Hematinics and Erythropoietin- Pharmacology of Hematinics
Seeking Input on Future PROMIS® Research: Educating Patients and Stakeholders about PCORI’s Request for Information (RFI)
1. Seeking Input on Future
PROMIS® Research: PCORI’s
Request for Information (RFI)
January 30, 2013
2. 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
3. Introductions: Speakers
3
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
5. About PCORI
5
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
6. Why PCORI?
6
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
7. 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.
8. 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
9. 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
10. 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
11. 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
12. 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.
15. 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
19. 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
22. Computer Adaptive Testing:
An Illustration
0123-1-2-3
high
physical
function
012
Question #2
12
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
26. Fatigue Item Bank
Lower Back
Pain
Same metric, same meaning
Depression
Heart Failure
Cancer
COPD
27. 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
29. 5035 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
30. 5035 40 45 55 60 65
PROMIS Fatigue Across Five Clinical Conditions
Average for General Population
COPD Stable (B) COPD Exacerbation (B
HF Pre-transplantHF 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
34. 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
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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
42. 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
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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
(help@assessmentcenter.net)
54. 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.
55. 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
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56. 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
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57. 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
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58. 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 PROMISRFI@pcori.org
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
60. Thank you!
60
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
lfrank@pcori.org
Seeking Input on Future PROMIS® Research: Educating Stakeholders
about PCORI’s Request for Information