For more information: Quality of Life. World Health Organization Definition of Health

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1 Health-Related Quality of Life - 10 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 1, 10 Quality of Life Community Education Family Life Friendships Health Housing Marriage Nation Neighborhood Self Standard of Living Work Source: Campbell, 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,

2 Health is Measured in Terms of: Bodily structure & function What you are able to do functioning How you feel distress & well-being Human Function And Well-Being What you say it is personal evaluation Sources: Understanding Health Outcomes Educational Series 4 What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Physical well-being Ease of interpretation Life satisfaction Physical activity limitations Symptoms of psychological distress Emotional behavior Role disability due to physical problems Psychological well-being General health perceptions Physical mobility Role disability due to emotional problems Satisfaction with physical condition Social activities with friends/relatives 5 Continuum of Disease-specific and Generic Health Measures - CKD Serum Creatinine CKD Symptoms Over the last 4 weeks I have had the following symptoms: Itching, muscle cramps, fatigue : Almost every day Several days a week A few days a month Not at all Another name: Patient-Reported CKD Outcomes Impact (PROs) How much did your lung/respiratory problems limit your usual activities or enjoyment of everyday life? Not at all A little Moderately Extremely Generic In general, would you say your health is Excellent Very good Good Fair Poor Specific Symptoms (1) (2) (3) (4) Adapted from: Wilson and Cleary, JAMA, 1995 Ware, Annual Rev. Pub. Health, 1995 Health-related QOL 6 6

3 Summary of Information About Widely-Used General Health Surveys NIH Roadmap PROMIS Initiative: Fatigue Negative affect Psychometric Utility Related Concepts and Characteristics SIP HIE NHP QLI COOP DUKE Pain MOS MOS QWB EURO HUI SF-6D FWBP Physical SF-36 Function -QOL CONCEPTS Social/role activity Physical functioning Social functioning Role functioning Psychological distress Health perceptions (general) Pain (bodily) Energy/fatigue Psychological well-being Sleep Cognitive functioning Quality of life Reported health transition SIP = Sickness Impact Profile (1976) MOS FWBP = MOS Functioning and Well-Being Profile (1992) HIE = Health Insurance Experiment surveys (1979) MOS SF-36 = MOS 36-Item Short-Form Health Survey (1992) NHP = Nottingham Health Profile (1980) QWB = Quality of Well-Being Scale (1973) QLI = Quality of Life Index (1981) EUROQOL = European Quality of Life Index (1990) COOP = Dartmouth Function Charts (1987) HUI = Health Utility Index (1996) DUKE = Duke Health Profile (1990) SF-6D= SF-36 Utility Index (Brazier, 02) Source: Ware JE, Jr.: Standards for validating health measures: Definition and content. Journal of Chronic Diseases 1987;: Adapted from Ware, SF-36 Health Survey Measures Physical Utility Index (Brazier et al., 02) Mental 8 There is More to the Continuum Specific Symptoms (1) (2) (3) (4) 9

4 Prediction and Risk Management: HRQOL is one of the Best Predictors (3) (4) Health-Related QOL (HR-QOL) Future health Inpatient expenditures Outpatient expenditures Job loss Response to treatment Return to work Work productivity Mortality 10 What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Ease of interpretation 11 What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Ease of interpretation 12

5 Problems with Short-Form Surveys Measuring Too Low - Ceiling Effect Some Thermometers Focus on a Very Narrow Range F C Cooking Thermometer 14 A Promising Solution in 1999: Computerized Adaptive Testing (CAT) Software Ceiling Effect Criterion Score r = N = SD units Criterion Score r = N = 1016 No Disability Skewed 5-Item Headache Pain Measure Ware JE, Jr, et al. Med Care. 00;38: Dynamic 5-Item Headache Pain Measure 15

6 Improving the Physical Function Ruler 1980 Old Ruler > Ceiling NEW Ruler > Ceiling Vigorous Activities, Not limited Norm Climbing several flights of stairs Walk one hundred yards 1,0 Limited 0,8 a lot Bathing or dressing, Limited a little 08 BETTER Ruler < 3 Ceiling Limited a little Not.76 Limited 0,6 0,4.24 0,2 0 a b Mean = SD = 10 What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Ease of interpretation 17 Original Thermoscope Results were not interpretable No marks on the ruler Poor reproducibility No interpretation guidelines Pain As Bad As It Could Be No Pain Visual Analogue Scale (VAS) 18

7 What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Ease of interpretation 19 Example: Cross-Calibrating Celsius and Fahrenheit Normal Human Blood Temperature F = 98.6 C = 37.0 Shirt Sleeve Weather Water Freezes Cross-Calibration Makes Scores Comparable and 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, 00 21

8 What do the results mean? What Do We Need to Measure Health Outcomes in the 21st Century? Outcomes that matter to patients Practical measures Coverage of a wide range Greater precision Comparability of scores Ease of interpretation 22 Standardization Scoring Software Health Profile Short Forms Long Forms Dynamic Forms Improvements in Short Form Measures of Health Status, J. Clinical Epidemiology, 08 Interpreting Health Measures Causes Diagnosis Disease severity Responders Treatments Gold Standard Measures In Question 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 Pharmacoeonomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1995: Chapter

9 What Do Changes in Health Mean? % reduction in physical disability 33% reduction in hospitalization Substantial increase in work productivity Reduced expenditures the following year Congestive Heart Failure Asthma Before Rx Asthma After Rx Chronic Lung Disease Diabetes Type II Treatment Average Adult Average Well Adult Physical Component Summary (PCS) 25 Advances & Solutions Business Week. November 26, 01. Improved psychometrics (Item response theory IRT) Computerized adaptive testing (CAT) software The Internet (and other connectivity) 26 Rheumatoid Precision Varies for Arthritis Static and Dynamic Forms and Score Levels for Physical Function Measures 6.0 PF-1 ( Static ) 5.0 PF-2 ( Static ) PF-10 ( Static ) Standard Error PF CAT-10 Reliability PF Criterion (Item Bank) Physical Function (PF), Mean = 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, 08, 61,

10 2 nd Solution: Assess Health Dynamically Patient scores here CAT CAT = Computerized Adaptive Testing 28 Dynamic Assessments Match Questions to Each Patient s Level 80 Severe Moderate Mild First Question Noisy Score Estimate (+/- 15) 80 Severe Moderate First Item Score = 62 +/- 15 Mild 10

11 Second Question: Standard Error Reduced by One Third 80 Severe Moderate Second Item Score = 64 +/- 10 Mild Third Question: Standard Error Cut in Half 80 Severe Moderate Third Item Score = 63 +/- 7 Mild Fourth Question: Standard Error Cut by Two Thirds 80 Severe Moderate Fourth Item Score = 62 +/- 5 Mild 10 33

12 Practical Implications of CAT in Health Assessment 34 Performance of 5-item CAT Scores Confirmed in NIH-Sponsored Studies r = 0.98 N = 2,753 r = 0.94 N = 1, r = 0.96 N = Mental Health Headache Disability Pediatric Disability 2 r = N = 1, Chronic Kidney Disease r = N = Diabetes Impact r = N = Post Acute Rehabilitation 35 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 numerous ways Markedly reduced data collection costs Monitor data quality in real time 36

13 3rd Solution: The Internet 37 Internet Health Portals for Sampling, Data Capture 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, 03, 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. 04;113: Health Outcomes Research Using ipods/iphones

14 Conclusions Patient-reported outcomes (PROs) are very useful Standardization of concepts & metrics is enabling comparisons & interpretation across applications Increasing widespread use proves that more practical tools will be adopted Technological advances include: item response theory (IRT), computerized adaptive testing (CAT) and electronic data capture Data quality studies support use of improved measurement tools across diverse populations