Preference-Based Health-Related Quality of Life Measures Ron D. Hays, Ph.D. January 26, 2015 (9:00-11:50 am) HPM 214

Preference-Based Health-Related Quality

of Life MeasuresRon D. Hays, Ph.D.

January 26, 2015 (9:00-11:50 am)

HPM 214 http://hpm214.med.ucla.edu/

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

Scale N Mean SD Min Max

Physical functioning (10 items) 13 97 4 90 100Role functioning - Physical (4 items) 13 91 23 19 100Pain scale (2 items) 13 89 11 68 100General health perceptions (5 items) 13 68 20 20 85Emotional well-being (MHI, 5 items) 13 75 19 30 90Role functioning - Emotional (3 items) 13 83 25 25 100Social functioning (2 items) 13 85 25 25 100Energy/fatigue (4 items) 13 58 23 6 81

2015 HPM214 SF-36 Scores on 0-100 Possible Range

2015 HPM 214 SF-36 T-Scores

Scale N Mean SD Min Max

Physical functioning (10 items) 13 56 2 53 57Role functioning - Physical (4 items) 13 53 9 25 57Pain scale (2 items) 13 56 6 46 62General health perceptions (5 items) 13 49 10 26 58Emotional well-being (MHI, 5 items) 13 50 11 25 64Role functioning - Emotional (3 items) 13 48 11 25 56Social functioning (2 items) 13 50 11 24 57Energy/fatigue (4 items) 13 50 11 24 61Physical health summary (PCS) 13 56 6 40 63Mental health summary (MCS) 13 47 14 14 59

2013 HPM 214 SF-36 Scores Compared to U.S. General

Population Scale Class Mean Gen. Population Mean

Physical functioning (10 items) 56 54*Role functioning - Physical (4 items) 53 53Pain scale (2 items) 56 52*General health perceptions (5 items) 49 52Emotional well-being (MHI, 5 items) 50 49Role functioning - Emotional (3 items) 48 51Social functioning (2 items) 50 51Energy/fatigue (4 items) 50 50Physical health summary (PCS) 56 54Mental health summary (MCS) 47 49* 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

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SF-36 PCS and MCSPCS_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) + 50MCS = (MCS_z*10) + 50

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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: PMC2065865]

Is New Treatment (X) Better Than Standard Care (O)?

0

10

20

30

40

50

60

70

80

90

100

XX

00XX

00

PhysicalPhysicalHealthHealth

X > 0X > 0

Mental Mental HealthHealth

0 > X0 > X

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Ultimate Use of HRQOL Measures--Helping to Ensure Access to

Cost-Effective Care

Cost ↓

Effectiveness ↑

35%35% 84%84% at least 1 moderate symptomat least 1 moderate symptom

7%7% 70% 70% at least 1 disability day at least 1 disability day

1%1% 11% 11% hospital admission hospital admission

2%2% 14% 14% performance of invasive performance of invasive diagnostic procedurediagnostic procedure

Perceived Health Index (n = 1,862; reliability = 0.94))

Highest Lowest Quartile on Index

Perceived Health Index = 0.20 Physical functioning + 0.15 Pain + 0.41 Energy +0.10 Emotional well-being + 0.05 Social functioning + 0.09 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, 716-731.

Single Weighted Combination of Scores

Is Medicine Related to Worse HRQOL?

1 No deaddead2 No deaddead

3 No 50 4 No 75 5 No 100 6 Yes 0 7 Yes 25 8 Yes 50 9 Yes 75 10 Yes 100

MedicationPerson Use HRQOL (0-100)

No Medicine 3 75Yes Medicine 5 50

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)

25

81

25

63 66

27

81

15

49

64

31

83

12

7585

0102030405060708090

Total HospitalCharges

Satisfactionwith Care

Mortality PhysicalFunction

EmotionalWell-Being

A B C

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, 95-108.

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Quality of Life for Individual Over Time

http://www.ukmi.nhs.uk/Research/pharma_res.asp

“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.

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Preference Elicitation• Standard gamble (SG)• Time trade-off (TTO)• Rating scale (RS)

– http://araw.mede.uic.edu/cgi-bin/utility.cgi

SG > TTO > RS SG = TTOa

SG = RSb (Where a and b are less than 1)

• Also discrete choice experiments18

Direct Preference Measures

• Underlying attributes unknown

Rating ScaleStandard gambleTime tradeoff

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Rating Scale

Overall, how would you rate your current health?(Circle One Number)

0 1 2 3 4 5 6 7 8 9 10

Worst possible health (as bad or

worse than being dead)

Half-waybetween worst

and best

Best possible

health

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Standard Gamble

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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

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http://araw.mede.uic.edu/cgi-bin/utility.cgi

http://araw.mede.uic.edu/cgi-bin/utility.cgi

24

Indirect Preference Measures

• Attributes know

• Based on “societal preferences” a single score is assignedQuality of Well-Being (QWB) ScaleEQ-5DHUI2 and HUI3SF-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

0 1

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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

012

43

5

78

6

910 Perfect Health

Death 27

Component Measures States Weights Physical activity Physical function In bed, chair, couch, or wheelchair* -.077

In wheelchair+ or had difficulty lifting,-.060

stooping, using stairs, walking, etc.

Mobility Ability to get around or In hospital, nursing home, or hospice. -.090 transport oneself Did not drive car or use public -.062

transportation

Social activity Role function and self-care Did not feed, bath, dress, or toilet -.106Limited or did not perform role -.061

Symptom/problem Physical symptoms and Worst symptom from loss of -.407 complexes problems consciousness to breathing -.101 smog or unpleasant air

* did not move oneself in wheelchair +moved oneself in wheelchair

Quality of Well-Being States and Weights

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EQ-5D (243 states, 3 levels each)

• Mobility

• Self-care

• Usual activities

• Pain/discomfort

• Anxiety/depression

http://www.euroqol.org/

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HUI-3

Vision

Hearing

Speech

Ambulation

Dexterity

Cognition

Pain and discomfort

Emotion

http://www.healthutilities.com/hui3.htmhttp://www.healthutilities.com/hui3.htm

972,000 states, 5-6 levels per attribute

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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 populationhttp://www.shef.ac.uk/scharr/sections/heds/mvh/sf-6d

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Health state 424421 (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-5D HUI2 HUI3 QWB-SA SF-6D

EQ-5D 1.00

HUI2 0.71 1.00

HUI3 0.68 0.89 1.00

QWB 0.64 0.66 0.66 1.00

SF-6D 0.70 0.71 0.69 0.65 1.00

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, 1162- 1170.

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Change in Indirect Preference Measures Over Time

Cataract (1 mon. – B) Heart F (6 mons. – B)

HUI3 0.05 0.02

HUI2 0.03 0.00

QWB-SA 0.02 0.03

EQ-5D 0.02 0.00

SF-6D 0.00 0.01

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, 497-506.

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), 273-286. 35

Perfect HRQOL

Dead

0.9

0.8

0.7

0.6

0.5

0.4

0.3

0.2

0.1

Exists Measured

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

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VFQ-UI Health State Classification

Near Vision

I have no difficulty doing work or hobbies that require seeing well up close, such as cooking, sewing, fixing things around the house or using hand tools.

I have a little difficulty doing work or hobbies that require seeing well up close, such as cooking, sewing, fixing things around the house or using hand tools.

I have moderate difficulty doing work or hobbies that require seeing well up close, such as cooking, sewing, fixing things around the house or using hand tools.

I have extreme difficulty doing work or hobbies that require seeing well up close, such as cooking, sewing, fixing things around the house or using hand tools.

Distance Vision

I have no difficulty going out to see movies, plays or sports events.

I have a little difficulty going out to see movies, plays or sports events.

I have moderate difficulty going out to see movies, plays or sports events.

I have extreme difficulty going out to see movies, plays or sports events.

Role Function

I am not limited in how long I can work or do other activities

I am limited in how long I can work or do other activities a little of the time

I am limited in how long I can work or do other activities some of the time

I am limited in how long I can work or do other activities

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VFQ-UI Health State Classification

Mental Health

I don’t worry about doing things that will embarrass me or others

I worry some of the time about doing things that will embarrass me or others

I worry most of the time about doing things that will embarrass me or others

I worry all of the time about doing things that will embarrass me or others

Vision Dependency

I don’t have to stay at home

I stay at home some of the time

I stay at home most of the time

I stay at home all of the time

Social Function

I have no difficulty at all seeing how people react to things I say.

I have a little difficulty at all seeing how people react to things I say.

I have moderate difficulty at all seeing how people react to things I say.

I have extreme difficulty at all seeing how people react to things I say.

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Example Health State (Perfect Vision)

Because of my eye sight…

I have no difficulty doing work or hobbies that require seeing well up close, such as cooking, sewing, fixing things around the house or using hand tools

I have no difficulty at all seeing how people react to things

I say

I have no difficulty going out to see movies, plays or sports events

I am not limited in how long I can work or do other

activities

I don’t have to stay at home I don’t worry about doing things that will embarrass me or

others

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 0.34-0.96

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, 323-334.

Rentz, A., et al. (2014). Development of a preference-based index from the National Eye Institute Visual Function Questionnaire-25. JAMA Ophthalmology. 132 (3), 310-318.

- e41

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VA VisionHRQOL

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), 1199-1206.

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 = 0.69 -> 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?

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Finding HRQOL Surveys

http://www.proqolid.org/

http://www.nihpromis.org/

http://www.healthmeasurement.org/

http://www.chime.ucla.edu/analysis.htm

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