Partnering with Patients for Science: Closing Evidence Gaps and Engaging Participants

1. Team Science: Partnering with Patients Adrian F. Hernandez, MD, MHS Director, Health Services and Outcomes Research Associate Director, DCRI

2. 1 What problems are we trying to solve?

3. A Persistent Problem – Major Gaps in Evidence Tricoci P et al. JAMA 2009;301:831-41 3

4. AF Heart failure PAD STEMI Perioperative Secondary prevention Stable angina SV arrhythmias UA/NSTEMI Valvular disease VA/SCD PCI CABG Pacemaker Radionuclide imaging Level of Evidence ACurrent Guidelines* *Guidelines expressing Level of Evidence 11.7% 26.4% 15.3% 13.5% 12.0% 22.9% 6.4% 6.1% 23.6% 0.3% 9.7% 11.0% 19.0% 4.9% 4.8% 0% 10% 20% 30%

5. Trial Hyperinflation Berndt E, Cockburn I. Monthly Labor Review, June 2014

6. The Driver is Complexity Berndt E, Cockburn I. Monthly Labor Review, June 2014

7. Surprisingly….. Less Participation Berndt E, Cockburn I. Monthly Labor Review, June 2014

8. 2 How Can We Engage Patients (Participants)?

9. Across the continuum? • Provide information on unmet need aand therapeutic burden • Interest of research question to patient community • Provide feedback on how the patient community will view results • Provide input on study design such as barriers to participation, study endpoints, and risk/benefit perception • Serve on FDA advisory committees or post-market surveillance initiatves • Recruit study participants • Serve as a peer advocate during the informed consent procedure Secure Funding Implement the Study Dissemi-nate study information Prepare the Study Protocol Create study procedure Monitor the study Analyze data and interpret results FDA Review and Approval Develop the study concept Post Approval Studies • Help finalize eligibility criteria within the study protocol • Assist in creating the informed consent form • Advise study recruitment • Work with research team to ensure study participants get feedback from study • Write newsletter articles or blog about results • Co-present results with researcher at a conference or support group • Serve on a Data Safety Monitoring Board • Provide recommendations for revising study protocol if changes need to be made • Work with researcher to secure NIH, PCORI, AHRQ funding • DART & Halo Therapeutics, e.g. raise seed funding • Fundraise for own research Courtesy: Bray Patrick-Lake, MFS

10. Common problems with protocols from patient perspective • Exclusion/inclusion criteria so stringent that no “real” patients meet criteria • Study not feasible • Procedural burden too high • Unmanageable dosing regimen • Too many study appointments • Location not convenient • Pediatric patients needing to be seen during school hours Courtesy: Bray Patrick-Lake, MFS

11. Perspectives on Patient Engagement Per Investigator (Worries) Per Patient (Benefits) Inspire scientists Improve plans for recruitment, retention, outreach, follow up, dissemination Provide “real life” perspectives on risk/benefits Serve as reminder to ultimate end product of concrete health benefits rather than an ‘academic’ only product • Single-minded focus on cures • Disrupt or delegitimize the peer review process • Bring an agenda or too much emotion • Lack sufficient experience to speak to essential issues of scientific merit

12. Meaningful Patient Engagement is often a foreign language Engagement Worst to Best Translation to Researchers 隨機雙盲對照研究 观察性研究 病例對照研究 病例對照研究 案例系列 病例報告 想法，意見 • Online reports & anecdotes • Patient visits • The sign off • Survey • Focus group • Patient Stakeholders • Co-investigators Courtesy: Bray Patrick-Lake, MFS

13. Patient Advocate Review of Research • Why is this study important? • Who does this study benefit? • Do patients agree that clinical equipoise exists? • Will this change the standard of care? • Who’s eligible for the study? Are they representative of the patient population? • Are there other similar trials? • Does this fill a gap in research? • Will patients be interested in enrolling? • Is the study feasible? • Is the informed consent readable and clear? • How will study results be communicated? Earp, J.L., French, E.A, and Gilkey, M.B. (2008) Research Advocacy in Traditional Settings: Questions of Influence and Legitimacy. In Patient Advocacy for Health Care Quality: Strategies for Achieving Patient-Centered Care (pp. 445-478). Sudbury, MA: Jones and Bartlett Publishers.

14. 3 Can Patient (Participant) Engagement Matter?

15. What is PROSPER? • Patient-centered Research into Outcomes Stroke Patients prefer and Effectiveness Research • Above all, PROSPER is a research collaboration • Patients • Doctors • Caregivers • Researchers • Multiple perspectives and experiences = better understanding!

16. Patient-Centered Research into Outcomes Stroke Patients Prefer and Effectiveness Research

17. Real, live patients and what they say… “The worse doctor was a… cardiologist” “The doctors didn’t give me the clot-buster because I was on coumadin” “Doctors didn’t listen to what I needed” PROSPER Interview with Lesley Maisch http://www.youtube.com/watch?v=KubuT1_6Lrw

18. A Few Questions… I’ve had a stroke and worry about falls. Will blood thinners hurt me? Which one is better? How do the new anticoagulants affect my likelihood of having another stroke? Can antidepressants improve my quality of life after stroke? What are the tradeoffs between long-term health and potential negative effects of my medications? Will statins be as good for me as someone younger? And what dose?

19. What outcomes are we addressing? • Days alive and at home (“home-time”) • Any fatigue or depression experienced as a result of the stroke • Quality of life • Mobility • Whether patient can complete normal activities like dressing, eating, shopping • How well patients are tolerating their medications/any side effects they are experiencing

20. PROSPER: Personalized Decisions http://www.prosper-stroke.org/

21. 4 Can we do Even better?

22. 75m+ Patients walk through the doors of hospitals and clinics each year with questions about their health and their care. ? How do we study their experiences to find answers and create solutionsthat change care and improve outcomes? http://www.cdc.gov/nchs/fastats/hospital.htm

23. A United Network: PCORnet PCORnet is a network of patients, clinicians, researchers, health systems and health plans united to efficiently generate evidence to improve patient-centered outcomes “Research Infrastructure Done Differently”

24. Hallmarks of PCORnet • Highly engaged patients, clinicians, health systems, researchers and other partners • A collaborativecommunity supported by robust governance • Analysis-ready standardized data with strong privacy protections • Oversight that protects patients, supports the timely conduct of research, and builds trust in the research enterprise • Research that is sustainably integrated into care settings and with communities of patients

25. Enabling Pragmatic Research: eScreening, eEnrollment and eFollowup 30 OR • Portal FOLLOW-UP • Patient Reported Outcomes • Medication use • Health outcomes • DCRI FOLLOW-UP • Patient Reported Outcomes • Medication use • Health outcomes ADAPTABLE Enrollee 12 16 20 …. 8 4 PCORNet Coordinating Center FOLLOW-UP • Via Common Data Model • Longitudinal health outcomes Baseline Data CMS & Payer Virtual Data Warehouse FOLLOW-UP • Longitudinal health outcomes

26. Conclusions • Patients are a driving force in improving care and will be for the research enterprise. • Meaningful engagement of patient stakeholders across the research continuum will improve quality, efficiency and translation of research. • Patient engagement requires an investment of time and resources just like any component of research • Engaging patients as partners for research is inspirational and fun!

27. Thanks! N Trials Trials