Transcript The Panel on Cost-Effectiveness in Health and

The Panel on Cost-Effectiveness in
Health and Medicine
Marthe Gold
City University of London
30 October, 2003
U.S. Department of Health and Human Services
Health and Human Services
ASPE
Public Health Service
NIH
CDC
NCHS
AHRQ
SAMHSA
HRSA
Health Care
Financing Administration
FDA
IHS
ACF
Context:
Federal Initiatives
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Office of Technology Assessment (Congress)
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Health Care Financing Administration
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Cost-effectiveness analyses of preventive services
Oregon Medicaid Waiver
Coverage decision regs
Agency for Health Care Policy and Research
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Guidelines
Technology assessments
Context:
Federal Initiatives
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National Institutes of Health
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Centers for Disease Control and Prevention
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Clinical trials
CEAs of preventive strategies
State requests for local decision making
Food and Drug Administration
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Regulatory review of drug marketing claims
Disarray in the Field…..
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Cost-effectiveness methods incomplete/non
standardized. Udvarhelyi S, et al, 1992
Breast cancer screening ratios range from
cost saving to $84k/YLS. Brown ML and L Fintor,
1993
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Oregon’s priority list a failure due to technical
problems in CEA. Eddy, D. 1991
Source of Problems
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Flaws in methods
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Perspective not identified
Inappropriate choice of comparator
Inadequate or non-generalizable cost/effectiveness
data
No “discounting”
Uncertainty unaccounted for
Source of Problems
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Differences in investigator approach
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Perspective differs
Non-comparable outcome measures
Differences in how future costs of health care
unrelated to the intervention are handled
The Panel On Cost-Effectiveness in
Health and Medicine: Charge

Assess the current practice of CEA
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Provide recommendations to improve quality,
comparability and utility of studies in the service of
decision making
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Identify unresolved methodological issues
PCEHM:
Reference Case Analysis
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“Base case” analysis for analyses designed to
inform resource allocation decisions
Defined by a standard set of methods and
assumptions
Recommendations for methods drawn from
consistent and theoretically grounded series of
considerations
A CEA may be valid without following RC
methodology.
Recommendation Rationales

Theoretical
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Ethical
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theoretical considerations drawn from welfare
economics and expected utility theory
ethical considerations justifying deviation from
strictly interpreted welfare economic theory
Accounting consistency
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logical consistency/avoidance of double counts
Recommendation Rationales
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Pragmatic
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Conventional
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best empirical evidence and consideration of the
practical limitations of current techniques
conformance to, or establishment of, a convention
to produce standardized procedure
User needs
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responds to particular needs of decision makers
PCEHM Recommendation:
Perspective
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The Reference Case should be based on the
societal perspective
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Everything counts - (costs and benefits)
“The public interest” viewed ex ante
Provides a benchmark against which to assess
results from other perspectives
PCEHM Recommendations:
Outcomes

Morbidity and mortality consequences
incorporated into a single measure using QALYs

Preferences (values) should be drawn from a
representative sample of the community
– Consistent with the societal perspective
HALYs for 5 Conditions using HALex, QWB, and DALY weights
300,000
250,000
200,000
YHL
150,000
QWB
DALY
100,000
50,000
Stroke
Diabetes
Gold MR and P Muennig. Med Care, 2002
COPD
PUD
Asthma
PCEHM Recommendations:
Costs
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Costs reflected in the numerator should
include: health care services; time patients
expend receiving care; care giving; other
related associated with the illness; non-health
impacts of the intervention
Include or exclude costs associated with
diseases other than those affected in added
years of life
PCEHM Recommendations: Comparators

The reference case should compare the health
intervention of interest to existing practice
(status quo)
Cost-effectiveness in decision making
for resource allocation
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CEA not an “answer” to a resource allocation
decision
Other values must enter in, including:
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Fairness in distribution of resources, priority to
disadvantaged (e.g., sick, poor, aged)
These values can not easily be embedded in the
CEA methodology
Decisions must represent the convergence of
many views
Seven years pass….
What’s new?
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In the medical literature, evidence that quality
of CEA studies has improved
AHRQ and CDC include information about CE
in their assessments of community-based and
clinical preventive services
No impact on Congressional decision-making
No (explicit) change in the policies of CMS
On the horizon….
Office of Management and Budget
“BCA is an evolving discipline, but one which the
administration believes provides important insight
into the design of smart regulations……OMB’s final
guidance will also promote CEA…it’s advantage is
it does not require analysts to determine the
monetary cost of life-saving: it reserves that
judgment for accountable policy officials.”
(Federal Register, March 2003)
On the horizon….?
Centers for Medicare and Medicaid
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Huge growth in program costs
Huge budget deficit
Addition of pharmaceutical benefits
How will the U.S. pay for this?
Health Care Spending per Capita
Adjusted for Cost-of-Living Differences, 2001
$6,000
$5,000
$4,887
$4,000
$2,808 $2,792
$3,000
$2,626
$2,350
$2,561
$1,984
$2,000
$2,191
$1,992
$1,710
$1,000
•2000 OECD estimate
OECD Data
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$0
U.S. Health Expenditures, 1965-2000
Trillions of Dollars
1.40
1.20
1.00
0.80
0.60
0.40
0.20
19
65
19
68
19
71
19
74
19
77
19
80
19
83
19
86
19
89
19
92
19
95
19
98
0.00
Source: National Expenditure Accounts