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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
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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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.
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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?
“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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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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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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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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Engagement Goals
Promote Dissemination and Implementation
Engage the PCOR Community in Research
Build a Patient-Centered Outcomes Research Community
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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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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
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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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
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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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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Engagement Principles
Trust
Transparency
Co-learning
ReciprocalRelationshi
ps
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) 19
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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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
Our Growing Research Portfolio
pfaawards.pcori.org
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
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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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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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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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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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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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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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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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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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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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
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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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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Application and Review Process
pcori.org/apply
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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Have a Question?
General Inquiries
[email protected] | (202) 827-7200
Research/Programmatic Questions
[email protected] | (202) 627-1884
Administrative/Financial/Technical Questions
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Connect with PCORI
Bryan Luce, PhD, MBAChief Science [email protected]
Regina L. Yan, MAChief Operating [email protected]
Joe V. Selby, MD, MPHExecutive [email protected]
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Find Us Online
www.pcori.org
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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.
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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)
IHO - Rural
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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
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 metrics2) Standards for implementation methods3) Reporting standards for implementation studiesNewhouse, 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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Committed to advance research to improve health care…………
Robin Newhouse, PhD, RN, NEA-BC, [email protected]
