From Populations to Patients
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Transcript of From Populations to Patients
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Patient-Centered Outcomes Research Institute
Joe V. Selby, PCORI Executive Director May 1, 2012
From Populations to Patients
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Imagine My Surprise!
3 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
“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 healthconditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriatenessof medical treatments, services.
Purpose
PCORI is a Research Institute
4 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
‘‘(2) COMPARATIVE CLINICAL EFFECTIVENESS RESEARCH; (A) IN GENERAL.—The terms ‘comparative clinical effectiveness research’ and ‘research’ mean research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of 2 or more medical treatments, services, and items described in subparagraph (B).
Purpose
PCORI is a Research Institute
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PCORI MISSION STATEMENT (adopted July 17, 2011)
The Patient-Centered Outcomes Research Institute (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.
Taking Patient-Centeredness Seriously
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Defining Patient-Centered Outcomes Research
Patient Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options. This research answers patient-centered questions such as:
“Given my personal characteristics, conditions and preferences:
1. What should I expect will happen to me?
2. What are my options and what are the potential benefits and harms of those options?
3. What can I do to improve the outcomes that are most important to me?
4. 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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Understanding the choices
patients face
Aligning research questionsand methods with
patient needs
Providing patients and providers with information
for better decisions
Patient EngagementPatient-Driven
Research Dissemination
Taking Patient-Centeredness Seriously
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Taking Patient-Centeredness Seriously
• Patients and other relevant stakeholders must be included as members of research teams and involved in all aspects of the project - from planning to dissemination
• Applications are evaluated and scored on patient and stakeholder engagement in the review process
• At least 3 trained patient/stakeholder reviewers serve on each review committee
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PCORI National Patient and Stakeholder Dialogue
Feb 27, 2012
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PCORI Hopes to Turn the Research World On Its Head
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Yes!! Including the patient and clinician perspectives in PCOR help to ensure that:
The research questions are genuinely practical
The range of outcomes important to patients are identified and studied
The patients studied are representative of those who require information and come from relevant settings
Possibilities of heterogeneity in effectiveness are carefully examined in various patient subgroups.
Is PCOR still CER?
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The Population and The Patient
The Population
Patients
Patients
13 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
“Research shall be designed, as appropriate, to take into account the potential for differences in the effectiveness of health care treatments, services, and items as used with various subpopulations, such as racial and ethnic minorities, women, age, and groups of individuals with different comorbidities, genetic and molecular sub-types, or quality of life preferences and include members of suchsubpopulations as subjects in the research as feasible and appropriate.
Purpose
The Legislation re: Treatment Heterogeneity
Treatment Heterogeneity – Type One
HeterogeneousPopulation
• Biomarker• Comorbidity• Literacy
Marker Positive:
Net Benefit
Marker Negative
Net Harm
Therapy withDifferential
Effectiveness
andnon-differential adverse effects
Treatment Heterogeneity – Type Two
Therapy withNon-differential
Effectiveness
And Non-differential Adverse Effects
HeterogeneousPopulation
High Risk
Medium Risk
Low Risk
Outcomes
High Risk:Net Benefit
Medium Risk:No Net Benefit
Low Risk:Net Harm
Heterogeneity in Treatment Effectiveness
Patient Population Intervention Marker
Breast Cancer Trastuzumab HER-2 Expression
Atrial fibrillation Warfarin VKORC1, CYP2C9
Symptomatic Ischemic HD
Coronary Bypass Surgery
Age, major Comorbidity
Type 2 Diabetes eHealth Education Literacy/Numeracy
What Works Best – For Whom?
• Identifying new markers to guide therapy• Biology-driven• Empirical: Re-examining clinical trials data
Evidence synthesesLarge EHR database studies Larger trials
• Focus on developing treatments and approaches for those who are NOT responding well to current therapies
• In the face of imperfect accuracy and ongoing uncertainty, how do we incorporate patient preferences for USING these markers to guide treatment decisions?
PCORI’s
• National Priorities for Research
• Research Agenda
• Funding Announcements
• PCORI Pilot Projects
• Primary Research Funding Announcements
The Funding
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National Priorities and Research Agenda
Incre
asin
g S
pecifi
city
National Priorities and Research Agenda Must be approved before major funding can begin
National Priorities Research Agenda
Individual PFAs
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Building on Other Recent Prioritization Efforts
Source
Preventio
n
Acute
Care
Chronic
Disease
Care
Palliative Care
Care Coordination
Patient
Engagement
Safety
Appropria
te Use
HIT to
improve pt.
experienc
e
Impact of New Technolo
gy
IOM 2009: Priorities for CER √ √ √ √ √ √ √ √ √
Federal Coordinating Committee for CER
√ √ √ √ √
AHRQ National Quality Strategy
√ √ √ √
AHRQ Effective Health Care Program
√ √ √ √ √ √
National Quality Forum √ √ √ √ √ √ √ √
National Prevention Council √ √
National Priorities Partnership
√ √ √ √ √ √20
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1. Comparing Preventive,
Diagnostic and Treatment Options
2. Improving Health Care Systems Better Decisions
Better Outcomes
5. Accelerating PCOR and Methodological Research- Data- Training- Methods
4. Addressing Disparities
3. Communication/ Dissemination Research
Producing and delivering information to support better health care decisions by individuals
PCORI’s National Priorities for Research
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Comparisons of alternative clinical options Identifying patient differences in response to therapyStudies of patient preferences for various outcomes
Improved support of patient self-managementCoordination of care for complex conditions Improvements in the effectiveness and efficiency of care Improved workforce deployment
Understanding and enhancing shared decision-makingAlternative strategies for dissemination of evidence
Alternative interventions/strategies to eliminate disparities Improvements in alignment of decisions with preferences
Improving study designs and analytic methods of PCORBuilding and improving clinical data networks Methods for training researchers, patients to participate in PCORFacilitating the study of rare diseases
Addressing Disparities
Improving Healthcare Systems
Accelerating PCOR and
Methodological Research
Communication & Dissemination
Research
Assessing Options for Prevention, Diagnosis, and
Treatment
PCORI’s National Priorities for Research
• Public Comment PeriodJanuary - March, 2012
• Analysis of public comment and revisionMarch-April 2012
• Approval of revised National Priorities / Research AgendaApril 25, 2012
May 2012
July 2012
December 2012
July 2012
December 2012
Next Steps:
National Priorities and Research Agenda
Funding
Stakeholder Engagement
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Building Infrastructure for a National Learning Healthcare System
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National Learning Health System
FDA
SentinelOMOP
ONC
Meaningful UseNationwide Health Information Network Federal Health ArchitectureEHR Certification programsStandards & Interoperability Framework
NIH
CTSA’sCollaboratoryCRN, CVRN
AHRQ
DRN’sRegistriesSPANPROSPECTEDM Forum
VA
Industry
SpecialtySocieties
PCORI
IOM
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• Research on the validity and efficiency of data sources that can be used to create a national infrastructure for PCOR;
– improving the volume, completeness, comprehensiveness, accuracy, or efficiency of use of clinical data collected across health care systems, clinical data networks, registries, or payer databases;
– Supporting the routine collection of key patient-reported and patient-centered outcomes
– Engaging patients in PCOR (governance, research prioritization, research participation, dissemination)
– Engaging front-line clinicians in generating research questions, disseminating findings?
Priority #5 Accelerating PCOR and Methodologic Research
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• A clearer long-term national vision and strategy for CER/PCOR
• Re-vamped notions of governance, with greater willingness of organizations to participate and contribute
• More research on data standards, inter-operability, data quality and comparability
• Advances in methods of data storage and analyses
• Greater engagement of patients, clinicians in governance, and as end-users
• Ability to contact patients, and to randomize
What Would it Take?
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Re-examining Ethics of Research in a Patient-Centered World
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Re-examining Ethics
Historical Backdrop:
• Belmont Report (1978): draws bright line between research and clinical care
• different ethical standards and oversight applied to research than to practice.
Current Understanding:• Much of medicine still
practiced without good evidence, exposing patients to avoidable risks;
• with advent of EHRs, other computerized data, health systems and clinicians generate and hold key data for increasing the evidence.
Near Future?• research does
become more patient-centric, addressing questions patients and clinicians raise, with patients participating in the research
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Re-examining Ethics: Questions
Physician and System Perspective: Is it ethical for clinicians and health systems NOT to participate in patient-centered research? Is it ethical to continue practicing with uncertainty and NOT put the data generated in this practice to research use.
Patient Perspective: Is it ethical (or reasonable) for patientsNOT to at least strongly consider participation in research as subjects, lending clinical data with appropriate safeguards and participating in appropriate clinical trials.
IRB Perspective: Given a change to patient-centered research, and the recognized need for representative, real world data, including both HER data and broad participation in clinical trials, do we need to re-think aspects of IRB policy and practice.
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A Research World on Its Head
• Patients and clinicians generating and prioritizing research questions, reviewing research applications, joining teams in conduct of research
• Analytic strategies based on finding differences in treatment response, focusing on patients not yet responding, and aiming to optimize treatments for each patient
• Health care systems participating in a national learning health system based on safety and patient-centered outcomes research
• Ethics re-cast to recognize the ethical imperatives of participation – with appropriate protections – by patients, clinicians and systems
• Growing body of relevant evidence, growing appreciation for research as a national endeavor to improve decision-making and outcomes
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QuestionsComments
….thanks!