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 ControlNone or minimal sx 58% 29%

PPI baseline $1213 per QALM --------

H2RA baseline $648 per QALM --------

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

• 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

• 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

• Practical Examples• Panel Discussion• Encourage Core Utilization• Ideas for future talks, symposia