(Northwestern University, CAPriCORN CDRN) David Cella, PhD (Northwestern University, CAPriCORN CDRN) Patient centeredness and patient reported outcomes: PROMIS and its place in PCORnet

PROMIS Cooperative Group 2004-2014 Highlights More than 50 research protocols aligned with evolving PROMIS standards More than 50 grants Roadmap/Common Fund Individual Institutes PCORI, DoD, VA, US Army, CDC, multiple foundations, industry More than 50,000 people have provided data Adults and Children English, Spanish, Chinese and other languages Qualitative and quantitative

PROMIS® Measures Science Software The resource that we developed is the Patient-Reported Outcome Measurement Information System, or [CLICK] “PROMIS.” [CLICK] PROMIS is a collection of measures of PROs: 22 measures for adults, such as physical functioning and depression, 9 measures for pediatric populations, and more. These measures were developed using the state-of-the-science. [CLICK] Science: PROMIS is an effort to advance the state-of-the-science in measurement of patient-reported health status. For example, there’s a collection of statistical models known as item response theory, that have been developed for educational testing. PROMIS has been instrumental in discovering the best way to adopt these statistical models for patient-reported health data. [CLICK] Readily available software to disseminate and administer PROMIS measures. We’ll go into more detail about PROMIS measures, PROMIS science, and PROMIS software soon. Now it did take some time to develop this. [TRANSITION]

PROMIS Measures Adult Health Measures Pediatric Health Measures More than 1,000 individual items (questions) 51 distinct item banks or scales 20 more coming soon 20 languages Pediatric Health Measures More than 150 items (questions) 18 distinct banks or scales 22 more on the way 8 languages

PROMIS is Domain-specific; not Disease-specific How do we actually measure each domain? For almost every domain, we use the most advanced measurement science to create the most flexible type of tool--the item bank.

Cuts across different diseases A domain is the specific feeling, function, or perception you want to measure. [CLICK] PROMIS Domains cut across different diseases. Talk about studying the domain of FATIGUE in people with osteoarthritis, migraine sufferers, and people undergoing chemotherapy. Cuts across different diseases

PROMIS Domain Framework Symptoms Physical Health Function Affect Self-Reported Health Mental Health Behavior Cognition Global Health Relationships Social Health Function

An item bank is a large collection of items measuring a single domain. [CLICK] Definition [CLICK] Any and all . . . Let’s look at an item bank for the domain of Physical Functioning. [NEXT] Any and all items can be used to provide a score for that domain.

T Score Referenced to the US General Population The PROMIS Metric Mean = 50 SD = 10 Referenced to the US General Population

PROMIS Basic Tools Global Health Index Global-10 Derived from Item Banks Computerized Adaptive Testing (CAT) Dynamic testing averaging 4-5 items per domain Fixed Length Forms By individual domain (4-10 items) 7-domain health profiles (-29, -43, -57)

PROMIS® Profile Short Forms (v1) (29-43-57 items) 8 6 4 Anxiety 29 Mental Depression 28 Fatigue 95 Pain Interference 41 Physical The 7 sub-domains were selected to represent three higher level PROMIS domains, Mental Health, Physical Health, and Social Health. The short forms of different lengths, 4, 6, and 8 items, were designed to provide incrementally more precision and coverage for clinical populations. Sleep Disturbance 27 Physical Function 124 Social Satisfaction with Roles 14 (+ pain intensity) 12

Cycle of Development and Validation Qualitative Research and Item Writing Testing General Population Clinical Samples Item Bank Item bank is not fixed. Don’t make and put on shelf. We can continually work to evaluate and improve it. Perhaps adding items to help measure the extremes of the domain. Analysis Interpretation Refining

Efficiency

Computerized Adaptive Tests Question #1 high physical function 3 1 2 Question #2 1 2 Question #3 2 1 Purpose of this slide is to walk the audience through the CAT process using an example …and thereby transmit the message of narrower measurement range and higher precision with each question … I typically ese e.g. an example for depression, or any other topic I want to focus on … like first question “do you feel depressed”, answer “some of the time” … than we would like to know how depressed and the CAT might choose a question asking about self worth, and if the patient indicates that this is diminished also, a third question asking about thoughts of suicide might be appropriate … in this case the answer would be no, never … And maybe compare it with another scenario like the participant would have answered the first question, no I am not at all depressed, asking about suicide would not make sense, instead the CAT might have chosen a question asking about happiness … bla bla The conclusion is, we can make precise test over the entire range … Questionnaire with a high precision - AND a wide range - 1 - 2 low physical function - 3

Validity

Responsiveness in Depressive Disorder: PROMIS Depression

Comparability

PROMIS Measures Tested in Six Conditions Relevant Item Banks COPD Physical Function Fatigue Pain Social Role Satisfaction Emotional Distress (Depression, Anxiety, Anger) Heart Failure Depression Low Back Pain Pain (Interference and Behavior) Sleep Disturbance Arthritis Cancer Emotional Distress (Depression, Anxiety)

Same metric, same meaning Fatigue Item Bank Chemotherapy trial Osteoarthritis trial Heart Failure trial Items 1-10 CAT Items 6-12 The same PROMIS item bank can be used across conditions for varying degrees of severity across conditions. Across all types of diseases, PROMIS Fatigue scores have same interpretation. Facilitates comparisons, meta-analyses, health policy, etc. Epilepsy trial Diabetes trial Items 2, 4, 9, 13 Items 1-2 Same metric, same meaning

PROMIS Fatigue Across Five Clinical Conditions Cancer w/ benefit (2 mos) Cancer Chemo (B) N = 310 N = 229 Back Pain (3 mos) Back Pain (1 mo) Back Pain (B) N = 114 Depression (3 mos) Depression (1 mo) Depression (B) N = 64 HF Post-transplant HF Pre-transplant Exacerbation to Stable N = 125 COPD Stable (B) COPD Exacerbation (B) 50 35 40 45 55 60 65 Average for General Population

PROsetta Stone® A Rosetta Stone for Linking Patient-Reported Outcome Measures www.prosettastone.org USPHS Grant No. RC4 CA157236-01

Raw Score to T-Score Conversion Table (IRT Fixed Parameter Calibration Linking) for PHQ-9 to PROMIS PHQ-9 Score PROMIS T-score SE 37.4 6.4 1 42.7 5.3 2 45.9 4.8 3 48.3 4.7 4 50.5 4.3 5 52.5 4.0 6 54.2 3.8 7 55.8 3.7 8 57.2 3.6 9 58.6 3.5 10 59.9 3.4 11 61.1 3.3 12 62.3 13 63.5 3.2 14 64.7 15 65.8 16 66.9 17 68.0 3.1 18 69.2 19 70.3 20 71.5 21 72.7 22 74.0 23 75.3 24 76.7

Choi et al, Psychological Assessment, 26(2): 513-527, 2014 Figure 3. Comparison of clinical cut scores on the PROMIS Depression metric. CES-D: 16 or higher for positive clinical depression. PHQ-9: 5-9 (mild), 10-14 (moderate), 15-19 (moderately severe), 20 or higher (Severe). BDI-II: 0-13 (minimal), 14-19 (mild), 20-28 (moderate), 29 or higher (severe). Choi et al, Psychological Assessment, 26(2): 513-527, 2014

Raw Score to T-Score Conversion Table (IRT Fixed Parameter Linking) SF-36/PF-10 to PROMIS Physical Function SF-36/PF Score PROMIS T-score SE 10 24.5 4.0 11 28.3 2.8 12 30.3 2.5 13 32.0 2.2 14 33.4 2.1 15 34.8 2.0 16 36.0 17 37.2 18 38.4 1.9 19 39.5 20 40.7 21 41.8 22 42.9 23 44.1 24 45.3 25 46.7 26 48.2 2.3 27 49.9 28 52.0 2.9 29 55.0 3.5 30 61.7 5.7

Three Diseases; Three Instruments; and a Tower of Babel Sample Instrument Baseline Follow up Difference (sig) Relative value? RheumatoidArthritis HAQ-DI 1.20 0.30 - 0.90 (p<.05) ? Lung Cancer SF-36 PF-10 20 25 + 5.0 Diabetes PROMIS PF CAT 45 50 Just a note, FYI, HAQ-DI is usually scored in .25 increments (0 to 3.0). We estimated in smaller increments in our chart, which is more accurate.

PROsetta Stone Allows Comparability for CER Physical Function Change Across Three Conditions 40 25 30 35 45 50 55 Average for General Population Baseline Follow-up Lung Cancer Rheumatoid Arthritis Diabetes

GI PROMIS® in Clinical Practice Integrating GI PROMIS into practice: Delivering to patients via free app called “My GI Health” Currently available in L.A. VA, UCLA and U. Michigan Initiating pilot RCT under PROMIS supplemental funding Working with Athena Health to connect to EHR via API In short, we are delivering GI PROMIS questionnaires and scores via a free app called "My GI Health." We are currently using a web-based portal as part of our supplemental funding from our PROMIS grant. The web-based portal is a non-tethered app that will, in time, be "baked" into the EHR via API programming. For the moment, the app can be accessed by a link within the EHR, shown in the example here with the VA CPRS system. We are about to start a pilot RCT of GI PROMIS vs. usual care in our GI clinics at the West Los Angeles VA Medical Center, UCLA, and U. Michigan. The study will evaluate patient satisfaction with care, their perceptions about their provider's interpersonal skills, and shared decision-making. We deliver the PROMIS scores using a modified "heat map," shown in this slide deck, which we crated after iterative dashboard development with rounds of provider interviews. Details probably not important here. We are also working with the EHR vendor, athenahealth, to write APIs between My GI Health and their EHR. We hope to test that in a large network of over 49,000 sites via the athenahealth network, and are seeking funding for that study. Finally, we are finalizing the consumer-facing version of My GI Health, shown in these slides, which will allow anyone to "take the test," so to speak, to measure their GI symptoms and compare against population benchmarks. We will also allow for longitudinal tracking on the app, also shown in the slides. If you looks closely at the tracker slides, you'll see our current visualization for longitudinal PROMIS score tracking. When there is a "+" in a dot (see slide), it means the patient has improved beyond the MCID for a scale. Same in reverse if there is a "-" in a dot. Users can select the scale of interest, and it will come forward in full color, with other scales falling behind in muted pastel colors.   Quick slide overview: Slide 1: high level summary of current activities Slide 2: Example screenshots a patient completed on "My GI Health: Slide 3: Example report on "heat map" Slide 4: Shows link to "My GI Health" in VA CPRS system Slide 5: Example reports available for provider, including heat map linked to a fully automated HPI triggered by the system based off PROMIS scores (this provides detailed clinical context to the PROMSI heat map) Slide 6: Screen shots of consumer app to be launched in late Fall or early Winter Slide 7: Wireframes of longitudinal tracking on consumer app

GI PROMIS Symptom Tracker

Adults who Report Good or Better Physical Health, 2010 HP2020 Target: 79.8 Increase desired Age (years) NOTES: Data (except data by age group) are age adjusted to the 2000 standard population. SOURCE: National Health Interview Survey (NHIS), CDC/NCHS. Obj. HRQOL/WB-1.1 30

Adults who Report Good or Better Mental Health, 2010 HP2020 Target: 80.1 Increase desired Age (years) NOTES: Data (except data by age group) are age adjusted to the 2000 standard population. SOURCE: National Health Interview Survey (NHIS), CDC/NCHS. Obj. HRQOL/WB-1.2 31

> 300 PROMIS Publications

Concluding remarks PROMIS provides reliable, flexible and responsive treatment options for several patient-centered health domains Assessment options powered by Epic, REDCap, Assessment Center and others have made PROMIS attractive for health outcomes, health services and population health applications. IRT modeling and linking enable flexibility in assessment options while retaining common reporting metrics

Final recommendations Common Measures Working Group Final recommendations

Common Measures Working Group Lead: Dave Cella, PhD (CAPriCORN CDRN) Members: Reesa Laws, Thilo Deckersbach, Michael Kappelman, James Willig, and Richard Mularski Task: Select 5-10 common measures for collection across all PCORnet sites Activities: Identifying measures of greatest significance to the most networks. Selecting common measure (single items) for data collection across networks based on priority domains Sorting core items and highly recommended items

Common Measures WG Recommendations Focus on domains rather than disease or conditions Core domains include pain, fatigue, depression, sleep, physical function and social function, and general (global) perceptions of health and life quality Additional domains include: anxiety, medication adherence, satisfaction with care PROMIS single item measures as common measures Can estimate scores on the same metric as more precise assessments of the same domains, through both static short forms and CAT. large sample sizes allows for very good estimates of group average score based on only one question.

Common Measures WG Recommendations Instrument linking through co-calibration of items across PROMIS other instruments via PROsetta Stone will enable PCORnet to emphasize domain metrics over measures.

Recommended Common Measure Items: Core for Adults Domain Time Point Question Answer Choices Recommendation Physical Function Current Are you able to run errands and shop? 5=Without any difficulty 4=With a little difficulty 3=With some difficulty 2=With much difficulty Core Depression In the past 7 days I felt depressed 1=Never 2=Rarely 3=Sometimes 4=Often 5=Always Fatigue I feel fatigued 1=Not at all 2=A little bit 3=Somewhat 4=Quite a bit 5=Very much Sleep Disturbance I had a problem with my sleep 5=Not at all 4=A little bit 2=Quite a bit 1=Very much Pain Interference How much did pain interfere with your day to day activities?

Recommended Common Measure Items: Highly Recommended for Adults Domain Time Point Question Answer Choices Recommendation Anxiety In the past 7 days I felt uneasy 1=Never 2=Rarely 3=Sometimes 4=Often 5=Always Highly Recommended Social Roles & Activities None (current ability) I have trouble doing all of my regular leisure activities with others 5=Never 4=Rarely 2=Usually 1=Always

Recommended Common Measure Items: Adults and Pediatrics Domain Time Point Question Answer Choices Recommendation General Health None (current perception) In general, would you say your health is 5=Excellent 4=Very good 3=Good 2=Fair 1=Poor Core Quality of Life In general, would you say your quality of life is Global Physical Health In general, how would you rate your physical health? Global Mental Health In general, how would you rate your mental health including your mood and your ability to think? Highly Recommended

Recommended Common Measure Items: Core for Pediatric Domain Time Point Question Answer Choices Recommendation Pain Interference In the past 7 days I had trouble sleeping when I had pain 0=Never 1=Almost Never 2=Sometimes 3=Often 4=Almost Always Core Fatigue I got tired easily Depression   How often do you feel really sad 1=Rarely 4=Always

Recommended Common Measure Items: Highly Recommended for Pediatric Domain Time Point Question Answer Choices Recommendation Social Health How often do you have fun with friends 0=Never 1=Rarely 2=Sometimes 3=Often 4=Always Highly Recommended Family Relationships   How often do your parents listen to your ideas?

Questions/Comments

Supplemental slides: PROMIS Measures in redcap Example screen shots of what the PROMIS measures look like in REDCAP Supplemental slides: PROMIS Measures in redcap

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