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Slide 1: How Much Benefit is Good Enough?

Muriel R. Gillick, MD
Department of Ambulatory Care and Prevention
Harvard Medical School/Harvard Pilgrim

Slide 2: Beyond the Randomized Trial

• Randomized trial may establish efficacy of treatment
• Trial data must be translated into policy
• Policy decisions are value-laden

Slide 3: Is the Intervention Good Enough?

• To make treatment widely available
• Develop new centers, train physicians
• To substitute new treatment for existing approach
• Change formularies; modify guidelines
• To pay for new intervention
• Third party payers (Medicare, health plans)

Slide 4: Current Decision-Making Process

• Practice guidelines
• Food and Drug Administration approval
• Centers for Medicare and Medicaid process for reimbursing new technology

Slide 5: Basis for Policy Decisions Today

• Utilitarian approach
• If on average patients who get new rx are better off, make rx available
• Acceptable to make some people worse off if there is net improvement

Slide 6: Values Implicit in Current Approach

• Some people may be harmed by intervention, but that's all right as long as treated group benefits on average
• If there is a risk of an adverse effect, intervention may be withheld from all
• Cost is not a relevant consideration
• Resource allocation, justice unimportant

Slide 7: Example: CMS Coverage Decisions

• CMS mandate: provide services that are reasonable and necessary
• For its 42 million beneficiaries
• Repeated efforts to define criteria have failed

Slide 8: CMS Approach

• Many decisions made by local carriers
• Controversial decisions referred to CMS for national decision
• Manufacturers, professional organizations can request policy decision
• Requesters submit outcomes data

Slide 9: CMS Decision-Making

• Exhaustive review of scientific evidence
• by Coverage and Analysis Group
• Technology assessment
• May be commissioned by AHRQ
• Comprehensive review
• by Medicare Coverage Advisory Committee

Slide 10: CMS Issues Decision Memorandum

• To inform public of decision
• Specify reasons for decision
• Outline process followed
• Summarize evidence considered
• May specify conditions for use
• May demand further clinical trials

Slide 11: Medicare Coverage Advisory Committee

• Chartered 1998
• Advisory role to CMS
• Composed of 100 representatives from science, medicine, economics, law
• Subcommittee chosen for each decision
• Includes non-voting representatives of public, industry
• Indicates whether reported benefits translate into improved
• net health outcomes

Slide 12: How CMS Approach Plays Out: Left Ventricular Assist Device

• Involves surgical implantation of prosthetic pump
• LVAD accepted rx for patients awaiting heart transplant
• New indication CMS asked to pay for: definitive rx for older, sicker patients

Slide 13: LVAD as "Destination Therapy"

• 68 patients assigned to device; 61 controls
• 48% decrease in all-cause mortality with rx
• Survival rate with device 52% at 1 year
• Survival rate with device 23% at 2 years
• Quality of life improved
• High morbidity: infections (28%), bleeding (42%)
• Rose et al; NEJM 2001

Slide 14: CMS Evaluation Process

• CMS receives request to reimburse for new indication
• Medicare Coverage Advisory Committee input (votes 6:1 to approve subject to constraints on eligibility)
• CMS repeatedly postpones decision
• CMS agrees to provide coverage
• CMS sets reimbursement at $70,000

Slide 15: Actual Cost of LVAD

• Device alone: $65,000
• Hospitalization total: $200,000
• Total cost/year to Medicare program:
• $350 million (5000 cases)
• $7 billion (100,000 cases)

Slide 16: Beyond Outcomes Data

• What if we consider cost?
• What if we factor in values?

Slide 17: Suppose Cost is Included in Decision-Making

• Cost-effectiveness is technique for selecting among competing options when resources are limited
• Cost-effectiveness ratio=
• Cost (new)- Cost (current)
• Effectiveness (new)- Effectiveness (current)

Slide 18: Quality-of-Life Considerations

• QALY=life expectancy x quality of remaining years
• Single score to evaluate health state based on mobility, pain, anxiety…
• Assigns utilities to health states
• Year of perfect health=1
• Death=0
• States may be worse than death (negative)

Slide 19: Standards for C-E Analysis

• Use societal perspective
• Include all health effects, costs that flow from intervention
• Include benefits and harms
• Use CE = Cost(new)- Cost (old)
• QALYs (new)-QALYs (old)

Slide 20: Incorporating Values into Policy

• Give preference to patients in poor health
• Give preference to vulnerable populations
• Give preference to interventions that are of large magnitude (even if few benefit)

Slide 21: What Might LVAD Decision-Making Look Like with Values, Cost?

• Start with scientific data
• Use cost-effectiveness threshold
• Vary C-E threshold depending on special circumstances

Slide 22: Cost-Effectiveness of LVAD

• CE ratio/QALY: best est. $802,700/QALY
• Low: $500,000/QALY
• High: $1.4 million/QALY
• Benchmark $50,000-$100,000/QALY
• Blue Cross/Blue Shield with Southern Permanente Health Plan and
• Kaiser Foundation

Slide 23: Policy Conclusions Using New Approach: Case of LVAD

• Start with scientific data; conclude small net benefit with many side-effects; consider rx
• Does intervention meet cost effectiveness threshold of $100,000/QALY?--NO
• Should threshold be modified to give preference to patients who are extremely ill?-YES
• Does intervention meet higher threshold, eg $200,000/QALY?-NO

Slide 24: From Observational Studies to Clinical Trials to Public Policy

• Start with science
• Add in cost-effectiveness
• Temper with explicit, previously agreed upon values

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