Team Science: Partnering with Patients Adrian F. Hernandez, MD, MHS Director, Health Services and Outcomes Research Associate Director, DCRI All Rights Reserved, Duke Medicine 2007 What problems are we trying to solve? 1 All Rights Reserved, Duke Medicine 2007 A Persistent Problem Major Gaps in Evidence 3 Tricoci P et al. JAMA 2009;301:831-41 All Rights Reserved, Duke Medicine 2007 Level of Evidence A Current 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%AF Heart failure PAD STEMI Perioperative Secondary prevention Stable angina SV arrhythmias UA/NSTEMI Valvular disease VA/SCD PCI CABG Pacemaker Radionuclide imaging All Rights Reserved, Duke Medicine 2007 Trial Hyperinflation Berndt E, Cockburn I. Monthly Labor Review, June 2014 All Rights Reserved, Duke Medicine 2007 The Driver is Complexity Berndt E, Cockburn I. Monthly Labor Review, June 2014 All Rights Reserved, Duke Medicine 2007 Surprisingly.. Less Participation Berndt E, Cockburn I. Monthly Labor Review, June 2014 All Rights Reserved, Duke Medicine 2007 HOW CAN WE ENGAGE PATIENTS (PARTICIPANTS)? 2 All Rights Reserved, Duke Medicine 2007 Post Approval Studies Create study procedure Implemen t the Study Monitor the study Analyze data and interpret results Dissemi- nate study information FDA Review and Approval Develop the study concept Secure Funding Prepare the Study Protocol Provide input on study design such as barriers to participation, study endpoints, and risk/benefit perception Recruit study participants Serve as a peer advocate during the informed consent procedure Recruit study participants Serve as a peer advocate during the informed consent procedure Provide feedback on how the patient community will view results 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 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 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 Work with researcher to secure NIH, PCORI, AHRQ funding DART & Halo Therapeutics, e.g. raise seed funding Fundraise for own research Help finalize eligibility criteria within the study protocol Assist in creating the informed consent form Advise study recruitment Help finalize eligibility criteria within the study protocol Assist in creating the informed consent form Advise study recruitment Across the continuum? Provide information on unmet need aand therapeutic burden Interest of research question to patient community Provide information on unmet need aand therapeutic burden Interest of research question to patient community Serve on FDA advisory committees or post-market surveillance initiatves Courtesy: Bray Patrick-Lake, MFS All Rights Reserved, Duke Medicine 2007 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 All Rights Reserved, Duke Medicine 2007 Perspectives on Patient Engagement Per Investigator (Worries) 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 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 All Rights Reserved, Duke Medicine 2007 Meaningful Patient Engagement is often a foreign language Engagement Worst to Best Online reports & anecdotes Patient visits The sign off Survey Focus group Patient Stakeholders Co-investigators Translation to Researchers Courtesy: Bray Patrick-Lake, MFS All Rights Reserved, Duke Medicine 2007 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? Whos 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 ). Sudbury, MA: Jones and Bartlett Publishers. All Rights Reserved, Duke Medicine 2007 CAN PATIENT (PARTICIPANT) ENGAGEMENT MATTER? 3 All Rights Reserved, Duke Medicine 2007 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! All Rights Reserved, Duke Medicine 2007 Patient-Centered Research into Outcomes Stroke Patients Prefer and Effectiveness Research All Rights Reserved, Duke Medicine 2007 Real, live patients and what they say The worse doctor was a cardiologist The doctors didnt give me the clot-buster because I was on coumadin Doctors didnt listen to what I needed PROSPER Interview with Lesley Maisch All Rights Reserved, Duke Medicine 2007 A Few Questions Will statins be as good for me as someone younger? And what dose? What are the tradeoffs between long-term health and potential negative effects of my medications? Ive had a stroke and worry about falls. Will blood thinners hurt me? Which one is better? Ive 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 antidepressant s improve my quality of life after stroke? All Rights Reserved, Duke Medicine 2007 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 All Rights Reserved, Duke Medicine 2007 PROSPER: Personalized Decisions All Rights Reserved, Duke Medicine 2007 CAN WE DO EVEN BETTER? 4 All Rights Reserved, Duke Medicine m+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 solutions that change care and improve outcomes? ? 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 Research Infrastructure Done Differently Hallmarks of PCORnet 1.Highly engaged patients, clinicians, health systems, researchers and other partners 2.A collaborative community supported by robust governance 3.Analysis-ready standardized data with strong privacy protections 4.Oversight that protects patients, supports the timely conduct of research, and builds trust in the research enterprise 5.Research that is sustainably integrated into care settings and with communities of patients Enabling Pragmatic Research: eScreening, eEnrollment and eFollowup DCRI FOLLOW-UP Patient Reported Outcomes Medication use Health outcomes Baseline Data ADAPTABLE Enrollee 8 20 . OR CMS & Payer Virtual Data Warehouse FOLLOW-UP Longitudinal health outcomes Portal FOLLOW-UP Patient Reported Outcomes Medication use Health outcomes PCORNet Coordinating Center FOLLOW-UP Via Common Data Model Longitudinal health outcomes All Rights Reserved, Duke Medicine 2007 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! All Rights Reserved, Duke Medicine 2007 Thanks! Trials N