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Health-Related Quality of Life (HRQOL) - 2014
John E. Ware, Jr., PhD
Introduction to the Principles and Practice of Clinical ResearchNational Institutes of Health - Warren G. Magnuson Clinical Center Building 10
Lipsett Amphitheater, Bethesda, MD, December 2, 2013
Professor & Division Chief, Dept. of Quantitative Health Sciences, UMass Medical School Chief Science Officer and Founder, JWRG, Incorporated, Worcester, MA
2
Quality and Outcomes
“The best measure of quality is not how well or how frequently a medical service is given, but how closely the result approaches the fundamental objectives of prolonging life, relieving distress, restoring function, and preventing disability.”
Lembcke, 1952
Lembcke , PA. Measuring the quality of medical care through vital statistics based on hospital service areas: 1. Comparative study of appendectomy rates. Am J Public Health 1952; 42: 276-86.
33
Quality of Life (QoL)
•Community•Education
•Family Life•Friendships
•Health•Housing
•Marriage•Nation•Neighborhood•Self•Standard of Living•Work
Source: Campbell, 1981
4
World Health Organization Definition of Health
“Health is a state of complete physical, mental, and social well-being and not merely the absence of disease or infirmity”
WHO, 1948
55
Health is Measured in Terms of:
• Bodily structure & function
• Specific symptoms
• What you do/are able to do – functioning
• How you feel – subjective ill- and well-being (+ and -)
• What you say it is – personal evaluation
66666
Clinical Markers
(1)
Specific Symptoms
(2)
Impact of Disease-specific
Problems
(3)
Arthritis SymptomsHave you had the following symptoms: Joint pain, swelling, burning sensation:
• Almost every day• Several days a week• A few days a month• Not at all
(4)
Generic Functioning, Well-being
and Evaluation
X-ray, Disease Progression
Continuum of Disease-specific and Generic Health Measures - Arthritis
Health-related QOL(HRQoL)
GenericHealth
In general, would you say your health is:
• Excellent• Very good• Good• Fair• Poor
How much does your arthritis limit your usual activities or enjoyment of everyday life?
• Not at all• A little• Moderately• Extremely
ArthritisImpact
Adapted from: Wilson and Cleary, JAMA, 1995Ware, Annual Rev. Pub. Health, 1995
7
There is More to the Continuum
Clinical Markers
Specific Symptoms
(1) (2) (4)
Generic Functioning, Well-being
and Evaluation
Impact of Disease-specific
Problems
(3)
8
Predictive Validity:HRQoL is One of the Best Predictors
Health-Related QOL (HRQoL)
Future healthInpatient expendituresOutpatient expendituresJob lossResponse to treatmentReturn to workWork productivityMortality
(3) (4)
Impact of Disease-specific
Problems
Generic Functioning, Well-being
and Evaluation
9
Summary of Content/Concepts for Widely-Used Generic Health Surveys
Source: Adapted from Ware, 1995
Reported health transition
SIP = Sickness Impact Profile (1976)HIE = Health Insurance Experiment surveys (1979)NHP = Nottingham Health Profile (1980)QLI = Quality of Life Index (1981)COOP = Dartmouth Function Charts (1987)DUKE = Duke Health Profile (1990)MOS FWBP = MOS Functioning & Well-Being Profile
(1992)
MOS SF-36 = MOS 36-Item Short-Form Health Survey (1992)
QWB = Quality of Well-Being Scale (1973)EUROQOL = European Quality of Life Index (1990)HUI = Health Utility Index (1996)SF-6D= SF-36 Utility Index (2002)
PsychometricUtility Concepts and Characteristics SIP HIE NHP QLI COOP DUKE MOS
FWBPMOSSF-36 QWB EQ-5D HUI SF-6D
CONCEPTSPhysical functioning Social functioning Role functioning Psychological distress Health perception (gen’l) Pain Vitality Psychological well-being Sleep Cognitive functioning Quality of life
PROMIS
PROMIS = Patient Reported Outcomes Measurement Information System (2008)
(-)
(-)(-)
(-)
(-)
(-)
10
www.nihpromis.orgPROMIS Website
Cella D, Yount S, Rothrock N, et al. PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care. 2007 May; 45(5 Sup 1):S3-S11.
Cella D, Riley W, Stone A, et al. PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. J Clin Epidemiol. 2010 Nov; 63(11):1179-94.
Rose M, Bjorner JB, Becker J, et al. Evaluation of a preliminary physical function item bank supported the expected advantages of the Patient-Reported Outcomes Measurement Information System (PROMIS). J Clin Epidemiol. 2008 Jan; 61(1):17-33.
Varni JW, Thissen D, Stucky BD, et al. PROMIS(®) Parent Proxy Report Scales: an item responsetheory analysis of the parent proxy report item banks. Qual Life Res. 2011 Oct 5.[Epub ahead of print]
PROMIS References
1111
Medical Outcomes Study 36-Item Health Survey (SF-36)
Utility Index(Brazier et al., 2002)
(Lam, Brazier, McGhee 2008)
Physical
Mental
12
Generic Health Profiles: Before & After Medication
Best Health
Poorest Health
Component Summaries
SF-36 Subscales
Treatment
Baseline
2530354045505560
2530354045505560
Arthritis
Norm
Physical Mental P M 2530354045505560
30354045505560
Depression
Component Summaries
SF-36 Subscales
Physical Mental P M
Treatment
Baseline
Best Health
Poorest Health
-
Next Step: Integrate and Standardize Disease-specific and Generic Measures
Best Health
Poorest Health
+
2530354045505560
Disease-SpecificImpact Scale
(QDIS)
2530354045505560
Example: Osteoarthritis
Physical HealthGeneric Summary
(PHGS)
US Norms(Mean=50
SD=10)
14
Comparison of Content of Generic And Disease-Specific Measures
Health transition (B/S/W)
Health Domains
Physical functioningSocial functioningRole functioningPsychological distressHealth perceptions (general)Pain (bodily)Energy/fatiguePsychological well‐being SleepCognitive functioning
SIP
Quality of life
PROMIS
Sexual functioningSpecific symptomsSpecific treatments
Generic*MOSFWBP
WO‐MAC
Disease‐Specific*
SAQ
KDQOL
StGeorg
DQOL
ML‐HFQ
DQOL ‐ Diabetes Quality of Life MeasureKDQOL ‐ Kidney Disease Quality of Life QuestionnaireMLHFQ ‐ Minnesota Living with Heart Failure QuestionnaireSAQ ‐ Seattle Angina QuestionnaireSt. George's Respiratory QuestionnaireWOMAC ‐ Western Ontario and Mac Master's Arthritis Index
SIP ‐ Sickness Impact Profile, 136 items (1976)MOS FWBP ‐MOS Functioning and Well‐Being Profile,
149 items (1992)PROMIS ‐ Patient Reported Outcomes Measurement
Information System (2007‐on)
*Source: Comparison adapted from Ware 1987; Ware, 1995
Content Areas* QOL Disease Impact Scale (QDIS)
1. Global (“activity” and “QOL”)2. Physical Functioning3. Mobility4. General Health 5. Fatigue6. Cognitive7. Emotional8. Sleep9. Role Functioning 10. Social Functioning
*Note: Bank of 49 items reduced to 25 items with attribution to specific condition
GenericAttribution
16
During the past 4 weeks, how often did your healthlimit your ability to do your everyday activities?
• Very often • Often • Sometimes• Rarely• Never
Anatomy of a Survey Item:QDIS Changes Attribution from Health
to Specific Disease
health
Disease-Specific Attribution
arthritis
Early Reference: Patrick DL, Deyo RA. Generic and Disease-specific Measures in Assessing Health Status and Quality of Life. Medical Care. 1989;27(3):S217-S232.
QDIS Reference: QOL Disease Impact Scale (QDIS); Ware JE, Guyer R, Harrington M, Boulanger R. Quality of Life Research, 2012.
1717
• Standardized Metrics
• Adaptive survey administrations
• Norm-based scoring
• Internet and mobile data collection
Better Measures Are Being ConstructedSource:
Business Week
11/26/01
1818
100
0
20
30
40
50
Mean = 50SD = 10
Climbing several flights of stairs
Walk one hundred yards
Bathing or dressing, Limited a little
NormVigorous Activities, Not limited
Improving the Physical Function “Ruler”
1980 Old “Ruler”
> 75%@ Ceiling
1990 NEW “Ruler”
> 30%@ Ceiling
00.2
0.4
0.6
10 20 30 40 50 60
.76
.24
.01a b
Limiteda little
Limiteda lot0.8
1.0 NotLimited
2008BETTER “Ruler”< 3 % @ Ceiling
20
30
40
50
70
60
19
We Need the Health Equivalent of a Two-Sided Tape Measure
52 centimeters = 20.5 inches
and Public-Private Partnerships Meetingthe Needs of Research and Business
20
Short-Form Surveysand Ceiling Effects
1
3
1
3
7
1
3
5
Measuring Too Low -CeilingEffect
21Cooking Thermometer
Some Thermometers Focus on a Very Narrow Range
130–190 °F54–88 °C
22
Temperature
F° = 98.6 C° = 37.0
Example: Cross-Calibrating Celsius and Fahrenheit
WaterFreezes
NormalHumanBlood
ShirtSleeve
Weather
23
CAT
Solution: Adaptive Survey Methods
Patient scores here
CAT = Computerized Adaptive Testing
24
Moderate
Adaptive Assessments of Disease ImpactMatch Questions to Each Patient’s Level
10
30
40
70
80
Mild
Severe
50
60
40
20
Higher is worse.Mean = 50SD = 10
25
First QuestionNoisy Score Estimate (+/- 15)
10
30
40
70
80
50
60
40
20
Score estimate,1st response = 62+/- 15
Moderate
Mild
Severe
Higher is worse.Mean = 50SD = 10
26
Second Question: Standard Error Reduced by One Third
Score estimate,2 responses = 64+/- 10
10
30
40
80
50
40
20
70
60
Moderate
Mild
Severe
Higher is worse.Mean = 50SD = 10
27
Third Question: Standard ErrorCut in Half
10
30
40
80
50
40
20
70
60
Moderate
Mild
Severe Score estimate,3 responses = 63+/- 7
Higher is worse.Mean = 50SD = 10
28
Fourth Question: Standard ErrorCut by Two Thirds
10
30
40
80
50
40
20
70
60
Moderate
Mild
Severe Score estimate,4 responses = 62+/- 5
Higher is worse.Mean = 50SD = 10
29
Practical Implications of CATin Health Assessment
30
Reference: Ware JE, Jr., Bjorner JB, Kosinski M: Practical implications of item response theory and computerized adaptive testing: A brief summary of ongoing studies of widely used headache impact scales. Medical Care 2000;38:II73-82.
Static 5-Item Headache Pain Measure
“Ceiling Effect”
r = 0.536N = 1016
Dynamic 5-Item HeadachePain Measure
r = 0.938N = 1016
CriterionScore
CriterionScore
A Promising Solution in 1999: CAT-Based Health Assessment
3 SD units
NoDisability
31
Performance of 5-item CAT Scores Confirmed in NIH-Sponsored Studies
r = 0.98N = 2,753
Mental Health Headache Disability
r = 0.94N = 1,016
-2-10123
-2 -1 0 1 2 3
DiabetesImpact
r = 0.93N = 100
20 30 40 50 60 70
Pediatric Disability
r = 0.96N = 263
10
20
30
40
50
60
70
Chronic Kidney Disease
-3
-2
-1
0
1
2
-4-3 -2 -1 0 1 2
r = 0.95N = 1,846
Post AcuteRehabilitation
15
20
25
30
35
40
45
50
20 25 30 35 40 45
r = 0.95N = 485
3232
What are the Advantages of Dynamic Assessments?
• More accurate risk screening • Reliable enough to monitor individual
outcomes• Brevity of a short form –
90% reduction in respondent burden• Elimination of “ceiling” & “floor” effects • Can be administered using various data
collection technologies• Markedly reduced data collection costs• Monitor data quality in real time
33
Matching Methods to Applications
1
2
3
4
5
6
7
1
3
5
7
Single-Item
1
2
3
4
5
Multi-ItemScale
PopulationSurveys
Group-LevelStudies
Patient-LevelAssessments
“Item Bank” (CAT Dynamic)
7
6
Most Functionally Impaired
NoisyIndividual
Classification
Very AccurateIndividual
Classification
34
Adaptive Survey Logic (ASLX ®) Flow Chart
ASLX® Screen Estimate Score & CI from Screen
NEG
POS
SupplementalDomain
Assessment(CAT/Static)*
Repeat Cycle for Each Generic Domain
Repeat Cycle for Each Chronic Condition
ConditionPresent?
PosSupplementalDisease ImpactAssessment
(CAT/Static)*
Follow‐upAssessmentModules
GenericAssessment
Survey ContentAnd User
Interface (UI)
GenericHealth
Assessments
QDISAssessments(QOLIX® CCC)
YES
ASLX® Screen
NONext Condition
Abbreviations: QOLIX® DIS; Chronic condition checklist QOLIX® CCC), Computer adaptive test (CAT), Confidence interval (CI), Diagnosis (Dx), Electronic data capture (EDC), User interface (UI); Follow-up assessment modules vary across
applications.
Impact Estimated
From Screening
Neg
353535
• Diagnosis• Disease severity• Responders • Treatments
HR-QOL
GoldStandard
• Work productivity• Costs of care• Mortality• Self- evaluated
health
OtherMeasures& Methods
• Diagnosis• Disease severity• Clinical endpoint• Treatment
• Work productivity• Costs of care• Mortality• Self-evaluated
health
GoldStandard
OtherMeasures& Methods
Adapted from: Ware JE, Jr. and Keller SD: Interpreting general health measures, in: Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1995: Chapter 47.
Clinical CausesEconomic & Social
Consequences
HRQoL Validation Strategies
363636
Interpreting HRQoL Scores
30 40 50
Chronic Lung
Disease
55
Average Well Adult
MOS Physical Component Summary (PCS)(Mean = 50, SD = 10)
DiabetesType II
Congestive Heart
Failure
Asthma
Average Adult
PopulationHealth Surveys
Chronic Disease Registry
General Population Health SurveysChronic Disease Registry
45
ChronicKidneyDisease
35
37373737
Interpreting HRQoL Scores
30 40 50
Chronic Lung
Disease
55
Average Well Adult
MOS Physical Component Summary (PCS)(Mean = 50, SD = 10)
DiabetesType II
Congestive Heart
Failure
Asthma
Average Adult
General Population Health SurveysChronic Disease Registry
45
Clinical Trials
ChronicKidneyDisease
35
RAAfterRx
RABefore
Rx
TreatmentEffect (net)
Strand etal Arth & Rheum, 1999;42(9): 1870-78
PopulationHealth Surveys
ClinicalTrials
Chronic Disease Registry
• Content of questionnaire items
• Statistically significant change
• Important reduction in disease burden
• Reduction in subsequent expenditures
• Substantial increase in work productivity
38383838
Are Generic HRQoL Measures Responsive?
Clinical Markers
Specific Symptoms
Impact of Disease-specific
Problems
Reference: Ware JE and Frendl D “Systematic review of the responsiveness of SF-36 HealthSurvey measures to efficacious pharmaceutical therapies in published double-blind randomized controlled trials.” Presentation at the 13th Annual ISPOR Congress, Prague CzechRepublic, 11/6/2010; Value in Health, Vol 13, (7): A239–A250.
(1) (2) (3) (4)
How much does your arthritis limit your usual activities or enjoyment of everyday life?
• Not at all• A little• Moderately• Extremely
ArthritisImpact
In general, would you say your health is:
• Excellent• Very good• Good• Fair• Poor
GenericHealth
Arthritis SymptomsHave you had the following symptoms: Joint pain, swelling, burning sensation:
• Almost every day• Several days a week• A few days a month• Not at all
SF-36 RCTs
Generic Functioning, Well-being
and Evaluation
X-ray, Disease Progression
SF-36 agreed with primary endpoint (across Rx):
• 219 of 253 RCTs• 86.6%
393939
Reference – Headache Impact: MS Bayliss, JE Dewey, R Cady etal., A.Study of the Feasibility of Internet Administration of a computerizedhealth survey: The Headache Impact Test (HIT), Quality of Life Research, 2003, 12: 953-961
References – Asthma Control: Nathan RA, Sorkness CA, Kosinski M et al., “Development of the Asthma Control Test: A survey for assessing asthma control. Journal of Allergy and Clinical Immunology. 2004;113: 59-65.
Internet Sampling and Data Collection
40
Health Outcomes Research Using Handhelds – Mobile Metrics
Ware NIH – Health-Related Quality of Life - 2014 40
41
Final Comment
For more information: john.ware@umassmed.edu
Business Week 11/26/2001
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