Comparative Effectiveness Research:

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• Comparative Effectiveness Research
• Payer Perspectives

Sean Tunis MD, MSc May 5, 2009
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Disclosure

• CMTP develops tools and strategies for
  comparative effectiveness research
• We receive funding from government, foundations,
  life sciences companies, health plans, and
  medical professional societies
• Aim is to serve as a neutral convener

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Great Expectations

• At the core of both the stimulus bill and
  Obamas budget is Orszags belief that a
  government empowered with research on the most
  effective medical treatments can, using the
  proper incentives, persuade doctors to become
  more efficient health care providers, thus saving
  billions of dollars. Obama is in effect betting
  his Presidency on Orszags thesis.
• The New Yorker. May 4, 2009.

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Medicare Coverage

• Sect. 1862 (a)(1)(A), Title 18, SSA
• no payment may be madefor items or services .
  . which are not reasonable and necessary for
  the diagnosis or treatment of illness or injury.
• Working definition of RN
• Adequate evidence to conclude that the item or
  service improves net health outcomes

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Illustrating the Problem Tx of Clinically
Localized Prostate Cancer

• Limited evidence on relative safety and
  effectiveness of major treatment options
• prostatectomy, brachytherapy, radiation, active
  surveillance
• New technologies rapidly spreading without data
• robotic surgery, proton beam
• Rigorous trials needed to compare treatment
  options, especially for side effects

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115 Technologies Reviewed by Kaiser
Paul Wallace, Permanente Federation
Generally not medically appropriate
Medically appropriate
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• Insufficient evidence because the evidence is
• Of insufficient quantity and/or quality
• B. Conflicting or inconsistent
• C. There is no evidence

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Critical Knowledge Gaps

• The paradox
• 18,000 RCTs published each year
• Available evidence is limited or poor quality
• Patients, settings, comparators, outcomes, timing
  often not aligned with decision makers
• Patients, clinicians, payers, policy makers
• Decision makers have limited traction
• didnt invite CMS because its a scientific mtg
• dont want patients messing up our protocols

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CMS Efforts to Improve Evidence

• NETT trial (1995)
• Category B IDE regulation (1996)
• Cover routine costs of clinical trials (2000)
• Coverage with evidence development (2003)
• Promote pragmatic clinical trials (2003)
• MCAC becomes MedCAC (2005)
• Ad hoc collaborations with NIH
• Creative application of existing authorities
• Functional equivalence, LCA, HCPCS coding

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Implantable Defibrillator Registry

• Medicare coverage expanded 01/05
• Registry intended for risk stratification
• 300k patients now in registry
• Baseline data interesting
• Median age 74 (vs 60 in trials) LVEF higher
• 3.6 complication rate
• No firing info or other outcomes data
• Low priority for NHLBI, Industry, ACC/HRS
• AHRQ/NIH have recently identified funds
• Small fraction of 12.5B could have major ROI
• Funding of CED studies high priority

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Impact of ARRA

• 1.1B is a good way to focus attention
• Multiple Coordinating Councils
• IOM priority setting committee
• Many position papers developed / underway
• New advocacy coalitions (e.g. PIPC)
• Lots of sleep-derived, newly-minted comparative
  effectiveness researchers

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IOM CER Working Definition

• The generation and synthesis of evidence that
  compares the effectiveness of alternative methods
  to prevent, diagnose, treat, monitor, and improve
  delivery of care for a clinical condition.
• The purpose of CER is to assist patients,
  clinicians, purchasers, and policy makers in
  making informed health decisions.

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Implications of CER Purpose

• If primary purpose of CER is to inform decisions
  by patients, clinicians, payers, policymakers,
  then
• CER requires high level of involvement of these
  decision makers
• Implies collaborative approach
• Inherent tension with current PI-driven model
• Best practices for meaningful engagement of
  patients/consumers, payers not yet clear

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CMTP Patient-Consumer Advisory Committee

• Jessie Berlin (chair of PCAC)
• Maureen Corry
• Gene Kazmierczak
• Jennifer Sweeney
• Larry Sadwin (CMTP board chair)
• Patience White

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Medicare Review of CCTA

• EPC report from Duke (April 2006)
• Limited evidence of clinical utility in any
  population
• MedCAC mtg (May 2006)
• Uncertain confidence about existing evidence
• Broad local coverage of CCTA
• Medicare draft policy in 12/07 proposed CED for
  CCTA in adequate studies

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Debate on CCTA Coverage

• Payers/researchers
• Propose RCT with death/AMI outcome
• 20k patients, 2-3 years of follow-up
• Vendors / clinicians
• existing evidence adequate for coverage
• Medicare final decision (March 2008)
• No adequately designed studies show improved
  outcomes
• We believe large, well-designed prospective
  trials needed
• Broad coverage by local contractors retained
• ICER review unproven / promising
• NHLBI currently reviewing 3 RCTs

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Cardiac Imaging Think Tank

• Goal was to move toward shared evidence framework
  for non-invasive cardiac imaging
• Engage decision makers in study design
• Broad involvement of experts / stakeholders
• Co-sponsors ICER, ACC, ACR, SCCT, ASNC
• private payers, CMS, imaging vendors, clinical
  researchers, consumers, AHRQ, VA, etc.
• Result
• Modest reduction in hostility / some conversation
• Now developing Effectiveness Guidance Document
  to guide future CER studies

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Contact Info

• sean.tunis_at_cmtpnet.org
• www.cmtpnet.org
• 443-759-3116 (D)
• 410-963-8876 (M)