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Seeking Input on Future PROMIS® Research: PCORI’s Request for Information (RFI)
January 30, 2013
Webinar Objectives
2
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
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information
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.
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information
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
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information
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
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information 8
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.
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information 9
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.
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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
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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
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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
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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
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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
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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
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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
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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
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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
assessmentcenter.net
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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
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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
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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
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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
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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
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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
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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
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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
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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
Summary of PCORI’s Request for Information
Purpose of this RFI
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information 54
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.
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information 57
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
Seeking Input on Future PROMIS® Research: Educating Stakeholders about PCORI’s Request for Information
Question and answer session
Thank you!
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Acknowledgements NIH staff
Stay current with email alerts at http://www.pcori.org/home/signup and follow us on Twitter @PCORI
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