JAMA Briefing on Comparative Effectiveness Research


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The Patient-Centered Outcomes Research Institute (PCORI) sponsored a webcast to unveil the April 18 issue of the Journal of the American Medical Association (JAMA). The issue focuses on comparative effectiveness research and features two articles authored by PCORI.

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  • Who are the individuals responsible for carrying out PCORI’s mission?PCORI is governed by a 21-member board that represents everyone in the health care community – patients, caregivers, doctors, nurses, researchers, public health officials, payers and industry.By law, PCORI’s Board is required to include:3 members representing patients and health care consumers5 members representing physicians and providers, including at least 1 surgeon, nurse, state-licensed integrative health care practitioner, and representative of a hospital3 members representing private payers, of whom at least 1 member shall represent health insurance issuers and at least 1 member shall represent employers who self-insure employee benefits3 members representing pharmaceutical, device, and diagnostic manufacturers or developers1 member representing quality improvement or independent health service researchers2 members representing the Federal Government or the States, including at least 1 member representing a Federal health program or agencyThe directors of the National Institutes of Health and the Agency for Healthcare Research and Quality (or their designees)PCORI has established three committees: Program Development Communication, Outreach and Engagement Finance, Audit and AdministrationPCORI also has a 17-member Methodology Committee to help PCORI develop and update methodological standards and guidance for comparative clinical effectiveness and outcomes research.
  • What is the specific work that PCORI will do? The law outlines several core duties (bulleted on slide).To support its work, the law also: Provides PCORI access to federal health data (adhering, of course, to confidentiality and privacy standards) Authorizes PCORI to appoint “expert advisory panels” – and support the participation of patients or consumer representatives on those panels Requires PCORI to provide an annual report to Congress. PCORI will issue its report on its 2011 activities in April 2012.
  • PCORI’s priorities and agenda are developed in an iterative processes. As a learning organization, PCORI will update the priorities and agenda based on community feedback and the results of our work.
  • We followed a careful, multi-step process in developing these draft “roadmaps” for our future work. We reviewed major comparative effectiveness research initiatives undertaken previously and under way now and their results. We reviewed previous national priority-setting processes for related health research to identify common priority areas that were informed by stakeholder input. These were evaluated against the criteria laid out for PCORI in the health care reform law. This led to a focus on five priority areas, which we informally vetted with stakeholders through small group meetings and patient and caregiver focus groups. We’re now ready to receive formal input from the entire health community.As part of this process, and in our efforts going forward, we’re taking great care to build on and add value to the best work that others have done and continue to do, while filling in gaps and supporting promising new initiatives.
  • We started by taking the patient centered perspective – how doe we get better information to patients so that they can indeed make better health care decisions. We could focus entirely on the kinds of comparisons represented in this first priority, but we all know that good research findings sometimes sit on the shelf, maybe for as long as 17 years, never influencing decision-making.
  • 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.
  • PCORI is currently reviewing all input received on the draft National Priorities and Research Agenda.A report will be published summarizing the feedback and how it led to changes in the priorities and agenda.The revised National Priorities and Research Agenda will be adopted by Board of Governors during a special meeting – a public teleconference – in April.PCORI’s largest engagement event to dateBoard Member Harlan Krumholz: “Our true North is our patients”Uniform support for putting patient at center of PCORI research.Future workshops and advisory panels will challenge stakeholders to solve problems together, on behalf of patients.
  • 12 groups total were conducted and 96 patients and caregivers participated. Nine of the groups were patients and three were caregivers. For the Atlanta groups and the Baltimore group on asthma, we oversampled African Americans to ensure we captured their perspective.For the Hispanic group in Phoenix, we conducted the event in Spanish, but the participants of their own choosing elected after the introductions to participate in English.
  • The clinician focus groups will allow us to have detailed, facilitated discussions (90 minutes to 2 hours) with small groups of physicians and nurses on the priorities and agenda.The feedback obtained through these groups will be incorporated into the public input process to revise the priorities and agenda.Tomorrow, in our committee workshop, we will discuss specifically which types of clinicians to recruit to participate in these focus groups – with an eye towards the populations they serve – and we will make recommendations to the staff, so that planning can proceed.We will begin work with the staff and Program Development Committee next week to develop the discussion guides that will be used in these focus groups.The goal is to complete all of the focus groups by February 15, so that a report can be provided to the Board by March 1.
  • JAMA Briefing on Comparative Effectiveness Research

    1. 1. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTE Setting PCORI’s Priorities – How the Patient-Centered Outcomes Research Institute Plans to Make a Difference Anne C. Beal, MD, MPH COO, Patient-Centered Outcomes Research Institute (PCORI) JAMA Media Briefing National Press Club, Washington, DC April 17, 2012
    2. 2. PCORI Purpose: Defining Legislation P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTE “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. Source: Affordable Care Act. Subtitle D—Patient-Centered Outcomes Research. PUBLIC LAW 111–148—MAR. 23, 2010.
    3. 3. PCORI Mission P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTE PCORI MISSION STATEMENT • 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.
    4. 4. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEWhy PCORI?• Patients’ individual needs, values and concerns have not been an “outcome measure” in traditional research.• Patients who face a range of treatment options, especially for serious conditions, want clinicians to help them figure out what different choices mean for them or people like them.• Clinicians often lack the high-quality evidence they could use to advise patients making complex clinical decisions.• Even the best traditional clinical effectiveness research has not had this “patient-centric” focus. 4
    5. 5. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEA Board Broadly Representing the Health Care Community • Patients • Caregivers • Physicians, nurses and clinicians • Hospitals and health systems • Health plans • Health services researchers • State and federal health officials • Pharmaceutical, device, and diagnostic manufacturers • Private payers • Employers 4
    6. 6. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTE What is patient-centered outcomes research?*Complements traditional CER; answers patient-centered questions like:• “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?” *From PCOR definition adopted by PCORI Board of Governors, March 5, 2012
    7. 7. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEMaking Outcomes Research Patient-Centered 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 6
    8. 8. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEPCORI seeks to do its work by:• Establishing national research priorities• Establishing and carrying out a research agenda• Developing and updating methodological standards• Disseminating research findings• Engaging patients, caregivers, clinicians and other stakeholders as its work evolves 7
    9. 9. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEPCORI’s Path from Priorities to ResearchPatients Can Use Future priorities and agendas will be informed by the development process, ongoing stakeholder engagement, and PCORI’s research results. 8
    10. 10. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTECriteria for Research Outlined by Law Addresses Impact on Health of Current Gaps in Individuals and Patient-Centeredness Knowledge/ Populations Variation in CareImprovability through Impact on Health Care Rigorous Research Research System Performance Methods Inclusiveness of Potential to Influence Efficient Use ofDifferent Populations Decision-Making Research Resources 9
    11. 11. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEEstablishing PCORI’s First National Prioritiesfor Research and Initial Research Agenda First primary 9 5 Corresponding Public input Priorities and fundingcriteria outlined draft priorities received and agenda revised agenda drafted announcements by law proposed evaluated and approved issued Aug-Dec 2011 Jan-Apr 2012 May 2012 10
    12. 12. National Priorities for Research P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEBetter Information Better Decisions 1. Comparing Better Decisions Preventive, Diagnostic and ? ? Better Outcomes Treatment Options
    13. 13. National Priorities for Research P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEProducing 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 Better OutcomesTreatment Options 4. Addressing Disparities 5. Accelerating PCOR and Methodological Research - Data - Training - Methods
    14. 14. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEA rigorous methodological basis for PCOR • To do CER and PCOR properly, methods matter. • PCORI’s Methodology Committee is established by statute. • Committee is charged with developing and periodically updating methodological standards for CER/PCOR. • Committee is required to deliver first report to PCORI’s Board in early May. – prioritizing research questions – using appropriate study designs – incorporating patient perspectives throughout the research continuum – fostering efficient dissemination and implementation of results.
    15. 15. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEA commitment to public engagementFormal 53-day Public Comment Period• Nearly 500 comments received through website, e-mail or postal mail• All comments will be posted at pcori.orgAdditional Forums• National Patient and Stakeholder Dialogue• Patient, caregiver and clinician focus groups• Individual meetings with diverse mix of stakeholders 14
    16. 16. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEPatient and Caregiver Focus Groups Baltimore, MD (Nov. 9) Atlanta, GA (Nov. 21) • Patients with Arthritis • Patients with Diabetes • Parents of children with Pediatric • Caregivers to Alzheimer’s patients Asthma (caregivers) • Patients with Chronic Pain • Patients with mix of chronic • Insured and uninsured conditions • Age: 21-75+ • Insured and uninsured • Age: 21-69 Columbus, OH (Dec. 7) Phoenix, AZ (Dec. 8) • Parents of children with ADD/ADHD • Patients with Respiratory Disease (caregivers) (chronic bronchitis, emphysema) • Patients with Mental Health • Hispanic patients with mix of conditions chronic conditions • Patients who survived Cancer • Patients with Heart Disease • Insured and uninsured • Insured and uninsured15 • Age: 30-70+ • Age: 21-69
    17. 17. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTEReceiving Public Feedback on the NationalPriorities and Research Agenda• Clinician Focus Groups o Clinician focus groups took place in cities across the US o Philadelphia o Birmingham o California o Chicago o Four groups of physicians and four groups of nurses o Behavioral Health Professionals o Physician Assistants16
    18. 18. P A T IENT -CENTERED O U T C OM ES R ES EA R CH IN S T ITUTENext Steps March-April 2012 • Analysis of public comment received on Priorities and Agenda April 2012 • Adoption of first National Priorities for Research and Research Agenda May 2012 • First Methodology Report posted for public comment May 2012 • Broad Funding Announcements Issued June 2012 • Patient and Stakeholder Recruitment and Training for Research Review July 2012 • Broad Funding Applications Due Summer 2012 • Advisory Groups Formed to Advance Agenda Specificity August 2012 • Targeted Funding Announcements Issued December 2012 • Broad Funding Awards Announced 17