1. Introduction to Human Subjects Protection Challenges in Patient- Centered Outcomes Research (PCOR) Toward Establishing a Stakeholder Community and Action Plan This project is funded by a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington Engagement Award Program (EAIN-2299, PI: Bevans)

2. Thank you for joining! This is the first of a 3-part webinar series on the protection of human subjects in patient-centered outcomes research (PCOR) Overview of site functionality -Polling feature -Comments/Questions -This session will be recorded

3. Stakeholders at the Table PCOR ResearchersIRB Members & Leaders Patients & Families Katherine Bevans Assistant Professor of Pediatrics, CHOP Amy Schwartzoff Director, Human Subjects Research CHOP Megan Kasimatis Singleton Associate Director University of Pennsylvania Amy Kratchman Family Consultant Darlene Barkman Family Consultant Anna de la Motte Project Manager, CHOP Researchers: 55 (55%) IRB Affiliates: 39 (39%) Patients & Families: 6 (6%) Megan Morash IRB Chair, Partners Healthcare Researchers have been asking questions like… Do I need IRB approval to engage patients and other stakeholders in my research? IRB members have been asking questions like… Under what circumstances are patients and other stakeholders "human subjects?" Patients and other stakeholder contributors have been asking questions like… Is my personal information kept safe if I’m not signing a formal consent form? Alison Rein AcademyHealth

4. What makes PCOR unique? Patient and other stakeholder engagement “meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders throughout the research process— from topic selection through design and conduct of research to dissemination of results. In unique ways, not typical of traditional research” – PCORI Traditional Research Patient-Engaged Research Patients and Families (Research Participants) Researchers Clinicians Community Leaders Researchers Patients and Families Policy Makers Educators

5. Challenges to Patient and Other Stakeholder Protection Patient and other stakeholder roles in PCOR are not limited to that of human subjects/research participants Significant challenges are:  Protection of patient and other stakeholder contributors to research (not acting as human subjects)  Preparing patient/other stakeholder contributors to uphold the principles of ethical research

6. Overview of Human Subjects Protection Research  Systematic investigation, including research development, testing and evaluation, designed to develop or contribute to generalizable knowledge.  An activity that involves a prospective research plan which incorporates data collection, either quantitative or qualitative, and data analysis to answer a research question.

7. Human Subject - a living individual about whom a researcher obtains: (1) Data through intervention or interaction with the individual, or (2) Identifiable private information. Overview of Human Subjects Protection

8. Evolution of This Work PCORI Pilot Project Leaning Network (PPPLN) - AcademyHealth  65% of Pilot Project teams experienced one or more human subjects protection or IRB-related challenged (considered unique to PCOR)  Investigator questionnaire (n=31) and IRB leader interviews (n=5) revealed 3 common challenges: 1.Is the activity research? 2.Is the patient/other stakeholder a “human subject?” 3.How can research teams prepare patient/other stakeholder partners to uphold the principles of human subject protection? Need for guidance/strategies on these and other issues:  Community of stakeholders with diverse perspectives  Identifying and prioritize concerns

9. Purpose of the Webinar Series 1.Develop and evaluate a process for sharing actionable strategies for addressing human subjects protection challenges in PCOR; 2.Establish a community of stakeholders who seek to advance the ethical conduct of PCOR; 3.Identify high priority challenges to the ethical conduct of PCOR.

10. Consider the Following Scenario: A team aims to evaluate the impact of diabetes self-management education programs on self-care behaviors, symptoms, functioning, and quality of life. The team met with a group of 5 people with diabetes on three occasions. These people are called “patient contributors.” During the meetings, the patient contributors responded to the team’s questions about…  Critical features of diabetes self-management education programs e.g., What should the programs cover?  Important outcomes of diabetes self-management education programs e.g., How do patients want the programs to improve their lives?  Ways of assessing patient outcomes e.g., Do the surveys do a good job of measuring outcomes that matter to patients?  Recruitment strategies e.g., What are the best ways to recruit patients into the study?  Helping researchers understand study results e.g., Why do some patients (but not others) have fewer symptoms after completing the program?

11. Polling Question #1 Do researchers need to obtain informed consent from the patient contributors for their involvement in this study?  YES  NO  MAYBE

12. Polling Question #1 Results yes27% no41% maybe32% Do researchers need to obtain informed consent from the patient contributors for their involvement in this study?

13. Stakeholder Perspectives: IRB Leaders The patient contributors are NOT human subjects. This is preliminary discussion with patient contributors to plan research project. It describes a prospective plan to evaluate the impact of diabetes education programs and their outcomes. Is this research ? No Patient contributor role ? Yes Study staff Study advisorHuman subject Involved in scientific design; collection of identifiable data; analysis of individual data; participation in research intervention Contribute to study planning or research question formulation; advise in recruitment strategies; participate in survey development Participation in focus group and sharing identifiable information Consent is not required. Consent required.

14. Stakeholder Perspectives: Researchers Patient partners’ expertise commonly stems from their personal experiences.  In sharing this expertise, patient advisors and study team members often disclose personal health information (even if they are not explicitly asked to do so).  Obtaining feedback from patient advisors often “feels” a lot like a focus group. E.g., obtaining patient feedback on survey content How can researchers protect patient advisor/team members’ privacy? What about dual roles?

15. Stakeholder Perspectives: Patient & Family Partners “I just assume I’m protected. We heard this a lot from other patient and family partners in research. The hope is that you and your information is being protected.” “Am I protected if I’m on the ‘other’ side (i.e. as an advisor)? I’m still giving information that’s personal information, and it’s being used to guide things. So is that protected information?”

16. A team aims to evaluate the impact of diabetes self-management education programs on self-care behaviors, symptoms, functioning, and quality of life. The researchers invited 5 people with diabetes to contribute to the study by:  Enrolling patient participants (human subjects) in the study e.g., providing potential participants with information about the study, engaging in the informed consent process  Collecting data e.g., Administering questionnaires to patient participants Consider the Following Scenario:

17. Polling Question #2 The patient contributors’ role in this scenario is best classified as:  HUMAN SUBJECT/RESEARCH PARTICIPANT  ADVISOR TO THE RESEARCH TEAM  MEMBER OF THE RESEARCH TEAM  I DON’T KNOW

18. Polling Question #2 Responses Don't Know4% Study Team Member67% Advisor22% Human Subject7% The patient contributors’ role in this scenario is best classified as:

19. Polling Question #3 Should the 5 patient contributors be required to complete training in the protection of human subjects?  YES  NO  MAYBE

20. Polling Question #3 Responses yes86% no9% maybe5% Should the 5 patient contributors be required to complete training in the protection of human subjects?

21. Polling Question #4 Are existing human subjects protection training programs appropriate for patient contributors?  YES  NO  I DON’T KNOW Please list recommended training programs in the comments box.

22. Polling Question #4 Response Yes31% No56% Don’t know13% Are existing human subjects protection training programs appropriate for patient contributors?

23. Stakeholder Perspectives: IRB Leaders & Researchers  In order to best protect the “subjects” of research some training in human subjects protections is felt to be important for all individuals who may interact with subjects or their identifiable data  Most of the training programs currently available are primarily designed to address the responsibilities of the researcher and are not appropriately tailored to the potential roles of community partners  Targeted training is needed that better addresses human subjects protections within the context of the roles community partners might have

24. Stakeholder Perspectives: Patient & Family Partners If I’m recruiting people, or collecting their information, shouldn’t I know about how to protect those subjects?  How can I learn how to protect research participants? If I don’t have training as a researcher or a research assistant, how am I expected to learn all of it? Who is responsible for making sure that I’m trained? Who do I go to if I have questions about the protection of human subjects?  Or, if there are parts of the training that you don’t understand who do you go to?

25. Summary of Notable Challenges Determining whether an activity that involves a patient or other stakeholder is “research.” Categorizing patients/other stakeholders according to their role(s) in research (e.g., human subject, advisor, study team member).  For each role, specifying…  Patient/other stakeholder responsibilities and limits  Principal Investigator responsibilities  Training needs

26. Upcoming Webinars Monday, February 8 th (3:00pm-5:00pm EST) Clarifying Definitions: Who is a human subject? Is it research? Monday, February 29 th (3:00pm-5:00pm EST) Preparing Patients and Other Stakeholders to Uphold Ethical Research Principles If you know of anyone who may be interested in attending a future webinar, please send them the handout provided. In particular, please help us to grow our community of patients and other stakeholders!

27. Next Steps: We need your input!  Help us revise topics and identify speakers for remaining webinars  Webinar satisfaction survey  Help us generate a stakeholder-informed, prioritized list of challenges  Brief (<10 minute) open-ended questionnaire or telephone- based interview to identify additional challenges  Online activity to generate, refine, and prioritize challenges Guidance may be developed for the most highly prioritized challenges.

28. Thank you! Contact us at: Katherine Bevans (Project Leader) bevans@email.chop.edu Anna de la Motte (Project Manager) delamottea@email.chop.edu