Comparative Effectiveness Research: The Big Picture

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Comparative Effectiveness ResearchThe Big
Picture

• Mark McClellan, M.D., Ph.D.
• Engelberg Center for Health Care Reform
• The Brookings Institution

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Comparative Effectiveness Research (CER)
Legislative Proposals

• Medicare Modernization Act (2003)
• Created AHRQ Effective Health Care Programto
  conduct secondary reviews of clinical
  effectiveness
• Childrens Health and Medicare Protection Act
  (2007)
• Proposed establishment of CER Commission within
  HHS and center for CER within AHRQ to conduct
  primary and secondary research, including
  cost-effectiveness analysis
• CER Act (2008)
• Proposed formation of non-government, non-profit
  CER Institute with Board of 21 Governors and
  mandate to perform primary and secondary research
  on comparative clinical effectiveness and
  cost-effectiveness of interventions and delivery
  system components

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New Legislation Would Add to Growing CER
Activities

• Studies for FDA Product Approval
• Studies to Support Reimbursement
• Other Randomized Clinical Trials
• Coverage with Evidence Development
• Public and Private Funding
• Studies of Actual Medical Practice

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What is CER?
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What Is CER?

• Alternative specific treatments

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What is CER?

• Specific treatment comparisons
• Broad vs. targeted populations

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Regional Variations in Per Capita Medicare
Spending, 2005
Note Unit of analysis is hospital referral region
Source Congressional Budget Office, Geographic
Variation in Health Care Spending (February
2008).
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What is CER?

• Specific treatment comparisons
• Broad vs. targeted populations
• Practice style comparisons

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The role of CER in a Learning Health Care
System
Develop evidence to improve quality and value
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Other Critical CER Policy Issues
Priorities
Incentives
Identify high-priority gaps in evidence
Coordinate new funding with existingincentives
GovernanceWho will oversee and who will pay
Methods
Impact
Select appropriate data and research designs
Apply evidence to improve quality, outcomes and
value
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Identify high-priority gaps in evidence
Priorities

• Identify gaps in both existing evidence and in
  current CER efforts that can clearly improve
  medical practice
• Integrate different types of evidence into an
  overall CER strategy
• Create a process for incorporating stakeholder
  input into a CER research agenda and adjusting
  priorities as needed

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Select appropriate data and research designs
Methods

• Describe strengths and weaknesses of current CER
  methods
• Randomized controlled trials vs. analysis of
  medical practice data
• Integration of public and private data sources
  and study centers

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Distributed Data Network Model for Studies Based
on Actual Medical Practice
Coordinating Center Drug Safety
Coordinating Center Effectiveness
Coordinating Center Quality
Query system pulls data on an as-needed basis (a
virtual layer / platform)
Institutional Firewalls
Registries
Payer Claims Data
Lab and Pharmacy Data
Ambulatory and Inpatient EMRs
Inpatient Data Warehouse
Source Composite of slides presented by Arnold
Chan and Richard Platt, 2008.
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Coordinate new funding with existing regulatory
and reimbursement incentives
Incentives

• Improve the business case for privately conducted
  CER value focus
• Identify the CER priorities that require public
  investment and can leverage private spending

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Follow through to assure new evidence has
positive impact on health care quality and value
Impact

• Communicate results effectively with patients and
  the wider public
• Evaluate impact on health care policies and
  practices
• Evaluate the impact of CER on valuable innovation

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CER Is Bigger Bang for the Buck Possible?

• Priorities Right Questions
• Not necessarily just a few more head to head
  trials
• CER for practices, policies, personalized care
• Methods Randomized Trials AND Learning from
  Actual Practice
• Incentives Leverage Better Research
• Impact Evaluate for Improving Care