So You Want to Do
Comparative Effectiveness
Research?
The Nuts and Bolts of CER
Introduction to Comparative
Effectiveness Research
Greg Cooper, MD
CER - ACA Definition
• Research evaluating and comparing
health outcomes and the clinical
effectiveness, risks and benefits of 2 or
more medical treatments, services and
items
Categories of CER Methods
• Systematic reviews of existing research
• Decision modeling, with or without cost
information
• Retrospective analysis of existing clinical
or administrative data
• Prospective, non-experimental studies,
including registries
• Experimental studies, including RCT’s
Pragmatic Clinical Trials
• Compared to explanatory
• Decision maker viewpoint – should we
provide intervention?
• Whether an intervention works in “real
world” conditions
• Whether it works, not how or why
Pragmatic Clinical Trials
• Patient selection reflects routine practice
– Exclude only if contraindicated
• Intervention to discretion of clinician
– Dose adjustment
• Control routine practice (vs. placebo)
• Patient centered outcomes
• Always ITT analysis
Pragmatic Clinical Trials
• Identify prevalent condition
– Often based on symptoms rather than explicit,
verifiable diagnoses
• Evaluate common clinical dilemma in that
condition
• Develop pragmatic study to understand
health outcomes between competing
management approaches
Primary Care Management of GERD
• Open label study of patients with
symptomatic GERD despite H2RA or
omeprazole
• Randomization at clinic level (n=226)
• Screened on basis of 5 item questionnaire
• Intervention esomeprazole at dose
selected by clinician (20 or 40 mg) or
continued care
• Measured HRQOL and utilities at 4 weeks
Moayeddi, et al: Am J Gastroenterol 2010
Results
Intervention
Control
58%
29%
PPI baseline
$1213 per QALM
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H2RA baseline
$648 per QALM
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None or minimal sx
Patient Centered Outcomes
Research Institute
PCORI Research Areas
• Prevention, Diagnosis and Treatment
Options
• Improving Healthcare Systems
• Communication and Dissemination
Research
• Addressing Disparities
• Accelerating Patient-Centered Outcomes
Research and Methodological Research
PCORI Funding
• Up to 3 years, $500K/year (direct costs)
– 8/15, 12/15, 4/15 deadlines
• Letter of Intent Required (2 months prior)
• Must have key stakeholders engaged
early and throughout research process
• Patient and stakeholder engagement plan
– Involved in study design and monitoring
conduct
– Involved in dissemination of findings
Stakeholders
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Patients
Caregivers
Clinicians
Patient advocacy groups
Community groups
Involved in advisory panels,
teleconferences, focus groups
CER and the CTSA
• Develop a CTSA Consortium-wide
framework for community-engaged and
CER that will support improvement in
healthcare access, quality, efficiency and
patient-centered outcomes and will
improve the health of diverse communities
and the public
• CER Key Function Committee (KFC)
CER and Case CTSC
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Pilot projects 2010
Core Utilization Pilots
Explorys Pilots
Research symposium 2010
Three focal areas
– Large database
– Economic analysis
– EHR
CER Services
• Assistance with study design
– Pilots
– Investigator initiated projects
• Grant preparation
• Facilitating referrals
• Through Request Management System
(RMS)
Symposium Objectives
• Overview of CER Methodology
– Electronic Health Records
– Large Database Research
– Systematic Review/Meta Analysis
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Practical Examples
Panel Discussion
Encourage Core Utilization
Ideas for future talks, symposia