PCD8 8-bit DMA Interface - EDT

Ware NIH Lecture Handouts 12/3/2012

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For more information:[email protected]@jwrginc.com

Health-Related Quality of Life (HRQoL) - 2013

Introduction to the Principles and Practice of Clinical ResearchNational Institutes of Health - Warren G. Magnuson Clinical Center Building 10

Lipsett Amphitheater, Bethesda, MD, December 3, 2012

John E. Ware, Jr., PhD, Professor and Division Chief

Department of Quantitative Health Sciences, Umass Medical School

Chief Science Officer, John Ware Research Group, Worcester, MA

Quality of Life (QoL)

•Community•Education

•Family LifeF i d hi

•Marriage•Nation•Neighborhood•Self

22

•Friendships•Health

•Housing

•Self•Standard of Living•Work

Source: Campbell, 1981

World Health Organization Definition of Health

“Health is a state of complete physical, mental, and social well-being and not

3

, gmerely the absence of disease or infirmity”

WHO, 1948


2


Health is Measured in Terms of:

• Bodily structure & function

• Specific symptoms

44

• What you do/are able to do – functioning

• How you feel – subjective ill- and well-being (+ and -)

• What you say it is – personal evaluation

Sources: Understanding Health Outcomes Educational Series

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

X-ray, Disease Progression

Continuum of Disease-specific and Generic Health Measures - Arthritis

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

55555

Clinical Markers

(1)

Specific Symptoms

(2)

Impact of Disease-specific

Problems

(3)

• Not at all

(4)

Generic Functioning, Well-being

and Evaluation

Health-related QOL(HRQoL)

Extremely

Adapted from: Wilson and Cleary, JAMA, 1995Ware, Annual Rev. Pub. Health, 1995

There is More to the Continuum

6

Clinical Markers

Specific Symptoms

(1) (2) (4)


and Evaluation


Problems

(3)


3


Predictive Validity:HRQoL is One of the Best Predictors

Future health

Inpatient expenditures

Outpatient expendituresImpact of Generic

7

Health-Related QOL (HRQoL)

p p

Job loss

Response to treatment

Return to work

Work productivity

Mortality

(3) (4)


Problems

Functioning, Well-being

and Evaluation

Summary of Content/Concepts for Widely-Used Generic Health Surveys

PsychometricUtility Concepts and Characteristics SIP HIE NHP QLI COOP DUKE MOS

FWBPMOSSF-36 QWB EQ-5D HUI SF-6D

CONCEPTS

Physical functioning Social functioning Role functioning Psychological distress Health perception (gen’l)

PROMIS

(-)(-)

(-)

( )

(-)

8Source: 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)

Pain Vitality Psychological well-being Sleep Cognitive functioning Quality of life

PROMIS = Patient Reported Outcomes Measurement Information System (2008)

(-)(-)

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

PROMIS References

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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]


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Medical Outcomes Study 36-Item Health Survey (SF-36)

Physical

1010

Utility Index(Brazier et al., 2002)

(Lam, Brazier, McGhee 2008)

Mental

Generic Health Profiles: Before & After Medication

Best Health

45

50

55

60

45

50

55

60

Arthritis

Norm45

50

55

60

45

50

55

60

Depression

11

Poorest Health

Component Summaries

SF-36 Subscales

Treatment

Baseline

25

30

35

40

45

25

30

35

40

45

Physical Mental P M25

30

35

40

30

35

40

Component Summaries

SF-36 Subscales

Physical Mental P M

Treatment

Baseline

Poorest Health

-

Next Step: Integrate and Standardize Disease-specific and Generic Measures

Best Health

+

45

50

55

60

45

50

55

60

US Norms(Mean=50

SD=10)

Best Health

Poorest Health

2530

35

40

Disease-SpecificImpact Scale

(QDIS)

2530

35

40

Example: Osteoarthritis

Physical HealthGeneric Summary

(PHGS)

)


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Comparison of Content of Generic And Disease-Specific Measures

Health Domains

Physical functioning

Social functioning

Role functioning

Psychological distress

Health perceptions (general)

Pain (bodily)

Energy/fatigue

SIP

PROMIS

Generic*

MOSFWBP

WO‐

MAC

Disease‐Specific*

SAQKD

QOL

StGeorg

DQOL

ML‐

HFQ

13

Health transition (B/S/W)

Energy/fatigue

Psychological well‐being

Sleep

Cognitive functioning

Quality of life

Sexual functioning

Specific symptomsSpecific treatments

DQOL ‐ Diabetes Quality of Life Measure

KDQOL ‐ Kidney Disease Quality of Life Questionnaire

MLHFQ ‐ Minnesota Living with Heart Failure Questionnaire

SAQ ‐ Seattle Angina Questionnaire

St. George's Respiratory Questionnaire

WOMAC ‐ 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 MeasurementInformation System (2007‐on)

*Source: Comparison adapted fromWare 1987; Ware, 1995

Content Areas* QOL Disease Impact Scale (QDIS)

1. Global (“activity” and “QOL”)2. Physical Functioning3. Mobility4. General Health 5 Fatigue5. Fatigue6. Cognitive7. Emotional8. Sleep9. Role Functioning 10. Social Functioning

*Note: Bank of 49 items reduced to 25 items with attribution to specific condition

Generic

During the past 4 weeks, how often did your healthlimit your ability to do your everyday activities?

• Very often

Anatomy of a Survey Item:QDIS Changes Attribution from Health

to Specific Disease

health

Disease-Specific Att ib ti

arthritis

Attribution

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• Very often

• Often

• Sometimes• Rarely

• Never

Attribution

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.


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• Standardized Metrics

• Adaptive survey administrations

Better Measures Are Being ConstructedSource:

Business Week

11/26/01

1616

Adaptive survey administrations

• Norm-based scoring

• Internet and mobile data collection

50 NormVigorous Activities, Not limited

Improving the Physical Function “Ruler”

1980 Old “Ruler”

> 75%@ Ceiling

1990 NEW “Ruler”

> 30%@ Ceiling

2008BETTER “Ruler”< 3 % @ Ceiling

50

70

60

1717

100

0

20

30

40

Mean = 50SD = 10

Climbing several flights of stairs

Walk one hundred yards

Bathing or dressing, Limited a little

00.2

0.4

0.6

10 20 30 40 50 60

.76

.24

.01a b

Limiteda little

Limiteda lot0.8

1.0 NotLimited

20

30

40

We Need the Health Equivalent of a Two-Sided Tape Measure

52 centimeters = 20.5 inches

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and Public-Private Partnerships Meetingthe Needs of Research and Business


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Short-Form Surveysand Ceiling Effects

7

Measuring Too Low -CeilingEffect

19

1

3

1

3

1

3

5

Some Thermometers Focus on a Very Narrow Range

130–190 °F54–88 °C

20Cooking Thermometer

Temperature

F° = 98.6 C° = 37.0

WaterFreezes

NormalHumanBlood

ShirtSleeve

Weather

21Example: Cross-Calibrating Celsius and Fahrenheit


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CAT

Solution: Adaptive Survey Methods

Patient scores here

22

CAT = Computerized Adaptive Testing

Adaptive Assessments of Disease ImpactMatch Questions to Each Patient’s Level

70

80

Severe60

23

Moderate

10

30

40

Mild

50

40

20

Higher is worse.Mean = 50SD = 10

First QuestionNoisy Score Estimate (+/- 15)

70

80

60

Score estimate,1st response = 62+/ 15

Severe

2410

30

40

50

40

20

+/- 15

Moderate

MildHigher is worse.Mean = 50SD = 10


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Second Question: Standard Error Reduced by One Third

Score estimate,2 responses = 64+/- 10

80

70

60

Severe

25

/ 10

10

30

40

50

40

20

Moderate

MildHigher is worse.Mean = 50SD = 10

Third Question: Standard ErrorCut in Half

80

70

60

Severe Score estimate,3 responses = 63

/ 7

2610

30

40

50

40

20

Moderate

Mild

+/- 7


Fourth Question: Standard ErrorCut by Two Thirds

80

70

60

Severe Score estimate,4 responses = 62

/ 5

2710

30

40

50

40

20

Moderate

Mild

+/- 5



10


Practical Implications of CATin Health Assessment

28

“Ceiling Effect”

r = 0.536N = 1016

r = 0.938N = 1016

CriterionScore

CriterionScore

A Promising Solution in 1999: CAT-Based Health Assessment

3 SD units

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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

Dynamic 5-Item HeadachePain Measure

ScoreScore

NoNoDisabilityDisability

Performance of 5-item CAT Scores Confirmed in NIH-Sponsored Studies

r = 0.98N = 2,753

r = 0.94N = 1,016

20 30 40 50 60 70

r = 0.96N = 263

10

20

30

40

50

60

70

30

Mental Health Headache Disability

-2

-1

0

1

2

3

-2 -1 0 1 2 3

DiabetesImpact

r = 0.93N = 100

Pediatric Disability

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


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What are the Advantages of Dynamic Assessments?

• More accurate risk screening

• Reliable enough to monitor individual outcomes

• Brevity of a short form –

3131

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

Matching Methods to Applications

5

6

7

5

7

5

PopulationSurveys

Group-LevelStudies

Patient-LevelAssessments

7

6

32

1

2

3

4

1

3

Single-Item

1

2

3

4

Multi-ItemScale

“Item Bank” (CAT Dynamic)

Most Functionally Impaired

NoisyIndividual

Classification

Very AccurateIndividual

Classification

Adaptive Survey Logic (ASLX ®) Flow Chart

ASLX® Screen Estimate Score & CI from Screen

NEG

POS

ConditionPresent?

Follow‐upAssessmentModules

GenericAssessment

Survey ContentAnd User

Interface (UI)

GenericHealth

Assessments

QDISAssessments(QOLIX® CCC)

YES

NONext Condition

33

SupplementalDomain

Assessment(CAT/Static)*

Repeat Cycle for Each Generic Domain

Repeat Cycle for Each Chronic Condition

Pos

SupplementalDisease ImpactAssessment

(CAT/Static)*

ASLX® Screen

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


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• Diagnosis

• Disease severityHR QOL

GoldStandard

• Work productivity• Costs of care

Mortality

• Diagnosis

• Disease severity

• Work productivity• Costs of care

M t lit

GoldStandard

Clinical CausesEconomic & Social

Consequences

HRQoL Validation Strategies

343434

• Responders

• Treatments

HR-QOL • Mortality• Self- evaluated

health

OtherMeasures& Methods

y

• Clinical endpoint

• Treatment

• Mortality• Self-evaluated

health

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.

Interpreting HRQoL Scores

Chronic Lung A th

PopulationHealth Surveys

Chronic Disease Registry

General Population Health SurveysChronic Disease Registry

353535

30 40 50

Lung Disease

55

Average Well Adult

MOS Physical Component Summary (PCS)(Mean = 50, SD = 10)

DiabetesType II

Congestive Heart

Failure

Asthma

Average Adult

45

ChronicKidneyDisease

35

Interpreting HRQoL Scores

Chronic Lung A th

General Population Health SurveysChronic Disease RegistryClinical Trials

RAAfterRx

RABefore

Rx

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

36363636

30 40 50

Lung Disease

55

Average Well Adult

MOS Physical Component Summary (PCS)(Mean = 50, SD = 10)

DiabetesType II

Congestive Heart

Failure

Asthma

Average Adult

45

ChronicKidneyDisease

35

RxRx

TreatmentEffect (net)

Strand etal Arth & Rheum, 1999;42(9): 1870-78


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Are Generic HRQoL Measures Responsive?

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

X-ray, Disease Progression

SF-36 agreed with primary endpoint (across Rx):

• 219 of 253 RCTs• 86.6%

37373737

Clinical Markers

Specific Symptoms


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)

SF-36 RCTs


and Evaluation

Internet Sampling and Data Collection

383838

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.

Health Outcomes Research UsingHandhelds –Mobile Metrics


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Thank You!

For more information: [email protected]

[email protected]

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