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

None 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