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 To help people say how good or bad a health state is, we have drawn a scale (rather like a thermometer) on which the best state you can imagine is marked 100 and the worst state you can imagine is marked 0. We would like you to indicate on this scale how good or bad your own health is today, in your opinion. Please do this by drawing a line from the box below to whichever point on the scale indicates how good or bad your health state is today.
Your own health state today
Best imaginable health state 100 9
0
8
0
7
0
6
0
5
0
4
0
3
0
2 0 1 0
0 Worst imaginable health 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 To help people say how good or bad a health state is, we have drawn a scale (rather like a thermometer) on which the best state you can imagine is marked 100 and the worst state you can imagine is marked 0. We would like you to indicate on this scale how good or bad your own health is today, in your opinion. Please do this by drawing a line from the box below to whichever point on the scale indicates how good or bad your health state is today.
Your own health state today
Best imaginable health state 100 9
0
8
0
7
0
6
0
5
0
4
0
3
0
2 0 1 0
0 Worst imaginable health 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 Intervention
$ / QALY
GM-CSF in elderly with leukemia
235,958
EPO in dialysis patients
139,623
Lung transplantation
100,957
End stage renal disease management
53,513
Heart transplantation
46,775
Didronel in osteoporosis
32,047
PTA with Stent
17,889
Breast cancer screening
5,147
Viagra
5,097
Treatment of congenital anorectal malformations
2,778
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
