GHS Research Lecture Series Greenville, SC June 10, 2014

1. Patient-Centered Outcomes Research Linda Crew McNamara, RN, MBA PSDTO Advisory Panel & Stakeholder Reviewer GHS Research Lecture Series Greenville, SC June 10, 2014

2. Overview of presentation Background (Evolving Research Paradigm) Patient Centered Outcomes Research Patient Centered Outcomes Research Institute (PCORI) Tips for strong proposal

3. Background Community-based Participatory Research: • Lack of trust (legacy of research abuse) • Helicopter researchers • Authority of community members less meaningful than researchers • Limited resource sharing • Lack of equitable compensation for patient partners

4. Background • Length of time from bench to bedside • Lack of relevance to real-life patient concerns • Research findings not disseminated broadly or not understandable • Rare diseases and subgroups of patients not addressed • Limited amount of behavioral health research

5. Background Patients wanted more active role: “Nothing about me without me.” “PhDs of the sidewalk.”

6. Background “Changing the way patients are thought about, as consumers of research rather than just study subjects, is a core principle for engaging patients in research.”

7. What is Patient-Centered Outcomes Research (PCOR)? “Research that sees through the eyes of patients.” Annals of Internal Medicine 18 Sept 2012 Relevant Usable Easily accessible

8. PCOR helps patients and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options. This work answers patient’s questions. What is Patient-Centered Outcomes Research (PCOR) 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?”

9. Examples of Patient-CenteredOutcomes • Quality of life and functional status • Psychological factors: anxiety, depression • Economic outcomes (work hours, income lost) • Treatment decision related (decisional conflict, regret)

10. Why PCOR? • Patients are one of healthcare’s richest, yet largely untapped resources. • Patients as meaningful partners can lead to more effective research, more useful evidence, and care than leads to improved outcomes. • Positive outcomes on a much faster track.

11. PCORI Has a Broad and Complex Mandate “The purpose of the Institute is to assist patients, clinicians, purchasers, and policy-makers in making informed health decisionsby 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...” -- from Patient Protection and Affordable Care Act

12. PCORI Is Accountable for Changing Practice “(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…” -- from Patient Protection and Affordable Care Act

13. PCORI Focus • Comparative Clinical Effectiveness Research • Patient-centered • Answering questions that matter to patients and other clinical decision makers • Comparisons of outcomes that matter to patients

14. National Priorities for Research 9

15. Key Feature: Emphasizes Engagementfor Getting to Practical, Useful Research Review and Conduct Research Generate and Prioritize Research Questions Review Proposals and Partner in Research Advise Us on What to Study Tell Us How We’re Doing Help Us Share the Findings Dissemination and Implementation Portfolio Review

16. Engagement Goals Build a Patient-Centered Outcomes Research Community Engage the PCOR Community in Research Promote Dissemination and Implementation 32

17. Eugene Washington PCORI Engagement Awards 33

18. The National Patient-Centered Clinical Research Network (PCORnet) Clinical Data Research Networks • 11 Networks • $76.8 Million Awarded System-based networks, such as hospital systems Patient-Powered Research Networks • 18 Networks • $16.8 Million Awarded Patients with a single condition form a research network • Coordinating Center • Provides technical and logistical assistance under the direction of the Steering Committee and PCORI staff 29

19. PCORI Funded Projects to Date Totalnumber 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) 15

20. How to Submit an Application • Visit pcori.org/apply • Key Dates • Funding Announcements • Application Guidelines and Templates • Guidance on the PCORI Methodology Standards • Sample Engagement Plans • Frequently Asked Questions 39

21. PCORI’s Merit Review Process 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 • Impact of the condition on the health of individuals and populations • Potential for the study to improve healthcare and outcomes • Technical merit • Patient-centeredness • Patient and stakeholder engagement 40

22. Tips for a Strong Proposal Meaningful engagement at each phase: • Topic generation • Research design/modification • Implementation • Dissemination • Outcomes

23. Tips for a Strong Proposal Identifying & selecting research questions. Ask the type of questions that patients can address. • “Patients don’t have research questions. They just have questions.” • What is your biggest health issue, and what do you need to know about it?”

24. Tips for a Strong Proposal Identifying & selecting research questions. Leverage the potential of “big data.” • Centers for Medicare & Medicaid Services (CMS) claims database to find the sickest people, and then identify the research questions that would provide the knowledge they need. • Mining social media, using algorithms to find the questions already being asked by patients.

25. Tips for a Strong Proposal Applications must answer major question related to decisions about clinical choices, healthcare delivery, or another relevant issue in the announcement. Gap analysis and systematic reviews must support the need for the study.

26. Tips for a Strong Proposal Must be comparative clinical effectiveness research: Evaluate and compare health outcomes and the clinical effectiveness, risks, and benefits of 2 or more medical treatments, services, and health care interventions protocols for treatment, care management, and delivery, procedures, medical devices, diagnostic tools, pharmaceuticals (including drugs and biologicals), integrative health practices, and any other strategies or items being used in the treatment, management, and diagnosis of, or prevention of illness or injury in individuals

27. Tips for a Strong Proposal Differentiate between patient engagement & patient-centeredness. Engagement: Inclusion of patients in the research process. Patient centeredness: addresses questions that patients and their families care about in clinical settings.

28. Tips for a Strong Proposal Proposals should demonstrate superior technical merit. Study design that best aligns with study topic and answers research question in most efficient and effective way. Refer to PCORI Methodology Standards as a guide.

29. Tips for a Strong Proposal • Research should adhere to PCORI’s Methodology Standards. • Methodology Standards: 11 Broad Categories 13

30. Tips PCORI Funding Center: Tools and resources for applicants. Help desk: Call 202.627.1884; complete an online inquiry form; or schedule a call with program staff. Institute of Medicine workshops and publications.

31. Pragmatic Clinical Studies and Large Simple Trials • 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. 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 30

32. Have a Question? General Inquiries info@pcori.org | (202) 827-7200 Research/Programmatic Questions sciencequestions@pcori.org | (202) 627-1884 Administrative/Financial/TechnicalQuestions pfa@pcori.org Engagement and PCORI Activities getinvolved@pcori.org 42

33. Thank You Linda Crew McNamara, RN, MBA