[Federal Register Volume 65, Number 83 (Friday, April 28, 2000)]
[Notices]
[Pages 24961-24963]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 00-10680]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Centers for Disease Control and Prevention

[Program Announcement 00048]


Patient Follow-Up Study of Prevention of the Joint Complications 
of Hemophilia; Notice of Availability of Funds

A. Purpose

    The Centers for Disease Control and Prevention (CDC) announces the 
availability of fiscal year (FY) 2000 funds for a cooperative agreement 
program for the Mountain State Regional Hemophilia Treatment Center 
(MSRHTC), University of Colorado Health Sciences Center to conduct a 
follow-up study of patients enrolled in a trial of primary prophylactic 
therapy for the prevention of joint disease in children with 
hemophilia. CDC is committed to achieving the health promotion and 
disease prevention objectives of ``Healthy People 2010,'' a national 
activity to reduce morbidity and mortality and improve the quality of 
life. This announcement is related to the focus areas of Diabetes. For 
the conference copy of ``Healthy People 2010'', visit the internet site 
http://www.health.gov/healthypeople.
    The purpose of the program is to provide continued assistance MSRHC 
in the follow-up of patients enrolled in the current randomized, 
controlled trial of primary prophylaxis in previously untreated 
patients with severe hemophilia A and no demonstrable factor VIII 
inhibitors. Cost and efficacy of early intervention to prevent joint 
complications should be determined in the treatment groups and should 
be compared to similar data from appropriately treated, control 
subjects. The cumulative risk of factor VIII inhibitor development 
should be determined for each treatment group and total costs and 
complication rates ascertained as well. Molecular characterization of 
factor VIII defects in an effort to predict which subjects will develop 
inhibitors should be carried out.
    Further, follow-up of patients will continue to improve 
understanding of specific public health issues and enhance preparedness 
to meet changes.

B. Eligible Applicants

    Assistance will be provided only to the MSRHTC. No other 
applications are solicited. The MSRHTC was the only applicant that 
applied under the original Program Announcement 95019 and was 
subsequently awarded. It is the only Hemophilia Treatment Center (HTC) 
capable of carrying out the follow-up of the hemophilia patients 
enrolled in this clinical trial because they began and continue the 
enrollment of patients under the original clinical trial. The 
enrollment of Hemophilia patients for this program will be completed on 
September 29, 2000, and it is the intention of CDC to allow for a 
smooth transition into this follow-up study.
    MSRHTC already possesses the necessary data required for the 
follow-up study for the prevention of the joint complications of 
hemophilia. MSRHTC's patient data is proprietary, and no other HTC 
would have ready access.

C. Availability of Funds

    Approximately $250,000 is available in FY 2000 to fund this award. 
It is expected that the award will begin on or about September 30, 2000 
and will be made for a 12-month budget period within a project period 
of up to 4 years. The funding estimate may change.
    Continuation awards within an approved project period will be made 
on the basis of satisfactory progress as evidenced by required reports 
and the availability of funds.

D. Program Requirements

    In conducting activities to achieve the purpose of this program, 
the recipient will be responsible for the activities under 1. 
(Recipient Activities), and CDC will be responsible for the activities 
listed under 2. (CDC Activities).

1. Recipient Activities

    a. Develop standardized study protocols, data collection 
instruments, interview questionnaires, progress report forms.
    b. Train study coordinators and medical personnel in methods of 
data collection and patient assessment in the use of standard data 
abstraction instruments, in techniques of reviewing medical records, in 
interviewing patients, and in other methods of data collection as 
appropriate and provided for in the study protocols. It is the 
responsibility of the recipient to ensure uniform training of study 
personnel at all data collection sites. The recipient must ensure that 
the data is collected in a uniform manner at all data collection sites.
    c. Develop appropriate management and evaluation systems to ensure 
that study personnel use data collection and interview instruments 
according to standard study protocols.
    d. Collect and edit all data from all sites, including cost 
effectiveness data.
    e. Obtain sufficient clinical specimens for specialized laboratory 
analysis and genetic testing, including plasma and cell pellets, to 
meet the requirements of the study.
    f. Develop papers and publish the results.

2. CDC Activities

    a. Provide consultation, scientific and technical assistance in 
planning and implementing the study protocol, as requested. This 
assistance may include the development of study protocols, data 
abstraction instruments, interview questionnaires, consent forms, 
support in statistical and epidemiologic methods to conduct data 
analysis, and development of the clinical laboratory specimen testing.
    b. Assist in the development of a research protocol for 
Institutional Review Board (IRB) review by all institutions 
participating in the research project. The CDC IRB will review and 
approve the protocol initially and on at least an annual basis until 
the research project is completed.
    c. Collaborate in the planning, coordination, and facilitation of 
initial and periodic meetings.
    d. Perform sufficient clinical specimens for specialized laboratory 
analysis and genetic testing, including plasma and cell pellets, as 
requested.

E. Application Content

    Use the information in the Purpose, Program Requirements, Other 
Requirements, and Evaluation Criteria sections to develop the 
application content. The application should describe MSRHTC's ability 
to address the purpose and required activities of this announcement. 
The application

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will be evaluated on the criteria listed, so it is important to follow 
them specifically in laying out the program plan. The narrative should 
be no more than 20 double-spaced pages, printed on one side, with one 
inch margins, and unreduced font.

F. Submission and Deadline

Application

    Submit the original and two copies of the PHS-5161-1 (OMB Number 
0920-0428). Forms are available at the following Internet address: 
www.cdc.gov/Forms, or in the application kit.
    On or before June 1, 2000, submit the application to the Grants 
Management Specialist identified in the ``Where to Obtain Additional 
Information'' section of this announcement.

G. Evaluation Criteria

    Each application will be evaluated individually against the 
following criteria by an independent review group appointed by CDC.

1. Background and Need: (10 Points)

    Extent to which applicant summarizes new information (literature) 
on the prevention of joint disease among persons with hemophilia; 
describes how this information enhances the need for the clinical 
trial; describes in as much detail as possible, how the study will 
enhance future management of children less than six years old with 
hemophilia.

2. Goals and Objectives: (15 Points)

    Extent to which the proposed goals and objectives meet the purpose 
of the collaborative agreement and the required activities specified in 
the ``Recipient Activities'' section of this announcement and that are 
measurable, specific, time-phased, and realistic.

3. Capacity: (35 Points)

    a. Extent to which knowledge, ability, and experience with primary 
prophylaxis studies initiated early in life as a form of ``low-dose'' 
immune tolerance are described in a plan to ensure a high level of 
follow-up among patients enrolled on the current study. Extent to which 
applicant describes, in detail, how a high loss to follow-up will 
affect the results and conclusions that can be drawn from the final 
analysis of the study. (30 points)
    b. Extent to which applicant details the qualifications of the 
current staff to meet the stated objectives and goals, and the 
availability of facilities to be used during the project follow-up 
period. The person (or persons) who will be directly responsible for 
the follow-up should be identified and their duties specified in 
detail. (5 points)

4. Methods and Activities: (25 Points)

    a. The quality of the applicant's plan for conducting program 
activities and the extent to which the study design proposed is (1) 
appropriate to accomplish the stated goals and objectives; (2) 
acceptable to the needs of the patient population (e.g., likely to 
produce compliance); and (3) feasible within the programmatic and 
fiscal restrictions. (10 points)
    b. Extent to which applicant demonstrates knowledge of implementing 
patient follow-up procedures and describes methods for patient follow-
up among all currently collaborating sites.

    Note: Description of patient follow-up protocol is critical for 
ascertaining that long-term follow-up of enrolled patients will 
occur over the project period. The applicant should indicate in the 
application how they plan to monitor sites to identify problems with 
maintaining an acceptable loss to follow-up rate as described in 
section 3.a. above. (15 points)

5. Program Management and Evaluation: (15 Points)

    a. Extent to which applicant should describes the information 
management systems currently available for this study, detailing any 
system modifications required to accomplish collecting follow-up data 
versus enrollment data. Extent to which applicant describes, in detail, 
specific plans to evaluate data on a quarterly basis, quality assurance 
measures to be used and how operations will be changed based on the 
above information. Extent to which applicant describes study close-out 
procedures once all patients have completed follow-up data collection. 
Extent to which applicant demonstrates adequate biostatistical support 
for study completion, data management and data analysis. (10 points)
    b. The degree to which the applicant has met the CDC Policy 
requirements regarding the inclusion of women, ethnic, and racial 
groups in the proposed research. This includes: (1) The proposed plan 
for the inclusion of both sexes, racial and ethnic minority populations 
for appropriate representation, (2) the proposed justification when 
representation is limited or absent, (3) a statement as to whether the 
design of the study is adequate to measure differences when warranted, 
and (4) a statement as to whether the plans for recruitment and 
outreach for study participants include the process of establishing 
partnerships with community(ies) and recognition of mutual benefits. (5 
points)

6. Budget

    The extent to which the budget is reasonable and consistent with 
the intended use of the cooperative agreement funds. (Not scored)

7. Human Subjects Requirements

    Does the application adequately address the requirements of Title 
45 CFR part 46 for the protection of human subjects? (not scored)

H. Other Requirements

Technical Reporting Requirements

    Provide CDC with original plus two copies of:
    1. Progress reports quarterly;
    2. Financial Status Report (FSR), no more than 90 days after the 
end of the budget period; and
    3. Final FSR 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 I 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
AR-9  Paperwork Reduction Act Requirements
AR-10  Smoke-Free Workplace Requirements
AR-11  Healthy People 2010
AR-12  Lobbying Restrictions
AR-15  Proof of Non-profit Status

I. Authority and Catalog of Federal Domestic Assistance Number

    This program is authorized under section 301(a) [42 U.S.C. 241(a)] 
and 317(k)(2) [42 U.S.C. 247b(k)], as amended. The Catalog of Federal 
Domestic Assistance number is 93.283.

J. Where To Obtain Additional Information

    To obtain additional information, contact: Merlin Williams, Grants 
Management Specialist, Grants Management Branch, Procurement and Grants 
Office, Centers for Disease Control and Prevention, 2920 Brandywine 
Road, Room 3000, Atlanta, GA 30341-4146, Telephone: (770) 488-2765, E-
mail address: [email protected].
    For program technical assistance, contact: Lisa Richardson, MD, 
MPH, Hematologic Diseases, Branch, Division of AIDS, STD, and TB 
Laboratory

[[Page 24963]]

Research, National Center for Infectious Diseases, Centers for Disease 
Control and Prevention (CDC), 1600 Clifton Road, NE, Mail-stop E-64. 
Atlanta, GA 30333, Telephone: (404) 371-5264, E-mail address: 
[email protected].
    The CDC Homepage address on the Internet is: http://www.cdc.gov

    Dated: April 24, 2000.
John L. Williams,
Director, Procurement and Grants Office, Centers for Disease Control 
and Prevention (CDC).
[FR Doc. 00-10680 Filed 4-27-00; 8:45 am]
BILLING CODE 4163-18-P