Health-Related Quality of Life (HRQoL)

Transcription

1 Health-Related Quality of Life (HRQoL) John E. Ware, Jr., PhD, Professor and Chief Measurement Sciences Division, Department of Quantitative Health Sciences University of Massachusetts Medical School, Worcester, MA Introduction to the Principles and Practice of Clinical Research National Institutes of Health - Warren G. Magnuson Clinical Center Building 10 Lipsett Amphitheater, Bethesda, MD, February 27, 2012

2 What Are The Advances? Standardization (concepts & metrics) Better measures Adaptive (dynamic) survey administration Internet and other technologies 2

3 Quality of Life (QoL) Community Education Family Life Friendships Health Housing Marriage Nation Neighborhood Self Standard of Living Work Source: Campbell,

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,

5 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 Sources: Understanding Health Outcomes Educational Series 5

6 Continuum of Disease-specific and Generic Health Measures es - Arthritis X-ray, Disease Progression Arthritis Symptoms Arthritis Impact Generic Health Have 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 How much does your arthritis limit your usual activities or enjoyment of everyday life? Not at all A little Moderately Extremely In general, would you say your health is: Excellent Very good Good Fair Poor Clinical Markers Specific Symptoms Impact of Diseasespecific Problems Generic Functioning, Well-being and Evaluation (1) (2) (3) (4) Adapted from: Wilson and Cleary, JAMA, 1995 Ware, Annual Rev. Pub. Health, 1995 Health-related QOL (HRQoL) 6 6

7 Summary of Content/Concepts for Widely-Used Generic Health Surveys Psychometric Utility Concepts and Characteristics SIP HIE NHP QLI COOP DUKE MOS MOS PROMIS QWB EURO HUI SF-6D FWBP SF-36 -QOL CONCEPTS Physical functioning (-) Social functioning (-) Role functioning (-) Psychological distress (-) Health perception (gen l) Pain (-) Vitality (-) Psychological well-being Sleep Cognitive functioning Quality of life 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) Source: Adapted from Ware, 1995 MOS SF-36 = MOS 36-Item Short-Form Health Survey (1992) PROMIS = Patient Reported Outcomes Measurement Information System (2008) 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) 7

8 PROMIS Website & References References: 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 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: J Clin Epidemiol Nov; 63(11): 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). JClin Epidemiol. pde 2008 Jan; 61(1): Varni JW, Thissen D, Stucky BD, et al. PROMIS( ) Parent Proxy Report Scales: an item response theory analysis of the parent proxy report item banks. Qual Life Res Oct 5. [Epub ahead of print] 8

9 Anatomy of a Survey Item Recall period Item stem During the past 4 weeks, how often did your health ATTRIBUTION heart problem limit your ability to do your everyday activities such as work, school or chores? Attribution Domain/concept Never Rarely Sometimes Often Very often Response categories 9

10 SF-36 Health Survey Utility Index (SF-6D) Physical Utility Index (Brazier et al., 2002) (Lam, Brazier, McGhee 2008) Mental 10

11 Generic Health Profiles: Before & After Medication Best Health Norm Poorest Health Arthritis Physical Mental 25 P M SF-36 Subscales Component Summaries Depression Physical Mental P M SF-36 Subscales Component Summaries Treatment Baseline Treatment Baseline 11

12 Standardizing and Norming Generic and Disease-Specific Measures Generic Profile and Summaries Arthritis-Specific Best Health Norms Poorest Health Physical Mental P M SF-36 Subscales Component Summaries DIS Disease Impact Summary (DIS) Most Impact Lower Least Impact

13 There is More to the Continuum Clinical Markers Specific Symptoms Impact of Diseasespecific Problems Generic Functioning, Well-being and Evaluation (1) (2) (3) (4) 13

14 Predictive Validity: HRQoL is one of the Best Predictors Impact of Disease- specific Problems Generic Functioning, Well-being and Evaluation (3) (4) Health-Related QOL (HRQoL) Future health Inpatient expenditures Outpatient expenditures Job loss Response to treatment Return to work Work productivity Mortality 14

15 Methodological Advances Standardization (norm-based scoring Item response theory (IRT) Computerized adaptive testing (CAT) Internet and handhelds 15

16 Improving the Physical Function Ruler 1980 Old Ruler > Ceiling NEW Ruler > Ceiling Vigorous Activities, iti Not limitedit Norm Climbing several flights of stairs Walk one 0.2 hundred yards Bathing or dressing, Limited a little 2008 BETTER Ruler < 3 Ceiling 1.0 Limited Limited Not alot a little.76 Limited a b Mean = 50 SD = 10 16

17 IRT-Based Item Bank Combining HAQ and MOS Items Yielded a Better Scale + = Source: Business Week 11/26/01 MHAQ MOS Better Scale Physical Functioning (PF) Reference: M Martin, M Kosinski, J Bjorner, J Ware, R Maclean and T Li. Item Response Theory Methods Can Improve the Measurement of Physical Function by Combining the Modified Health Assessment Questionnaire and the SF-36 Physical Function Scale. Quality of Life Research, 2007; 16:

18 Cross-Calibration Makes Scores Comparable, More Interpretable Theta (θ) [Best Possible Estimate] Scales HDI HIMQ MIDAS MSQ DYNHA-5 (+) Note: Direction of scoring shown with arrows Source: Ware, Bjorner & Kosinski, Medical Care,

19 We Need the Health Equivalent of a Two-Sided d Tape Measure 52 centimeters = 20.5 inches and Public-Private Partnerships Meeting the Needs of Research and Business 19

20 Short-Form Surveys and Ceiling Effects Measuring Too Low - Ceiling Effect

21 Some Thermometers Focus on a Very Narrow Range F C Cooking Thermometer 21

22 Example: Cross-Calibrating Celsius and Fahrenheit Normal Human Blood Temperature Shirt Sleeve Weather F = 98.6 C = 37.0 Water Freezes 22

23 Solution: Assess Health Dynamically y Patient scores here CAT CAT = Computerized Adaptive Testing 23

24 Logic of Computerized Adaptive Testing (CAT) 1. Begin with initial iti score estimate t 2. Select & present 3. Score response optimal survey item No 5. Is stopping rule satisfied 4. Re- estimate health score and confidence interval Yes 6. End scale No 8. Administer next 7. End of battery? assessment scale Yes Source: Adapted from Wainer et al. (2000) 9. Stop 24

25 Dynamic Assessments Match Questions to Each Patient s Level el 80 Severe Moderate Mild

26 First Question Noisy Score Estimate (+/- 15) 80 Severe Moderate First Item Score = 62 +/- 15 Mild

27 Second Question: Standard Error Reduced d by One Third 80 Severe Moderate Second Item Score = 64 +/- 10 Mild

28 Third Question: Standard Error Cut in Half 80 Severe Moderate Third Item Score = 63 +/- 7 Mild

29 Fourth Question: Standard Error Cut by Two Thirds 80 Severe Moderate Fourth Item Score = 62 +/- 5 Mild

30 Practical Implications of CAT in Health Assessment 30

31 A Promising Solution in 1999: CAT-Based Health Assessment Ceiling Effect Criterion Score r = N = SD units No Disability Criterion Score r = N = 1016 Static 5-Item Headache Pain Measure Dynamic 5-Item Headache Pain Measure 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:II

32 Performance of 5-item CAT Scores Confirmed in NIH-Sponsored Studies r = 0.98 r = 0.94 r = N = 2,753 N = 1,016 N = Mental Health Headache Disability Pediatric Disability 50 2 r = 0.95 r = 0.93 r = N = 1,846 N = 100 N = Chronic Kidney Disease Diabetes Impact Post Acute Rehabilitation 32

33 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

34 Matching Methods to Applications Population Group-Level Patient-Level Surveys Studies Assessments Noisy Individual Classification Very 4Accurate Individual 3 Classification 2 Most Functionally Impaired Single-Item Multi-Item Scale Item Bank (CAT Dynamic) 34

35 Dynamic Short-Forms are More Precise Difference Missed Difference Detected Norm Static Short Forms Norm Dynamic Short Forms Improvements in Short Form Measures of Health Status, J. Clinical Epidemiology,

36 HRQoL Validation Strategies Gold Standard Diagnosis Disease severity Responders Treatments t HR-QOL Work productivity Costs of care Mortality Self- evaluated health Other Measures & 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

37 Rheumatoid Arthritis Using Comprehensive Item Banks as Criteria in Validating Physical Function Measures 6.0 Reliable 0.90 PF-10 Range 5.0 Reliable 0.90 CAT Range PF-2 ( Static ) PF-10 ( Static ) Standard Reliable 0.95 Error CAT Range PF CAT-10 Reliability Rheumatoid Arthritis (RA) General Population Physical Function (PF), Mean = Gold Standard (136-Item Bank, N=17,726) 726) Source: Rose M, Bjorner JB, Becker J, Fries JF and Ware JE. Evaluation of a preliminary physical function item bank supported expected advantages of the Patient-Reported Outcomes Measurement Information System (PROMIS). Journal of Clinical Epidemiology, 2008, 61,

38 HRQoL Validation Strategies Clinical Causes Diagnosis Disease severity Responders Clinical endpoint Treatments t t Gold Standard HR-QOL Economic & Social Consequences Work productivity Costs of care Mortality Self-evaluated evaluated health Other Measures & 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

39 Standardizing Metrics Across Applications General Chronic Population Disease Health Registry Surveys Chronic Disease Registry Population Health Surveys Congestive Heart Failure Chronic Lung Disease Chronic Kidney Disease Asthma Diabetes Type II Average Adult Average Well Adult MOS Physical Component Summary (PCS) (Mean = 50, SD = 10) 39

40 Standardizing Metrics Across Applications RA Before Rx General Chronic Population Clinical Disease Trials Health Registry Surveys Congestive Heart Treatment Failure Effect (net) Strand etal Arth & Rheum, 1999;42(9): RA Chronic After Lung Rx Disease Chronic Kidney Disease Content of questionnaire items Statistically significant change Chronic Clinical Disease Population Trials Registry Important t reduction in disease Health burden Surveys Reduction in subsequent expenditures Diabetes Type II Substantial increase in work productivity Asthma Average Adult Average Well Adult MOS Physical Component Summary (PCS) (Mean = 50, SD = 10) 40

41 Are Generic HRQoL Measures Responsive? X-ray, Disease Progression Arthritis SF-36 agreed with primary Arthritis Symptoms endpoint (across Rx): Impact Have you had the 219 of 253 RCTs following symptoms: Joint pain, swelling, burning sensation: 86.6% Almost every day Several days a week A few days a month Not at all How much does your arthritis limit your usual activities or enjoyment of everyday life? Not at all A little Moderately Extremely Generic Health In general, would you say your health is: Excellent Very good Good Fair Poor Clinical Markers Specific Symptoms Impact of Diseasespecific Problems Generic Functioning, Well-being and Evaluation (1) (2) (3) (4) Reference: Ware JE and Frendl D Systematic review of the responsiveness of SF-36 Health Survey measures to efficacious pharmaceutical therapies in published double-blind blind randomized controlled trials. Presentation at the 13th Annual European ISPOR Congress, Prague Czech Republic, November 6-9, 2010 SF-36 RCTs 41

42 Internet: Sampling, Data Collection and Reporting Reference Headache Impact: MS Bayliss, JE Dewey, R Cady etal., A.Study of the Feasibility of Internet Administration of a computerized health survey: The Headache Impact Test (HIT), Quality of Life Research, 2003, 12: 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:

43 Health Outcomes Research Using Handhelds Mobile Metrics

44 Final Comments Continuum of outcomes (specific-generic) Standardization of concepts and metrics Advances in psychometric methods Advances in data collection methods Comprehensive validation 44

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