The Patient-Centered Outcomes Research Institute...A web-based patient portal collects...

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:


• 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



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




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





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





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)


Examples of Projects


• Rural Health• Aging and Conditions of Aging• Disability and Chronic Disease

Better Engagement Through Research


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


• 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

http://www.pcori.org/assets/2014/02/PCORI-Patient-and-Family-Engagement-Rubric.pdf






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

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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

[email protected]

Thank You

M. Suzanne Schrandt, JDDeputy Director, Patient [email protected]

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