Measuring the “Q” in QALYs for cost- effectiveness analysis: the EuroQol Group’s approach Valuing health outcomes for healthcare decision making using

Measuring the “Q” in QALYs for cost-effectiveness analysis: the EuroQol

Group’s approach

Valuing health outcomes for healthcare decision making using the EQ-5D: 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]

3

Slides: www.busschbach.com

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 ownhealth statetoday

Best imaginable health state

Worst imaginable health state

9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0






9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

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

1) Two versions in target language

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

and fluent in target language

2) Comparison to the original English version

3) Second consensus version


1) Test second consensus by 8 lay respondents

- Native to the target language- Patients and healthy persons- Range of socio-demographic characteristics

2) Third consensus version


1. Forward Translation

2. Backward Translation

3. Respondent Testing

Final translation of EQ-5D

Translation Protocol

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






9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0






9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

9

8

7

6

5

4

3

2

1

0

1 0 0

0

0

0

0

0

0

0

0

0

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

• Example – Blindness– Time trade-off value is 0.5– Life span = 80 years– 0.5 x 80 = 40 QALYs

Quality Adjusted Life Years (QALY)

0.00

1.00

X

Life years40 80

0.5 x 80 = 40 QALYs

Area under the curve

30

QALY league tables

Intervention $ / QALYGM-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

32

9000 Citations in PubMed

0

200

400

600

800

1000

1200

1970 1980 1990 2000 2010 2020

Pu

bli

cati

on

s

1980[pdat] AND (QALY or QALYs)

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


38


– 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?





50

Coping: can be a problem in the patient perspective….

• 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

• Need for indirect valuation

Healthy

Death

51






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






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?





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

-0.8

-0.6

-0.4

-0.2

0

0.2

0.4

0.6

0.8

1

1111

1

2111

1

1211

1

1121

1

1112

1

1111

2

1112

2

2123

2

3221

1

2232

3

2223

3

3332

1

3333

3

DenmarkGermanyJapanNetherlandsSpainUKUSAZimbabwe

-0.4

-0.2

0

0.2

0.4

0.6

0.8

1

1111

1

2111

1

1211

1

1121

1

1112

1

1111

2

1112

2

2123

2

3221

1

2232

3

2223

3

3332

1

3333

3

BelgiumDenmarkFinlandNew ZealandSloveniaSpainUKEurope

Health State

Val

ue

Val

ue

Health State

64

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 1:1– EQ-5D 3L 1:5– QWB 1:44– EQ-5D 5L 1:62– SF-36 1:360– HUI (Mark III) 1:19,400– 15D 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 Actual levels– PF 6 5– RL 4 2– SF 5 5– PN 6 5– MH 5 4– VI 5 3

• Proposed Levels : 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

Documents

Measuring the “Q” in QALYs for cost- effectiveness analysis: the EuroQol Group’s approach Valuing health outcomes for healthcare decision making using