[Federal Register Volume 60, Number 101 (Thursday, May 25, 1995)]
[Notices]
[Pages 27733-27735]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 95-12835]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
[Announcement 519]
Prevention of the Complications of Hemophilia
Introduction
The Centers for Disease Control and Prevention (CDC) announces the
availability of fiscal year (FY) 1995 funds for a cooperative agreement
program to conduct a trial of primary prophylaxis therapy for the
prevention of joint disease and/or inhibitor formation in children with
hemophilia.
The Public Health Service (PHS) is committed to achieving the
health promotion and disease prevention objectives of Healthy People
2000, a PHS-led national activity to reduce morbidity and mortality and
improve the quality of life. This announcement is related to the
priority area of Diabetes and Chronic Disabling Conditions. (For
ordering a copy of Healthy People 2000, see the section Where to Obtain
Additional Information.)
Authority
This program is authorized under Sections 301(a) and 317(k)(2) of
the Public Health Service Act, as amended [42 U.S.C. 241(a) and
247b(k)(2)]. Applicable program regulations are found in 42 CFR Part
51b--Project Grants for Preventive Health Services.
Smoke-Free Workplace
PHS strongly encourages all grant recipients to provide a smoke-
free workplace and to promote nonuse of all tobacco products, and
Public Law 103-227, the Pro-Children Act of 1994, prohibits smoking in
certain facilities that receive Federal funds in which education,
library, day care, health care, and early childhood development
services are provided to children.
Eligible Applicants
Because of the low prevalence of hemophilia, competition is limited
to hemophilia treatment centers (HTCs) that routinely access and
administer comprehensive health care to sufficient numbers of
previously untreated patients with severe hemophilia each year. Since
HTCs are the only health care facilities administering to the numbers
of hemophiliacs required for this study, assistance will be provided
only to hemophilia treatment centers.
Availability of Funds
Approximately $500,000 is available in FY 1995 to fund up to two
awards. It is expected that the award will begin on or about September
30, 1995, and will be made for a 12-month budget period within a
project period of up to 5 years. Funding estimates may vary and are
subject to change. Continuation awards within the project period will
be made on the basis of satisfactory programmatic progress and the
availability of funds.
Purpose
The purpose of this hemophilia cooperative agreement program is to
assist recipients in the implementation of and analysis of data from a
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 should be
determined in the treatment group and should be compared to similar
data from appropriately treated, control subjects. In addition to
objective measures of joint function and mobility, the cumulative risk
of factor VIII inhibitor development should be determined for each
treatment group and total costs and complication rates ascertained.
Molecular characterization of factor VIII defects and detailed
molecular HLA typing should be determined for all subjects in an effort
to predict which subjects will develop inhibitors.
Program Requirements
In conducting activities to achieve the purpose of this program,
the recipient shall be responsible for the activities under A. below,
and CDC shall be responsible for conducting activities under B. below:
A. Recipient Activities
1. Develop standardized study protocols, data collection
instruments, interview questionnaires, progress report forms, and amend
previous protocols with new activities or procedures incorporating all
changes agreed to at assistance meetings.
2. 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 and to ensure that the data is
collected in a uniform manner at all locations.
3. Develop appropriate management and evaluation systems to ensure
that study personnel use data collection and interview instruments
according to standard study protocols.
4. Collect and edit all data from all sites, including cost
effectiveness data.
5. Obtain and transmit to CDC sufficient clinical specimens for
specialized laboratory analysis and genetic testing, including whole
blood, plasma, cell pellets or joint tissue/fluid, to meet the
requirements of the study.
6. Publish the results of the study using a writing committee to
determine the inclusion and order of authors on all publications.
B. CDC Activities
1. Provide consultation, and scientific and technical assistance in
planning and implementing the study protocol. This assistance will
include the development of standard study protocols, data abstraction
instruments, interview questionnaires, consent and progress report
forms.
2. Participate in the planning, coordination, and facilitation of
initial and periodic meetings with recipients to exchange operational
experiences, and to provide consultation and assistance in the
modification of standard study protocols as needed.
3. Provide the required software and technical assistance in
statistical and epidemiologic methods to conduct data analysis.
4. The coagulation research laboratory at CDC will be responsible
for confirmation of factor VIII inhibitor levels and will serve as the
central reference laboratory for molecular analysis of all study
participants. CDC will be responsible for epitope typing of all
inhibitors and other specialized immunological/genetic testing.
Evaluation Criteria
Applications will be reviewed and evaluated according to the
following criteria: (Total 100 points)
A. Capacity
1. The capacity of the applicant to accrue a minimum total of 40
boys with severe factor VIII (<1%) who="" are="" 30="" months="" old="" or="" less="" without="" a="" history="" of="" joint="" hemorrhage="" from="" multiple="" htcs="" to="" each="" treatment="" arm="" of="" the="" protocol.="" each="" participating="" htc="" must="" be="" able="" to="" enroll="" a="" minimum="" of="" 5="" previously="" untreated="" patients="" who="" meet="" the="" above="" criteria.="" (20="" points)="" 2.="" the="" capacity="" to="" accrue="" and="" maintain="" patients="" on="" trials="" will="" be="" measured="" by="" (a)="" the="" number="" of="" patients="" [[page="" 27734]]="" eligible="" for="" randomization="" into="" the="" trial="" that="" are="" seen="" annually="" at="" each="" htc,="" (b)="" the="" number="" of="" patients="" entered="" and="" successfully="" followed="" in="" previous="" similar="" trials="" and="" (c)="" the="" publication="" of="" the="" results="" of="" other="" such="" trials="" in="" peer="" reviewed="" journals.="" such="" publications="" should="" demonstrate="" that="" the="" applicant="" is="" capable="" of="" enrolling="" and="" following="" young="" hemophiliacs="" in="" a="" clinical="" trial.="" (20="" points)="" 3.="" qualifications="" of="" proposed="" staff="" to="" meet="" stated="" objectives="" and="" goals,="" and="" the="" availability="" of="" facilities="" to="" be="" used="" during="" the="" project="" period.="" (10="" points)="" b.="" goals="" and="" objectives="" the="" extent="" to="" which="" the="" applicant's="" proposed="" goals="" and="" objectives="" meet="" the="" required="" activities="" specified="" under="" section="" a.="" ``recipient="" activities''="" of="" this="" announcement,="" and="" that="" are="" measurable,="" specific,="" time-phased,="" and="" realistic.="" (10="" points)="" c.="" methods="" and="" activities="" 1.="" the="" quality="" of="" the="" applicant's="" plan="" for="" conducting="" program="" activities="" and="" the="" extent="" to="" which="" the="" clinical="" trial="" design="" proposed="" is:="" (a)="" appropriate="" to="" accomplish="" stated="" goals="" and="" objectives;="" (b)="" acceptable="" to="" the="" needs="" of="" the="" patient="" population="" (e.g.,="" likely="" to="" produce="" compliance);="" (c)="" feasible="" within="" programmatic="" and="" fiscal="" restrictions.="" (30="" points)="" 2.="" the="" recipient="" should="" demonstrate="" a="" basic="" knowledge="" of="" the="" methods="" of="" randomized,="" clinical="" trials="" and="" describe="" how="" they="" will="" implement="" a="" standardized="" protocol="" at="" various="" htcs;="" (a)="" develop="" standardized="" progress="" report="" forms;="" (b)="" collect,="" edit,="" and="" transmit="" appropriate="" data="" to="" the="" cdc.="" (10="" points)="" d.="" budget="" the="" extent="" to="" which="" the="" budget="" is="" reasonable="" and="" consistent="" with="" the="" intended="" use="" of="" the="" cooperative="" agreement="" funds.="" (not="" scored)="" funding="" priorities="" in="" order="" to="" maximize="" the="" probability="" of="" developing="" meaningful="" conclusions="" from="" this="" randomized="" trial="" in="" the="" shortest="" possible="" time,="" funding="" priorities="" will="" take="" into="" consideration="" the="" ability="" of="" the="" htc="" (including="" geographical="" representation="" of="" all="" participating="" htcs)="" to="" accrue="" up="" to="" 40="" previously="" untreated="" hemophilia="" patients="" in="" each="" treatment="" arm="" of="" the="" protocol.="" interested="" persons="" are="" invited="" to="" comment="" on="" the="" proposed="" funding="" priorities.="" all="" comments="" received="" on="" or="" before="" june="" 26,="" 1995="" will="" be="" considered="" before="" the="" final="" funding="" priorities="" are="" established.="" written="" comments="" should="" be="" addressed="" to:="" clara="" m.="" jenkins,="" grants="" management="" officer,="" grants="" management="" branch,="" procurement="" and="" grants="" office,="" centers="" for="" disease="" control="" and="" prevention="" (cdc),="" 255="" east="" paces="" ferry="" road,="" ne.,="" room="" 300,="" mailstop="" e-18,="" atlanta,="" georgia="" 30305.="" executive="" order="" 12372="" review="" this="" program="" is="" not="" subject="" to="" executive="" order="" 12372="" review.="" public="" health="" system="" reporting="" requirements="" this="" program="" is="" not="" subject="" to="" the="" public="" health="" system="" reporting="" requirements.="" catalog="" of="" federal="" domestic="" assistance="" number="" the="" catalog="" of="" federal="" domestic="" assistance="" number="" is="" 93.283,="" centers="" for="" disease="" control="" and="" prevention="" (cdc)--investigations="" and="" technical="" assistance.="" other="" requirements="" paperwork="" reduction="" act="" projects="" that="" involve="" collection="" of="" information="" from="" 10="" or="" more="" individuals="" and="" funded="" by="" the="" cooperative="" agreements="" will="" be="" subject="" to="" review="" by="" the="" office="" of="" management="" and="" budget="" (omb)="" under="" the="" paperwork="" reduction="" act.="" human="" subjects="" if="" the="" proposed="" project="" involves="" research="" on="" human="" subjects,="" the="" applicant="" must="" comply="" with="" the="" department="" of="" health="" and="" human="" services="" regulations="" (45="" cfr="" part="" 46)="" regarding="" the="" protection="" of="" human="" subjects.="" assurance="" must="" be="" provided="" which="" demonstrate="" that="" the="" project="" will="" be="" subject="" to="" initial="" and="" continuing="" review="" by="" an="" appropriate="" institutional="" review="" committee.="" the="" applicant="" will="" be="" responsible="" for="" providing="" evidence="" of="" this="" assurance="" in="" accordance="" with="" the="" appropriate="" guidelines="" and="" forms="" provided="" in="" the="" application="" kit.="" all="" information="" obtained="" in="" connection="" with="" this="" prevention="" trial="" shall="" not,="" without="" such="" individual's="" consent,="" be="" disclosed="" except="" as="" may="" be="" necessary="" to="" provide="" services="" to="" him="" or="" her="" or="" as="" may="" be="" required="" by="" a="" law="" of="" a="" state="" or="" political="" subdivision="" of="" a="" state.="" information="" derived="" from="" any="" such="" program="" may="" be="" disclosed:="" (1)="" in="" summary,="" statistical,="" or="" other="" form,="" or="" (2)="" for="" clinical="" or="" research="" proposed,="" but="" only="" if="" the="" identity="" of="" the="" individuals="" under="" such="" program="" is="" not="" disclosed.="" application="" submission="" and="" deadline="" the="" original="" and="" five="" copies="" of="" the="" application="" phs="" form="" 398="" (omb="" number="" 0925-0001)="" must="" be="" submitted="" to="" clara="" m.="" jenkins,="" grants="" management="" officer,="" grants="" management="" branch,="" procurement="" and="" grants="" office,="" centers="" for="" disease="" control="" and="" prevention="" (cdc),="" 255="" east="" paces="" ferry="" road,="" ne.,="" room="" 300,="" mailstop="" e-18,="" atlanta,="" georgia="" 30305,="" on="" or="" before="" july="" 7,="" 1995.="" 1.="" deadline:="" applications="" shall="" be="" considered="" as="" meeting="" the="" deadline="" if="" they="" are="" either:="" (a)="" received="" on="" or="" before="" the="" deadline="" date;="" or="" (b)="" sent="" on="" or="" before="" the="" deadline="" date="" and="" received="" in="" time="" for="" submission="" to="" the="" objective="" review="" group.="" (applicants="" must="" request="" a="" legibly="" dated="" u.s.="" postal="" service="" postmark="" or="" obtain="" a="" legibly="" dated="" receipt="" from="" a="" commercial="" carrier="" or="" u.s.="" postal="" service.="" private="" metered="" postmarks="" shall="" not="" be="" acceptable="" as="" proof="" of="" timely="" mailing.)="" 2.="" late="" applications:="" applications="" which="" do="" not="" meet="" the="" criteria="" in="" 1.(a)="" or="" 1.(b)="" above="" are="" considered="" late="" applications.="" late="" applications="" will="" not="" be="" considered="" in="" the="" current="" competition="" and="" will="" be="" returned="" to="" the="" applicant.="" where="" to="" obtain="" additional="" information="" a="" complete="" program="" description="" and="" information="" on="" application="" procedures="" are="" contained="" in="" the="" application="" package.="" business="" management="" technical="" assistance="" may="" be="" obtained="" from="" locke="" thompson,="" grants="" management="" specialist,="" grants="" management="" branch,="" procurement="" and="" grants="" office,="" centers="" for="" disease="" control="" and="" prevention="" (cdc),="" 255="" east="" paces="" ferry="" road,="" ne.,="" room="" 300,="" mailstop="" e-18,="" atlanta,="" georgia="" 30305,="" telephone="" (404)="" 842-6595.="" programmatic="" technical="" assistance="" may="" be="" obtained="" from="" bruce="" evatt,="" m.d.,="" division="" of="" hiv/aids,="" national="" center="" for="" infectious="" diseases,="" centers="" for="" disease="" control="" and="" prevention="" (cdc),="" 1600="" clifton="" road,="" ne.,="" mailstop="" e-64,="" atlanta,="" georgia="" 30333,="" telephone="" (404)="" 639-3925.="" please="" refer="" to="" announcement="" number="" 519="" when="" requesting="" information="" and="" submitting="" an="" application.="" potential="" applicants="" may="" obtain="" a="" copy="" of="" healthy="" people="" 2000="" (full="" report,="" stock="" no.="" 017-001-00474-0)="" or="" healthy="" people="" 2000="" (summary="" report,="" stock="" no.="" 017-001-00473-1)="" referenced="" in="" the="" introduction="" through="" the="" superintendent="" of="" documents,="" government="" printing="" office,="" washington,="" dc="" 20402-9325,="" telephone="" (202)="" 512-="" 1800.="" [[page="" 27735]]="" dated:="" may="" 19,="" 1995.="" joseph="" r.="" carter,="" acting="" associate="" director="" for="" management="" and="" operations,="" centers="" for="" disease="" control="" and="" prevention="" (cdc).="" [fr="" doc.="" 95-12835="" filed="" 5-24-95;="" 8:45="" am]="" billing="" code="" 4163-18-p="">