Supported by an unrestricted educational grant from sanofi-aventis and by the ALS Association. Endorsed by the World Federation of Neurology 1 Amyotrophic Lateral Sclerosis Clinical Assessment, Research and Education & The ALS Patient Care Database

ROGRAM The ALS C. A. R. E P 2 ALS C.A.R.E. Program ALS Patient Care Database Improved outcomes Educational programs OutcomesEducation

ROGRAM The ALS C. A. R. E P 3 Overview  Voluntary, confidential, outcomes database  Mechanism for evaluating the impact of diagnostic and therapeutic decisions  Foundation for assessing current patterns of clinical practice  Effort to improve outcomes for patients with ALS and their caregivers

ROGRAM The ALS C. A. R. E P 4 Objectives  Develop benchmarks  Provide individual and aggregate data to neurologists  Publish overall findings on issues such as compliance with AAN practice parameters  Improve outcomes

ROGRAM The ALS C. A. R. E P 5 Design Principles  Observational cohort study  Physician-, patient-, and caregiver-reported data  Standard data collection instruments  Broad participation  Uniform diagnostic and assessment measures  Data collected at each routine patient visit  Quarterly reports delivered to participating neurologists

ROGRAM The ALS C. A. R. E P 6 ALS C.A.R.E. Program Medical Advisory Board Robert G. Miller Fred Anderson Linda Boynton de Sepulveda Mark B. Bromberg Benjamin Rix Brooks Michael Graves Yadalloh Harati Terry Heiman-Patterson Sharon Matland Hiroshi Mitsumoto Dan H. Moore Eric Pioro Steven P. Ringel Jeffrey Rosenfeld Mark A. Ross Robert L. Sufit Ashok Verma November 2007

ROGRAM The ALS C. A. R. E P 7 Study Coordinating Center  Center for Outcomes Research - University of Massachusetts Medical School  Prepare quarterly reports  Provide scientific support for data analysis  Assure confidentiality

ROGRAM The ALS C. A. R. E P 8 Physician Confidentiality  Use of coded physician and clinic ID numbers ID numbers  Physician names kept in locked file  Provisions approved by UMass Medical School’s IRB  Expedited local IRB approval (typical)

ROGRAM The ALS C. A. R. E P 9 Patient and Caregiver Confidentiality  Sites allocate patient ID numbers  Provisions approved by UMass Medical School’s IRB  Verbal informed patient consent per local IRB (typical)

ROGRAM The ALS C. A. R. E P 10 Data Collection Instruments  Health Professional Form  Patient Form  Caregiver Form  Completion Form

ROGRAM The ALS C. A. R. E P 11 Physician-Reported Data  Type of ALS  El Escorial diagnostic criteria  Atypical features  Regions affected  ALS functional rating score  Forced vital capacity  ALS-related conditions  Current management

ROGRAM The ALS C. A. R. E P 12 Patient Self-Reported Data  Demographics (age, sex, etc)  Personal information (insurance, income, employment status, etc)  General health status (capacity to perform ADL, psychosocial status, etc)  Diagnostic factors (presenting symptoms)  Activities of daily living  Use of services (number of physician visits, satisfaction with medical care, etc)

ROGRAM The ALS C. A. R. E P 13 Caregiver-Reported Data  Relationship to patient  Source of payment (if paid caregiver)  General health status of caregiver  Psychosocial impact on caregiver  Employment status of caregiver

ROGRAM The ALS C. A. R. E P 14 Quarterly Reports  Confidential  Include individual physician/site and aggregate North American data  Allow participants to compare their outcomes with North American benchmarks

ROGRAM The ALS C. A. R. E P 15 History of ALS C.A.R.E. Program  First board meetingNovember 1995  Pilot trialDecember 1995  Modify data formsJanuary 1996  Begin data collectionSeptember 1996  First reportFebruary 1997  >5,000 patientsFebruary 2003  New report designFebruary 2003  Electronic data captureSeptember 2004  >6,000 patientsNovember 2005  Data collection completedJune 2007 November 2007

ROGRAM The ALS C. A. R. E P 16 Enrollment Summary  Clinics enrolled 323  Clinics submitting data 109  Neurologists enrolled 381  Patients enrolled 6337 November 2007

ROGRAM The ALS C. A. R. E P 17 Distribution of Enrolled Sites November 2007

ROGRAM The ALS C. A. R. E P 18 Follow-Up Data November 2007

ROGRAM The ALS C. A. R. E P 19 Demographics At Study Enrollment  Age, years –Median60 –Range20-90  Caucasian92%  Female 41% November 2007

ROGRAM The ALS C. A. R. E P 20 Type of ALS At Study Enrollment November 2007

ROGRAM The ALS C. A. R. E P 21 Limitations  Observational, no control group  Mainly academic practices  No data on pathological confirmation  More longitudinal data needed

ROGRAM The ALS C. A. R. E P 22 Conclusions  A North American database has been established for studying ALS  The database is informative about practice patterns (diagnostic tests, treatment, etc.)  Patient satisfaction and patient needs are reflected in the database