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The Patient-Centered Outcomes Research Institute
M. Suzanne Schrandt, JD
Deputy Director, Patient Engagement
November 11, 2015
Who We Are and What We Do
About Us
• An independent research institute authorized by Congress in 2010 and governed by a 21-member Board of Governors representing the entire healthcare community
• Funds comparative clinical effectiveness research (CER) that engages patients and other stakeholders throughout the research process
• Seeks answers to real-world questions about what works best for patients based on their circumstances and concerns
Why Is Our Work Needed?
• For all the advances it produces, traditional healthcare research has not answered many questions patients face.
• People want to know which preventive, diagnostic, or treatment option is best for them.
• Patients and their clinicians need information they can understand and use.
Our Broad and Complex Mandate
“The purpose of the Institute is to assist patients, clinicians, purchasers, and policy-makers in making informed health decisionsby 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...
--from PCORI’s authorizing legislation
… and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments, services...”
Who Are Our Stakeholders?
Purchasers
Caregivers/Family MembersPayers
Patients/Consumers
Clinicians
Training Institutions
Policy Makers
Hospitals/Health SystemsIndustry
Patient/Caregiver Advocacy Organizations
How We Engage Stakeholders
Advisory Panels
Pipeline to Proposal Awards
Webinars and Workshops
AmbassadorsEngagement Awards
Merit Review Panels
Speakers Bureau
Our Research Focus
Adapted from Initial National Priorities for Comparative Effectiveness Research, Institute of Medicine of the National Academies
We Fund Comparative Clinical Effectiveness Research
• Measures benefits in real-world populations
• Describes results in subgroups of people
• Generates and synthesizes evidence comparing benefits and harms of at least two different methods to prevent, diagnose, treat, and monitor a clinical condition or improve care delivery
• Helps consumers, clinicians, purchasers, and policy makers make informed decisions that will improve care for individuals and populations
• Informs a specific clinical or policy decision
Note: We do not fund cost-effectiveness research
We Fund Patient-Centered Outcomes Research
PCOR is a relatively new form of CER that….
• Considers patients’ needs and preferences, and the outcomes most important to them
• Investigates what works, for whom, under what circumstances
• Helps patients and other healthcare stakeholders make better-informed decisions about health and healthcare options
We Fund Research That…
• Affecting large numbers of people across a range of population
• Placing a heavy burden on individuals, families, specific populations, and society
• Including rare diseases, which are difficult to study
Focuses on high-priority conditions:
We Fund Research That…
• Racial and ethnic minorities
• Older adults
• Low-income
• Residents of rural areas
• Women
• Children
• Individuals with special healthcare needs, including individuals with disabilities, individuals with multiple chronic diseases, individuals with rare diseases, and individuals whose genetic makeup affects their medical outcomes
Pays particular attention to specific populations:
• Patients with low health literacy/numeracy and limited English proficiency
• Lesbian, gay, bisexual, transgender (LGBT) persons
• Veterans and members of the armed forces and their families
Our National Priorities for Research
Assessment of Prevention, Diagnosis, and Treatment Options
Research that:
• Compares the effectiveness and safety of alternative prevention, diagnosis, and treatment options
• Determines which ones work best for different people with a particular health problem
Our National Priorities for Research
Assessment of Prevention, Diagnosis, and Treatment Options
Improving Healthcare Systems
Research that:
• Compares health system–level approaches to improving access
• Supports patient self-care, innovative use of health information technology, care coordination for complex conditions, and effective workforce deployment
Our National Priorities for Research
Assessment of Prevention, Diagnosis, and Treatment Options
Improving Healthcare Systems
Communication and Dissemination Research
Research on:
• Providing information produced by CER
• Empowering people to ask for and use the information
• Supporting shared decision making between patients and their providers
Our National Priorities for Research
Assessment of Prevention, Diagnosis, and Treatment Options
Improving Healthcare Systems
Communication and Dissemination Research
Addressing Disparities
Research on:
• Prevention, diagnosis, or treatment effectiveness
• Preferred clinical outcomes across patient populations
• Health care required to achieve best outcomes in each population
Our National Priorities for Research
Assessment of Prevention, Diagnosis, and Treatment Options
Improving Healthcare Systems
Communication and Dissemination Research
Addressing Disparities
Accelerating Patient-Centered Outcomes Research and Methodological Research
Research on:
• Building data infrastructure
• Improving analytic methods
• Training researchers, patients, and other stakeholders to participate in this research
The National Patient-Centered Clinical Research Network (PCORnet)
20 Patient-Powered Research NetworksPatients with a single condition form a research network
Coordinating CenterProvides technical and logistical assistance under the direction of a steering committee and PCORI staff
13 Clinical Data Research NetworksHealth system-based networks, such as hospital systems
The National Patient-Centered Clinical Research Network (PCORnet)
• Improve the nation’s capacity to conduct clinical research faster, more efficiently and less expensively, with greater power
• Establish a large, highly representative, national patient-centered clinical research network with a focus on conducting randomized and observational comparative studies
• Support a learning US healthcare system, which would allow for large-scale research to be conducted with greater accuracy and efficiency within real-world care-delivery systems
Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-term Effectiveness (ADAPTABLE)
Matthew T. Roe, MD, MHS Associate Professor of Medicine, Duke Cardiology
Potential Impact
• Demonstrate PCORnet’s capability to conduct important CER efficiently and economically
• Identify the optimal dose of aspirin for secondary prevention of heart attacks and stroke in patients with heart disease
Engagement
• ADAPTORS patient group involved throughout the trial, contributing to design, start-up, enrollment, follow-up, analysis, and dissemination
Methods
• Individual-randomized pragmatic clinical trial to compare the effectiveness of two doses of aspirin, using the PCORnet Common Data Model as a key data source
An innovative pragmatic clinical trial conducted within the PCORnet infrastructure to determine the optimal daily aspirin dose (325 mg versus 81 mg) for patients with heart disease. The trial leverages existing electronic health records, which link to insurance claims. A web-based patient portal collects patient-reported outcomes and additional patient-encounter data. The trial engages patients, their healthcare providers, and researchers in using the infrastructure that PCORnet has developed and continues to refine.
CER Methods and Infrastructure,awarded April 2015
• Broad PFAs, three cycles per year• Targeted PFAs, variable• Large Pragmatic Clinical Studies, variable
We Fund Research Through Three Mechanisms
Snapshot of Funded Projects
Number of projects: 468
Amount awarded: $1.18 billion
Number of states where we are funding research: 41 (plus the District of Columbia)
As of September 28, 2015
Highest Concentration of Funded Projects
By number of projects By amount awarded (in millions)
As of September 28, 2015
Examples of Projects
As of September 28, 2015
• Rural Health• Aging and Conditions of Aging• Disability and Chronic Disease
Better Engagement Through Research
We Fund Research That…
Meets these criteria:1:Potential for the study to fill critical gaps in evidence
Is associated with a significant burden in the US population (prevalence, mortality, morbidity, individual suffering, or loss of productivity)
2: Potential for the study findings to be adopted into clinical practice and improve delivery of care
Has the potential to lead to meaningful improvement in the quality and efficiency of care and to improvements in outcomes important to patients
3: Scientific merit (research design, analysis, outcomes)Has a research design of sufficient technical merit to ensure that the study goals will be met
4: Patient-centerednessFocuses on questions relevant to outcomes of interest to patients and caregivers
5: Patient and stakeholder engagementIncludes patients and other healthcare stakeholders as partners in every stage of the research
We Fund Research That…
• Patients are partners in research, not just “subjects”
• Active and meaningful engagement between scientists, patients, and other stakeholders
• Community, patient, and caregiver involvement already in existence or a well-thought-out plan
“Patient and stakeholder engagement”
What we mean by…
• The project aims 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
“Patient-centeredness”
Why Engage?
To establish trust and a sense of legitimacy in research findings
To encourage successful uptake and use of research results
To influence research to be patient-centered, relevant, and useful
A systematic review* provides the first international evidence of the impact of patient and public involvement on research on health and social-care research.
Does Engagement Make a Difference?
*Health Expectations 2014; 17(5): 637–650.
• Literature search from 1995-2009 identified 66 studies
• Analysis showed patient and public involvement enhanced quality and appropriateness of research
• Impacts were described for all stages of research
• But authors note the evidence base on impact of engagement still needs significant enhancement
Engagement as a Path to Useful, High-Quality Research
Evaluation
Proposal Review; Design and Conduct of Research
Topic Selection and Research Prioritization
Dissemination and Implementation of Results
Our Engagement Rubric–A Valuable Resource
Provides practical guidance to applicants, merit reviewers, awardees, and engagement/program officers on effective engagement in research
• Planning the Study: How patient and stakeholder partners will participate in study planning and design
• Conducting the Study: How patient and stakeholder partners will participate in the conduct of the study
• Disseminating the Study Results: How patient and stakeholder partners will be involved in plans to disseminate study findings and ensure that findings are communicated in understandable, usable ways
• PCOR Engagement Principles: Reciprocal relationships, co-learning, partnership, trust, transparency, honesty
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Show Your Work
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The Rubric
Why develop a rubric?
What is the rubric?
How will the rubric be used?
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The rubric is a collection of examples, intended to provide guidance to applicants, merit reviewers, awardees, and engagement/program officers
It is not intended to be comprehensive or prescriptive Applicants can choose to include some (but not all) activities
illustrated, and they can include additional innovative approaches not included
The examples provided are from real PCORI-funded projects
Overarching Rubric Principles
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Elements of the Rubric
Planning the Study
Conducting the Study
Disseminating the Study Results
Potential activities include Identifying the topic and developing the research question and
comparators to be studied Defining the characteristics of study participants
Examples of how to demonstrate this in your proposal Providing letters of support from patient and stakeholder partners that
clearly describe the origin of the study topic and the role of the patient and stakeholder partners in defining the question, outcomes, comparators, goals and outcomes, and so on
Planning the Study
Real-World Examples Mental health study: Patient partners and community members helped
craft the study name and materials to reduce the potential for stigma and to reframe the goal of the study as a movement toward emotional well-being rather than away from a mental health challenge.
Diabetes study: Clinicians who reviewed the initial study design indicated that clinical practice is quite variable and suggested that a three-arm approach would be more appropriate for the study. The study design was revised accordingly.
Planning the Study (Cont’d)
Potential activities Drafting or revising study materials and protocols Assisting with the recruitment of study participants
Example of how to demonstrate this in your proposal Providing letters of support from patient and stakeholder partners
that clearly describe the role of these partners in conducting and monitoring the study
Clearly articulating in the application the roles of the patient and stakeholder partners in each component of study conduct (e.g., helping draft survey tools and focus group questions, reviewing participant materials for readability), including the dissemination and implementation assessment
Conducting the Study
Real-World Examples Chronic pain study: The informed consent document was developed with
patient partners to make it understandable to study participants. Preeclampsia study: The study team is recruiting via a national network of
local health departments and community health centers, as well as through a preeclampsia advocacy group’s website and Facebook page.
Asthma study: Clinicians and patients both provided guidance on who should deliver the intervention, when it should be provided during the process of care, and how it should be delivered.
Conducting the Study (Cont’d)
Potential activities include Identifying partner organizations for dissemination Planning dissemination efforts
Examples of how to demonstrate this in your proposal Clearly identifying the role of patient and stakeholder partners
in planning the dissemination of the study’s findings Including patient and stakeholder partners on a project
committee that will oversee dissemination
Dissemination of the Study Results
Real-World Examples Trauma study: The research team is convening a policy
summit with relevant professional societies during the third year of the study to focus on identifying ways to speed the implementation of findings into practice.
Neurology study: The research team presented at a neurology patient advocacy conference to inform the community that this research was ongoing and to stay tuned for future results.
Dissemination of the Study Results (Cont’d)
Six Engagement Principles Partnership Co-learning Reciprocal Relationships Trust Transparency Honesty
Engagement Principles
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Putting the Rubric to Work
Greater understanding of
engagement
Stronger engagement in
proposals
Strong engagement
during research?
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• Helping Awardees to outline engagement milestones
• Participating in the Awardees' kick-off and interim phone calls with research team members including key patient and stakeholder partners
• Facilitating communication between Awardees to troubleshoot engagement challenges
• Gathering promising engagement practices from the portfolio to feature in webinars and for use in updating or expanding the Engagement Rubric
Engagement Officers
Engagement Funding Programs
The Engagement Awards Program
• Provides a platform to expand the role of all stakeholders in research and to support PCORI’s Engagement Priorities
• Launched in February 2014• $15.5 Million (FY 2015)• Awards up to 2 years, $250,000
– Knowledge, Training and Development, Dissemination Awards– EAIN: Patient-Centered Outcomes Research/Clinical Comparative
Effectiveness Research Meeting and Conference Support– EAIN: Supporting Dissemination and Implementation Activities of the
PCORI Pilot Projects Learning Network
Engagement Awards Strengthen the PCORI Research Enterprise
• Build knowledge about how consumers of healthcare information view PCOR/CER; how they receive and make use of PCOR/CER findings; and/or what evidence they need to reach health and healthcare decisions.
• Grow capacity for healthcare community engagement in PCOR/CER; train patients and other stakeholders to participate in PCOR/CER; create methods to connect patients and other stakeholders with the research community; and/or facilitate patient and other stakeholder engagement in PCOR/CER.
• Develop channels for D&I of PCOR/CER evidence; identify, build, and strengthen partnerships and facilitators for spreading PCOR/CER findings; and/or D&I PCOR/CER findings in creative ways, especially within PCORI’s priority populations.
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Build Knowledge Grow Capacity Develop Channels
48
Engagement Awards Strengthen the PCORI Research Enterprise
Capacity Channels
Engagement Award Program Processes
• Standing call for LOIs for Engagement Awards and Applications for PCOR/CER Meeting and Conference Support
• Internal merit review by Engagement team; consult with Science• CMA conducts budget review and makes awards• Engagement Team Program Officer(s) monitor projects• Evaluation plan in development• Program platform - SalesForce Foundation Connect • Opportunities for new EAINs
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Engagement Awards to Date
• The University of Alabama - The Alabama Initiative for Integrated Primary Care-Behavioral Health Services
• Franciscan Hospital for Children - Empowering Adolescents with Asthma and their Caregivers to Make Shared Decisions with their Doctors
• American College of Physicians, American Academy of Pediatrics, American Osteopathic Association, American Academy of Family Physicians - Measuring Physicians' Opinions of CER to Strengthen Its Role in Patient-Centered Care
• Health Research and Educational Trust - Integrating Patient-Centered Outcomes Research into Hospital CHNAs
• National Academy for State Health Policy - PCOR Roadmap for State Policy Makers
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Pipeline to Proposals
Tier IUp to $15,000Up to 9 month
term
Tier IIUp to $25,000
Up to 12 month term
Tier IIIUp to $50,000
Up to 12 month term
PCORI Funding
Advancement to Tier II depends on
successful completion of Tier I
Advancement to Tier III is a competitive
application process
Or other funders of PCOR/CER
Pipeline to Proposal Awards
• Tier I: building community and capacity around a topic of interest to patients, researchers or other stakeholders.
• Tier II: formalizing partnerships, identifying potential CER question to be researched, developing infrastructure or governance processes.
• Tier III: creating research proposal, including robust engagement plan for later submission to PCORI or other funders of PCOR.
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First Thirty Tier I Awards Announced December 23, 2013
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Western Region Tier I Pipeline Pilot
Pipeline to Proposal Program Offices
Strengthening PCOR Nationwide
Number of projects: Tier I – 77Tier II - 27
Amount awarded: $1,813,999(Tier I Cycles 1 & 2 and Tier II Cycle 1)
Number of states where we are funding projects: 30 states and Puerto Rico
As of March 27, 2015
Our Pipeline to Proposal Awards encourage PCOR in comparative clinical effectiveness research.
Upcoming Opportunities
• Tier I funding will be available in December
• “Independent” Tier III funding will be available in Spring 2016
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Evaluating Our Impact
Evaluating Our Impact: Does “Research Done Differently” Make a Difference?
• How well are we carrying out our current activities?
• Are our activities moving us toward our goals?
• What are the effects of our unique, patient-centered approach on the research we fund and its results?
• Is the research we fund ultimately having a positive impact on health decisions, the quality of health care, and health outcomes?
To Answer These Questions, We Have Begun an Extensive Evaluation Process
We evaluate progress toward our goals.Each of three strategy committees focuses on the metrics and evaluation activities associated with one of our goals.
Our evaluation process is organized into a framework.We’ve organized hundreds of questions and indicated our initial approach to answering them.
Our evaluation process flows from our strategic plan.Are we following our strategies, and are they having the intended effect?
Mandated Assessment by GAO
Our authorizing legislation requires the US Government Accountability Office to review our progress. The GAO’s initial assessment, released in March 2015, found our efforts to date are consistent with legislative requirements in these key areas:
• Establishment of research priorities and a research agenda
• Establishment of processes for funding research and monitoring contractors
• Development of dissemination plans and coordination of dissemination activities with AHRQ
The GAO report also described the status of our efforts to fund research and our plans to evaluate the effectiveness of our work.
Contact Us
Thank You
M. Suzanne Schrandt, JDDeputy Director, Patient [email protected]