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GHS Research Lecture Series Greenville, SCJune 10, 2014
Linda Crew McNamara, RN, MBAPSDTO Advisory Panel & Stakeholder Reviewer
Patient-Centered Outcomes Research
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• Background (Evolving Research Paradigm)
• Patient Centered Outcomes Research• Patient Centered Outcomes Research
Institute (PCORI)• Tips for strong proposal
Overview of presentation
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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
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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
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Background
Patients wanted more active role:
“Nothing about me without me.”
“PhDs of the sidewalk.”
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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.”
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“Research that sees through the eyes of patients.”
Annals of Internal Medicine 18 Sept 2012
RelevantUsableEasily accessible
What is Patient-Centered Outcomes Research (PCOR) ?
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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?”
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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Examples of Patient-CenteredOutcomes
• Quality of life and functional status• Psychological factors: anxiety,
depression• Economic outcomes (work hours,
income lost)• Treatment decision related
(decisional conflict, regret)
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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.
“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
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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PCORI Focus
Patient-centered
Answering questions that matter to patients and other clinical decision makers
Comparisons of outcomes that matter to patients
Comparative Clinical Effectiveness Research
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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Help Us Share the Findings
Tell Us How We’re Doing
Advise Us on What to
Study
Review Proposals
and Partner in Research
Key Feature: Emphasizes Engagementfor Getting to Practical, Useful Research
Generate and Prioritize Research Questions
Portfolio Review
Review and Conduct Research
Dissemination andImplementation
Engagement Goals
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Promote Dissemination and Implementation
Engage the PCOR Community in Research
Build a Patient-Centered Outcomes Research Community
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
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The National Patient-Centered Clinical Research Network (PCORnet)
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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
• 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
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)
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How to Submit an Application
Visit pcori.org/applyKey Dates
Funding Announcements
Application Guidelines and Templates
Guidance on the PCORI Methodology Standards
Sample Engagement Plans
Frequently Asked Questions
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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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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.
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Formulating Research Questions
Patient-Centeredness
Data Integrity and Rigorous Analyses
Preventing/Handling Missing Data
Heterogeneity of Treatment Effects
Tips for a Strong Proposal
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
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
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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Have a Question?
General Inquiries
[email protected] | (202) 827-7200
Research/Programmatic Questions
[email protected] | (202) 627-1884
Administrative/Financial/Technical Questions
Engagement and PCORI Activities
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Linda Crew McNamara, RN, MBA
Thank You
