Comparative Effectiveness Research: Payer Perspectives 1 Sean Tunis MD, MSc May 5, 2009

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 2

Great Expectations “At the core of both the stimulus bill and Obama’s budget is Orszag’s 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 Orszag’s thesis.” –The New Yorker. May 4,

Medicare Coverage Sect (a)(1)(A), Title 18, SSA –“…no payment may be made…for items or services.. [which] are not reasonable and necessary for the diagnosis or treatment of illness or injury.” Working definition of R&N –“Adequate evidence to conclude that the item or service improves net health outcomes”

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

115 Technologies Reviewed by Kaiser 6 Insufficient evidence because the evidence is: A.Of insufficient quantity and/or quality B. Conflicting or inconsistent C. There is no evidence Medically appropriate Generally not medically appropriate Paul Wallace, Permanente Federation

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 –“didn’t invite CMS because it’s a scientific mtg” –“don’t 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

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

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 11

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.”

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 13

CMTP Patient-Consumer Advisory Committee Jessie Berlin (chair of PCAC) Maureen Corry Gene Kazmierczak Jennifer Sweeney Larry Sadwin (CMTP board chair) Patience White 14

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

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

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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