Introduction to Outcomes and Effectiveness Research and Patient-Reported Outcomes (PROs)

1. Introduction to Outcomes and Effectiveness Research and Patient-Reported Outcomes (PROs) Professor of Medicine and Public Health, Dept Med, Div GIM/HSR; Dept Health Services, UCLA William Cunningham MD, MPH wcunningham@mednet.ucla.edu HPM 214 http://hpm214.med.ucla.edu/ January 6, 2014

2. Two Major Teaching Goals: • Measures of Health-Related Quality of Life (HRQOL) • Application of HRQOL measures to outcomes research studies HS 214, Winter 01·09·12

3. Randomized Trial Design Intervention Group • Measure and Compare Outcomes • HRQOL • Symptoms • Mortality • Complications • Laboratory data • Little control for case-mix required Study Population Randomize Control Group

4. Analytic Model for anOutcomes Study • Observation of groups • Nonrandomized assignment: intervention vs. control group • Outcomes • HRQOL • Lab data • Symptoms • Mortality • Utilization • *Control for case-mix needed • Severity • Comorbidity • Socio-demographics • other covariates HS 214, Winter 01·09·12

5. Health Services Background Concepts to HRQOL • Health status, quality of life and health services: - needs, outcomes and policy • Conceptualizing health: HRQOL • Measuring health: HRQOL - reliability, validity, acceptability • Applications to health services research HS 214, Winter 01·09·12

6. Key Problems With US Health Care System • Access to care • ~ 45 million people without health insurance • Costs of care • expenditures ~ $ 2.7 Trillion • Effectiveness (quality) of care • not all care delivered is beneficial • not all needed care is delivered HS 214, Winter 01·09·12

7. Behavioral Model of Health Services Utilization (Access) Health Status Predisposing Enabling Need Use Costs Satisfaction Perceived Health Personal Health Practices Personal/family Demographic Evaluated Health Community [Process] Social Structure Health Beliefs Adapted from Andersen 1968

8. Defining Health Outcomes • Health and Disease vs. Illness • Meeting Health Needs Results in Better Health • Use of Services or Process of Care Results in Better Health • Focus is on Intervention • Clinical • Community-Based • Policy HS 214, Winter 01·09·12

9. History of Health and Quality of Life Concepts: A Paradigm Shift • Changes in survival, epidemiology • Limits of medical technology • Public health and policy: populations • social indicators of health status, QOL • Chronic disease treatment: individuals • measures of function and well-being (HRQOL) • Cost-containment and consumerism • demonstration of health care value HS 214, Winter 01·09·12

10. WHO Definition of Health • Health is a total state of physical, mental and social well-being, and not merely the absence of disease - WHO, 1949 HS 214, Winter 01·09·12

11. How Do We Know If Care Is Effective? • Effective care maximizes probability of desired outcomes • Outcomes are markers of whether or not care is effective HS 214, Winter 01·09·12

12. Quality of Care Model Structure Process Outcomes HealthCareOrganizationCharacteristics Clinical Status • TechnicalExcellence • Appropriate • Effective ProviderCharacteristics HRQOL CommunityCharacteristics Satisfaction • Interpersonalexcellence • Patient-centered • Responsive PopulationCharacteristics Mortality

13. What Are Health Outcomes? • Traditional clinical endpoints • Death, disease occurrence, other adverse events • Clinical measures/biological indicators • blood pressure • blood hemoglobin level • symptoms, (e.g. fever) • Health-Related Quality of Life HS 214, Winter 01·09·12

14. Definition of Health-Related Quality of Life (HRQOL) • Ability to engage in daily functions: • physical (e.g. self-care) • role • social • Perceived well-being versus distress • Multi-dimensional HRQOL Physical Health Mental Health HS 214, Winter 01·09·12

15. Quality of environment Type of housing Level of income Social Support HRQOL is Not HS 214, Winter 01·09·12

16. Quality of Life, Health Status, & Health-Related Quality of Life (HRQOL) QOL • Life Satisfaction • Happiness • Environmental • Socio-economic • Community/Family • Political Health Status • Disease • Illness, Morbidity • Comorbidity • Physiologic, Anatomic • Mortality • Survival HRQOL HS 214, Winter 01·09·12

17. Valid Measurement of Health Is Critical • Develop/ select HRQOL measures for specific purpose • Evaluate performance of measures in target group, disease, or context • acceptability • reliability and validity HS 214, Winter 01·09·12

18. Example of HRQOL Measure Overall, how would you rate your current health? (Circle One Number) 0 1 2 3 4 5 6 7 8 9 10 Worst possible health (as bad or worse than being dead) Half-way between worst and best Best possible health HS 214, Winter 01·09·12

19. Introduction to Patient-Reported Outcomes (PROs)

20. Patient-Reported Outcomes (PROs) “Any report coming from patients about a health condition and its treatment” (U.S. FDA, 2006) Including • Health-related quality of life (HRQOL) • Satisfaction with treatment • Patient reports about care • Needs assessment • Adherence to treatment HS 214, Winter 01·09·12

21. Quality of Care Model for HIV Health IT Project ENSStructure IT NetworkEnhancements: -Bi-directional lab interface. -E-prescriptions ordering and re-fill interface. - Bi-directional referral and medical record linkage with LB Health Dept. Outcomes -HRQOL (SF-12) -Reports of care (CAHPS) -ARV use/adherence -Viral load count -CD4 count -Dr/Hospital/ER visits -Access to care -Stigma -Trust -Willingness to share PHI (preferences) Process of Care -CD4 testing -Viral load testing -Obtaining lab results -E-prescribing -Pharmacy re-fills -Referrals Patient Characteristics -Age -Race/ethnicity -Gender -Risk group -Income -Education -Insurance -CD4 -AIDS DX Conceptual model adapted from Donabedian HS 214, Winter 01·09·12

22. Patient-Reported Measures (PRMs) • Outcomes • HRQOL • Satisfaction with care • Mediators • Health behaviors (adherence) • Health Care Process • Reports about care (e.g., communication) • Needs assessment (preferences for care) HS 214, Winter 01·09·12

23. PRO Domains • HRQOL • Functioning (Behavior) • Physical functioning • Social/role participation • Symptoms and Well-Being (Feelings) • Pain, fatigue, etc. • General or global health perceptions • Satisfaction with treatment HS 214, Winter 01·09·12

24. Satisfaction with Treatment for Diabetes Insulin Treatment Questionnaire (22 items, alpha = 0.93) • “How satisfied are you with the stability of your blood sugar levels?” (not at all-completely) • Inconvenience of Regimen • 5 items, alpha = 0.87 • Lifestyle Flexibility • 3 items, alpha = 0.85 • Glycemic Control • 3 items, alpha = 0.86 • Hypoglycemic Control • 5 items, alpha = 0.81 • Insulin Delivery Device Satisfaction • 6 items, alpha = 0.83 • Andersen, RT 2004 HS 214, Winter 01·09·12

25. Other Important PRMs • Patient preferences for care • Needs assessment • Patient reports about care • Communication, coordination of care, getting needed care • Patient health behaviors • Taking medicines (medication adherence), exercising, not smoking • Other patient characteristics • Values and Beliefs (e.g., desire to participate in care) • Perceived social support HS 214, Winter 01·09·12

26. Communication (Neurologic Care) • In the last 6 months, how often did your personal doctor explain things in a way that was easy to understand? • In the last 6 months, how often did your personal doctor listen carefully to you? Never | Almost Never | Sometimes | Usually | Almost Always | Always HS 214, Winter 01·09·12

27. Coordination of Care • When you need your doctor to authorize more refills for a prescription, how often does it take more than two days? Never | Rarely | Sometimes | Most of the time |Always HS 214, Winter 01·09·12

28. Getting Needed Care • How much of a problem, if any, was it to get a regular HIV provider you are happy with? • In the last 6 months, how much of a problem, if any, was it to get a referral to a specialist that you wanted to see? • In the last 6 months, how much of a problem, if any, was it to get the care you or your doctor believed necessary? A big problem | A small problem | Not a problem HS 214, Winter 01·09·12

29. Other Important PRMs • Patient preferences for care • Needs assessment • Patient reports about care • Communication, coordination of care, getting needed care • Patient health behaviors • Taking medicines (medication adherence), exercising, not smoking • Other patient characteristics • Values and Beliefs (e.g., desire to participate in care) • Perceived social support HS 214, Winter 01·09·12

30. Medication Adherence • How often during the past week were you able to take your medication exactly as your doctor or nurse told you?  Would you say: None of the time | A little of the time | Some of the time | A good bit of the time | Most of the time | All of the time • Do you ever forget to take your medications? • Sometimes if you feel worse, do you stop taking your medications? • Did you not take any of yours medications over the past weekend? Never | Rarely | Sometimes | Often | Always HS 214, Winter 01·09·12

31. Other Important PRMs • Patient preferences for care • Needs assessment • Patient reports about care • Communication, coordination of care, getting needed care • Patient health behaviors • Taking medicines, exercising, not smoking (medication adherence) • Other patient characteristics • Values and Beliefs (e.g., desire to participate in care) • Perceived social support HS 214, Winter 01·09·12

32. Social Support People sometimes look to others for companionship, assistance, or other types of support.  How often were each of the following kinds of support available to you if you needed it during the past four weeks? • Someone to turn to for help with a personal problem? • Someone to help with daily chores if you were sick? • Someone to love and make you feel wanted? All of the Time | Most of the Time | Some of the Time |A Little of the Time |None of the Time HS 214, Winter 01·09·12

33. How is the Patient Doing? • Temperature • Respiration • Pulse • Weight • Blood pressure HS 214, Winter 01·09·12

34. What they are able to do And how they feel about their life Asking Her or Him About... “QOL is determined by its activities” - Me HS 214, Winter 01·09·12

35. What You Can Do (Functioning) • Physical functioning • Self-care -> vigorous activities • Role • Social • During the 4 weeks prior to your hospitalization for your stroke or TIA, how much of the time were you limited in the kind of work or other regular daily activities you do as a result of your physical health? All of the Time | Most of the Time | Some of the Time |A Little of the Time |None of the Time HS 214, Winter 01·09·12

36. How You Feel (Well-Being) • Emotional well-being • Pain • Energy • How much of the time during the past 4 weeks have you been happy? All of the Time | Most of the Time | Some of the Time | Little of the Time |None of the Time HS 214, Winter 01·09·12

37. Uses of HRQOL Measures • Monitoring population (and subgroups) • Observational studies • Clinical trials • Clinical practice HS 214, Winter 01·09·12

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39. Behavioral Risk Factor Surveillance System (BRFSS) • Nationwide survey of U.S. adults • Telephone interview (random digit dialing) • Percent poor or fair health about 16% HS 214, Winter 01·09·12

40. Greater % of fair or poor health reported by older adults (32% for 75+ vs. 8% for 18-24) HS 214, Winter 01·09·12

41. Burden of HIV Compared to Other Conditions and General Population Hays, Cunningham, et al. (2000), American Journal of Medicine HS 214, Winter 01·09·12

42. Uses of HRQOL Measures • Monitoring population (and subgroups) • Observational studies • Clinical trials • Clinical practice HS 214, Winter 01·09·12

43. The Effect of Engagement with Provider on HRQOL Outcomes * p<0.05 HS 214, Winter 01·09·12

44. Uses of HRQOL Measures • Monitoring population (and subgroups) • Observational studies • Clinical trials • Clinical practice HS 214, Winter 01·09·12

45. HRQOL Assessment by Providers May: • Facilitate patient-physician communication • Improve clinician understanding of patients’ problems (particularly those of a psychosocial nature) Detmar SB, Aaronson NK. Quality of life assessment in daily clinical oncology practice: a feasibility study. Eur J Cancer. 1998;34(8):1181-6. Detmar SB, Muller MJ, Schornagel JH, Wever LD, Aaronson NK. Health-related quality-of-life assessments and patient-physician communication: a randomized controlled trial. J Am Med Assoc. 2002;288(23):3027-34. Velikova G, Brown JM, Smith AB, Selby PJ. Computer-based quality of life questionnaires may contribute to doctor-patient interactions in oncology. Br J Cancer. 2002;86(1):51-9. Velikova G, Booth L, Smith AB, et al. Measuring quality of life in routine oncologypractice improves communication and patient well-being: a randomized controlled trial. JClin Oncol. 2004;22(4):714-24. HS 214, Winter 01·09·12

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47. Links http://gim.med.ucla.edu/FacultyPages/Hays/ http://www.fda.gov/CDER/GUIDANCE/5460dft.pdf http://gim.med.ucla.edu/kdqol/ https://www.cahps.ahrq.gov/ http://www.chime.ucla.edu/measurement/measurement.htm http://www.dartmouth.edu/~coopproj/more_charts.html http://www.facit.org/about/welcome.aspx http://healthmeasurement.org/ http://www.cdc.gov/hrqol/ http://www.cdc.gov/nchs/otheract/IAWG/IAWG.htm http://www.mapivalues.com/ http://www.nihpromis.org/ http://www.springer.com/medicine/journal/11136 http://www.sf-36.org/demos/SF-36.html http://www.gim.med.ucla.edu/apps/seminar/sem_ListSchedule.php HS 214, Winter 01·09·12