“Update on PCORI: a new era in Research” Clyde W. Yancy, MD, MSc, FACC, FAHA, MACP Magerstadt Professor of Medicine Adjunct, Department of Medical Social Sciences Chief of Cardiology Northwestern University, Feinberg School of Medicine & Associate Medical Director Bluhm Cardiovascular Institute Chicago, IL cyancy@nmff.org Now we would like to focus on the clinical case for Get With the Guidelines and take you through some of the literature and experience that we've had in our program. 1

DISCLOSURES Consultant/speaker/honoraria: none Editorial Boards: American Heart Journal, American Journal of Cardiology (associate editor); Circulation; Circulation-Heart Failure; Circulation- Quality Outcomes; Congestive Heart Failure Guideline writing committees: Chair, ACC/AHA, chronic HF; member, hypertrophic cardiomyopathy; member, ACC/AHA Guideline Taskforce, chair, methodology subcommittee Federal appointments: FDA: Chair, Cardiovascular Device Panel; ad hoc consultant; NIH CICS study section; advisory committee to the Director; AHRQ- adhoc study section chair; NHLBI- consultant; PCORI- methodology committee member Volunteer Appointments: American Heart Association- President, American Heart Association, 2009-2010; American College of Cardiology, Founder- CREDO

PCORI What is PCORI? What is different about PCORI? How will PCORI accomplish its work? What has PCORI done?

PCORI Mission Statement The 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. PCORI is an independent, non-profit organization authorized by Congress committed to continuously seeking input from patients and a broad range of stakeholders to guide its work.

Taking Patient-Centeredness Seriously Patient-Driven Research Patient Engagement Dissemination 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. Understanding the choices patients face Aligning research questions and methods with patient needs Providing patients and providers with information for better decisions

PCOR Definition Defining 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: Expectations Options Outcomes Decisions “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 help me make the best decisions about my health and healthcare?” Joe’s slide Here is a definition of patient-centered outcomes research that guides the work PCORI does. 6

In order to answer these patient-focused questions, PCOR: PCOR Definition In order to answer these patient-focused questions, PCOR: Assesses the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions to inform decision making, highlighting comparisons and outcomes that matter to people; Is inclusive of an individual's preferences, autonomy and needs, focusing on outcomes that people notice and care about such as survival, function, symptoms, and health-related quality of life; Incorporates a wide variety of settings and diversity of participants to address individual differences and barriers to implementation and dissemination; and Investigates (or may investigate) optimizing outcomes while addressing burden to individuals, resource availability, and other stakeholder perspectives.

. National Priorities for Research and Research Agenda Assessment of Options for Prevention, Diagnosis, and Treatment Comparisons of alternative clinical options to support personalized decision-making and self-care Identifying patient differences in response to therapy Studies of patient preferences for various outcomes Improving Healthcare Systems Improving support of patient self-management Focusing on coordination of care for complex conditions and improving access to care Comparing alternative strategies for workforce deployment Communication & Dissemination Research Understanding and enhancing shared decision-making Alternative strategies for dissemination of evidence Exploring opportunities to improve patient health literacy Addressing Disparities Understanding differences in effectiveness across groups Understanding differences in preferences across groups Reducing disparities through use of findings from PCOR Accelerating PCOR and Methodological Research Improving study designs and analytic methods of PCOR Building and improving clinical data networks Methods for training researchers, patients to participate in PCOR Establishing methodology for the study of rare diseases . 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. 8

Criteria for Research Outlined by Law Impact on Health of Individuals and Populations Addresses Current Gaps in Knowledge/ Variation in Care Patient-Centeredness Improvability through Research Impact on Health Care System Performance Rigorous Research Methods Inclusiveness of Different Populations Potential to Influence Decision-Making Efficient Use of Research Resources 8

PCORI What is PCORI? What is different about PCORI? How will PCORI accomplish its work? What has PCORI done?

“This is going to be research done differently!” PCORI Board Member Harlan Krumholz, MD National Patient and Stakeholder Dialogue  National Press Club, Washington, DC February 27,2012

What Makes PCORI Funding Different? Special features include: Patient & Stakeholder Engagement Plan Dissemination and Implementation Assessment Reproducible and Transparent Research Plan PCORI Criteria Outlined by Statute Complies with Methodology Standards User-friendly announcements to encourage broader range of applicants 12 Source: PCORI PFA Application Guidelines http://www.pcori.org/assets/PFAguidelines.pdf

Stakeholder Engagement in PCORI-funded Research Key stakeholders are engaged early and throughout the research process. PCORI will score applications on how meaningfully patients and stakeholders are engaged. Key stakeholders include those for whom the results of the research will be relevant: Patients Nonprofessional Caregivers Clinicians (e.g. Physicians, Nurses, Pharmacists, Counselors, and other providers of care and support services) Patient-Advocacy Groups Community Groups Researchers Health-Related Associations Policy Makers Institutions, Including Organizational Providers, Purchasers, Payers, and Industry A key goal of patient engagement in research is to present information that best supports health decisions through generation of evidence relevant to patients, their caregivers, and clinicians. Patients and other key stakeholders should be meaningfully involved in the research team. The specific members of the team will vary from study to study. 13

The engagement of patients and stakeholders should include: What roles should patients and stakeholders play in research teams? The engagement of patients and stakeholders should include: Participation in formulation of research questions; Defining essential characteristics of study participants, comparators, and outcomes; Monitoring of study conduct and progress; and Dissemination of research results. A key goal of patient engagement in research is to present information that best supports health decisions through generation of evidence relevant to patients, their caregivers, and clinicians. Patients and other key stakeholders should be meaningfully involved in the research team. The specific members of the team will vary from study to study. 14 Source: PCORI PFA Application Guidelines (Sec. 3.1.3.4) http://www.pcori.org/assets/PFAguidelines.pdf

Patient and Stakeholder Engagement 2012 (placeholder slide) Building communities of patients and stakeholders – using website, social media, face-to-face meetings Strengthening ties with advocacy associations, professional clinician organizations, purchaser organizations, research community Refining the PCORI Research Agenda Convening multi-stakeholder workshops focused on each of the National Priorities Forming multi-stakeholder Advisory panels Using social media, surveys to obtain broad input

PCORI What is PCORI? What is different about PCORI? How will PCORI accomplish its work? What has PCORI done?

The First Methodology Committee Report Lori to introduce webinar and welcome listeners.

Methodology Report Chapter 1. Introduction Chapter 2. How the Methodology Committee Developed the Recommended Standards Chapter 3. Overview of the Standards Chapter 4. Methodological Standards for Patient- Centeredness of Research Proposals and Protocols Chapter 5. Methods for Prioritizing Patient-Centered Outcomes Research Chapter 6. Choosing Data Sources, Research Design, and Analysis Plan: Translation Framework and Development of a Translation Table Chapter 7. General and Cross-Cutting Research Methods Chapter 8.  Design-Specific Methods Chapter 9.     Next Steps

Methodology Report The mandate for PCORI’s Methodology Committee is to define methodological standards, recommended actions and a translation table to guide health care stakeholders towards the best methods for patient-centered outcomes research (PCOR). Rigorous methods are essential to building trust in research findings. The report is the necessary catalyst for scientifically rigorous, patient-centered outcomes research that can inform decision- making. Once Report is revised and accepted by the PCORI Board of Governors, future PCORI funding applicants will be expected to reference the Standards in their applications and use the Standards in their PCORI funded research. Robin: The mandate for the Methodology Committee of PCORI is to define methodological standards and develop a translation table to guide health care stakeholders towards the best methods for patient-centered outcomes research (PCOR). The MC has four specific roles: Provide guidance about the appropriate use of methods in patient-centered outcomes research Establish priorities to address gaps in research methods or their application Recommend actions to support standards Map research methods to specific research questions via Translation Table The methodology report is the first deliverable to address PCOR methods. The report includes the first set of methodological standards and a translation table to guide health care stakeholders towards the best methods for patient-centered outcomes research (PCOR). Use of these standards will promote Rigorous methods that are essential to building trust in research findings. Using scientifically rigorous methods will produce patient-centered outcomes research results that can inform decision-making. Once the Report is revised and accepted by the PCORI Board of Governors, future PCORI funding applicants will be expected to reference the Standards in their applications and use the Standards in their PCORI funded research. 19 19

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Selected Milestones in Health Care Interventions and Delivery Strategies and in Research Methods. Gabriel SE, Normand ST. N Engl J Med 2012;367:787-790.

Information Gathering Methodology Report Development Methods Selection Working groups identified and prioritized major research methods questions to be addressed Researchers contracted to address selected topics Contractors developed research materials (e.g., reports, summary templates for proposed standard) MC solicited for external feedback on the translation table (RFI) Workshops held to discuss contractor findings, with invited experts in attendance Information Gathering Committee Expertise Internal Review MC conducted in-depth internal review of materials developed by contractors, and support staff MC independently submitted preliminary votes on proposed standards MC deliberated to reach consensus on recommendations to be endorsed in the report Robin The process for developing the first methodology report over our first year together as the methodology committee involved four phases: method selection, information gathering, internal review, and report generation. Method selection involve working in groups to prioritize the major research methods questions to be addressed. Second, we entered an Information gathering phase in which request for proposals were publicly solicited for selected topics, contractors were selected and completed their reports with summary templates of proposed standards. In addition to methodology committee solicited external feedback on the translation table through a request for information. Workshops were held to discuss contractor findings with invited experts in attendance. Third, the methodology committee began a phase of internal review. The committee conducted an in-depth internal review of the materials that were developed by the contractors and support staff. We then independently submitted are pulmonary votes on the proposed standards and then met in person to deliberate and reach consensus on the recommendations to be endorsed in the report. The report was then generated and refined based on committee of evaluations and discussions. Report Generation Refined recommendations and report content per committee evaluations and discussions 22

Methodology Report – Information Gathering 17 reports* addressing 15 topics, from MC-led contracted research, informed 1st Methodology Report Topics Design, Conduct, and Evaluation of Adaptive Randomized Clinical Trials Conduct of Registry Studies Design of Patient-Reported Outcomes Measures (PROMS) Use of Collaborative or Distributed Data Networks Prevention and Handling of Missing Data Design, Conduct and Evaluation of Diagnostic Testing Causal Inference Methods in Analyses of Data from Observational and Experimental Studies Addressing Heterogeneity of Treatment Effects: Observational and Experimental PCOR *Reports are available on PCORI’s website (www.pcori.org)

Methodology Report – Information Gathering Contracted Research Reports (Cont’d) Topics Involving Patients in Topic Generation Value-of-Information in Research Prioritization Peer Review as a Method for Research Prioritization Examination of Research Gaps in Systematic Reviews for Research Prioritization Integrating Patients' Voices in Study Design Elements with a Focus on Hard- to-Reach Populations Eliciting Patient Perspective PCORI Expert Interviews *Reports are available on PCORI’s website (www.pcori.org)

Methods for Involving Patients in Topic Generation for Patient-Centered Comparative Effectiveness Research – An International Perspective Petra Nass, Susan Levine, Clyde Yancy

Project Framework Discuss the levels of engagement Summarize qualitative research strategies and methods Provide specific examples Discuss facilitators of public engagement Describe three types of scientific research data as part of the engagement process Propose a process of engagement

Levels of Engagement

Public Engagement as Research Is the objective study of the individual experience Uses mostly qualitative research strategies and methods

Scientific Strategies the Framework of Engagement Phenomenology the study of experiences Ethnography the study of cultural phenomena Grounded theory the study of theory through analysis of data Action research the study of focused problem solving Survey

Methods and Processes Interviews (one-on-one or group interviews, photovoice) Observation Documents Questionnaires Public-physician partnerships Consultation Collaboration

Views, Opinions, Experience as Research Data Generates mostly textual data Can be categorized into themes And can be translated into research areas and topics

Case 1 In-depth one-on-one interviews and focus group interviews 40 patients with ulcerative colitis Patients identified 9 research areas Only during in-depth interview patients asked about prenatal genetic testing for a possible termination of pregnancy if the fetus was affected

Case 2 Public-Clinician Partnership to develop research topics for urinary incontinence (James Lind Alliance) Lay members and clinicians consult with their peers to include diverse views Systematic reviews are used to generate additional topics and to avoid duplication of research Nominal Group Technique to reach a consensus and prioritize topics E.g., creating a“Top 10” list of research questions

Case 3 Advisory patient/stakeholder panel to identify research topics and research priorities related to urinary incontinence in women What can researchers study to make your life better? What should we measure to see if your life is better? E.g., Patients considered quality of life the most important outcome measures.

Facilitators that Overcome the Barriers to Public Engagement Creating a patient-centered organizational structure Supporting members of the public Communicating clear expectations Provide training Using processes that give an equal voice to professional and lay participants Using a variety of engagement methods

PCORI What is PCORI? What is different about PCORI? How will PCORI accomplish its work? What has PCORI done?

PCORI Awards The first experience – cycle 1 “Title Slide” format

4 9 6 6 25 Slate includes all applications scoring 30 or better. Addressing disparities (6% of PFA total) 9 Assessing options (4% of PFA total) 6 Communication & dissemination (7% of PFA total) We want to mainly focus on 2, 4 and 7 6 Improving healthcare systems (6% of PFA total) 25 Total (5% of total) % of total means of those applications deemed responsive

Addressing Disparities Conditions Addressing Disparities Comm. & Dissem. We want to mainly focus on 2, 4 and 7 Assessing Options Improving Systems “Other” typically indicates a non-condition response to the question. Responses include: insurance coverage, primary care, surgical decision making, clinical management, comprehensive health systems., etc.

Populations Overall We want to mainly focus on 2, 4 and 7 Other Population includes women, disabled persons, and veterans.

Locations We want to mainly focus on 2, 4 and 7

Project Titles: Assessing Options A Comparison of Non-Surgical Treatment Methods for Patients with Lumbar Spinal Stenosis. Cognitive AED Outcomes in Pediatric Localization Related Epilepsy (COPE) Comparative effectiveness of adolescent lipid screening and treatment strategies Comparative Effectiveness of Intravenous v. Oral Antibiotic Therapy for Serious Bacterial Infections Comparative effectiveness of rehabilitation services for survivors of an acute ischemic stroke Evaluation of a Patient-Centered Risk Stratification Method for Improving Primary Care for Back Pain Improving Psychological Distress Among Critical Illness Survivors and Their Informal Caregivers Selection of Peritoneal Dialysis or Hemodialysis for Kidney Failure: Gaining Meaningful Information for Patients and Caregivers Shared Decision Making in the Emergency Department: The Chest Pain Choice Trial We want to mainly focus on 2, 4 and 7

Improving Palliative and End-of-Life Care in Nursing Homes Project Titles: Improving Healthcare Systems Creating a Clinic-Community Liaison Role in Primary Care: Engaging Patients and Community in Health Care Innovation Improving Palliative and End-of-Life Care in Nursing Homes Innovative Methods for Parents And Clinics to Create Tools (IMPACCT) for Kids' Care Optimizing Behavioral Health Homes by Focusing on Outcomes that Matter Most for Adults with Serious Mental Illness Relative patient benefits of a hospital-PCMH collaboration within an ACO to improve care transitions The Family VOICE Study (Value Of Information, Community Support, and Experience): a randomized trial of family navigator services versus usual care for young children treated with antipsychotic medication We want to mainly focus on 2, 4 and 7

Shared Medical Decision Making in Pediatric Diabetes Project Titles: Communications & Dissemination Decision Support for Parents Receiving Genetic Information about Child’s Rare Disease Extension Connection: Advancing Dementia Care for Rural and Hispanic Populations Patient-Identified Personal Strengths (PIPS) vs. Deficit-Focused Models of Care Presenting Patient-Reported Outcomes Data to Improve Patient and Clinician Understanding and Use Relapsed childhood neuroblastoma as a model for parental end-of-life decision-making Shared Medical Decision Making in Pediatric Diabetes   We want to mainly focus on 2, 4 and 7

Project Titles: Addressing Disparities Cultural tailoring of educational materials to minimize disparities in HPV vaccination Long-term outcomes of community engagement to address depression outcomes disparities Reducing Disparities with Literacy-Adapted Psychosocial Treatments for Chronic Pain: A Comparative Trial Reducing Health Disparities in Appalachians with Multiple Cardiovascular Disease Risk Factors We want to mainly focus on 2, 4 and 7

Examples of Patient Centeredness in Funded Applications

The Chest Pain CHOICE Trial Assessment of Prevention, Diagnosis and Treatment Options “We first developed a Web-based tool to reliably determine the future risk of a heart attack. Then, incorporating feedback from patients, doctors, and researchers, developed a patient education tool—Chest Pain Choice—to help patients better understand the tests that are being performed to determine the cause of their chest pain, what these tests might show, their individualized 45- day risk of a heart attack, and the available management options.” $2.03 million

Assessment of Prevention, Diagnosis and Treatment Options Preventing Venous Thromboembolism: Empowering Patients and Enabling Patient-Centered Care via Health Information Technology Assessment of Prevention, Diagnosis and Treatment Options “Patient-led, health educator–moderated training sessions for nurses will promote improved communication about VTE with patients. Informational materials developed with partnering patient stakeholders, including self-monitoring tools, will be provided to all hospitalized patients as a part of the admission package, empowering patients to take an active role in VTE prevention”. $1.5 million

Ovarian Cancer Patient-Centered Decision Aid Assessment of Prevention, Diagnosis and Treatment Options “The objective of this study is to develop and test a new decision aid—named Patient Centered Outcome Aid (PCOA)—that will allow patients to assimilate information and identify trade-offs about the impact of IP/IV therapy versus IV-only therapy on their QOL and survival, based on their own preferences and personal clinical characteristics, described in terms that are meaningful to them”. $1.9 million

Improving Healthcare Systems The Family VOICE Study: A Randomized Trial of Family Navigator Services Versus Usual Care for Young Children Treated with Antipsychotic Medication Improving Healthcare Systems “In this study, we are partnering with parents/family advocates, child-serving agencies, and health providers to develop a Family Navigator Service to link with this medication program. Family Navigators are individuals who have cared for their own child with mental illness. The navigators will support parents, provide information on psychosocial treatment options, and address barriers to using services”. $1.4 million

PATient Navigator to rEduce Readmissions (PArTNER) Improving Health Care Systems “We propose to engage stakeholder groups at an MSI,(minority serving institution) including patients/caregivers, in an iterative process to develop a CHW-based Patient Navigator (CHW- Navigator- community health worker) toolkit tailored to their needs to augment the benefits of a QI program to reduce readmission. We will compare the effectiveness of an integrated CHW- Navigator on the patient experience, 30-day readmissions rates, and other outcomes”. $2.0 million

Reverse Innovation and Community Engagement to Improve Quality of Care and Patient Outcomes Communication and Dissemination Research “We propose to adapt a World Health Organization community engagement approach to support Hospital Community Health Partnership (J-CHiP), an initiative targeting high-risk adults with chronic conditions who reside in surrounding ZIP codes. The overall goals are to improve the health of residents by enhancing communication and “co-developing” a community engagement partnership between hospitals and clinics, CBOs, and community”. $2.03 million

Addressing Healthcare Disparities Reducing Health Disparities in Appalachians with Multiple Cardiovascular Disease Risk Factors Addressing Healthcare Disparities “We will compare (a) the standard of care alone and referral to a primary care provider for management of CVD risk factors with (b) standard of care supplemented by patient- centered, culturally appropriate, self-care CVD risk reduction intervention (HeartHealth) designed to improve multiple CVD risk factors while overcoming barriers to success”. $2.09 million

PCORI What is PCORI? What is different about PCORI? How will PCORI accomplish its work? What has PCORI done?

Sebastian Schneeweiss PCORI METHODOLOGY COMMITTEE Al Berg Brian Mittman Ethan Basch Sherine Gabriel Jean Slutsky Michael Lauer Naomi Aronson Clyde Yancy Mary Tinetti David Flum Robin Newhouse Mark Helfand Sharon Lise-Normand Sebastian Schneeweiss Steven Goodman John Ioannidis David Meltzer

Thank You!