Robin Newhouse 4/7/14 PCORI Seminar series

1. Healthcare Decisions and Methods
that Matter:
The Link to Patient
Centered Outcomes Research
Methodological Standards
Robin Newhouse, PhD, RN, NEA-BC, FAAN
University of Arkansas for Medical Sciences
Translational Research Institute
April 7, 2014

2. Key Points
PCOR methods matter…they
1. affect design, methods
and outcomes.
2. produces evidence for practice
that can be used to make
decisions by patients, providers,
caregivers and policy makers.
3. use outcomes that people care
about.
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3. 3

4. The Patient Centered Outcomes
Research Institute
Robin Newhouse is Chair of the Methodology Committee of the Patient
Centered Outcomes Research Institute (PCORI).The views expressed in
this presentation are those of the authors and not necessarily those of
PCORI.

5. 5
• Research has not
answered many
questions patients face.
• People want to know
which treatment is right
for them.
• Patients need information
they can understand and
use.
Why PCORI?

6. “The purpose of the Institute is to assist
patients, clinicians, purchasers, and policy-
makers in making informed health
decisions by advancing the quality and
relevance of evidence concerning the manner
in which diseases, disorders, and other health
conditions can effectively and appropriately
be prevented, diagnosed, treated,
monitored, and managed through research
and evidence synthesis...and the
dissemination of research findings with
respect to the relative health outcomes, clinical
effectiveness, and appropriateness of the
medical treatments, services...”
PCORI Has a Broad and Complex Mandate
-- from Patient Protection and Affordable
Care Act
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7. PCORI Is Accountable for Changing Practice
-- from Patient Protection and Affordable
Care Act
“(g) FINANCIAL AND GOVERNMENTAL
OVERSIGHT. …
(2) REVIEW AND ANNUAL REPORTS.
…
(iv) Not less frequently than every 5
years … the overall effectiveness of
activities conducted under this section …
such review shall include an analysis of the
extent to which research findings are
used by health care decision-makers,
the effect of the dissemination of such
findings on reducing practice variation
and disparities in health care…”
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8. Mission
PCORI helps people make informed health care decisions, and improves health
care delivery and outcomes, by producing and promoting high integrity, evidence-
based information that comes from research guided by patients, caregivers and
the broader health care community.
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9. Strategic Goals
Influence Research Funded by Others
Speed the Implementation and
Use of Evidence
Increase Quantity, Quality and
Timeliness of Research Information
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10. Engagement Goals
Promote Dissemination and
Implementation
Engage the PCOR Community in Research
Build a Patient-Centered Outcomes
Research Community
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11. National Priorities for Research
Assessment of Prevention,
Diagnosis, and Treatment
Options
Improving Healthcare
Systems
Communication &
Dissemination Research
Addressing Disparities
Accelerating PCOR and
Methodological Research
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12. 6
Patients Are Our “True North”

13. Study Design/
Implementation
Evaluation
Topic
Selection and
Research
Prioritization
Merit Review
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We engage stakeholders at every step
Key Features of PCORI Research

14. Key Features of PCORI Research
Our work answers patient’s questions.
“Given my personal characteristics, conditions and
preferences…
…what should I
expect will
happen to me?”
…what are my
options and
what are the
potential
benefits and
harms of those
options?”
.. what can I do
to improve the
outcomes that
are most
important to
me?”
…how can
clinicians and the
care delivery
systems they work
in help me make
the best decisions
about my health
and healthcare?”
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15. Key Features of PCORI Research
Study the benefits and harms of
interventions and strategies
delivered in real-world settings
Compare at least two alternative
approaches
Research Should:
Special Topics of Interest:
Be based on health outcomes that
are meaningful to the patient
population
Be likely to improve current clinical
practices
Conditions that heavily burden
patients, families and/or the health
care system.
Chronic or multiple chronic
conditions
Rare and understudied conditions
Conditions for which outcomes
vary across subpopulations
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Formulating Research Questions
Patient-Centeredness
Data Integrity and Rigorous
Analyses
Preventing/Handling Missing Data
Heterogeneity of Treatment
Effects
Key Features of PCORI Research
Research should adhere to PCORI’s Methodology
Standards.
Data Networks
Data Registries
Adaptive and Bayesian Trial
Designs
Causal Inference
Studies of Diagnostic Tests
Systematic Reviews
Methodology Standards: 11 Broad Categories

17. Patient Engagement vs. Patient-Centeredness
 Patient engagement is about having patients as partners in
research as opposed to merely subjects
 Active engagement between scientists, patients, and
stakeholders
 Community, patient, and caregiver involvement already in
existence or a well-thought out plan
 Patient- Centeredness is a component of what PCOR is looking
for in research applications
 Does the project aim to answer questions or examine outcomes
that matter to patients within the context of patient
preferences?
 Research questions and outcomes should reflect what is
important to patients and caregivers
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18. Engage Patients and Stakeholders
in the Research Process
 Patients and Caregivers
 Patient Organizations
 Practice-Based Research Networks
 Medical Groups
 Health Plans
 Integrated Delivery Systems
 Disease Registries
 Centers for Medicaid and Medicare
Services
 State and Local Health Agencies
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19. Engagement Principles
Trust
Transparency
Co-learning
Reciprocal
Relationships
Partnerships
Honesty
Incorporates the Engagement Principles* and conceptual framework of patient
engagement in research developed by PCORI’s scientific team (Source: Curtis, P,
Slaughter-Mason, S, Thielke, A, Gordon, C, Pettinari, C, Ryan, K, Church, B, King, V(2012).
PCORI Expert Interviews Project)
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20. Examples of Promising Practices from First
150 Awards
Patients participate in:
Determining what needs
to be researched
The creation of the intervention to
be studied
Defining outcomes important to
patients
Leading the project as Co-PI,
consultant or advisory committee
member
Determining methods and
processes of research study
Recruiting and retaining
participants
Collecting data and, co creating
privacy and confidentiality policies
Creation of patient friendly
informed consent forms
Evaluating the patient engagement
throughout the life of the project
Disseminating research results to
advocacy organizations and
research participant community
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21. Methodology Committee of the Patient Centered Outcomes Research Institute. (2013). Hickam, D., Totten, T., Berg, A., Rader, K.,
Goodman, S., Newhouse, R. (Eds). Section II, Prioritizing Research Questions and Development of the Translation Table, pages
11-20.
http://www.pcori.org/assets/2013/11/PCORI-Board-Meeting-Methodology-Report-for-Acceptance-1118131.pdf
PCORI Translation Table

22. Our Growing Research
Portfolio
pfaawards.pcori.org

23. Funded Projects to Date
Total number of research
projects awarded : 279
Total funds awarded:
$464.2 million
Number of states where we
are funding research:
38 states (plus the District of Columbia
and Quebec, Canada)
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Snapshot of Funded Projects

25. Assessment of Prevention, Diagnosis, and
Treatment Options
Seeks to fund investigator-
initiated research that:
• Compares the effectiveness of
two or more options that are
known to be effective but have not
been adequately compared in
previous studies.
• Among compared population
groups, investigates factors that
account for variation in treatment
outcomes that may influence
those outcomes in the context of
comparing at least two treatment
approaches.
Portfolio Snapshot
By primary health topic
• 65 Projects
• $117 Million Awarded
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26. Selection of Peritoneal Dialysis or Hemodialysis for Kidney Failure:
Gaining Meaningful Information for Patients and Caregivers
Francesca Tentori, MD, MS,
Arbor Research Collaborative for Health
Engagement
• Interviews over 130 patients with
kidney disease to determine how to
address personal preferences in
choosing type of dialysis:
hemodialysis or peritoneal dialysis.
Potential Impact
• Over 100,000 patients start dialysis
in the United States each year.
• This research could provide
practical information regarding the
consequences (outcomes) of each
choice for patients with kidney
disease and their families.
Methods
• Research is completed through
qualitative methods.
Aims to identify factors that
matter to most to patients with
kidney disease and study how they are
impacted by different types of dialysis,
focusing on hemodialysis and
peritoneal dialysis. Today, patients
often choose a dialysis type without
fully understanding how it will impact
their lives
Assessment of Prevention, Diagnosis and Treatment Options,
awarded December 2012
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27. Improving Healthcare Systems
Seeks to fund investigator-initiated
research on effects of system
changes on :
• Patients’ access to high quality,
support for self-care, and
coordination across healthcare
settings.
• Decision making based on patients’
values.
• Experiences that are important to
patients and their caregivers, such
as overall health, functional ability,
quality of life, stress, and survival.
• The efficiency of healthcare delivery,
as measured by the amount of
ineffective, duplicative, or wasteful
care provided to patients.
Portfolio Snapshot
By primary health topic
• 41 Projects
• $76.5 Million Awarded
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28. Evaluating the Impact of Patient-Centered
Oncology Care
Sarah Scholle, MPH, DPH
National Committee for Quality Assurance
Engagement
• A broad multi-stakeholder advisory
group will help define the Patient-
Centered Oncology Care model.
Potential Impact
• Could change practice by
addressing current gaps in cancer
care and providing patients and
clinicians with important
information about what kind of
care is possible.
Methods
• Research is completed through
qualitative and quantitative
analysis.
Tests and evaluates using the
patient-centered medical home
(PCMH) model of care for treating
oncology patients. The goal is to
examine whether the model improves
patient experiences and quality of
care, reduces events such as ED visits
and hospital stays, and whether its
adoption varies across practices.
Improving Healthcare Systems,
awarded May 2013
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29. Communication and Dissemination
Research
Portfolio Snapshot
• 25 Projects
• $43.2 Million Awarded
Seeks to fund investigator-
initiated research in:
• Clinician engagement with CER.
• Translating research, decision
support interventions, and risk
communication.
• For this funding
announcement, studies of
decision support aids are not
encouraged.
• Distribution of CER to patients,
caregivers, and providers.
By population; some projects address
multiple populations.
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30. Patient-Identified Personal Strengths (PIPS) vs. Deficit-Focused
Models of Care
Kurt Stange, MD, PhD,
Case Western Reserve University
Engagement
• Engages patients, caregivers, and
primary care clinicians in identifying
mechanisms by which leveraging
focusing on patient-identified
strengths might affect processes
and patient-centered outcomes of
care.
Potential Impact
• Could change practice by
demonstrating a method for
motivating positive change and
engaging patients in ways that the
usual deficit-based model of
chronic care cannot.
Methods
• Uses a mixed methods approach.
Compares a model of chronic care
focused on patient-identified
personal strengths vs. deficit-focused
models of care to leverage strengths
that can help patients to live a fulfilling
life with multiple chronic illnesses.
Aims to build simulation models for
how focusing on patient-identified
strengths can change outcomes.
Communication and Dissemination,
awarded December 2012
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31. Addressing Disparities
Portfolio Snapshot
• 31 Projects
• $53 Million Awarded
By primary health topic
Seeks to fund investigator-initiated
research that:
• Compares interventions to reduce
or eliminate disparities in patient-
centered outcomes.
• Identifies/compares promising
practices that address contextual
factors and their impact on
outcomes.
• Compares health care options
across different patient
populations.
• Compares and identifies best
practices within various patient
populations for information sharing
about outcomes and research.
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32. Long-Term Outcomes of Community Engagement to
Address Depression Outcomes Disparities
Kenneth Brooks Wells, MD, MPH
University of California, Los Angeles
Engagement
• Community agencies collaborate to
tailor depression toolkits to needs
and strengths of community.
Potential Impact
• Could change practice by providing
information about how depressed
patients prioritize outcomes and
make decisions. Also, could impact
practice by showing how clinicians
respond to patients’ preferences.
Methods
• Research is completed both a
mixed methods approach and a
randomized controlled trial.
Project looks at whether a
community engagement
intervention or technical assistance
model will improve clients’ mental
health and physical functioning and
reduced risk factors for homelessness.
It will also identify patient preferences
and priorities and assess community
capacity to respond to these priorities.
Addressing Disparities Research Project,
awarded December 2012
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33. Portfolio Snapshot
• 30 Projects
• $28.1 Million Awarded
Seeks to fund investigator-initiated
research that:
Improving Methods for Conducting Patient-
Centered Outcomes Research
• Addresses gaps in
methodological research relevant
to conducting patient-centered
outcomes research (PCOR).
Results of these projects will
inform future iterations of
PCORI’s Methodology Report.
• Focuses on Patient-Reported
Outcome Measurement
Information System (PROMIS)-
related research.
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34. The National Patient-Centered Clinical
Research Network (PCORnet)
System-based networks, such as
hospital systems
Patient-Powered Research Networks
Coordinating Center
Provides technical and logistical
assistance under the direction of the
Steering Committee and PCORI Staff.
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• 11 Networks
• $76.8 Million Awarded
• 18 Networks
• $16.8 Million Awarded
Patients with a single condition
form a research network
Clinical Data Research Networks

35. Pragmatic Clinical Studies and Large
Simple Trials
Opportunity Snapshot
Number of Anticipated
Awards: Six to Nine
Funds Available: $90 Million
Maximum Project Duration:
5 Years
Maximum Direct Costs Per
Project: $10 Million
Seeks to fund investigator-initiated
research that compares two or
more alternatives for:
• Addressing prevention, diagnosis,
treatment, or management of a disease
or symptom
• Improving health care system–level
approaches to managing care; or
• Eliminating health or healthcare
disparities.
• Research topics of particular
interest identified by stakeholders,
or questions included in IOM’s Top
100 Topics for CER or AHRQ’s
Future Research Needs.
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36. Eugene Washington PCORI
Engagement Awards
Smaller awards, up to $250,000 total, to provide “wrap-around”
support and enhance impact of PCORI’s scientific research.
Objectives
 Engage new groups who have not previously been involved with PCORI
 Develop new mechanisms for disseminating research findings
 Promote research done differently by supporting the engagement and
partnering
Three Types of Awards
 Knowledge: Support knowledge of PCORI’s work, and inform about our
program efforts
 Training and Development: Training and development of “non-usual
suspects” and non-traditional researchers
 Dissemination and Implementation: Disseminate the results of our
research to promote implementation into practice
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37. Application and Review
Process
pcori.org/apply

38. How to Submit an Application
Visit pcori.org/apply
Key Dates
Funding Announcements
Application Guidelines and Templates
Guidance on the PCORI
Methodology Standards
Sample Engagement Plans
Frequently Asked Questions
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39. PCORI’s Merit Review Process
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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
Applications are reviewed
against five criteria:
Applications are reviewed
by a committee of two
scientists, one patient,
and one other stakeholder
PCORI’s Board of
Governors makes funding
decisions based on merit
review and staff
recommendations
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40. Have a Question?
General Inquiries
info@pcori.org | (202) 827-7200
Research/Programmatic Questions
sciencequestions@pcori.org | (202) 627-1884
Administrative/Financial/Technical Questions
pfa@pcori.org
43

41. Connect with PCORI
Bryan Luce, PhD, MBA
Chief Science Officer
bluce@pcori.org
Regina L. Yan, MA
Chief Operating Officer
ryan@pcori.org
Joe V. Selby, MD, MPH
Executive Director
jselby@pcori.org
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42. Find Us Online
www.pcori.org
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44. Straus, S.E., Tetroe, J. & Grahm, I.D. (2009). Knowledge to action: What it is and what it isn’t. In
Knowledge Translation in Health Care: Moving from Evidence to Practice. Hoboken,
N.J.:Wiley-Blackwell, pp. 3-9.
Johantgen, M., Newhouse, R. P. (2013). Participating in a Multi-Hospital Study to Promote Adoption
of Heart Failure Guidelines: Lessons Learned for Nurse Leaders. Journal of Nursing
Administration, 43(12), 660-666.
44

45.  2 hospitals
 HF patients (N=40, 20 each hospital)
 Nurses who care for HF patients on study units
This study is funded by AHRQ as a subproject (Newhouse, PI) in PATient-centered
Involvement in Evaluating the effectiveNess of TreatmentS (PATIENTS)
Program.
( PATIENTS PI, Mullins, 1R24 HS22135-01)
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Expanding PCOR through Advancements in
Implementation & Dissemination Science
• Develop infrastructure for dissemination and implementation
of research products and findings to patients, patient
advocates, clinicians, and healthcare systems
• Build sustainable digital infrastructure for data mining, data set
integration, and virtual collaborations for training and research
• Provide mentorship for faculty research development
• Offer tools and methods for implementation and dissemination
PATient-centered Involvement in Evaluating the effectiveNess of TreatmentS
(PATIENTS) Program. AHRQ (PI, Mullins).
(1R24 HS22135-01)

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PCOR is a Win for Everyone
PATIENT RESEARCHER
Informed decision making
Meaningful
research
Not a “one-
size-fits-all”
result
Interaction
Continuous
Topics and research
questions
Translate/disseminate
results
Comparators and
outcomes

48. Implementation Science:
Best Practices
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• Mixed methods
• Engagement of target audience and stakeholders
• Conceptual models and implementation frameworks (RE-AIM)
• Tailoring interventions to context
• Fidelity
• Measurement (context and outcomes)
• Evidence-based interventions
Required to build the science:
1) Core set of implementation concepts and metrics
2) Standards for implementation methods
3) Reporting standards for implementation studies
Newhouse, R.P., Bobay, K, Dykes, P.C., Stevens, K.R., Titler, M. (2013).
Methodology Issues in Implementation Science. Medical Care, 51,S32-S40.
doi: 101097/MLR.0b013e31827feeca

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50. Committed to advance research to
improve health care…………
Robin Newhouse, PhD, RN, NEA-BC, FAAN
newhouse@son.umaryland.edu