supported by an unrestricted educational grant from sanofi-aventis and by the als association....

11

Supported by an unrestricted educational grant fromSupported by an unrestricted educational grant fromsanofi-aventis and by the ALS Association. sanofi-aventis and by the ALS Association.

Endorsed by the World Federation of NeurologyEndorsed by the World Federation of Neurology

AAmyotrophic myotrophic LLateral ateral SSclerosisclerosisCClinical linical AAssessment, ssessment, RResearch and esearch and EEducationducation

&&

The ALS Patient Care DatabaseThe ALS Patient Care Database

www.alscare.orgwww.alscare.org

2ROGRAMROGRAMTheThe

ALS CALS C..AA..RR..E PE P

ALS C.A.R.E. ProgramALS C.A.R.E. Program

ALS Patient Care ALS Patient Care DatabaseDatabase

Improved Improved outcomesoutcomes

Educational Educational programsprograms

OutcomesOutcomes EducationEducation

3ROGRAMROGRAMTheThe


OverviewOverview

Voluntary, confidential, outcomes databaseVoluntary, confidential, outcomes database

Mechanism for evaluating the impact of diagnostic Mechanism for evaluating the impact of diagnostic and therapeutic decisionsand therapeutic decisions

Foundation for assessing current patterns Foundation for assessing current patterns of clinical practice of clinical practice

Effort to improve outcomes for patients with Effort to improve outcomes for patients with ALS and their caregiversALS and their caregivers

4ROGRAMROGRAMTheThe


ObjectivesObjectives

Develop benchmarksDevelop benchmarks Provide individual and aggregate Provide individual and aggregate

data to neurologistsdata to neurologists Publish overall findings on issues such as Publish overall findings on issues such as

compliance with AAN practice parameterscompliance with AAN practice parameters Improve outcomesImprove outcomes

5ROGRAMROGRAMTheThe


Design PrinciplesDesign Principles

Observational cohort studyObservational cohort study Physician-, patient-, and caregiver-reported dataPhysician-, patient-, and caregiver-reported data Standard data collection instrumentsStandard data collection instruments Broad participationBroad participation Uniform diagnostic and assessment measuresUniform diagnostic and assessment measures Data collected at each routine patient visitData collected at each routine patient visit Quarterly reports delivered to participating Quarterly reports delivered to participating

neurologistsneurologists

6ROGRAMROGRAMTheThe


ALS C.A.R.E. ProgramALS C.A.R.E. ProgramMedical Advisory BoardMedical Advisory Board

Robert G. MillerRobert G. Miller

Fred AndersonFred Anderson

Linda Boynton de SepulvedaLinda Boynton de Sepulveda

Mark B. BrombergMark B. Bromberg

Benjamin Rix BrooksBenjamin Rix Brooks

Michael GravesMichael Graves

Yadalloh HaratiYadalloh Harati

Terry Heiman-PattersonTerry Heiman-Patterson

Sharon MatlandSharon MatlandHiroshi MitsumotoHiroshi MitsumotoDan H. MooreDan H. MooreEric PioroEric PioroSteven P. RingelSteven P. RingelJeffrey RosenfeldJeffrey RosenfeldMark A. RossMark A. RossRobert L. SufitRobert L. SufitAshok VermaAshok Verma

November 2007

7ROGRAMROGRAMTheThe


Study Coordinating CenterStudy Coordinating Center

Center for Outcomes Research - University of Center for Outcomes Research - University of

Massachusetts Medical SchoolMassachusetts Medical School

Prepare quarterly reportsPrepare quarterly reports

Provide scientific support for data analysisProvide scientific support for data analysis

Assure confidentialityAssure confidentiality

8ROGRAMROGRAMTheThe


Physician ConfidentialityPhysician Confidentiality

Use of coded physician and clinic Use of coded physician and clinic

ID numbersID numbers

Physician names kept in locked filePhysician names kept in locked file

Provisions approved by UMass Medical Provisions approved by UMass Medical School’s IRBSchool’s IRB

Expedited local IRB approval (typical)Expedited local IRB approval (typical)

9ROGRAMROGRAMTheThe


Patient and Caregiver Patient and Caregiver ConfidentialityConfidentiality

Sites allocate patient ID numbersSites allocate patient ID numbers

Provisions approved by UMass Medical Provisions approved by UMass Medical

School’s IRBSchool’s IRB

Verbal informed patient consent per local Verbal informed patient consent per local

IRB (typical)IRB (typical)

10ROGRAMROGRAMTheThe


Data Collection InstrumentsData Collection Instruments

Health Professional FormHealth Professional Form

Patient FormPatient Form

Caregiver FormCaregiver Form

Completion FormCompletion Form



Physician-Reported DataPhysician-Reported Data

Type of ALSType of ALS El Escorial diagnostic criteriaEl Escorial diagnostic criteria Atypical featuresAtypical features Regions affectedRegions affected ALS functional rating scoreALS functional rating score Forced vital capacityForced vital capacity ALS-related conditionsALS-related conditions Current managementCurrent management



Patient Self-Reported DataPatient Self-Reported Data

Demographics (age, sex, etc)Demographics (age, sex, etc) Personal information (insurance, income, Personal information (insurance, income,

employment status, etc)employment status, etc) General health status (capacity to perform ADL, General health status (capacity to perform ADL,

psychosocial status, etc)psychosocial status, etc) Diagnostic factors (presenting symptoms)Diagnostic factors (presenting symptoms) Activities of daily livingActivities of daily living Use of services (number of physician visits, Use of services (number of physician visits,

satisfaction with medical care, etc)satisfaction with medical care, etc)



Caregiver-Reported DataCaregiver-Reported Data

Relationship to patientRelationship to patient

Source of payment (if paid caregiver)Source of payment (if paid caregiver)

General health status of caregiverGeneral health status of caregiver

Psychosocial impact on caregiverPsychosocial impact on caregiver

Employment status of caregiverEmployment status of caregiver



Quarterly ReportsQuarterly Reports

ConfidentialConfidential

Include individual physician/site and Include individual physician/site and

aggregate North American dataaggregate North American data

Allow participants to compare their Allow participants to compare their

outcomes with North American outcomes with North American

benchmarksbenchmarks



History of History of ALS C.A.R.E. ProgramALS C.A.R.E. Program

First board meetingFirst board meeting November 1995November 1995

Pilot trialPilot trial December 1995December 1995

Modify data formsModify data forms January 1996January 1996

Begin data collectionBegin data collection September 1996September 1996

First reportFirst report February 1997February 1997

>5,000 patients>5,000 patients February 2003February 2003

New report designNew report design February 2003February 2003

New report designNew report design February 2003February 2003

Electronic data captureElectronic data capture September 2004September 2004

>6,000 patients>6,000 patients November 2005November 2005

Data collection completedData collection completed June 2007June 2007

November 2007



Enrollment SummaryEnrollment Summary

Clinics enrolledClinics enrolled 323 323

Clinics submitting dataClinics submitting data 109 109

Neurologists enrolledNeurologists enrolled 381 381

Patients enrolledPatients enrolled 6337 6337

November 2007



Distribution of Enrolled SitesDistribution of Enrolled Sites

November 2007



Follow-Up DataFollow-Up Data

56805291

4327

2589 2556 2369

1731 1643 1449

0

1000

2000

3000

4000

5000

6000

7000

Health Professional Patient Caregiver

Nu

mb

er o

f F

orm

s

Enrolled 6-month follow-up 12-month follow-up

November 2007



DemographicsDemographicsAt Study EnrollmentAt Study Enrollment

Age, yearsAge, years

– MedianMedian 6060

– RangeRange 20-9020-90

CaucasianCaucasian 92%92%

FemaleFemale 41% 41%

November 2007



94%

6%

Sporadic Familial

Type of ALSType of ALS At Study EnrollmentAt Study Enrollment

November 2007



LimitationsLimitations

Observational, no control groupObservational, no control group

Mainly academic practicesMainly academic practices

No data on pathological confirmationNo data on pathological confirmation

More longitudinal data neededMore longitudinal data needed



ConclusionsConclusions

A North American database has been A North American database has been

established for studying ALSestablished for studying ALS

The database is informative about practice The database is informative about practice

patterns (diagnostic tests, treatment, etc.)patterns (diagnostic tests, treatment, etc.)

Patient satisfaction and patient needs are Patient satisfaction and patient needs are

reflected in the databasereflected in the database

supported by an unrestricted educational grant from sanofi-aventis and by the als association....

Documents

als association

data type of als

confidentiality slide

als functional rating

routine patient

local irb typical slide

aggregate data

data analysis