Transcript Intro QALY & need assessment
Health Economics
Comparing different allocations
Should we spent our money on • Wheel chairs • Screening for cancer Comparing costs Comparing outcome
Outcomes must be comparable
Make a generic outcome measure 1
Outcomes in health economics
Specific outcome are incompatible
Allow only for comparisons within the specific field • Clinical successes: successful operation, total cure • Clinical failures: “events”
Generic outcome are compatible
Allow for comparisons between fields • Life years • Quality of life
Most generic outcome
Quality adjusted life year (QALY) 2
Quality Adjusted Life Years (QALY)
Multiply life years with quality index
Quality of life index
1.0 = normal health 0.0 = death (extremely bad health)
Example
Losing sense of sight Quality of life index is 0.5
Life = 80 years 0.5 x 80 = 40 QALYs 3
Which health care program is the most cost-effective?
A new wheelchair for elderly (iBOT)
Special post natal care
4
Which health care program is the most cost-effective?
A new wheelchair for elderly (iBOT)
Increases quality of life = 0.1 10 years benefit Extra costs: $ 3,000 per life year QALY = Y x V(Q) = 10 x 0.1 = 1 QALY Costs are 10 x $3,000 = $30,000 Cost/QALY = 30,000/QALY
Special post natal care
Quality of life = 0.8
35 year Costs are $250,000 QALY = 35 x 0.8 = 28 QALY Cost/QALY = 8,929/QALY 5
QALY league table Intervention
GM-CSF in elderly with leukemia EPO in dialysis patients Lung transplantation End stage renal disease management Heart transplantation Didronel in osteoporosis PTA with Stent Breast cancer screening Viagra Treatment of congenital anorectal malformations
$ / QALY
235,958 139,623 100,957 53,513 46,775 32,047 17,889 5,147 5,097 2,778 6
1.0
Egalitarian Concerns: Burden of disease 0.0
A B C
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CE-ratio by equity
8
Burden as criteria 30 25 20 15 10 5 0 Accepted High burden Rejected Low burden Pronk & Bonsel, Eur J Health Econom 2004, 5: 274-277
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What form of equity?
Choice
5 year old 35 year old 2 year old Single Smoker Heavy drinker Woman Unemployed Employed Director 70 year old
First strongly preferred to second
57 60 year old 8 year old Married Non-Smoker Light drinker Man Unskilled 29 4 3 6 5 4 3 5 Lorry driver Teacher With children No children 2 27
First slightly preferred to second
23 42 5 3 8
Unable to choose
14 27 70 69 43
Second slightly preferred to first
5 1 16 20 30 6 4 3 8 2 38 25 90 81 83 86 31 41 1 10 3 8 3
Second strongly preferred to first
2 0 5 5 12 23 0 2 0 3 1 10
3500 Citations in PubMed 1980[pdat] AND (QALY or QALYs)
500 450 400 350 300 250 200 150 100 50 0 1980 1985 1990 1995 2000 2005 2010 11
Top 6 journals Cost Utility Analysis Am J Med Med Decis Making Int J Technol Assess JAMA Ann Intern Med Pharmacoeconomics 0 www.tufts-nemc.org/cearegistry 10 20 30 # publications 1976 - 2003 40 50
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Most debate about the QoL estimates
Unidimensional QoL
In QALY we need a unidimensional assessment of Quality of life
Rules out multidimensional questionnaires
SF-36, NHP, WHOQOL
100 90 80 70 60 50 40 30 20 10 0 P hy si ca l f un ct ion ing S oc ia l Fu nc tion in g R ol e P hy si ca R ol l e em ot io na l M ent al he al th V ita lit y B od ily pa in G ene ra l H ea lth General pop.
Diabetes II Growth hormon def.
Depression
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Utility assessment
Unidimensional QoL
Often called ‘utility’
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Who to ask?
The patient, of course!
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The clinical perspective
Quality of life is subjective…..
“Given its inherently subjective nature, consensus was quickly reached that quality of life ratings should, whenever possible, be elicited directly from patients themselves. “ • (Niel Aaronson, in B. Spilker: Quality of life and Pharmacoeconomics in Clinical Trails, 1996, page 180)
…therefore ask the patient!
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Patient values count….
[…] the best way to do this, the technology, is a patient-based assessment. They report, they evaluate, they tell you in a highly standardized way, and that information is used with the clinical data and the economic data to get the best value for the health care dollar.”
John Ware
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A problem in the patient perspective….
Healthy
Stensman
Scan J Rehab Med 1985;17:87-99.
Scores on a visual analogue scale
36 subjects in a wheelchair 36 normal matched controls
Mean score
Wheelchair: 8.0
Health controls: 8.3
Death 18
The economic perspective
In a normal market: the consumer values count
The patient seems to be the consumer
Thus the values of the patients….
If indeed health care is a normal market…
But is it….?
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Health care is not a normal market
Supply induced demands
Government control
Financial support (egalitarian structure)
Patient
Consumer
The patient does not pay
Consumer = General public
Potential patients are paying
Health care is an insurance market
A compulsory insurance market 20
Health care is an insurance market
Values of benefit in health care have to be judged from a insurance perspective
Who values should be used the insurance perspective?
21
Who determines the payments of unemployment insurance?
Civil servant
Knowledge: professional But suspected for strategical answers • more money, less problems • identify with unemployed persons
The unemployed persons themselves
Knowledge: specific But suspected for strategical answers
General public (politicians)
Knowledge: experience Payers 22
Who’s values (of quality of life) should count in the health insurance?
Doctors
Knowledge: professional But suspected for strategical answers • See only selection of patient • Identification with own patient
Patients
Knowledge: disease specific But suspected for strategical answers But coping
General public
Knowledge: experience Payers Like costs: the societal perspective 23
Validated questionnaires
MOBILITY I have no problems in walking about I have some problems in walking about I am confined to bed SELF-CARE I have no problems with self-care I have some problems washing or dressing myself I am unable to wash or dress myself USUAL ACTIVITIES (e.g. work, study, housework family or leisure activities) I have no problems with performing my usual activities I have some problems with performing my usual activities I am unable to perform my usual activities PAIN/DISCOMFORT I have no pain or discomfort I have moderate pain or discomfort I have extreme pain or discomfort ANXIETY/DEPRESSION I am not anxious or depressed I am moderately anxious or depressed I am extremely anxious or depressed 24
Validated Questionnaires
Describe health states
Have values from the general public
Rosser Matrix QWB 15D HUI Mark 2 HUI Mark 3 EuroQol EQ-5D 25
EQ-5D, HUI and SF-36 Of the shelf instruments….
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Validated questionnaires Rosser EuroQol EQ-5D QWB SF-36 (SF-6D) HUI Mark 2 HUI Mark 3 15D www.euroqol.org www.sf-36.org www.15d-instrument.net
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The Rosser & Kind Index
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The Rosser & Kind index
One of the oldest valuation
1978: Magnitude estimation
Magnitude estimation PTO N = 70: Doctors, nurses, patients and general public
1982: Transformation to “utilities”
1985: High impact article
Williams A. For Debate... Economics of Coronary Artery Bypass Grafting. British Medical Journal 291: 326-28, 1985.
Survey at the celebration of 25 years of health economics: chosen most influential article on health economics 29
More health states
Criticism on the Rosser & Kind index
Sensitivity (only 30 health states) The unclear meaning of “distress” The compression of states in the high values The involvement of medical personnel
New initiatives
Higher sensitivity (more then 30 states) More and better defined dimensions Other valuation techniques • Standard Gamble, Time Trade-Off Values of the general public 30
Validated questionnaires Questionnaire Rosser EuroQol EQ-5D Number of health state 30 245 QWB SF-36 (SF-6D) HUI Mark 2 HUI Mark 3 15D 2,200 9,000 24,000 972,000 3,052,000,000
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No longer value all states
Impossible to value all health states
If one uses more than 30 health states
Estimated the value of the other health states with statistical techniques
Statistically inferred strategies • Regression techniques • EuroQol, Quality of Well-Being Scale (QWB) Explicitly decomposed methods • Multi Attribute Utility Theory (MAUT) • Health Utility Index (HUI) 32
Statistically inferred strategies
Value a sample of states empirically
Extrapolation
Statistical methods, like linear regression 11111 = 1.00
11113 = .70
11112 = ?
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Explicitly Decomposed Methods
Value dimensions separately
Between the dimensions What is the relative value of: • Mobility…... 20% • Mood…….. 15% • Self care.… 24%.
Value the levels
Within the dimensions What is the relative value of • Some problems with walking…… 80% • Much problems with walking…... 50% • Unable to walk…………………….10% 34
Explicitly Decomposed Methods
Combine values of dimensions and levels with specific assumptions
Multi Attribute Utility Theory (MAUT) • Mutual utility independence • Structural independence 35
Explicitly Decomposed Methods
Health Utilities Index (Mark 2 & 3)
Torrance at McMaster 8 dimensions Mark 2: 24.000 health states Mark 3: 972.000 health states
The 15-D
Sintonen H.
15 dimensions 3,052,000,000 health states (3 billion) 36
More health states, higher sensitivity ? (1)
EuroQol criticised for low sensitivity
Low number of dimensions • Development of EQ-5D plus cognitive dimension Low number of levels (3) • Gab between best and in-between level 37
More health states, higher sensitivity ? (2)
Little published evidence
Sensitivity EQ-5D < SF-36 • Compared as profile, not as utility measure Sensitivity EQ-5D HUI
Sensitivity
the number of health states
How well maps the classification system the illness?
How valid is the modelling?
How valid is the valuation? 38
More health states, more assumptions
General public values at the most 50 states
The ratios empirical (50) versus extrapolated
Rosser & Kind 1:1 EuroQol QWB SF-36 HUI (Mark III) 15D 1:5 1:44 1:180 1:19,400 1:610,000,000
What is the critical ratio for a valid validation?
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SF-36 as utility instrument
Transformed into SF6D
SG
N = 610
Inconsistencies in model
18.000 health states regression technique stressed to the edge
Floor effect in SF6D
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Conflicting evidence sensitivity SF-36 Liver transplantation, Longworth et al., 2001
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Conclusions
More states
better sensitivity
The three leading questionnaires
have different strong and weak points 42
Value a health state
Wheelchair
Some problems in walking about Some problems washing or dressing Some problems with performing usual activities Some pain or discomfort No psychosocial problems 43
Uni-dimensional value
Like the IQ-test measures intelligence
Ratio or interval scale
Difference 0.00 and 0.80 must be 8 time higher than 0.10
Three popular methods have these pretensions
Visual analog scale Time trade-off Standard gamble 44
Visual Analogue Scale
VAS
Also called “category scaling” From psychological research
“How is your quality of life?”
“X” marks the spot
Rescale to [0..1]
Different anchor point possible:
Normal health (1.0) versus dead (0.0) Best imaginable health versus worse imaginable health
Normal health
X
Dead
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Time Trade-Off
TTO
Wheelchair
With a life expectancy: 50 years
How many years would you trade-off for a cure?
Max. trade-off is 10 years
QALY(wheel) = QALY(healthy)
Y * V(wheel) = Y * V(healthy) 50 V(wheel) = 40 * 1
V(wheel) = .8
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Standard Gamble
SG
Wheelchair
Life expectancy is not important here
How much are risk on death are you prepared to take for a cure?
Max. risk is 20% wheels = (100%-20%) life on feet V(Wheels) = 80% or .8
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Consistent picture of difference
1.0
0.9
0.8
0.7
0.6
0.5
0.4
0.3
0.2
0.1
0.0
103 students
SG TTO VAS 12 11 1 11 21 1 21 11 1 11 12 1 11 11 2 11 12 2 32 21 1 21 23 2 33 32 1 22 32 3 22 23 3 33 33 3
EQ-5D health states
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Health economics prefer TTO/SG
Visual analogue scale
Easy No trade-off: no relation to QALY • No interval proportions
Standard Gamble / Time trade-Off
Less easy Trade-off: clear relation to QALY • Interval proportions
Little difference between SG and TTO
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Little difference between Cost/Life Year and Cost/QALY Richard Chapman et al, 2004, Health Economics
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Difference in QALYs makes little difference in outcome
Richard Chapman et al, 2004
“In a sizable fraction of cost-utility analyses, quality adjusting did not substantially alter the estimated cost-effectiveness of an intervention, suggesting that sensitivity analyses using ad hoc adjustments or 'off-the-shelf' utility weights may be sufficient for many analyses.” “The collection of preference weight data should […] should only be under-taken if the value of this information is likely to be greater than the cost of obtaining it.” 51
QALYs make a difference when:
Chronic disease
Palliative
Long term negative consequences
52
Conclusions
SG/TTO are preferred in Health economics
Reproducible results
Problems in QALYs are overestimated
Difference in QALYs makes little difference in outcome • Compared to cost per life year • With exception of chronic illness 53