Patient-Centered Outcomes Research
Linda Crew McNamara, RN, MBA
PSDTO Advisory Panel & Stakeholder Reviewer
GHS Research Lecture Series
Greenville, SC
June 10, 2014
1
Overview of presentation
• Background (Evolving Research
Paradigm)
• Patient Centered Outcomes Research
• Patient Centered Outcomes Research
Institute (PCORI)
• Tips for strong proposal
2
Background
Community-based Participatory Research:
• Lack of trust (legacy of research abuse)
• Helicopter researchers
• Authority of community members less
meaningful than researchers
• Limited resource sharing
• Lack of equitable compensation for patient
partners
3
Background
• Length of time from bench to bedside
• Lack of relevance to real-life patient concerns
• Research findings not disseminated broadly or
not understandable
• Rare diseases and subgroups of patients not
addressed
• Limited amount of behavioral health research
4
Background
Patients wanted more active role:
“Nothing about me without me.”
“PhDs of the sidewalk.”
5
Background
“Changing the way patients are thought about,
as consumers of research rather than just study
subjects, is a core principle for engaging patients
in research.”
6
What is Patient-Centered Outcomes
Research (PCOR) ?
“Research that sees through the
eyes of patients.”
Annals of Internal Medicine 18 Sept 2012
Relevant
Usable
Easily accessible
7
What is Patient-Centered Outcomes
Research (PCOR)
PCOR helps patients and their caregivers communicate and make
informed healthcare decisions, allowing their voices to be heard in
assessing the value of healthcare options.
This work answers patient’s questions.
Given my personal
characteristics,
conditions, and
preferences, what
should I expect will
happen to me?”
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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?”
Examples of Patient-Centered
Outcomes
• Quality of life and functional status
• Psychological factors: anxiety,
depression
• Economic outcomes (work hours,
income lost)
• Treatment decision related
(decisional conflict, regret)
9
Why PCOR?
• Patients are one of healthcare’s richest,
yet largely untapped resources.
• Patients as meaningful partners can lead
to more effective research, more useful
evidence, and care than leads to
improved outcomes.
• Positive outcomes on a much faster
track.
10
PCORI Has a Broad and Complex Mandate
“The purpose of the Institute is to assist
patients, clinicians, purchasers, and policymakers 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...”
-- from Patient Protection and Affordable
Care Act
PCORI Is Accountable for Changing Practice
“(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…”
-- from Patient Protection and Affordable
Care Act
PCORI Focus
Comparative Clinical Effectiveness Research
Patient-centered
Answering questions that matter to patients and
other clinical decision makers
Comparisons of outcomes that matter to patients
13
National Priorities for Research
Assessment of Prevention,
Diagnosis, and Treatment
Options
Improving Healthcare
Systems
Addressing Disparities
9
Communication &
Dissemination Research
Accelerating PCOR and
Methodological Research
Key Feature: Emphasizes Engagement
for Getting to Practical, Useful Research
Generate and
Prioritize Research
Questions
Portfolio Review
Review and Conduct
Research
Advise Us
on What to
Study
Review
Proposals
and Partner
in Research
Tell Us How
We’re Doing
Help Us Share
the Findings
Dissemination and
Implementation
Engagement Goals
Build a Patient-Centered Outcomes Research
Community
Engage the PCOR Community in Research
Promote Dissemination and Implementation
32
Eugene Washington PCORI
Engagement Awards
Awards of up to $250,000
to provide “wrap-around”
support and enhance
impact of PCORI’s
research initiatives.
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 engagement and partnering
in the conduct and usage of
comparative effectiveness research
Three Types of Awards
 Knowledge
 Training and Development
 Dissemination and Implementation
33
The National Patient-Centered Clinical
Research Network (PCORnet)
Clinical Data Research Networks
System-based networks, such as
hospital systems
• 11 Networks
• $76.8 Million Awarded
Patient-Powered Research Networks
Patients with a single condition
form a research network
Coordinating Center
Provides technical and logistical
assistance under the direction of the
Steering Committee and PCORI staff
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• 18 Networks
• $16.8 Million Awarded
PCORI 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)
15
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
39
PCORI’s Merit Review Process
Applications are reviewed
against five 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
40
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
21
Tips for a Strong Proposal
Meaningful engagement at each phase:
•
•
•
•
•
Topic generation
Research design/modification
Implementation
Dissemination
Outcomes
Tips for a Strong Proposal
Identifying & selecting research questions.
Ask the type of questions that patients can
address.
“Patients don’t have research questions. They just
have questions.”
What is your biggest health issue, and what do you
need to know about it?”
Tips for a Strong Proposal
Identifying & selecting research questions.
Leverage the potential of “big data.”
• Centers for Medicare & Medicaid Services (CMS)
claims database to find the sickest people, and
then identify the research questions that would
provide the knowledge they need.
• Mining social media, using algorithms to find the
questions already being asked by patients.
Tips for a Strong Proposal
Applications must answer major question related
to decisions about clinical choices, healthcare
delivery, or another relevant issue in the
announcement.
Gap analysis and systematic reviews must support
the need for the study.
Tips for a Strong Proposal
Must be comparative clinical effectiveness
research:
Evaluate and compare health outcomes and the clinical
effectiveness, risks, and benefits of 2 or more medical
treatments, services, and health care interventions protocols
for treatment, care management, and delivery, procedures,
medical devices, diagnostic tools, pharmaceuticals (including
drugs and biologicals), integrative health practices, and any
other strategies or items being used in the treatment,
management, and diagnosis of, or prevention of illness or
injury in individuals
Tips for a Strong Proposal
Differentiate between patient engagement &
patient-centeredness.
Engagement: Inclusion of patients in the research
process.
Patient centeredness: addresses questions that
patients and their families care about in clinical
settings.
Tips for a Strong Proposal
Proposals should demonstrate superior technical
merit.
Study design that best aligns with study topic and
answers research question in most efficient and
effective way.
Refer to PCORI Methodology Standards as a guide.
Tips for a Strong Proposal
Research should adhere to PCORI’s Methodology
Standards.
Methodology Standards: 11 Broad Categories
13
Formulating Research Questions
Data Networks
Patient-Centeredness
Data Registries
Data Integrity and Rigorous
Analyses
Adaptive and Bayesian Trial
Designs
Preventing/Handling Missing Data
Causal Inference
Heterogeneity of Treatment
Effects
Studies of Diagnostic Tests
Systematic Reviews
Tips
PCORI Funding Center: Tools and resources for
applicants.
Help desk: Call 202.627.1884; complete an online
inquiry form; or schedule a call with program staff.
Institute of Medicine workshops and publications.
Pragmatic Clinical Studies and Large
Simple Trials
Seeks to fund investigator-initiated
research that compares two or
more alternatives for:
Opportunity Snapshot
Number of Anticipated
Awards: Six to Nine
•
Addressing prevention, diagnosis,
treatment, or management of a disease
or symptom
•
Improving health care system–level
approaches to managing care; or
Funds Available: $90 Million
•
Eliminating health or healthcare
disparities.
•
Maximum Project Duration:
5 Years
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.
Maximum Direct Costs Per
Project: $10 Million
30
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
Engagement and PCORI Activities
getinvolved@pcori.org
42
Thank You
Linda Crew McNamara, RN, MBA