Preference-Based Health-Related Quality of Life Measures Ron D. Hays, Ph.D. January 26, 2015 (9:00-11:50 am) HPM

Where we are now in HPM214 1.Introduction 2.Profile Measures (SF-36 due) 3.Preference-Based Measures  4.Designing Measures 5.Evaluating Measures 6.PROMIS/IRT/Internet Panels 7.Reviews of Manuscripts 8.Course Review (Cognitive interviews due) 9.Final Exam (3/16/15) 2

ScaleNMeanSDMinMax Physical functioning (10 items) Role functioning - Physical (4 items) Pain scale (2 items) General health perceptions (5 items) Emotional well-being (MHI, 5 items) Role functioning - Emotional (3 items) Social functioning (2 items) Energy/fatigue (4 items) HPM214 SF-36 Scores on Possible Range

2015 HPM 214 SF-36 T-Scores ScaleNMeanSDMinMax Physical functioning (10 items) Role functioning - Physical (4 items) Pain scale (2 items) General health perceptions (5 items) Emotional well-being (MHI, 5 items) Role functioning - Emotional (3 items) Social functioning (2 items) Energy/fatigue (4 items) Physical health summary (PCS) Mental health summary (MCS)

2013 HPM 214 SF-36 Scores Compared to U.S. General Population ScaleClass MeanGen. Population Mean Physical functioning (10 items) 5654* Role functioning - Physical (4 items) 53 Pain scale (2 items) 5652* General health perceptions (5 items) 4952 Emotional well-being (MHI, 5 items) Role functioning - Emotional (3 items) Social functioning (2 items) Energy/fatigue (4 items) 50 Physical health summary (PCS) 5654 Mental health summary (MCS) * P < 0.05

SF-12 Items by Scale –General health (1) –Physical functioning (3b, 3d) –Role-Physical (4b, 4c) –Role-Emotional (5b, 5c) –Bodily pain (8) –Emotional well-being (9d, 9f) –Energy/fatigue (9e) –Social functioning (10) 6

7 SF-36 PCS and MCS PCS_z = (PF_Z * 0.42) + (RP_Z * 0.35) + (BP_Z * 0.32) + (GH_Z * 0.25) + (EF_Z * 0.03) + (SF_Z * -.01) + (RE_Z * -.19) + (EW_Z * -.22) MCS_z = (PF_Z * -.23) + (RP_Z * -.12) + (BP_Z * -.10) + (GH_Z * -.02) + (EF_Z * 0.24) + (SF_Z * 0.27) + (RE_Z * 0.43) + (EW_Z * 0.49) PCS = (PCS_z*10) + 50 MCS = (MCS_z*10) + 50

8 Farivar et al. alternative weights PCS_z = (PF_z *.20) + (RP_z *.31) + (BP_z *.23) + (GH_z *.20) + (EF_z *.13) + (SF_z *.11) + (RE_z *.03) + (EW_z * -.03) MCS_z = (PF_z * -.02) + (RP_z *.03) + (BP_z *.04) + (GH_z *.10) + (EF_z *.29) + (SF_z *.14) + (RE_z *.20) + (EW_z *.35) Farivar, S. S., Cunningham, W. E., & Hays, R. D. (2007). Correlated physical and mental health summary scores for the SF-36 and SF-12 health survey, V. 1. Health and Quality of Life Outcomes, 5: 54. [PMCID: PMC ]

Is New Treatment (X) Better Than Standard Care (O)? X 0 X 0 PhysicalHealth X > 0 MentalHealth 0 > X

10 Ultimate Use of HRQOL Measures-- Helping to Ensure Access to Cost-Effective Care Cost ↓ Effectiveness ↑

35%84%at least 1 moderate symptom 35%84%at least 1 moderate symptom 7% 70% at least 1 disability day 1% 11% hospital admission 1% 11% hospital admission 2% 14% performance of invasive 2% 14% performance of invasive diagnostic procedure diagnostic procedure ) Perceived Health Index (n = 1,862; reliability = 0.94) Highest Lowest Quartile on Index Perceived Health Index = 0.20 Physical functioning Pain Energy Emotional well-being Social functioning Role functioning. Bozzette, S.A., Hays, R.D., Berry, S.H., & Kanouse, D.E. (1994). A perceived health index for use in persons with advanced HIV disease: Derivation, reliability, and validity. Medical Care, 32, Single Weighted Combination of Scores

Is Medicine Related to Worse HRQOL? dead 1 Nodead dead 2 Nodead 3 No50 4 No75 5 No100 6 Yes0 7 Yes25 8 Yes50 9 Yes75 10 Yes100 Medication Person Use HRQOL (0-100) No Medicine375 Yes Medicine550 Group n HRQOL

Dead 0.0 Alive 1.0 Marathoner 1.0 Person in coma 1.0 Survival Analysis

Charges, Satisfaction, Mortality and HRQOL for Acute MI (n=133) Nelson, E. C., et al. (1995). Comparing outcomes and charges for patients with acute myocardial infarction in three community hospitals: An approach for assessing "value.” International Journal for Quality in Health Care, 7,

15 Quality of Life for Individual Over Time

“QALYs: The Basics” Value is … –Preference or desirability of health states Preferences can be used to –Compare different interventions on a single common metric (societal resource allocation) –Help make personal decisions about whether to have a treatment Milton Weinstein, George Torrance, Alistair McGuire, Value in Health, 2009, vol. 12 Supplement 1. 17

Preference Elicitation Standard gamble (SG) Time trade-off (TTO) Rating scale (RS) –  SG > TTO > RS  SG = TTO a  SG = RS b (Where a and b are less than 1) Also discrete choice experiments 18

Direct Preference Measures Underlying attributes unknown  Rating Scale  Standard gamble  Time tradeoff 19

Rating Scale Overall, how would you rate your current health? (Circle One Number) Worst possible health (as bad or worse than being dead) Half-way between worst and best Best possible health 20

Standard Gamble 21

p = probability of perfect health p = 1.00  QALY = 1.00 p = 0.50  QALY = 0.50 p = 0.25  QALY = 0.25 p = 0.00  QALY = 0.00 Standard Gamble (SG)

Time Tradeoff Alternative 1 is current health for time “t” (given), followed by death. Alternative 2 is full health for time “x” (elicited), followed by death. x/t = preference for current health 23

bin/utility.cgi 24

Indirect Preference Measures Attributes know Based on “societal preferences” a single score is assigned  Quality of Well-Being (QWB) Scale  EQ-5D  HUI2 and HUI3  SF-6D 25

Summarize HRQOL in QALYs – Mobility (MOB) – Physical activity (PAC) – Social activity (SAC) – Symptom/problem complexes (SPC) Well-Being Formula: w = 1 + MOB + PAC + SAC + SPC Quality of Well-Being (QWB) Scale Dead Well-Being 01 26

Each page in this booklet tells how an imaginary person is affected by a health problem on one day of his or her life. I want you to look at each health situation and rate it on a ladder with steps numbered from zero to ten. The information on each page tells 1) the person's age group, 2) whether the person could drive or use public transportation, 3) how well the person could walk, 4) how well the person could perform the activities usual for his or her age, and 5) what symptom or problem was bothering the person. Adult (18-65) Drove car or used public transportation without help (MOB) Walked without physical problems (PAC) Limited in amount or kind of work, school, or housework (SAC) Problem with being overweight or underweight (SYM) Quality of Well-Being Weighting Procedure Perfect Health Death 27

ComponentMeasuresStatesWeights Physical activityPhysical functionIn bed, chair, couch, or wheelchair*-.077 In wheelchair+ or had difficulty lifting,-.060 stooping, using stairs, walking, etc. MobilityAbility to get around orIn hospital, nursing home, or hospice transport oneselfDid not drive car or use public transportation Social activityRole function and self-careDid not feed, bath, dress, or toilet Limited or did not perform role Symptom/problem Physical symptoms andWorst symptom from loss of complexes problems consciousness to breathing smog or unpleasant air * did not move oneself in wheelchair +moved oneself in wheelchair Quality of Well-Being States and Weights

29 EQ-5D (243 states, 3 levels each) Mobility Self-care Usual activities Pain/discomfort Anxiety/depression

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HUI-3 Vision Hearing Speech Ambulation Dexterity Cognition Pain and discomfort Emotion 972,000 states, 5-6 levels per attribute

32 SF-6D Brazier et al. (1998, 2002) — 6-dimensional classification (collapsed role scales, dropped general health) — Uses 12 SF-36 items (PF: 3a, b, j; R: 4c, 5b; SF: 10; BP: 7, 8; MH: 9b, f; EN: 9e) --- About 18,000 possible states -— 249 states rated by sample of 836 from UK general population

33 Health state (0.59) Your health limits you a lot in moderate activities (such as moving a table, pushing a vacuum cleaner, bowling or playing golf) You are limited in the kind of work or other activities as a result of your physical health Your health limits your social activities (like visiting friends, relatives etc.) most of the time. You have pain that interferes with your normal work (both outside the home and housework) moderately You feel tense or downhearted and low a little of the time. You have a lot of energy all of the time

Correlations Among Indirect Measures EQ-5DHUI2HUI3QWB-SASF-6D EQ-5D1.00 HUI HUI QWB SF-6D Fryback, D. G. et al., (2007). US Norms for Six Generic Health-Related Quality-of-Life Indexes from the National Health Measurement Study. Medical Care, 45,

Change in Indirect Preference Measures Over Time Cataract (1 mon. – B)Heart F (6 mons. – B) HUI HUI QWB-SA EQ-5D SF-6D Kaplan, R. M. et al. (2011). Five preference-based indexes in cataract and heart failure patients were not equally responsive to change. J Clinical Epidemiology, 64, ICC for change was 0.16 for cataract and 0.07 for heart failure. Feeny, D. et al. (2011). Agreement about identifying patients who change over time: Cautionary results in cataract and heart failure patients. Medical Decision Making, 32 (2),

Perfect HRQOL Dead ExistsMeasured P3, M3 P2, M3 P3, M2 P3, M1 P1, M2 P1, M1 P1, M3 P2, M2 P2, M1 Mapping Health States to Preference Score

“Disease-Targeted” Preference- Based Measure (VFQ-UI) 6. How much difficulty do you have doing work or hobbies that require you to see well up close, such as cooking, sewing, fixing things around the house, or using hand tools? 14. Because of your eyesight, how much difficulty do you have going out to see movies, plays, or sports events ? 18. Are you limited in how long you can work or do other activities because of your vision? 25. I worry about doing things that will embarrass myself or others, because of my eyesight. 20. I stay home most of the time because of my eyesight. 11. Because of your eyesight, how much difficulty do you have seeing how people react to things you say? Near vision, distance vision, role function, mental health, dependency, social function 37

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VFQ-UI -6 item subset of NEI VFQ-25 (11 scales) -general vision, near vision, distance vision, driving, peripheral vision, color vision, ocular pain, role function, dependency, social function, mental health -8 vision-related health states (out of 15,625) ranging from no difficulty to stopped doing work -TTOs from sample of 607 from Australia, Canada, U.K. and U.S. ranged from Kowalski, J.W., et al. (2012). Rasch analysis in the development of a simplified version of the National Eye Institute Visual-Function Questionnaire-25 for utility estimation. Quality of Life Research, 21, Rentz, A., et al. (2014). Development of a preference-based index from the National Eye Institute Visual Function Questionnaire-25. JAMA Ophthalmology. 132 (3), e 41

42 VA Vision HRQOL EQ-5D Self-Care Pain Anxiety/Dep Mobility Usual Activities TTO

Comprehensive Geriatric Assessment (n = 363 community-dwelling older persons) lead to improvements in SF-36 energy, social functioning, and  Physical functioning (4.69 points) in 64 weeks  Cost of $746 over 5 years beyond control group Keeler, E. B., et al. Cost-effectiveness of outpatient geriatric assessment with an intervention to increase adherence. Med Care, 1999, 37 (12), Ad Hoc Preference Score Estimates

Is CGA worth paying for? Change in QALYs associated with 4.69 change in SF-36 physical functioning scale  r = > b =.003   QWB = 4.69 x.003 =.014 .014 x 5 yrs. = 0.07 QALYs  Cost/QALY: $10,600+ <$20,000 per QALY worthwhile

Quiz 1)What is the difference between a profile and preference-based measure? 2)Name a profile measure. 3)Name a preference-based measure. 4)What is a quality-adjusted life year? 45

Finding HRQOL Surveys