. Program Requirements

In conducting activities to achieve the purpose of this program, the recipient will be responsible for the activities under 1. Recipient Activities for Type I projects or 2. Recipient Activities for Type II projects. CDC will be responsible for the activities listed under 3. CDC Activities.

1. Recipient Activities for Type I Projects

a. Develop, implement and evaluate methods and approaches that will improve or expand the capacity of the applicant's existing surveillance system to ascertain cases and generate timely population-based data of DBMD and other single gene disorders and their complications.

b. Establish or enhance collaborative relationships with appropriate stakeholders, i.e., specialty treatment centers (e.g., MDA clinics, other muscular dystrophy clinics), state or regional chapters or associations related to genetic conditions, hospitals, emergency care centers, private physicians, managed care organizations, clinical and diagnostic laboratories that provide diagnosis of genetic conditions (e.g., creatine kinase measurements, muscle biopsy analysis, genetic analysis, etc.), and others.

c. Collaborate with other Type I funded recipients to design and develop one common protocol for all recipients to implement and evaluate.

d. Implement active case ascertainment of DBMD and other genetic condition(s) among reporting sources to determine the prevalence of the genetic condition(s) in the defined geographic area, including a complete count of all prevalent cases, including ages birth to 21 years, and supplemented in later years by newly diagnosed cases.

e. Describe the source, frequency, and type of preventive and medical care among persons with DBMD and other genetic condition(s), among three populations: (a) Those who access care at specialty clinics (e.g., MDA or other muscular dystrophy clinics), (b) those who receive their care elsewhere, and (c) those who are not receiving care or are undiagnosed.

f. Determine the prevalence of related complications.

g. Conduct population-based long-term follow-up of persons with DBMD and other genetic condition(s) to relate health outcomes to the source, frequency, and type of preventive and therapeutic care.

h. Obtain buccal samples or other biologics, as agreed upon by awardees, from children with DBMD and genetic conditions and other family members.

i. Evaluate and disseminate the findings.

2. Recipient Activities for Type II Projects

a. Collaborate with other Type II funded recipients to design and develop one common protocol for all recipients to implement and evaluate.

b. Describe the source, frequency, type of and access to preventive and medical care among persons with the genetic condition(s), and determine the prevalence of related complications.

c. Conduct population-based long-term follow-up of persons with genetic condition(s) to relate health outcomes to the source, frequency, type of, and access to preventive and therapeutic care, including both primary and specialty care settings.

d. Assess the option of obtaining buccal samples or other biologics, as agreed upon by awardees, from children with the genetic condition(s) and other family members.

e. Evaluate and disseminate the findings.

3. CDC Activities

a. Participate in designing, developing, and evaluating methodologies and approaches used for population-based surveillance of genetic conditions.

b. Participate in the collection, management, and analysis of surveillance data related to genetic conditions.

c. Participate in the development and planning of the study protocol. Provide final approval for the study protocol.

d. Assist in the analysis and reporting of aggregate surveillance data collected from funded initiatives; coordinate and consolidate the transfer of tabulated data, analyses, and conclusions among recipients.

e. Assist national, state, or regional programs in the use of data to develop or improve care programs for genetic conditions.

f. Assist recipients in developing a plan for the collection, storage and access of biologic samples.

g. Assist recipients in the evaluation and dissemination of the findings.

F. Content

Letter of Intent (LOI)

A LOI is requested for this program. The LOI will not be used to eliminate potential applicants, but it will enable CDC to determine the level of interest and plan the review more efficiently. The LOI should be no more than two, double-spaced pages, printed on one side, with one-inch margins and 12 point font. The LOI should denote the intent to submit a Type I or Type II application, and should include the following information: program announcement number; applicant's name and address; project director's name, phone number, and e-mail address; a brief description of the number of births in the defined geographic region; and a brief description of the planned cooperative agreement activities.

Application

Use the information in the Program Requirements, Other Requirements, and Evaluation Criteria sections to develop the application content. Your application will be evaluated on the criteria listed, so it is important to follow them in laying out your program plan.

The proposal narrative (excluding budget narrative and any appendices) should be no more than 35 double-spaced pages, printed on one side, with one inch margins, and 12 point font. Number each page consecutively and provide a complete table of contents.

The application should contain the following:

1. Executive Summary (one page, may be single spaced):

This section should briefly summarize:

a. Type of request (I or II).

b. Amount of federal assistance requested.

c. Existing capacity.

d. Key objectives and activities.

2. Proposal Narrative:

a. Introduction, statement of need, proposed goals and objectives.

b. Existing program and capacity.

c. Proposed methods and activities.

d. Project management and project staff.

e. Proposed methods to evaluate the attainment of objectives.

3. Budget Narrative.

4. Human Subjects.

5. Appendices, which may include letters of commitment from key collaborators (including specialty clinics such as MDA clinics and other muscular dystrophy clinics), resumes of key staff, brief summary reports of analyses of surveillance data for other genetic conditions.

G. Submission and Deadline

Letter of Intent

On or before June 28, 2002, submit the LOI to the Program Officer listed as the point of contact for programmatic technical assistance identified in the Where to Obtain Additional Information section of this announcement.

Application

On or before 5:00 p.m. Eastern Time August 2, 2002, submit the original and two copies of PHS-5161 (OMB Number 0920-0428). Forms are available at the following Internet address: www.cdc.gov/​od/​pgo/​forminfo.htm.

Submit the application to: Technical Information Management-PA02172, Procurement and Grants Office, Centers for Disease Control and Prevention, 2920 Brandywine Rd, Room 3000, Atlanta, GA 30341-4146.

Deadline: Applications shall be considered as meeting the deadline if they are received before 5:00 p.m. Eastern Time on the deadline date. Applicants sending applications by the United States Postal Service or commercial delivery services must ensure that the carrier will be able to guarantee delivery of the application by the closing date and time. If an application is received after closing due to: (1) Carrier error, when the carrier accepted the package with a guarantee for delivery by the closing date and time, or (2) significant weather delays or natural disaster, CDC will upon receipt of proper documentation, consider the application as having been received by the deadline.

Applications that do not meet the above criteria will not be eligible for competition and will be discarded. Applicants will be notified of their failure to meet the submission requirements.

H. Evaluation Criteria

Applicants are required to provide measures of effectiveness that will demonstrate the accomplishment of the various identified objectives of the cooperative agreement. Measures of effectiveness must relate to the performance goals as stated in section “A. Purpose” of this announcement. Measures must be objective/quantitative and must measure the intended outcome. These Measures of effectiveness shall be submitted with the application and shall be an element of evaluation.

Each application will be evaluated and scored individually by an objective review panel. Evaluations and scoring will be conducted according to the following criteria:

1. Methods and Activities (30 Points)

a. The quality of the applicant's plan for conducting program activities and the extent to which surveillance methods proposed are: (1) Appropriate to accomplish stated goals and objectives; (2) adaptable to a variety of health care settings, and to the collection of longitudinal data; (3) accurate to produce valid and reliable data, and (4) feasible within programmatic and fiscal restrictions.

b. The applicant's willingness to cooperate with CDC and other funded applicants to (1) identify optimal surveillance methods, (2) develop standardized surveillance protocols, data collection instruments, interview questionnaires, progress report forms, and database software, and (3) modify proposed methods and activities to conform to standardized protocols.

2. Capacity (20 Points)

a. The extent to which the applicant can access the state or regional community with genetic conditions that is receiving care within and outside of the specialty clinics (e.g., MDA and other muscular dystrophy clinics), as measured by (1) the extent that this proposal incorporates shared responsibility between specialty clinics and state or local health departments as delineated in letters of agreement; and (2) the extent of collaboration obtained from these entities with other organizations involved in the delivery of care and/or services to persons with genetic conditions.

b. A copy of the State Legislation that allows the authority for State Health Departments to collect information on birth defects, genetic diseases or related conditions needs to be included.

3. Goals and Objectives (20 Points)

The extent to which the project goals and objectives are relevant, specific, achievable, measurable, time-linked and can be addressed through the proposed methods.

4. Management and Staffing (20 Points)

a. The extent to which the scientific resources for project planning and data management/analysis are demonstrated within the applicant's organization or through collaboration with universities or other agencies.

b. The extent to which proposed staffing, staff qualifications and experience, and project organization indicates ability to accomplish the active case findings and other objectives of the program.

5. Evaluation (10 Points)

The degree to which the applicant includes plans to evaluate the attainment of proposed objectives and to evaluate the quality of the data collected.

6. Human Subjects (Not Scored)

Does the application adequately address the requirements of Title 45 CFR part 46 for the protection of human subjects? (Not scored; however, an application can be disapproved if the research risks are sufficiently serious and protection against risks are so inadequate as to make the entire application unacceptable.)

7. Budget (Not Scored)

The budget will be evaluated for the extent to which it is reasonable, clearly justified, and consistent with the intended use of the cooperative agreement funds.

I. Other Requirements

Technical Reporting Requirements

Provide CDC with original plus two copies of:

1. Semiannual progress reports which should include:

a. Brief project description.

b. Comparison of the actual accomplishments to the goals and objectives established for the period.

c. Data requirement that demonstrates measures of effectiveness. In the case that established goals and objectives may not be accomplished or are delayed, documentation of both the reason for the deviation and the anticipated corrective action or a request for deletion of the activity from the project.

d. Other pertinent information, including preliminary findings from the analysis of available data.

e. Financial recap of obligated dollars to date as a percentage of total available funds.

2. Financial status report, no more than 90 days after the end of the budget period.

3. Final financial status and performance reports, no more than 90 days after the end of the project period.

Send all reports to the Grants Management Specialist identified in the “Where to Obtain Additional Information” section of this announcement.

The following additional requirements are applicable to this program. For a complete description of each, see Attachment II in the Application Kit.

AR-1—Human Subjects Requirements

AR-2—Requirements for Inclusion of Women and Racial and Ethnic Minorities in Research

AR-7—Executive Order 12372 Review

AR-9—Paperwork Reduction Act Requirements

AR-10—Smoke-Free Workplace Requirements

AR-11—Healthy People 2010

AR-12—Lobbying Restrictions

AR-22—Research Integrity

J. Where To Obtain Additional Information

This and other CDC announcements can be found on the CDC home page Internet address http://www.cdc.gov. Click on Funding then Grants and Cooperative Agreements.

If you have questions after reviewing the contents of all the documents, business management technical assistance may be obtained from: Sheryl L. Heard, Grants Management Specialist, Acquisition and Assistance Branch B, Centers for Disease Control and Prevention, Announcement 02172, 2920 Brandywine Road, Room 3000, Atlanta, GA 30341-4146, Telephone: [phone], Email address: [e-mail].

Programmatic technical assistance may be obtained from: William Ramsey, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 4770 Buford Highway N.E. MailStop F-15, Atlanta, GA 30341-3724, Telephone: [phone], Email address: [e-mail].

Sandra R. Manning,

CGFM, Director, Procurement and Grants Office, Centers for Disease Control and Prevention.