Measuring the “Q” in QALYs for costeffectiveness analysis: the EuroQol
Group’s approach
Valuing health outcomes for healthcare decision making using the EQ5D: a symposium for policy makers and researchers in Asia
Friday, 22 March 2013
Falcon Room, Level 3, Grand Copthorne Waterfront Hotel, Singapore
Prof. Dr. Jan J.V. Busschbach
• Chair of the EuroQol Research Foundation
• Erasmus MC
– Psychiatry
• Section Medical Psychology and Psychotherapy
– [email protected]
Slides: www.busschbach.com
3
The EQ-5D-3L questionnaire
‘Simplified’ Chinese version
The EQ-5D-3L questionnaire
T o h elp p eo p le sa y h o w go o d o r b ad a h ealth state is,
w e h ave d raw n a scale (rath er lik e a th erm o m eter) o n
w h ich th e b est state yo u can im agin e is m ark ed 1 0 0
an d th e w o rst state yo u ca n im agin e is m ark ed 0 .
W e w o u ld lik e yo u to in d icate o n th is scale h o w g o o d
o r b ad yo u r o w n h ealth is to d a y, in yo u r o p in io n .
P lease d o th is b y d raw in g a lin e fro m th e b o x b elo w to
w h ich ever p o in t o n th e sc ale in d icates h o w go o d o r b ad
yo u r h ealth state is to d a y.
Y ou r ow n
h ealth state
tod ay
B est
im agin ab le
h ealth state
100
9
0
8
0
7
0
6
0
5
0
4
0
3
0
2 0
0
1
0
W orst
im agin ab le
h ealth state
5
EuroQol jargon: state 11232
6
Moving from 3 levels to 5…
7
New developments
• Developing the 5 level ‘EQ-5D-5L’
• Improving the validation
– New methodology
• Standardizing the validation
– Standardizing methodology
– Allowing cultural values
8
The EuroQol Group
• Founded 25 years ago
• A network of about 100…
– International,
– multi-disciplinary,
– researchers
• Devoted to…
– measurement of health status,
– health related Quality of Life
9
Still ‘Euro’?
•
•
•
•
•
•
•
•
•
•
Australia
Canada
Denmark
Finland
France
Germany
Greece
Italy
Netherlands
New Zealand
•
•
•
•
•
•
•
•
•
•
Norway
Poland
Singapore
Slovenia
South Africa
Spain
Sweden
Trinidad & Tobago
United Kingdom
United States
10
Non commercial
• An non profit organization
– A foundation
– No stock holders
– Members votes for a Executive Board
• The EuroQol Office
– Executive Director: Dr. Bernhard Slaap
– www.euroqol.org
• All money goes into research
11
Income
• Pharmaceutical industry
– Subscriptions for 3 years
• Non commercial users
– Sometimes fees
• Any research
– Free
12
EuroQol Membership
• Reserved for those who actively support the
work of the EuroQol Group and make a
positive and sustained commitment to it
• Attend and scientifically contribute to the
EuroQol Plenary Meetings and participate in
Working Groups
• Access to research grants and annual
meeting
13
EuroQol Annual Plenary Meeting
14
EuroQol Annual Meeting
• Present papers and posters on:
–
–
–
–
Methodological / valuation aspects of EQ-5D
Development of new EQ-5D versions
Alternative modes of administration
Use of EQ-5D in health population surveys
15
EuroQol Executive Office
16
EuroQol Office
• Handles EQ-5D license requests
• Scientific support clients
• Scientific and operational support EQ sponsored
studies
• EQ members support
• Based in Rotterdam
– The Netherlands
• 5.0 FTE + contractors
17
Translation Protocol
1) Two versions in target language
1. Forward Translation
Translators should be native in target
language and fluent in English
2) First consensus version
3) Report to EuroQol Group
1) Two versions in English
Translators should be native in English
2. Backward Translation
and fluent in target language
2) Comparison to the original English version
3) Second consensus version
4) Report to EuroQol Group
1) Test second consensus by 8 lay respondents
3. Respondent Testing
- Native to the target language
- Patients and healthy persons
- Range of socio-demographic characteristics
2) Third consensus version
3) Report to EuroQol Group
Final translation of EQ-5D
18
Certified language versions
• All produced following recommended guidelines for cultural
adaptation + rating scale exercise
• Translation certificates provided for all versions
-19-
EQ-5D User Guides
20
EQ-5D Paper version
EQ-5D-3L descriptive system
EQ-5D-3L VAS
T o h elp p eo p le sa y h o w go o d o r b ad a h ealth state is,
w e h ave d raw n a scale (rath er lik e a th erm o m eter) o n
w h ich th e b est state yo u can im agin e is m ark ed 1 0 0
an d th e w o rst state yo u ca n im agin e is m ark ed 0 .
W e w o u ld lik e yo u to in d icate o n th is scale h o w g o o d
o r b ad yo u r o w n h ealth is to d a y, in yo u r o p in io n .
P lease d o th is b y d raw in g a lin e fro m th e b o x b elo w to
w h ich ever p o in t o n th e sc ale in d icates h o w go o d o r b ad
yo u r h ealth state is to d a y.
Y ou r ow n
h ealth state
tod ay
B est
im agin ab le
h ealth state
100
9
0
8
0
7
0
6
0
5
0
4
0
3
0
2 0
0
1
0
W orst
im agin ab le
h ealth state
21
Other formats
Tablet, PDA, Web
-22-
Other formats
Tablet, PDA, Web
-23-
EQ-5D Web
24
EuroQol instruments
• EQ-5D-3L Translations
– More than 160 languages in Self-complete paper format
– Also available in; Telephone, Face-to-face, Proxy, IVR, Web
and Tablet format
• EQ-5D-5L Translations
– More than 90+ languages in self-complete paper format
– Also available in Web and Tablet format
• EQ-5D-Y Translations
– Available in more than 20 languages
– Youth between 7-12 years
25
Overview of the EQ-5D
Purpose and origins of the
descriptive system
26
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
27
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”
– “Hart failure” versus “second psychosis”
• Generic outcome are compatible
– Allow for comparisons between fields
• Life years
• Quality of life
• Most generic outcome
– Quality adjusted life year (QALY)
28
Quality Adjusted Life Years
(QALY)
• Example
1.00
–
–
–
–
Blindness
Time trade-off value is 0.5
Life span = 80 years
0.5 x 80 = 40 QALYs
X
0.5 x 80 = 40 QALYs
0.00
40
80
Life years
Area under the curve
30
QALY league tables
In te rv e n tio n
$ / QALY
G M -C S F in e ld e rly w ith le u ke m ia
2 3 5 ,9 5 8
E P O in d ia lys is p a tie n ts
1 3 9 ,6 2 3
L u n g tra n sp la n ta tio n
1 0 0 ,9 5 7
E n d sta g e re n a l d is e a s e m a n a g e m e n t
5 3 ,5 1 3
H e a rt tra n sp la n ta tio n
4 6 ,7 7 5
D id ro n e l in o ste o p o ro sis
3 2 ,0 4 7
P T A w ith S te n t
1 7 ,8 8 9
B re a st ca n c e r scre e n in g
5 ,1 4 7
V ia g ra
5 ,0 9 7
T re a tm e n t o f co n g e n ita l a n o re cta l m a lfo rm a tio n s
2 ,7 7 8
31
9000 Citations in PubMed
1980[pdat] AND (QALY or QALYs)
1200
Publications
1000
800
600
400
200
0
1970
1980
1990
2000
2010
2020
32
Most controversy about the ‘Q’
in QALY
• An uni-dimensional value
– Like temperature, of km/h
– Like the IQ-test measures intelligence
• Ratio or interval scale
– Difference 0.00 and 0.80…
– … must be 8 time higher than 0.10
33
Unidimensional, ratio scales
• Two popular methods have these
pretensions
– Time trade-off
– Standard gamble
• Two methods are less clear….
– Visual analog scale
– Paired comparison
• Conjoint analysis; DCE, etc
34
The Rosser & Kind Index
35
The Rosser & Kind index
• One of the oldest valuation
• 1978: Magnitude estimation
– Magnitude estimation  PTO / VAS
– N = 70
• Doctors, nurses, patients and general public
• 1982: Transformation to “utilities”
– On a 0.00 to 1.00 scale
– Could be used for QALYs
36
1985: High impact article
37
1985: High impact article
38
1985: High impact article
– Survey at the celebration of 25 years of health
economics in the UK (HESG): chosen most influential
article on health economics
39
Criticism on the Matrix
• Sensitivity
– only 30 health states
• The unclear meaning of “distress”
• The involvement of medical personnel
• No clear way how to classify the patients
– into the matrix
• Only British values
• The compression of states in the high
values
40
Value compression
41
New initiatives
• Higher sensitivity (more then 30 states)
• More and better defined dimensions
• Other valuation techniques
– Standard Gamble, Time Trade-Off, Visual Analogue Scale
• Values of the general public
• A questionnaire…
– to allow patients to ‘self classify’ themselves
• An international standard
– to allow international comparisons
– That is at that time “Europe”
42
EuroQoL Group
• First meeting 1987
• Participants from
– UK, Finland, Sweden, The Netherlands
• A common core instrument
– To standardize the instrument
• But allow different national values
– To allow international comparisons
– To allow linking of international results
• Instrument should be small
• Suitable for sever ill patients
– The emerging of high tech medicine, especially
transplantation
43
The first EuroQol
• Higher sensitivity (more then 30 states)
– 216 states
• More and better defined dimensions
– 6 dimensions (EQ-6D)
–
–
–
–
–
–
Mobility;
Daily activity and self care;
Work performance
Family and leisure performance
Pain/discomfort
Present mood
• Visual Analogue Scale
44
The first EuroQol
• Values of the general public
– Values from general public
– But also values from patients (!)
• A questionnaire
– to allow patients to ‘self classify’ themselves
• A international standard
– to allow international comparisons
– That is at that time “Europe”
45
Values from the patients
46
Values from the general public
47
First values general public
Why values of the general public?
• Original: To avoid ‘strategic responses’
– Patients pressure groups
• To avoid coping
– Underestimating the value of health
• To allow complex utility assessments
–
–
–
–
–
Time Trade Off
Standard Gamble
Willingness to pay
Person Trade off
Paired comparisons (DCE)
• To allow for societal values of health states
– Like costs: the societal perspective
49
Why indirect utility measures?
• Original: To avoid ‘strategic responses’
– Patients pressure groups
• To avoid coping
– Underestimating the value of health
• To allow complex utility assessments
–
–
–
–
–
Time Trade Off
Standard Gamble
Willingness to pay
Person Trade off
Paired comparisons (DCE)
• To allow for societal values of health states
– Like costs: the societal perspective
50
Coping: can be a problem in the
patient perspective….
• Stensman
Healthy
– 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
• Need for indirect
valuation
Death
51
Why values of the general public?
• Original: To avoid ‘strategic responses’
– Patients pressure groups
• To avoid coping
– Underestimating the value of health
• To allow complex utility assessments
–
–
–
–
–
Time Trade Off
Standard Gamble
Willingness to pay
Person Trade off
Paired comparisons (DCE)
• To allow for societal values of health states
– Like costs: the societal perspective
52
Time Trade-Off
• TTO: alternative for VAS
• 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) = .80
53
Health economics prefer TTO
• Visual analogue scale
– No trade-off: no relation to QALY
• No interval proportions
– Easy
• Time trade-Off
– Trade-off: clear relation to QALY
• Interval proportions
– Less easy
• Time consuming in patients
• Need for indirect valuation
54
Why values of the general public?
• Original: To avoid ‘strategic responses’
– Patients pressure groups
• To avoid coping
– Underestimating the value of health
• To allow complex utility assessments
–
–
–
–
–
Time Trade Off
Standard Gamble
Willingness to pay
Person Trade off
Paired comparisons (DCE)
• To allow for societal values of health states
– Like costs: the societal perspective
55
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….?
56
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
57
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?
58
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
59
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
60
The general public should be
informed…
• Valuing without knowledge makes no
sense
– Thyroid Eye Disease
• Give description of the disease
– For instance in terms of the EQ-5D
A patient with bilateral thyroid
eye disease with upper lid
retraction and exophthalmos.
61
Why indirect utility measures?
• Original: To avoid ‘strategic responses’
– Patients pressure groups
• To avoid coping
– Underestimating the value of health
• To allow complex utility assessments
–
–
–
–
–
Time Trade Off
Standard Gamble
Willingness to pay
Person Trade off
Paired comparisons (DCE)
• To allow for societal values of health states
– Like costs: the societal perspective
62
Indirect utility measrue
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
63
EQ-5D-3L Value Sets
TTO Value Sets
VAS Value Sets
1
1
0.8
0.8
0.6
0.4
0.2
0
-0.2
-0.4
Value
Denmark
Germany
Japan
Netherlands
Spain
UK
USA
Zimbabwe
Belgium
Denmark
Finland
New Zealand
Slovenia
Spain
UK
Europe
0.4
0.2
0
-0.2
-0.6
-0.8
Health State
Health State
64
33333
33321
22233
22323
32211
21232
11122
11112
11121
11211
12111
21111
11111
33333
33321
22233
22323
32211
21232
11122
11112
11121
11211
12111
21111
-0.4
11111
Value
0.6
Values from the patients
65
Values from the general public
66
Why use the EQ-5D?
What are the alternatives?
67
Validated questionnaires
Rosser & Kind
EuroQol EQ-5D
www.euroqol.org
QWB
SF-36 (SF-6D)
www.sf-36.org
HUI Mark 2
HUI Mark 3
www.healthutilities.com
15D
www.15d-instrument.net
68
The Rosser & Kind Index
69
The Rosser & Kind index
• Criticism on the Rosser & Kind index
– Sensitivity (only 30 health states)
• New initiatives
– Higher sensitivity (more then 30 states)
• EuroQol Group
– EQ-5D-3L and the EQ-5D-5L
• McMaster University
– Health Utility Index 2 & 3
• SF-36
– SF-6D
70
Health Utility Index
• Developed from pediatric care
– Strong proxy versions
• Symptom driven:
– “Outside the skin” instead of “inside the skin”
• EQ-5D: “problems with daily activity”
• HUI: “Unable to read ordinary newsprint…”
• Commercial
– All user have to pay
• 35 Translations
71
HUI 2
72
HUI 3
73
Increasing number of health states
Questionnaire
Number of states
Rosser & Kind Matrix
30
EQ-5D 3L
243
Quality of Well Being Scale (QWB)
2,200
EQ-5D 5L
3,125
SF-6D (SF-36)
18,000
HUI Mark 2
24,000
HUI Mark 3
972,000
15 D
3,052,000,000
74
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)
75
Regression techniques
• Value a sample of states empirically
• Extrapolation
– Statistical methods, like linear regression
– 11111 = 1.00
– 11113 = .70
– 11112 = ?
76
Gets complex if states increases
• Moving from 3 levels to 5….
• Extrapolation
– Statistical methods, like linear regression
– 11111 = 1.00
– 11115 = .70
– 11112 = ?
– 11113 = ?
– 11114 = ?
77
More health states, more
assumptions
• General public values at the most 50 states
• The ratios empirical (50) versus
extrapolated
–
–
–
–
–
–
–
Rosser & Kind
EQ-5D 3L
QWB
EQ-5D 5L
SF-36
HUI (Mark III)
15D
1:1
1:5
1:44
1:62
1:360
1:19,400
1:610,000,000
• What is the critical ratio for a valid
validation?
78
Conflicting evidence sensitivity SF-6D
Liver transplantation, Longworth et al., 2001
79
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
80
Some levels in the SF-6D do not
work…
Dimension: Physical Functioning (PF)
If PF=1
decrement: 0
If PF=2
decrement: - 0.056
If PF=3
decrement: - 0.056
If PF=4 *
decrement: - 0.072
If PF=5 *
decrement: - 0.080
If PF=6 *
decrement: - 0.134
Dimension: Role Limitations (RL)
If RL=1
decrement: 0
If RL=2
decrement: - 0.073
If RL=3 *
decrement: - 0.073
If RL=4 *
decrement: - 0.073
81
SF-6D loses a lot of levels
• Proposed Levels
–
–
–
–
–
–
PF
RL
SF
PN
MH
VI
Actual levels
6
4
5
6
5
5
• Proposed Levels
5
2
5
5
4
3
: 18.000
– 6x4x5x6x5x5
• Actual levels
: 480
– 5x2x5x5x4x3
82
EQ-5D
• Strong punts
–
–
–
–
–
–
–
Very sensitive in the low
Measures subjective burden (inside the skin)
Low administrative burden
Many translations
Cheap
Most used QALY questionnaire
Most international validations
• Weak points
– Only there levels per dimensions
– Insensitive in the high regions
83
HUI
• Strong punts
–
–
–
–
Sensitive
Measures objective burden (outside the skin)
Well developed proxy versions
Well developed child versions
• Weak points
– Expensive
– Only a few valuation studies
84
SF-6D
• Strong punts
– Probably sensitive in the high regions
– Often already include in trials (SF-36)
– Many translations
• Weak points
– Insensitive in the low regions
– Only a few validation study
– Might be expensive
85
Conclusions
• More states  better sensitivity
• The three leading questionnaires
– have different strong and weak points
86
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The EQ-5D-3L questionnaire