Working Paper No.7 22 November 2005 STATISTICAL COMMISSION and STATISTICAL OFFICE OF THE UN ECONOMIC COMMISSION F

1. Working Paper No.7 22 November 2005 STATISTICAL COMMISSION and STATISTICAL OFFICE OF THE UN ECONOMIC COMMISSION FOR EUROPEAN COMMUNITIES EUROPE (EUROSTAT) CONFERENCE OF EUROPEAN WORLD HEALTH STATISTICIANS ORGANIZATION (WHO) Joint UNECE/WHO/Eurostat Meeting on the Measurement of Health Status (Budapest, Hungary, 14-16 November 2005) Session 2 – Invited paper Overview of PROMIS Network University of Washington

2. PROMIS This work was funded by the National Institutes of Health through the NIH Roadmap for Medical Research. Principal InvestigatorDeborah N. Ader, Ph.D.Director, Behavioral and Prevention Research ProgramNational Institute of Arthritis and Musculoskeletal and Skin DiseasesGoals, Network, TasksPresented by:Dagmar Amtmann, Ph.D.PI University of Washington Center for Outcomes in Rehabilitation Research

3. Patient-Reported Outcomes Measurement Information System PROMIS

4. The PROMIS Outcome… To improve assessment of self-reported symptoms and other health-related quality of life measures across disabilities. Primary uses: • Enhance research • Improve clinical decision-making • Facilitate policy-making by health plan and systems and public programs

5. The PROMIS is on “the map” … The PROMIS is one of the key projects under the “Re-engineering the Clinical Research Enterprises” branch of the trans-NIH Roadmap Initiative. All NIH institutes contribute funds to the initiative.

6. PROMIS will develop: A publicly available, adaptable and sustainable Internet-based system that will: • Administer individually “tailored” questionnaires (using Computer Adaptive Testing (CAT) technology) to measure health status • Collect and analyze responses

7. PRS PROMIS Network PRS S C C SC PRS SAB NIH Science Officers The Structure of the PROMIS Network

8. University of Washington ● Stony Brook University Evanston Northwestern Healthcare ● ● ● ● ● ● ♦ ● ● ▲ NIH ● ● University of Pittsburgh Stanford University ● ● UNC –Chapel Hill Duke University ● The PROMIS Network

9. Goals of PROMIS • Item banks for specific domains • Relevant for a variety of chronic illnesses • Relevant for a variety of literacy levels • English, Spanish, and then other languages if funding can be acquired • Ability to “cross-walk” between PROMIS score and selected Legacy Measures

10. Items from Instrument A Items from Instrument B Items from Instrument C New Items Item Pool Content Expert Review Focus Groups Cognitive Testing Secondary Data Analysis      Questionnaire administered to large representative sample         Item Response Theory (IRT) Item Bank (IRT-calibrated items reviewed for reliability, validity, and sensitivity) CAT Short Form Instruments

11. PROMIS Data Repository Assessment Module Central Database Report Module Item Banks Study Protocols Web Laptop PDA IVR Patients Users Import Module Patients Clinicians Patient Reported Data Paper and Pencil Study Coordinators

12. What is the PROMIS Time Line? 2005-06: Choose specific domains Identify, review instruments and items April 2006: Collect pilot response data 2007: Continue data collection Create alpha version of CAT 2008: Conduct final calibration process, Put CAT into final form 2009: Carry out feasibility testing Build sustaining partnerships

13. PROMIS PRS’s • Each PROMIS primary research sites (PRS) has its own Independent Project, in addition to contributing to the Network goal of developing an item bank and CAT module for measuring patient reported outcomes in adults.

14. PROMIS Independent Projects and PIs • Duke University:Kevin Weinfurt, PhD Challenges for Using IRT-Based Assessments in Multi-center Clinical Trials • UNC, Chapel Hill:Harry A. Guess, MD, PhD Pediatric Reported Outcomes Assessment Using CAT ("Kitty") • University of Pittsburgh:Paul A. Pilkonis, PhD Psychiatric Symptoms and Social Functioning: IRT and DIF

15. PROMIS Independent Projects and PIsContinued • Stanford University:James F. Fries, MD Improved Outcome Assessment in Arthritis and Aging • Stony Brook University:Arthur A. Stone, PhD Ecological Validity in Patient-Reported Chronic Disease Outcomes • University of Washington:Dagmar Amtmann, PhD Improving Measurement of Pain and Fatigue and Increasing the Scientific Understanding of Pain and Fatigue in Children and Adults with Disabilities

16. Upper Extremities (ADL): grip, buttons, etc Lower Extremities (ADL): walking, arising, etc. Function/Disability Central (ADL): neck & back (twisting, bending) Activities: IADL (e.g. errands) Anxiety Depression Anger/Aggression Alcohol & Substance Use Negative Impacts of illness PROMIS Domain Framework General Health Physical Health Pain Symptoms Fatigue Other Satisfaction HealthPRO Emotional Distress Mental Health Cognitive Function Subjective Well-Being (positive effect) Satisfaction Meaning and Coherence (spirituality) Positive Psychological Functioning Mastery and Control (self-efficacy) Positive Impacts of Illness Performance Role Participation Social Health Satisfaction Social Support Satisfaction

17. Domain Definition: Physical Function Physical Function is defined as one's ability to carry out various activities, ranging from self-care (activities of daily living) to more challenging and vigorous activities that require increasing degrees of mobility, strength or endurance (Stewart & Kamberg, 1992; Haley, Coster & Binda-Sundberg, 1994; Haley, McHorney & Ware, 1994; Wilson & Cleary, 1995)*. Physical Function items, when considered as an outcome endpoint for clinical research in chronic illness, have a “capability” stem and a corresponding “capability” set of response items (e.g., “Are you able to…normally, with some difficulty, with moderate difficulty, with great difficulty, unable to do.”). This specifically excludes some items that may have great utility in other settings, as with “performance” items with the "Do you?" type of stem, which get at social or psychological issues, and “satisfaction” items (e.g., “How satisfied are you with your disability?”), which get at coping, stress, anxiety, etc. Also, since Physical Function/Disability is a much more fixed latent trait in chronic disease than Pain and some other domains, the response options will seldom be best expressed as "frequency" (“A little bit of the time”, etc.). Further, since many persons with a chronic disease will have more than one chronic disease and cannot distinguish the fraction of a problem attributable to each disease, Physical Function items attempt to quantitate the sum of these disease effects, leaving the teasing out of relative contributions to the analysis stage.

18. Domain Definition: Pain Pain is an unpleasant sensory and emotional experience associated with actual or potential tissue damage, or described in terms of such damage (Sherbourne, 1992a; Merskey & Bogduk, 1994; Chang, 1999; Meuser, et al, 2001). Pain is what the patient says it is (that is, the "gold standard" of pain assessment is self-report).

19. Domain Definition: Fatigue Fatigue is defined as an overwhelming, debilitating and sustained sense of exhaustion that decreases one’s ability to carry out daily activities, including the ability to work effectively and to function at one’s usual level in family or social roles (Stewart, Hays & Ware, 1992; North American Nursing Diagnosis Association, 1996; Glaus, 1998).

20. Domain Definition: Emotional Distress Emotional distress refers commonly to unpleasant emotions or cognitions that may interfere with the ability to cope with a disease, its physical symptoms, and its treatment. It covers a wide range of feelings, including worry, powerlessness, sadness, fear, depression, anxiety and panic (Schag et al, 1994; Lawton, Parmellee, Katz & Nesselroade, 1996; van’t Spijker, et al, 1997; Bottomley, 1998a; 1998b; Stark, et al, 2002)*. Problems in mental health may also be manifested, however, in maladaptive behaviors less commonly associated with subjective distress. For example, research in psychiatric nosology (Krueger, 1999) suggests that the “structure of common mental disorders” (excluding psychosis) can be captured at a general (second-order) level by two dimensions of psychopathology: an internalizing dimension reflected in unipolar depression and anxiety disorders and an externalizing dimension reflected in alcohol and substance use and antisocial behavior.

21. The PROMIS Core Domain Definition: Social Role Participation Social Role Participation: Role participation refers to involvement in, and satisfaction with, one’s usual social roles, including marital relationships, parental responsibilities, work abilities and social activities (Sherbourne, 1992b; McDowell & Newell, 1996; Dijkers, Whiteneck & El-Jaroudi, 2000)*. This has also been referred to as social adjustment (McDowell & Newell, 1996). *References provided in the slide notes

22. Current State of PROMIS Items • Items mostly from classical test theory • Created for a specific scale • Large variety of response options • Large variety of recall periods • Various methodologies in the development and validation of items

23. Qualitative Item Review Process • Identification of extant items • “Binning and Winnowing” • Legacy scales • Expert item revision • Focus groups on domain coverage • Cognitive interviews for individual items • Integrate quantitative analysis from archival data • Final revision

24. Expert Item Revision • Put item in the PROMIS format • 7 day recall period • Preferred response options • Stand alone on one screen

25. Revision Example

26. Final Revisions • Revise based on focus group feedback, cognitive interview results • Use Lexile framework to rate readability • Send on to field testing (April 2006)

27. PROMIS Website http://www.nihPROMIS.org/ Contact Information: Shani Rolle, M.S. NIH Coordinator RolleS@mail.nih.gov