Seeking Input on Future PROMIS® Research: Educating Patients and Stakeholders about PCORI’s...

Seeking Input on Future PROMIS® Research: PCORI’s Request for Information (RFI)

January 30, 2013

Webinar Objectives

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Describe PCORI’s unique mission and activities

Describe PROMIS®

Summarize PCORI’s Request for Information (RFI) seeking input on future PROMIS® research

Question and answer session

Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information

Introductions: Speakers

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Lori Frank, PhD Director, Research Integration and Evaluation Patient-Centered Outcomes Research Institute (PCORI)

Laura Lee Johnson, PhD

Biostatistician, Science Officer for the PROMIS® Statistical Center National Center for Complementary and Alternative Medicine (NCCAM) National Institutes of Health James Witter, MD, PhD, FACR

Chief Science Officer for PROMIS® Medical Officer/Rheumatic Diseases Clinical Program National Institute of Arthritis and Musculoskeletal and Skin Disease (NIAMS) National Institutes of Health


PCORI’s unique mission and activities

About PCORI

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An independent non-profit research organization authorized by Congress as part of the 2010 Patient Protection and Affordable Care Act (ACA).

Committed to continuously seeking input from patients and a broad range of stakeholders to guide its work.


Why PCORI?

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Patients have questions that research can answer People want to know which treatment is right for them Patients need information they can understand and use


Our Mission

Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information 7

PCORI helps people make informed healthcare decisions, and improves healthcare delivery and outcomes, by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community.

Research PCORI Supports

Comparative Effectiveness Research (CER) Patient-centered Answering questions that matter to patients and other

healthcare decision makers


PCORI Merit Review Process

PCORI review committees include scientists, patients, and other stakeholders to bring diverse perspectives to the review process. PCORI’s unique merit review criteria ensure that research funded by PCORI is scientifically rigorous and patient-centered.


Merit Review Criteria

1. Impact of the condition on the health of individuals and populations

2. Potential for the study to improve healthcare and outcomes

3. Technical Merit 4. Patient-centeredness

5. Patient and stakeholder engagement

10 Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information

Our Growing Research Portfolio

11 Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information

Total number of research projects awarded to date: 297 research projects

Total funds committed to date: $464 million

Number of states where we are funding research: 41 states

Background & Opportunities January 30, 2014

James Witter, MD, PhD, FACR: NIAMS/NIH

Laura Lee Johnson, PhD: NCCAM/NIH

Dynamic Tools to Measure Health Outcomes from the Patient Perspective

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Vision and Mission

• Vision – The Patient-Reported Outcomes Measurement

Information System (PROMIS®), funded by the National Institutes of Health, aims to provide clinicians and researchers access to efficient, precise, valid, and responsive adult- and child-reported measures of health.

• Mission – PROMIS uses measurement science to create an

efficient state-of-the-art assessment system for self-reported health.


PROMIS Resources

Advancing Knowledge >240 Peer-Reviewed Articles >100 journals >40,000 participants >10,000 children

Informatics Assessment Center Supports >800 active studies in past year alone

Tools (30 domains) 40+ Adult Measures

20+ Pediatric Measures

Translations Goal 100% Spanish

(Over 40 other languages)

Cooperative Group 12 Research Sites

3 Centers 150+ Scientists

NIH funding (>$100M) Numerous RFAs and supplements since 2004 to support Center & Sites

Applications Assess health domains of patients and healthy people in clinical trials and research as well as healthcare delivery and population surveys


Advancing Patient-Centered Outcomes

PROMIS: A Common Standard of PROs

Clinical Practice Clinical research

Surveys (CDC, NIH)

NIH FDA

Clinic Hospital

Industry

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Common Resource To Advance Patient-Centered Outcomes

•Patient-Centered •Cross Sectional

•Cognitive testing •Focus groups

Psychometrically focused testing Clinically focused testing

Fully develop domain

Fully test domain

•Patient Centered •Longitudinal

•NIH studies •Industry studies •National surveys •Clinical care

•Web based follow-up


PROMIS® Combines

• Item Response Theory (IRT) and Computer Adaptive Testing (CAT)

• Together, IRT and CAT provide precise measurement of individual symptoms

Low High

Levels of Physical Function

Item Difficulty

IRT models Latent Traits: People and Items Represented on the Same Scale

Are you able to run 5 miles

Are you able to

get out of bed

Low High


Computerized Adaptive Testing (CAT)

Integrates IRT with computers to administer a PRO instrument

• Selects questions on the basis of a patient’s response to previously administered questions

• Measurement is “adapted” to individual

• Skips uninformative items to minimize response burden

• Allows determination of person’s standing on a domain without a loss in measurement precision

Computer Adaptive Testing: An Illustration

0 1

2 3

- 1

- 2

- 3

high physical function

0 1

2

Question #2

1 2

Question #3

High Precision Minimal Respondent Burden

low physical function

Question #1

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Evolving Concept of Health

DOMAIN

(widely or universally relevant)

vs. DISEASE attributable


PROMIS Adult Fatigue Bank

• The fatigue (95) item bank evaluates a range of self-reported symptoms – Mild subjective feelings of tiredness – Overwhelming, debilitating, and sustained sense of

exhaustion – Decreases one’s ability to execute daily activities and

function normally in family or social roles

Fatigue Item Bank

Lower Back Pain

Same metric, same meaning

Depression

Heart Failure

Cancer

COPD

PROMIS Measures Tested in Six Conditions Condition Relevant Item Banks

COPD Physical Function Fatigue Pain Social Role Satisfaction Emotional Distress (Depression, Anxiety, Anger)

Heart Failure Physical Function Fatigue Social Role Satisfaction Depression

Low Back Pain Pain (Interference and Behavior) Physical Function Depression Fatigue Sleep Disturbance

Depression Emotional Distress (Depression, Anxiety, Anger) Sleep Disturbance Fatigue Physical Function Pain

Arthritis Physical Function

Cancer Pain Fatigue Emotional Distress (Depression, Anxiety) Physical Function


The PROMIS Metric

T Score Mean = 50 SD = 10

Reference: US General Population

50 35 40 45 55 60 65

PROMIS Fatigue Across Five Clinical Conditions

Average for General Population

COPD Stable (B) COPD Exacerbation (B)

Exacerbation to Stable

N = 125

50 35 40 45 55 60 65

PROMIS Fatigue Across Five Clinical Conditions

Average for General Population

COPD Stable (B) COPD Exacerbation (B

HF Pre-transplant HF Post-transplant

Exacerbation to Stable

Depression (B)

Depression (1 mo)

Depression (3 mos)

Cancer Chemo

(B)

Cancer w/ benefit (2 mos)

Back Pain (B)

Back Pain (1 mo)

Back Pain (3 mos)

N = 64

N = 310

N = 114

N = 229

N = 125


Child-Adult Linkage Studies

• Render child and

adult editions comparable • Same scale

• Enable life course outcome assessment

PROMIS® Profile Short Forms (29-43-57 items) (+ pain intensity)

Anxiety 29

Depression 28

Fatigue 95

Pain Interference 41

Sleep Disturbance 27

Physical Function 86

Satisfaction with Roles 14

4 6

8

Mental

Physical

Social


Sample Question

• PROMIS Short Form v1.0 – Physical Function 12a

• Are you able to get in and out of bed? – Without any difficulty – With a little difficulty – With some difficulty – With much difficulty – Unable to do

• http://www.nihpromis.org/measures/SampleQuestions


Assessment Center

• Online research management tool • Enables study-specific websites

– Secure data capture • Clinical studies can be customized • Includes PROMIS instruments

– Short forms, CATs, and Profiles • Detailed statistical/development history • Real-time scoring • www.assessmentcenter.net

http://www.assessmentcenter.net/

assessmentcenter.net


Demonstrated Capacity and Future Interest

Current Usage • Over 3,000 registered research users • Over 800 active PRO research protocols • Over 6.5 million patient responses collected

Additional Interest • Competitive grants funded • PROMIS short forms in REDCap library • Epic and other EHR vendors • NIH Clinical Center, Cleveland Clinic, other

clinical settings • US Department of Defense

PROMIS can be used with different modes of

administration


Clinical Settings and Systematic Integration of PROs

• Broderick, J. E., Morgan DeWitt, E., Rothrock, N., Crane, P. K., & Forrest, C. B. (2013). Advances in patient reported outcomes: The NIH PROMIS measures. eGEMs, 1(1), article 12. http://repository.academyhealth.org/egems/vol1/iss1/12/

• Examples – Cleveland Clinic, University of Washington,

Cincinnati Children’s Hospital, Northwestern University


University of Washington Outpatient HIV Clinic

• PRO measures integrated into clinic visits • Allotting time prior to the visit for assessment • General health-related quality of life • PRO measures have drawn provider attention to

depressive symptoms, poor medication adherence, and at-risk behaviors


Cincinnati Children’s Hospital Medical Center

• Using PROs in 12 subspecialty clinics • System-wide rollout planned • Over time will monitor health of the

medical center’s population as a whole by assessing general health-related quality of life


Kaplan et al Associate Change in Treatment for Inflammatory Bowel Disease with Outcomes

• Using individual patients as the unit of measure • Mobile and web-based data collection of

PROMIS and other measures • Generated graphical display of PRO data with

statistical process control charts • Determine when changes in medical therapy

were reflected in meaningful changes in PROs • Used the data to identify the most effective

treatment for a given patient in order to deliver personalized care


Robert H. Lurie Comprehensive Cancer Center of Northwestern University

• PROMIS CATs screen for distress, other outcomes, conduct needs assessment in gynecologic oncology patients

• Complete measures via EHR patient portal before visit

• Scores exceeding an established threshold or requests for services generate messages within EHR for appropriate clinical care team member

• Addresses accreditation standard set by American College of Surgeons Commission on Cancer for routine screening of distress

CAT Graph


Contributions to Future Clinical Research & Care

• Precision – improved measurement precision across the full range of patient-reported outcomes

• Efficiency – less respondent burden • Standardization – more interpretable

research with standard terminology and metrics • Common language between research and

practice fosters CER • International clinical trial applications


PROMIS: The Road Ahead

• Person-Centered Outcomes Research Resource (PCORR) – http://grants.nih.gov/grants/guide/rfa-files/RFA-CA-13-008.html

– Trans-NIH – PROMIS, NIH Toolbox, Neuro-QoL, ASCQ-Me

• Using patient-centered outcomes • Transition to long-term sustainability

• Ongoing implementation and validation – Investigator Initiated R01s – NIH issued Funding Opportunity

Announcements


Where to Find More Information

• PROMIS www.nihpromis.org – FAQs, publications, instructional videos – Twitter (@promisNIH) – Newsletter

• Assessment Center http://www.assessmentcenter.net/ • Assessment Center Help Desk

([email protected])

http://www.nihpromis.org/

http://www.assessmentcenter.net/

mailto:[email protected]

Summary of PCORI’s Request for Information

Purpose of this RFI


Gather input and ideas about opportunities for future research related to PROMIS® Opportunities for supporting and/or expanding

existing PROMIS® research areas Opportunities for supporting new research

incorporating PROMIS® measures

Submissions to the RFI will assist PCORI with understanding the healthcare community’s perspective on opportunities to support PROMIS-focused research.

RFI Submitters

PCORI is interested in input from stakeholders who use or may use PROMIS® measures including: Patients/Consumers Caregivers/Family members of patients Patients/Caregiver Advocacy Organizations Clinicians Clinics/Hospitals/Health Systems Purchasers Payers Industry Researchers Policy Makers Training Institutions

Seeking Input on Future PROMIS Research: Educating Stakeholders about PCORI’s Request for Information 55

Information Requested

Research gaps in patient-centered CER for which PROMIS® measures could contribute to outcomes assessment Opportunities to expand use of PROMIS® measures in clinical care This may include opportunities related to platforms for

clinical use of PROMIS® measures Opportunities to evaluate performance of PROMIS® item banks across diverse clinical populations and settings

Seeking Input on Future PROMIS Research: Educating Stakeholders about PCORI’s Request for Information 56

Information Requested (continued)

Opportunities to develop new item banks within PROMIS® to aid with evaluation of care quality and the patient experience of clinical care Opportunities to maintain or expand the PROMIS® infrastructure with a focus on models for sustainability Opportunities for use of PROMIS® measures in regulatory approval for new drugs, devices, and diagnostics, including opportunities for measure qualification by the Food and Drug Administration.


Submission Guidelines

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Available on PCORI’s website: http://www.pcori.org/assets/2014/01/PCORI-RFI-PROMIS-011514.pdf Responses due by 5 p.m. (ET) on Feb. 12, 2014 E-mail responses to [email protected] All responses must include: Names of submitter(s) Organization affiliation(s) if applicable Contact e-mail address(es) and phone number(s)

Consenting submitters may be acknowledged by name on PCORI’s website or in PCORI events


http://www.pcori.org/assets/2014/01/PCORI-RFI-PROMIS-011514.pdf

http://www.pcori.org/assets/2014/01/PCORI-RFI-PROMIS-011514.pdf

Question and answer session

Thank you!

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Acknowledgements NIH staff

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Please send questions or comments to: Lori Frank, PhD Director of Research Integration and Evaluation [email protected] Seeking Input on Future PROMIS® Research: Educating Stakeholders

about PCORI’s Request for Information

http://www.pcori.org/home/signup



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Seeking Input on Future PROMIS® Research: Educating Patients and Stakeholders about PCORI’s...

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