STATISTICAL COMMISSION and UN ECONOMIC COMMISSION FOR EUROPE CONFERENCE OF EUROPEAN STATISTICIANS

Joint UNECE/WHO/Eurostat Meeting on the Measurement of Health Status (Budapest, Hungary, 14-16 November 2005)

Session 2 – Invited paper

Working Paper No.7

22 November 2005

STATISTICAL OFFICE OF THE EUROPEAN COMMUNITIES (EUROSTAT) WORLD HEALTH ORGANIZATION (WHO) Overview of PROMIS Network

University of Washington

PROMIS

This work was funded by the National Institutes of Health through the NIH Roadmap for Medical Research. Principal Investigator Deborah N. Ader, Ph.D.

Director, Behavioral and Prevention Research Program National Institute of Arthritis and Musculoskeletal and Skin Diseases

Goals, Network, Tasks

Presented by:

Dagmar Amtmann, Ph.D.

PI University of Washington Center for Outcomes in Rehabilitation Research

PROMIS

P atient R eported O utcomes M easurement I nformation S ystem

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

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.

PROMIS will develop:

A publicly available, adaptable and sustainable Internet-based system that will: 1.

Administer individually “tailored” questionnaires (using Computer Adaptive Testing (CAT) technology) to measure health status 2. Collect and analyze responses

The Structure of the PROMIS Network

PRS PRS PRS S C C SC NIH Science Officers SAB

The PROMIS

Network

●

University of Washington

●

Stanford University

● ●

Evanston Northwestern Healthcare

♦ ● ● ●

University of Pittsburgh Stony Brook

●

University

● ●

▲ NIH UNC –Chapel Hill

●●

Duke University

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

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 1.0

0.8

0.6

0.4

0.2

0.0

-3 -2

  

-1 0 Theta 1

 

2

 

3 Questionnaire administered to large representative sample

 

2.5

   

2.0

Item Response Theory (IRT) 1.5

1.0

0.5

0.0

-3 -2 -1 0 Theta 1 Item Bank (IRT-calibrated items reviewed for reliability, validity, and sensitivity)

CAT

2 Short Form Instruments 3

PROMIS Data Repository

Assessment Module

Laptop PDA Phone Web

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

What is the PROMIS Time Line?

2005-06: 2007:

Choose specific domains Identify, review instruments and items

April 2006:

Collect pilot response data

2008:

Continue data collection Create alpha version of CAT Conduct final calibration process, Put CAT into final form

2009:

Carry out feasibility testing Build sustaining partnerships

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.

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

PROMIS Independent Projects and PIs

Continued •

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

Health PRO

PROMIS Domain Framework

General Health Physical Health Satisfaction Function/Disability Symptoms Mental Health Satisfaction Emotional Distress Cognitive Function Positive Psychological Functioning Social Health Satisfaction Role Participation Social Support Upper Extremities (ADL): grip, buttons, etc Lower Extremities (ADL): walking, arising, etc.

Central (ADL): neck & back (twisting, bending) Activities: IADL (e.g. errands) Pain Fatigue Other Anxiety Depression Anger/Aggression Alcohol & Substance Use Negative Impacts of illness Subjective Well-Being (positive effect) Meaning and Coherence (spirituality) Mastery and Control (self-efficacy) Positive Impacts of Illness Performance Satisfaction

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.

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).

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).

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.

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

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

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

Expert Item Revision

• Put item in the PROMIS format • 7 day recall period • Preferred response options • Stand alone on one screen

Revision Example

Original Due to your feet How much difficulty did you have climbing up or down curbs 101 point scale VAS (0 = No difficulty to 100 = unable) Revised Over the last 7 days: Could you climb up or down one step?

0 = Without difficulty 1 = With some difficulty 2 = With much difficulty 3 = Unable to do

Final Revisions

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

PROMIS Website http://www.nihPROMIS.org/

Contact Information:

Shani Rolle, M.S.

NIH Coordinator [email protected]