Sharing Data:
Observations From a
Recovering Registry Addict
Christopher Bredeson, MD, MSc., FRCPC
Director, Hematologic Malignancies
Professor of Medicine
Medical College of Wisconsin
DBV06_1.ppt
TODAY’S TOPIC IS - REGISTRIES
DBV06_2.ppt
TODAY’S TOPIC IS - REGISTRIES
DBV06_2.ppt
What I Am Supposed To Cover
Overview
Funding
Privacy
Security
Governance
Issues / challenges
Results
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What Is The Purpose Of The Registry?
Research
Natural history
Rare events
Tracking product
Safety
Lobbying
Quality of care / standards
Definitions
A patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes.
The patient registry database describes a file (or files) derived from the registry.
We Need Data in Medicine
Med/Scientific
Community Payors Industry Patient
Assess trends ++ ++ +++ +
Determine efficacy +++ ++ +++ +++
Track product +++ +++ ++ ++
Allocate resources ++ +++ +++ +
Monitor outcomes + +++ + +++
DBV06_4.ppt
Multiple Stakeholders may use the same registry!
DBV06_23.ppt
International BMT Registry
Established in 1972 to monitor and study outcomes of bone marrow transplants
Maintains a database of clinical information on recipients of autologous and allogeneic hematopoietic stem cell transplants in ~500 centers in >50 countries
Collates basic data set on all patients in member centers (registration) and comprehensive data (research) on a subset
Provides scientific and statistical support for analyzing those data
Primary purpose outcomes research
IBMTR – 1985 (1985 year of first major NIH funding)
Mortimer M. Bortin, MD
Scientific Director
Statistician
1
Data Management
3
AdministrativeAsst
1
1970-1985:
•200 centers•1,000 transplants•35 publications
Bortin Transplantation 1970 Vol. 9 p 571
Annual Numbers of Blood and Marrow Transplantations, 1970-2006 - Worldwide -
0
5,000
10,000
15,000
20,000
25,000
30,000
35,000
40,000
1970 1975 1980 1985 1990 1995 2000 2005
Year
Allogeneic
Autologous
Nu
mb
er o
f Tran
sp
lan
ts p
er Y
ear
SHORT HISTORY OF IBMTR / ABMTR
IBMTR
ABMTR
ACS/NIHBMTreg
(1970)
IBMTR to
Milwaukee (1972)
20,000 cases
1st
publications(1996)
1970 1980 1990 2000
Risk factorsDescriptive analyses
Study designData collection forms
Technology assessment
MethodologyTechnology assessment
Risk factorsDescriptive analyses
Mmh01_2.ppt
Clinical Trials
Evolution Leads To Complexity
Someone Has To Own It!
IBMTR
ABMTR
ACS/NIHBMTreg
(1970)
IBMTR to
Milwaukee (1972)
20,000 cases
1st
publications(1996)
1970 1980 1990 2000
Risk factorsDescriptive analyses
Study designData collection forms
Technology assessment
MethodologyTechnology assessment
Risk factorsDescriptive analyses
Mmh01_2.ppt
Clinical Trials
(Benign) Dictatorship Oligarchy Democracy
Centralized Operations
Structure and Governance
Oversight and
Guidance
•Independent
•Elected and
career people
Central leadership
•Career
•Content and
methodologic
expertise
Functional
working unit
for observational
research
•Stat Center staff
•Elected Directors
•Open to all
•Patient and donor
perspective
•Assist in guiding
research agenda
•Communicate
with non-medical
community
Types of
research activities
Collaboration Between Different Entities/Organizations
IBMTR
NMDP
EMMES
Overall Coordination Statistical
Design/Analysis
Protocol Development/
Implementation
Trial Oversight/Monitoring
Patient Advocacy
Contracting
Lab/Repository
Management
DCC02_3.ppt
Scientific Leadership
Medical Monitoring
ElectronicCommunications
DataManagement
MILWAUKEE WASHINGTON
MINNEAPOLIS
Money Issues
Cost ~ personnel and Data
Infrastructure / operations
Data acquisition
Technology
Paper is cheap up front, electronic data cheaper in the long-term
Life cycle considerations
Does the registry have a finite life?
Data access in the afterlife?
Open access?
Real-time or later?
Data = money
Amount of Funding
Hassle
Factor
Federal
Contracts
Industry
Unrestricted
Industry
Contract
Philanthropy
Peer Rev
Grants
Funding
Data and Privacy
Which patients?
All patients?
All consenting patients?
Selected or randomly selected patients?
You want to be able to update data
Need a link between center and registry database
What are you sending?
Data +/- tissue
Who owns the data?
Does the registry give the data back to the center?
Data and Consent
Anonymous data
De-identified data
Center sends with unique ID#
Center knows ID#=patient name
Data with Identifiers included
Patient consent
Higher risk of disclosure of personal health information
Usually still communicate with ID#
Ethical / Legal Issues
Transparency
Consent
Incentives are dodgy
Require review of IRB policies and Canadian regulations
Should also pass the newspaper test
If activity was described in the local paper would it be viewed favourably?
DBV06_30.ppt
CIBMTR Data Available
Registration Database
Basic (essential) information on consecutive transplant recipients in participating institutions
Age, sex, disease, disease stage and duration, graft type and treatment, conditioning regimen, posttransplant disease status, GVHD, survival, cause of death, new cancers
Research Database
Comprehensive patient, disease, treatment and outcome data
Data elements selected to allow investigation of important issues in the field
CIBMTR Data Validity
Data dictionary
Data review / cleaning
Computerized
E.g. transplant date after birth date
Manual
Data up to date
Calendar driven
Carrots and sticks
Data audits
All centers every three years
All critical data elements
Sampling of others
Data Forms
Data forms design is a difficult
Data forms central to success or failure of registry
Leave nothing to interpretation
Minimal free text options
Comprehensive data manual to accompany form
“help” line
“So much for computer dating!”
Technology Is Not Smart
There Are Still Barriers To Overcome
Data Dictionary:Speaking The Same Language
08/10/09
August 10, 2009
8th of October 2009
October 9th, 2008
Getting The Data…
Sharing The Rewards
Acute Leukemia
Chronic Leukemia
Lymphoma
Plasma Cell Disorders
Solid Tumors
Pediatric Cancer
Non-Malignant Marrow Disorders
Immune Deficiencies / Inborn Errors
Autoimmune Diseases
Graft Sources/Manipulation
GVHD
Late Effects & QOL
Immunobiology
Infection / Immune Reconstitution
Regimen-related Toxicity
Emerging Cellular Therapies
Health Services & Psychosocial Issues
Donor Health & Safety
International Studies
WORKING COMMITTEESParticipants Include Clinicians and Scientists
from >300 institutions worldwide
Finding A Niche
“You call this a niche!”
Accomplishments
0 4 872 65
0
20
40
60
80
100
PR
OB
AB
ILIT
Y O
F R
EL
AP
SE
, %
YEARS
P
Longterm Prognosis After BMT
EHA00_22.ppt
YEARS
PR
OB
AB
ILIT
Y O
F S
UR
VIV
AL
, %
0
20
40
60
80
100
0 1 2 3 4 5 6 7 8 9 10 11 12
P = 0.0001
CML (N = 2,146)
ALL (N = 1,458)
AML (N = 2,058)SAA (N = 1,029)
NEJM 1999
Cu
mu
lati
ve I
ncid
en
ce,
%
Years
1.0%
2.2%
6.6%
0
2
4
6
8
0 2 4 6 8 10 12 14 16
Solid cancers (N = 80)
PTLD (N = 78)
DBV06_54.ppt
Cumulative Incidence of PTLD and Invasive Solid Cancers Following Allogeneic BMT
Center For International Blood And Marrow Transplant Research (CIBMTR)
Established July 2004
A research affiliation between the IBMTR and
the NMDP to support clinical research in BMT
& related fields
Clinical Research includes
Observational Studies (including immunobiologic correlates)
Clinical Trials
Health Services Research
Statistical Methodology
CIBMTR Clinical Research 2008
19 Working Committees
>200 Active Studies
>7,600 samples distributed by the NMDP repository for immunobiology working committee studies (2006)
56 scientific papers submitted or accepted for publications in 2006.
ASH meeting presentation: 2006: 10 selected studies (2 posters, 8 oral)
2007: 18 selected studies (12 posters, 6 oral)
2008: 24 selected studies (11 posters, 13 oral)
Summary
A registry can be a powerful tool
Someone must own it
Fund with a balanced portfolio
Form follows function
Data elements:
“complete parsimony” is better
Play nicely in the sandbox
it is their sand!
