health-related quality of life (hrqol) - 2014 quality of life (hrqol) - 2014 john e. ware, jr., phd...
TRANSCRIPT
![Page 1: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/1.jpg)
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
![Page 2: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/2.jpg)
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.
![Page 3: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/3.jpg)
33
Quality of Life (QoL)
•Community•Education
•Family Life•Friendships
•Health•Housing
•Marriage•Nation•Neighborhood•Self•Standard of Living•Work
Source: Campbell, 1981
![Page 4: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/4.jpg)
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
![Page 5: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/5.jpg)
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
![Page 6: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/6.jpg)
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
![Page 7: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/7.jpg)
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)
![Page 8: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/8.jpg)
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
![Page 9: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/9.jpg)
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)
(-)
(-)(-)
(-)
(-)
(-)
![Page 10: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/10.jpg)
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
![Page 11: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/11.jpg)
1111
Medical Outcomes Study 36-Item Health Survey (SF-36)
Utility Index(Brazier et al., 2002)
(Lam, Brazier, McGhee 2008)
Physical
Mental
![Page 12: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/12.jpg)
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
![Page 13: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/13.jpg)
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)
![Page 14: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/14.jpg)
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
![Page 15: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/15.jpg)
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
![Page 16: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/16.jpg)
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.
![Page 17: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/17.jpg)
1717
• Standardized Metrics
• Adaptive survey administrations
• Norm-based scoring
• Internet and mobile data collection
Better Measures Are Being ConstructedSource:
Business Week
11/26/01
![Page 18: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/18.jpg)
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
![Page 19: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/19.jpg)
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
![Page 20: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/20.jpg)
20
Short-Form Surveysand Ceiling Effects
1
3
1
3
7
1
3
5
Measuring Too Low -CeilingEffect
![Page 21: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/21.jpg)
21Cooking Thermometer
Some Thermometers Focus on a Very Narrow Range
130–190 °F54–88 °C
![Page 22: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/22.jpg)
22
Temperature
F° = 98.6 C° = 37.0
Example: Cross-Calibrating Celsius and Fahrenheit
WaterFreezes
NormalHumanBlood
ShirtSleeve
Weather
![Page 23: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/23.jpg)
23
CAT
Solution: Adaptive Survey Methods
Patient scores here
CAT = Computerized Adaptive Testing
![Page 24: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/24.jpg)
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
![Page 25: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/25.jpg)
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
![Page 26: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/26.jpg)
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
![Page 27: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/27.jpg)
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
![Page 28: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/28.jpg)
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
![Page 29: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/29.jpg)
29
Practical Implications of CATin Health Assessment
![Page 30: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/30.jpg)
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
![Page 31: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/31.jpg)
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
![Page 32: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/32.jpg)
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
![Page 33: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/33.jpg)
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
![Page 34: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/34.jpg)
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
![Page 35: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/35.jpg)
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
![Page 36: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/36.jpg)
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
![Page 37: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/37.jpg)
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
![Page 38: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/38.jpg)
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%
![Page 39: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/39.jpg)
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
![Page 40: Health-Related Quality of Life (HRQOL) - 2014 Quality of Life (HRQOL) - 2014 John E. Ware, Jr., PhD Introduction to the Principles and Practice of Clinical Research National Institutes](https://reader034.vdocuments.us/reader034/viewer/2022051602/5ae49d557f8b9a9e5d8b5850/html5/thumbnails/40.jpg)
40
Health Outcomes Research Using Handhelds – Mobile Metrics
Ware NIH – Health-Related Quality of Life - 2014 40