1. 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

2. Prof. Dr. Jan J.V. Busschbach Chair of the EuroQol Research Foundation Erasmus MC –Psychiatry Section Medical Psychology and Psychotherapy –J.vanbusschbach@erasmusmc.nl

3. Slides: www.busschbach.com 3

4. The EQ-5D-3L questionnaire ‘Simplified’ Chinese version

5. The EQ-5D-3L questionnaire 5

6. EuroQol jargon: state 11232 6

7. Moving from 3 levels to 5… 7

8. New developments Developing the 5 level ‘EQ-5D-5L’ Improving the validation –New methodology Standardizing the validation –Standardizing methodology –Allowing cultural values 8

9. 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

10. 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

11. 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

12. Income Pharmaceutical industry –Subscriptions for 3 years Non commercial users –Sometimes fees Any research –Free 12

13. 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

14. EuroQol Annual Plenary Meeting 14

15. 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

16. EuroQol Executive Office 16

17. 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

18. 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 4)Report to EuroQol Group 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 3)Report to EuroQol Group 1. Forward Translation 2. Backward Translation 3. Respondent Testing Final translation of EQ-5D Translation Protocol 18

19. Certified language versions All produced following recommended guidelines for cultural adaptation + rating scale exercise Translation certificates provided for all versions -19-

20. EQ-5D User Guides 20

21. EQ-5D Paper version EQ-5D-3L descriptive systemEQ-5D-3L VAS 21

22. Other formats Tablet, PDA, Web -22-

23. Other formats Tablet, PDA, Web -23-

24. EQ-5D Web 24

25. 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

26. Overview of the EQ-5D Purpose and origins of the descriptive system 26

27. 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

28. 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

29. 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 years 40 80 0.5 x 80 = 40 QALYs

30. Area under the curve 30

31. QALY league tables 31

32. 32 9000 Citations in PubMed

33. 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

34. 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

35. The Rosser & Kind Index 35

36. 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

37. 1985: High impact article 37

38. 1985: High impact article 38

39. 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

40. 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

41. Value compression 41

42. 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

43. 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

44. 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

45. 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

46. Values from the patients 46

47. Values from the general public 47

48. First values general public

49. 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

50. 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

51. 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. 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

53. 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

54. 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. 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

56. 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

57. 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

58. 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

59. 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

60. 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

61. 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

62. 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

63. 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

64. EQ-5D-3L Value Sets TTO Value SetsVAS Value Sets Health State Value Health State 64

65. Values from the patients 65

66. Values from the general public 66

67. Why use the EQ-5D? What are the alternatives? 67

68. Validated questionnaires 68

69. The Rosser & Kind Index 69

70. 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

71. 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

72. HUI 2 72

73. HUI 3 73

74. Increasing number of health states QuestionnaireNumber of states Rosser & Kind Matrix30 EQ-5D 3L243 Quality of Well Being Scale (QWB)2,200 EQ-5D 5L3,125 SF-6D (SF-36)18,000 HUI Mark 224,000 HUI Mark 3972,000 15 D3,052,000,000 74

75. 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

76. Regression techniques Value a sample of states empirically Extrapolation –Statistical methods, like linear regression –11111 = 1.00 –11113 =.70 –11112 = ? 76

77. 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

78. More health states, more assumptions General public values at the most 50 states The ratios empirical (50) versus extrapolated –Rosser & Kind1:1 –EQ-5D 3L1:5 –QWB1:44 –EQ-5D 5L1:62 –SF-361:360 –HUI (Mark III)1:19,400 –15D1:610,000,000 What is the critical ratio for a valid validation? 78

79. Conflicting evidence sensitivity SF-6D Liver transplantation, Longworth et al., 2001 79

80. 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

81. Some levels in the SF-6D do not work… 81

82. SF-6D loses a lot of levels Proposed Levels Actual levels –PF6 5 –RL 42 –SF 55 –PN 65 –MH 54 –VI 53 Proposed Levels: 18.000 –6x4x5x6x5x5 Actual levels: 480 –5x2x5x5x4x3 82

83. 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

84. 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

85. 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

86. Conclusions More states  better sensitivity The three leading questionnaires –have different strong and weak points 86