Quality of Life Measurement

Robert M. Kaplan
University of California, San Diego
Department of Family and Preventive
Medicine
NIH RCT Summer Training Course
August 5, 2002
 Where This is Going
Profile Approach
z SF-36
Utility Approach
z QWB
Preference Assessment
Health-Related Quality of Life is:
What the person can DO (functioning)
z Self-care
z Role
z Social
How the person FEELs (well-being)
z Emotional well-being
z Pain
z Energy
 HRQOL is Multi-
dimensional
HRQOL

Physical Mental Social

Types of HRQOL Measures

Profile
Generic 
Targeted
Preference-based
 RAND-36 Scales (Items)
Physical functioning (10 items)
Role limitations/physical (4 items)
Role limitations/emotional (3 items)
Social functioning (2 items)
Emotional well-being (5 items)
Energy/fatigue (4 items)
Pain (2 items)
General health perceptions (5 items)
Physical Functioning Item
Does your health
now limit you in
bathing or
dressing yourself?

Yes, limited a lot

Yes, limited a little
No, not limited at all
Emotional Well-Being Item
How much of the time
during the past 4
weeks have you been
a very nervous
person?

None of the time; A little

of the time; Some of
the time; A good bit of
the time; Most of the
time; All of the time
Scoring RAND-36 Scales
Average or sum all items in the same
scale.

Transform raw average or sum to 0-

100 possible range (linear
transformation)

z (raw score – minimum)* 100/(max – min)

HRQOL of HIV Infected Adults

Emotional MS
Physical
ESRD
Diabetes
Depression
Prostate disease
GERD
Epilepsy
General Pop

CDC C
CDC B
CDC A

0 20 40 60 80 100
Hays, et al. (2000), American Journal of Medicine
Course of Emotional Well-being Over 2-
years for Patients in the MOS General
Medical Sector
Hypertensio
81 n
79 Diabetes
77
75
73
71
69 Subthreshol
X d Depression
67
65 X
Major Depression
63
61
59
57
55
Baseline 2-Years
Hays, R.D., Wells, K.B., Sherbourne, C.D., Rogers, W., & Spritzer, K. (1995).
Functioning and well-being outcomes of patients with depression compared
to chronic medical illnesses. Archives of General Psychiatry, 52, 11-19.
 Two Underlying
RAND-36 Dimensions
• Hays, R.D., and Stewart, A.L. (1990). The
structure of self-reported health in chronic
disease patients. Psychological Assessment, 2,
22-30.
• Hays, R. D., Marshall, G. N. et al. (1994). Four-
year cross-lagged associations between physical
and mental health in the Medical Outcomes
Study. Journal of Clinical Psychology, 62, 441-
449.
Indicators of Physical Health

Physical Health

Role
Physic
functio
al General
n- Pain
functio Health
physica
n
l
Indicators of Mental Health

Mental Health

Role
Emotion function
Social
al Well- - Energy
function
Being emotion
al
RAND-36 Summary Scores
Q Physical Health Composite
) Physical functioning, role—physical, pain,
general health perceptions
X Mental Health Composite
) Emotional well-being, role—emotional,
social functioning, energy/fatigue
) Intercorrelation = 0.66; reliability >= 0.91

Hays, R. D., Embury, S. & Chen, H. (1998). RAND-36 Health Status

Inventory. San Antonio: The Psychological Corporation.
 Range of Treatment Impacts on PCS

12
Duodenal Ulcer
10 Medication

8 Shoulder Surgery
mpact on SF-
6
36 PCS
4 Heart Value
Replacement
2
0 Total Hip Replacement
Treatment Outcomes
 Range of Treatment Impacts
on MCS

12 Stayed the same

10 Low back pain

therapy
8
mpact on SF- Hip replacement
6
36 MCS
4 Ulcer maintenance

2 Recovery from
Depression
0
Treatment Outcomes
Samsa et al. (1999).
Pharmacoeconomics
Q MCID for SF-
36 is “typically
in the range of
3 to 5 points”
(p. 149).
Q .09->0.28 ES
 Caution in Using
SF-36 PCS and MCS
Simon et al. (1998, Med Care); 536 primary care
patients initiating antidepressant tx.
z Physical functioning, Role—physical, pain, and general
health perceptions improved significantly and by 0.28 to 0.49
SDs, but PCS did not change!
Nortvedt et al. (2000, Med Care); 194 MS patients
z Emotional well-being was 0.3 SD lower, role-emotional 0.7
SD lower, energy/fatigue 1.0 SD lower, and social
functioning 1.0 SD lower than general US population, but
MCS was only 0.20 SD lower.
 Limitation of RAND-36:
Is New Treatment (X) Better
Than Standard Care (O)?
100
90
80 0
70 X X
0 X 0
60
50 X
0
40
30
20
10
0
Physical Pain Emotional Social
Functioning Well-being Functioning

X>0 0>X 0 >X X=0

Fryback et al. Prediction of
QWB from SF-36
Q 56.9% of the observed QWB variance;
49.5% on on cross-validation
QWB~ = 0.59196
+ (PF * 0.0012588)
- (EWB * 0.0011709)
- (BP * 0.0014261)
+ (RP x GH * 0.00000705)
+ (PF x BP * 0.00001140)
+ (BP x EWB * 0.00001931)
 Summary of RAND-36
Generic profile measure
Single integrated score
z Preference-based measure
z Estimate of preference-based measure
QWB Approach
 Outcomes Measurement
Does the health care you give, affect
patient health status?
How do you know?
How do you distinguish between + and -
effects on health status?
OVERALL, does the patient benefit from
the health care they are given?

(From Kind, 1995)

Traditional

• LifeExpectancy
• Infant Mortality
• Disability Days
 Survival Analysis

• Alive1.0
• Dead 0.0
 Problem with Survival
Analysis

• Tennis player 1.0

• Man in coma 1.0
Purpose of Quality Adjusted
Survival Analysis

• To summarize
life expectancy
with
adjustments for
quality of life
Quality of Well-being Scale
Currently two versions
z Interviewer
z Self-Report

Takes about 10 minuets

Automated scoring, low cost
About 200 published papers describe
use
 QWB Components
Functional Scales
z Mobility (MOB )
z Physical Activity (PAC)

z Social Activity (SAC)

Symptom/Problem Complexes (CPX)

Purpose of Quality Adjusted
Survival Analysis

• To summarize
life expectancy
with
adjustments for
quality of life
 Mobility Scale

• No limitations in travel
• Did not drive or use public
transportation
 Physical Activity Scale

• Walked without physical

problems
• Walked with limitations
• Moved own wheelchair without
help
• Confined to bed or chair
 Social Activity Scale
• Did work, school or housework and other activities
• Did work, school or housework, but limited in other activities
• Limited in amount or kind of work, school, or housework
• Performed self-care, but not work, school, or housework
• Had help with self care
 Symptoms or Problems (selected)
• coma
• trouble learning, remembering, or thinking clearly
• pain in back or neck
• sick or upset stomach
• coughing wheezingof breath
• spells of feeling upset, depressed or of crying
• overweight
• runny nose
• problems with sexual interest or performance
 Quality-Adjusted Life Year
Combines morbidity and mortality into a
single index
Represents life expectancy with
adjustments for quality of life
Is defined as a year of life free of all
disabilities and symptoms
 Example Case: 68 year old COPD patient
Description

• Shortness of breath
• Drove Car
• In Bed or Chair for Most of Day
• Performed No Major Role Activity, but did perform
self-care
• Weight
• Peer Rating equals .605
• For each year in this state, the patient loses 1-
.605 = .395 well years
 Sinus Disease and Diabetes in
the General Population
Source: Erickson, 1980 NHIS, Preliminary

1.00
Sinusitis
0.95 Diabetes
Emphysema
0.90

0.85
Average QWB

0.80

0.75

0.70

0.65

0.60

0.55

0.50
15-44 45-64 65+

Age
QWB by Level of Cognitive
Impairment in Alzheimer’s
800

775
750

725
700

675
650

625

600
575

550
525

500
475

450
425

400
Control Mild Moderate Severe

Group
 QWB and Serum Beta 2
Microglobulin in HIV
QWB by Serum Beta2 Microglobulin

0.85

0.80
QWB

0.75

0.70

0.65

0.60
1 2 3 4
Serum Beta2 Microglobulin Quartile
 QWB and Neurological
Evaluation in HIV
QWB by neurologist rating of central impairment

0.80
QWB
0.75
0.70
0.65
QWB

0.60
0.55
0.50
0.45
0.40
1 2 3 4 5
Rating of Central Impairment
Atrial Fibrillation (Ganiats et al,
1992)

44
42
40
FSI SCore

38
36
34
32
30
1 2 3 4 5
QWB Quintile
 QWB and Survival in HIV
0.80

0.75

0.70
QWB

0.65

0.60

0.55

0.50
Dead, N=46 Alive, N=466

Status
Estimating treatment effects

Quality Adjusted Life

Expectancy for
QWB Non-smokers
Quality Adjusted
Life Expectancy
for Smokers

Effect of Smoking
in QALYs

Years
 Patients Undergoing Sinus Surgery Vs
Control (Hodgson, 1994)
0.74
0.72
0.7
0.68
0.66
QWB

0.64
0.62
0.6
0.58
0.56
0.54

Follow-up
QWB Before and After Ciprofloxacin Treatment
for Exacerbations of CF (Orenstein et al, 1990)

0.80
0.70
0.60
0.50
QWB

0.40
0.30
0.20
0.10
0.00
Pre Post
Evaluation
Issues in Child Health:
Chronic Episodic
Chronic Asthma

1.0

0.0

Weeks
QWB has been criticized for
Excluding mental health
Excluding sensory function
Excluding social health
Excluding disease specific information,
and
Being to long
Is Mental Health Excluded
from the QWB?
 QWB by SAPS Patient Groups and
Controls

0.8 Overall F = 28.49, df 3/118, p<.001

Linearity F = 81.6, df 1/118, p<.001
0.7
0.7051
0.6605
0.6

0.5 0.5486
0.5196
QWB

0.4

0.3

0.2

0.1

0
Controls Mild Moderate Severe
(0 - 2) (3 - 6) (7 - 14)

Number of Positive Psychiatric Symptoms

Figure 1
QWB by Hamilton Depression
(from Rubin et al 1994)
0.9
0.8
0.7
QWB Score

0.6
0.5
0.4
0.3
0.2
0.1
0
Control 0-9 10 to 16 >=17
Hamilton Depression Groups
 QWB-SA Mental Health
trouble sleeping change in sexual
felling upset and interest or
blue performance
excessive worry memory loss
feeling no control thoughts images
feeling lonely mediation
frustration loss of appetite
hangover
Correlations with Depression
QWB-Hamilton .70
QWB-Beck .58
Beck-Hamilton .69
Using older QWB weights r
HamD=.33, Beck=.30
 Frequency
0 10 20 30 40 50 60 70 80 90
0.29

0.34

0.39

0.44

0.49
0.54

0.59

0.64

0.69

0.74

0.79

0.84

0.89

0.94
1998, N=301)

0.99

1
QWB-SA Distribution (Andresen
 Summary
QWB and SF-36 have some common roots
Correlations between QWB and some SF-36
components are substantial
QWB now can be self-administered
QWB can be used to estimate QALYs for policy
analysis
Several theoretical and technical issues must be
resolved in future studies
Utility Assessment Issues
The difficult task of development
of population utility weight
for a health condition
0 1.0 0 1.0

0.72
0 1.0
Population Population
utility distribution
weight

Individuals
 Preference and Utility
Assessment
Standard Gamble
Time Trade-off
Rating Scales
 State A for
Choice A Rest of
Life

Prob. p Healthy
for Rest
Choice B of Life
Prob. p-1
Death

Example of Standard Gamble (Torrance &

Feeny, 1989)
Category Scale
Perfect Health

Visual Analog

Death Perfect
Health

Death
 Approaches for describing health states

Brief Outline Narrative text Multimedia

Age: 40-65 yr. old employee

I am in the age rage of 40-64
housekeeper
years I live alone and am
Mobility: in house
confined to my home. I have
Physical activity: walks but lost 35 pounds in the last 6
has limitations. months. I am able to only eat
Social activity: did not small amounts of food at
perform but performed present and I vomit
self-care activities occasionally. I am tired and
Symptoms/problems: sick or weak and walk with the aid
upset stomach, of a walker. I require
vomiting, or diarrhea assistance to get into and out
of the bathtub. Social contact
with my family and friends is
infrequent.

What is an appropriate strength of stimulus to form preferences?

 Perceived Utility
state attributes Risk attitude
Time preference

Health
Health State preferences
Cognition
Description
Elicitation Utility
procedure measurements

Emotions and
prejudices Numeracy
Random error
Logical error
Cross method
inconsistency
Anchoring on
single values
Potential Sources of Variability
In Preference Measurements
Descriptions of states
Scaling methods
Measurement or assignment
procedures
Health Status and Social factors
 Validity
No absolute standard or threshold
Valid aggregate utilities can only arise from
valid measurements within individuals
Validity of measurements within an
individual measurable by
z Ability to discriminate among states within the
protocol
z Internal consistency of individual responses
 Discrete distributions of utilities make
can assessment of reliability difficult.

quality of life assigned

some other value of 1.0:
value:18% 15%
Results of the
Beaver Dam study
(Fryback et al.
Medical Decision
Making
1993) rated their
quality of life
either 0.9 or
0.5: 23% rated their
quality of life
at 1.0: 44%
 Procedural Invariance and the Organization
Of Preferences within an Individual

100
100
90
90
r=0.62 r=0.015
80
80
70
70
VAS Rating

60

VAS Rating
60
50
50
40
40
30
30
20
20
10
10
0
0
0 10 20 30 40 50 60 70 80 90 100
0 10 20 30 40 50 60 70 80 90 100
SG Rating
SG Rating

Satisfy Fail
Lenert and Treadwell, Medical Decision Making, 1999
 Wheelchair versus Not in
Comparison between ever and never in wheelchair or walker for 31 items:
Data from Oregon Health Services Commission
100 Chair
r=.97

80
Never in Wheelchair or Walker

60

40

20

0
0 20 40 60 80 100

Ever in Wheelchair or Walker

 1.0
Impaired in
2 Dimensions
0.9
Standard Impaired in
Gamble0.8 1 Dimension
Utility
Minimal
0.7 Impairment

0.6
States with States with States with
impairments impairments in Minimal
in 2 dimensions 1 dimension of impairments
of health health
Additive Independence and
Interval Scaling
0.47
In House
In Hospital

0.46
QWB Rating

0.45

0.44

0.43

0.42
In bed Wheelchair Limited Walking

Physical Activity
Potential minimally important
differences
$100,000 per QLAY
$10K

Annualized $50,000 per QLAY

marginal cost
$10,000 per QLAY

$1K

0.01 0.05 0.1

Annualized gain in utility
 Summary
Competing methods (RS,TTO, SG) are really
Complementary methods
z all have some evidentiary basis.
z Each capture different aspects of preferences.
There is no gold standard for measurement of preferences.
The focus of research needs to be:
z understanding of the implications of choice of a particular scaling
method
z effects of changes in procedures for elicitation.