Comparative Effectiveness Research: Key Issues and Controversies

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• Comparative Effectiveness ResearchKey Issues
  and Controversies
• Consumer-Purchaser Disclosure Project Discussion
  Forum
• May 5, 2009

Steven D. Pearson, MD, MSc, FRCP
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Background

• Policy givens
• Unsustainable cost increases
• Unexplainable variation in practice patterns
• Not enough evidence for decisions about new
  treatments
• International efforts (health technology
  assessment)
• NICE in England
• Comparative Effectiveness
• Stark bill
• Baucus bill
• American Recovery and Reinvestment Act (ARRA)
  stimulus bill funding for Comparative
  Effectiveness Research (CER)

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10-Year Impact on Spendingof a Center for
Comparative Effectiveness
Dollars in billions
SAVINGS COSTS
Source Based on estimates by The Lewin Group for
The Commonwealth Fund, 2007.
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Chief remaining questions on CER

• Stimulus spending
• Priorities for spending at AHRQ and NIH
• Secretary of HHS 400 million
• Inclusion of cost and/or cost-effectiveness
• CER 2.0
• Structure
• Governance
• Funding
• Priority Setting
• Research Methods (cost-effectiveness)
• Implementation

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Stimulus spending

• Priorities for spending at AHRQ and NIH
• Mix of systematic reviews and prospective studies
• Framing of topics as drug vs. drug or broader
  pathways of care
• Studies of health plan policies such as prior
  authorization
• Secretary of HHS 400 million
• Inclusion of cost-effectiveness

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Weighing up costs and effects
Cost ()
Effectiveness
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Why Costs?

• Not to consider costs is delusional
• Costs should be considered transparently and
  always in the context of clinical effectiveness
• Without consideration of cost
• No societal support for explicit cost
  considerations in clinical decisions and medical
  policies
• All explicit health plan efforts will be suspect
• Continued difficulty negotiating prices in
  relation to evidence of incremental benefit
• Marginal benefit at high price will continue to
  be a dominant market signal for manufacturers

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How to do Costs?

• Carve-out
• Commissioned by individual payers, including
  Medicare
• Arms length
• Funded as part of CER stream but function
  delegated to an allied yet separate organization
• Carve-in
• Distrust of clinical effectiveness judgments if
  mixed with costs
• More efficient to nest within same effort to
  generate a systematic review of the clinical
  evidence
• Benefits from the objectivity and transparency of
  a federal comparative effectiveness initiative to
  gain broad acceptance

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Legislation for CER 2.0

• Structure
• Inside or attached to government vs. independent?
• Governance
• Stakeholders on the Governing Board or only on
  Advisory Committees?
• Funding
• How much from private health plans and
  purchasers?
• Priority Setting
• Who and how?
• Research Methods
• Cost-effectiveness yea or nea?
• Implementation

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• http//www.politico.com/singletitlevideo.html?bcpi
  d1155201977bctid21157881001

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How will CE information be used?

• Concerns
• Limit access to life-saving treatments just
  because of cost
• One-size-fits-all methodologies and
  applications to coverage policies
• Cost-effectiveness applied as a strict cut-off
  for coverage
• Cost-effectiveness devalues older, sicker
  patients
• Put governmental bureaucrats between you and your
  doctor
• Stifle innovation

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How CER should be used

• Too cold
• Dissemination of information to patients and
  clinicians
• Too hot
• Direct mandates for all-or-nothing coverage
  decisions
• Just right
• Providing guidance to patients, clinicians, and
  payers
• Application by payers to create value-based tools
  and policies in support of optimal care and to
  ensure best use of every health care dollar
• Patient-clinician decision support
• Reimbursement policy
• Value-based insurance design
• Physician group compensation (P4P)

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Application of Cost-effectiveness

• Help identify the least costly alternative among
  equivalent treatment options
• Provide some context for the additional cost paid
  for very marginal clinical benefits
• Help anchor initial pricing for new technologies
  in evidence of their marginal (if any) benefit
• Tools
• Patient-clinician decision tools
• Reimbursement policy
• Value-based insurance design
• Physician group compensation (P4P) to align
  incentives

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• For further information
• spearson_at_icer-review.org
• www.icer-review.org