From Populations to Patients


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Plenary V - SELBY

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  • One of PCORI’s earliest priorities is to distinguish itself from other entities that are, increasingly, sponsoring and conducting CER – NIH, AHRQ, the VA – to name 3. And if it does, it will do so in 3 areas: Engaging patients; this will likely be a major research focus, as well as a key day-to-day activity of PCORI. This will lead to a research agenda that is more closely aligned with the questions patients need answers and hopefully will involve more patients in the research itself. The result will be better information to disseminate to support health decision-making for both patients and their providers.
  • All relevant stakeholders came togetherUniform support for concept of putting patient in the center of PCORI research Divergent views expressed on priorities - including cost control Participants heard each other.Future workshops and advisory panels will challenge stakeholders to solve problems together, on behalf of patients.
  • Our premise is that it is by engaging patients more extensively that we realize the promise of CER – for pragmatic studies that are generalizable to real patients and account for patient differences – both in treatment responses and in preferences for various outcomes.
  • So, we believe and stakeholders seem to concur that we also need to learn what works better in the areas of improving health care delivery systems, what works better for disseminating research findings to clinicians and to patients and what works better in communicatings between providers and patients in the shared d-m process.
  • What is the purpose of the Research Agenda?The Research Agenda describes the types of research questions that can be addressed under each of the five priorities. The agenda also outlines nine criteria, taken from PCORI’s establishing legislation, which will be used to evaluate research proposals. Studies that meet most or all of these criteria will be those most likely to be funded.Why doesn’t the Research Agenda name specific conditions or treatments that PCORI is interested in funding or studying?PCORI believes there are important comparative clinical effectiveness research questions to be answered across a wide range of conditions. Focusing on a narrower set of diseases or conditions at the start of PCORI’s research funding would mean that some patients and health care issues might have no chance of benefitting from PCORI’s existence. Also, there are issues that affect patients across a variety of conditions. For example, we have heard from the patient focus groups about challenges with communication and interaction with their providers. These are problems that are not specific to one condition, but have been identified by a variety of patients as being important to them. Involving patients and other stakeholders in a meaningful process of considering which conditions or questions deserve more PCORI funding is important and will begin immediately and continue throughout PCORI’s lifetime. However, it will take time to hear all views on these issues, to weigh the input, and to begin making these choices. This must be done in a transparent manner so that all can understand how we reached the narrower, targeted set of research questions we focus on. Over time, PCORI’s research portfolio will become more focused, but PCORI will continue to reserve a portion of its funding for addressing the broader set of possible questions that patients, researchers and stakeholders may raise.
  • From Populations to Patients

    1. 1. Patient-Centered Outcomes Research Institute From Populations to Patients Joe V. Selby, PCORI Executive Director May 1, 20121
    2. 2. Imagine My Surprise!2
    3. 3. PCORI is a Research Institute “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 Purpose conditions 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 appropriateness of medical treatments, services. 3 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
    4. 4. PCORI is a Research Institute ‘‘(2) COMPARATIVE CLINICAL EFFECTIVENESS RESEARCH; (A) IN GENERAL.—The terms ‘comparative clinical effectiveness research’ and ‘research’ Purpose 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). 4 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
    5. 5. Taking Patient-Centeredness Seriously 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. 5
    6. 6. 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 6 healthcare?”
    7. 7. Taking Patient-Centeredness Seriously Patient-DrivenPatient Engagement Research Dissemination Understanding Aligning research questions Providing patients and the choices and methods with providers with information patients face patient needs for better decisions 7
    8. 8. 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 8
    9. 9. PCORI National Patient and Stakeholder Dialogue Feb 27, 2012 9
    10. 10. PCORI Hopes to Turn the Research World On Its Head 10
    11. 11. Is PCOR still CER? 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.11
    12. 12. The Population and The Patient Patients Patients The Population12
    13. 13. The Legislation re: Treatment Heterogeneity “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 Purpose 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 such subpopulations as subjects in the research as feasible and appropriate. 13 Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
    14. 14. Treatment Heterogeneity – Type One Marker Positive: Therapy with Heterogeneous Differential Net Benefit Population Effectiveness • Biomarker Marker Negative • Comorbidity and • Literacy non-differential adverse effects Net Harm
    15. 15. Treatment Heterogeneity – Type Two Heterogeneous Outcomes Population Therapy with High Risk: High Risk Non-differential Net Benefit Effectiveness Medium Risk: Medium Risk And No Net Benefit Non-differential Adverse Effects Low Risk: Low Risk Net Harm
    16. 16. Heterogeneity in Treatment Effectiveness Patient Population Intervention Marker Breast Cancer Trastuzumab HER-2 Expression Atrial fibrillation Warfarin VKORC1, CYP2C9 Symptomatic Coronary Bypass Age, major Ischemic HD Surgery Comorbidity Type 2 Diabetes eHealth Education Literacy/Numeracy
    17. 17. What Works Best – For Whom? • Identifying new markers to guide therapy • Biology-driven • Empirical: Re-examining clinical trials data Evidence syntheses Large 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?
    18. 18. The Funding PCORI’s• National Priorities for Research• Research Agenda• Funding Announcements • PCORI Pilot Projects • Primary Research Funding Announcements
    19. 19. National Priorities and Research AgendaNational Priorities and Research Agenda Must be approved before major funding can begin National Increasing Specificity Priorities Research Agenda Individual PFAs 19
    20. 20. Building on Other Recent PrioritizationEfforts HIT to improve Impact of New pt. experience Palliative Care Coordination Disease Care Engagement Appropriate Technology Prevention Acute Care Chronic Patient Safety Care UseSourceIOM 2009: Priorities for CER √ √ √ √ √ √ √ √ √Federal Coordinating √ √ √ √ √Committee for CERAHRQ National Quality √ √ √ √StrategyAHRQ Effective Health Care √ √ √ √ √ √ProgramNational Quality Forum √ √ √ √ √ √ √ √National Prevention Council √ √National Priorities √ √ √ √ √ √Partnership 20 20
    21. 21. PCORI’s National Priorities for ResearchProducing and delivering information to support better healthcare decisions by individuals 2. Improving Health Care Systems 1. Comparing Better Decisions Preventive, 3. Communication/ Dissemination Diagnostic and Research BetterTreatment Options Outcomes 4. Addressing Disparities 5. Accelerating PCOR and Methodological Research - Data - Training - Methods 21
    22. 22. PCORI’s National Priorities for Research Assessing Options for Prevention,  Comparisons of alternative clinical options  Identifying patient differences in response to therapy Diagnosis, and  Studies of patient preferences for various outcomes Treatment  Improved support of patient self-management Improving  Coordination of care for complex conditions Healthcare  Improvements in the effectiveness and efficiency of care Systems  Improved workforce deployment Communication &  Understanding and enhancing shared decision-making Dissemination  Alternative strategies for dissemination of evidence Research  Alternative interventions/strategies to eliminate disparities Addressing  Improvements in alignment of decisions with preferences Disparities Accelerating  Improving study designs and analytic methods of PCOR PCOR and  Building and improving clinical data networks Methodological  Methods for training researchers, patients to participate in PCOR 22 Research  Facilitating the study of rare diseases
    23. 23. Next Steps:National Priorities and Research Agenda January - March, 2012 • Public Comment Period March-April 2012 • Analysis of public comment and revision April 25, 2012 • Approval of revised National Priorities / Research Agenda Funding May 2012 • First Funding Announcements Issued – Priorities 1 - 4 July 2012 • Applications due December 2012 • Awards announced Stakeholder Engagement July 2012 • First multi-stakeholder workshops convened December 2012 • First Advisory Panels Formed
    24. 24. Building Infrastructure for a National Learning Healthcare System24
    25. 25. National Learning Health System FDA ONC NIH Meaningful Use Nationwide Health Information NetworkSentinel Federal Health Architecture CTSA’s EHR Certification programs Collaboratory OMOP Standards & Interoperability Framework CRN, CVRN AHRQ VA IOM Specialty DRN’s Registries Industry Societies SPAN PROSPECT EDM Forum 25
    26. 26. Priority #5 Accelerating PCOR and Methodologic Research• 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? 26
    27. 27. What Would it Take? • 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 27
    28. 28. Re-examining Ethics of Research in a Patient-Centered World28 28
    29. 29. Re-examining EthicsHistorical Backdrop: Current Understanding: Near Future? • Much of medicine still • research does• Belmont Report become more practiced without good (1978): draws patient-centric, evidence, exposing bright line between addressing patients to avoidable research and questions risks; clinical care • with advent of patients and• different ethical EHRs, other clinicians raise, standards and computerized with patients oversight applied data, health systems participating in to research than and clinicians generate the research to practice. and hold key data for increasing the29 evidence.
    30. 30. 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 patients NOT 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 30 to re-think aspects of IRB policy and practice.
    31. 31. 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 31
    32. 32. ….thanks!32