The Crohn ’ s and Colitis Foundation of America Patient Powered Research Network Kick-off Meeting

1. The Crohn’s and Colitis Foundation of America Patient Powered Research Network Kick-off Meeting Michael D. Kappelman University of North Carolina February 3, 2014

2. Objectives • Celebrate the success of our proposal! • Get to know one another • Review and refine scope of our project • Leave with a shared vision • Understand the role that each collaborator/team will play in the development of our network • Understand how our network fits to PCORI’s larger National Patient-Centered Clinical Research Network Program

3. An Amazing Team! CCFA CCFA Partners Crohnology Patients Know Best Validic Data architecture and integration team UNC Qualitative Research and Intervention Core Industry stakeholder PCORI Most importantly, our patients!

4. CCFA • Balfour Sartor • Tania Kamphaus • Project management staff (Amanda Singer/Douglas) • Other CCFA staff in attendance: • Marie Granieri • Marjorie Merrick • CCFA consultants • Kelly Myers • Seth Myers • Jennifer Feikin

5. CCFA Partners Bob Sandler Myself Millie Long Chris Martin Wenli Chen Lucy Goble Beth Jaeger Tom Caruso

6. Crohnology Sean Ahrens

7. Patients Know Best Mohammad Al-Ubaydli Emily Zhao

8. Validic Ryan Beckland Brent Fagg

9. Data architecture and integration Kristen Anton Dan Crichton

10. UNC Qualitative Research and Intervention Team Maihan Vu Darren DeWalt

11. Industry Stakeholder Suzanne Cook

12. PCORI staff Sarah Daugherty Jaye Bea Smalley

13. Patient Governance and Engagement Sue Johnson Andy Garb Nick Uzl Jessica Burris Brian Price

14. Ambitious Goals Radically transformthe CCFA Partners Internet Cohort! • Enhance network growth, diversity, and retention; • Build a robust network community, including patient governance structures that allow greater involvement of patients in research • Expand network data to include electronic health records, data from mHealth apps and devices, and biological samples • Develop a customized, yet scalable and adaptable, distributed data network (i.e. virtual database) by repurposing NASA technology • Develop and test patient and provider-focused tools that utilize individual patient data to improve health behaviors, healthcare decisions, and, ultimately, outcomes • Further engage the scientific community through open collaboration and data sharing • Rapidly disseminate new knowledge to patients, enabling them to improve their health

15. Logistics for Today • Brief presentations from each of our network partners/teams • Skills, technology, assets, role in the project • Presentations meant to facilitate conversation! • Please react, respond, ask questions, make suggestions, brainstorm • I’m more concerned with having productive discussion than staying on schedule • Breaks, bathrooms, lunch • Breakout sessions • Some may need to leave early for flights • Cars at Carolina Inn • Be in car 90 minutes before flight

16. CCFA Introductions

17. High level project goals Integrate communities Expand capacity Grow membership Become part the U.S. National Patient Centered Research Infrastructure

18. High level project goals • Integrate communities • Membership • Data • Functionality • Expand capacity • Grow membership • Become part the U.S. National Patient Centered Research Infrastructure

19. Integration of communities • “Whole is greater than the sum of its parts” • Today CCFA Partners and Crohnology are quite distinct • Comprised of largely different populations • Survey of 2,000 CCFA Partners participants estimated that only 2% were Crohnology members • Serve different functions • CCFA Partners: primarily a research network; patient support, engagement and education secondary focus • Crohnology: Greater focus on community (interactive, facilitates sharing) and patient self-management/tracking (tools, etc.); secondary focus on research • Collect different types of data • CCFA Partners: Detailed (but infrequent) surveys to collect research data (i.e. validated instruments) • Crohnology: More frequent, less detailed data; focus is more practical

20. Integration • Therefore, integration will: • Grow membership • Enhance data assets • Improve user experience • Add value for both networks • With incremental costs • Details of this are beyond the scope of today’s meeting!

21. Integration • Therefore, integration will: • Grow membership • Enhance data assets • Improve user experience • Add value for both networks • With incremental costs • Details of this are beyond the scope of today’s meeting! • Technical • Practical • Legal • IP

22. High level project goals • Integrate communities • Expand capacity • New types of data • New functionality/tools • Enhanced patient engagement and governance • Grow membership • Become part the U.S. National Patient Centered Research Infrastructure

23. New types of data mHealth apps and devices personal health records (PHRs) biosamples

24. mHealth apps and devices • Quantified Self: a movement to incorporate technology into data acquisition on aspects of a person's daily life in terms of inputs (e.g. food consumed, quality of surrounding air), states (e.g. mood, arousal, blood oxygen levels), and performance (mental and physical). Such self-monitoring and self-sensing combines wearable sensors (EEG, ECG, video, etc.) and wearable computing • Wikipedia

25. mHealth apps and devices • Hundreds of products for use in general population • Activity monitors, sleep monitors, temp/HR monitors, diet trackers, etc. • IBD specific apps as well • Crohnology • CCFA GI Buddy • A treasure chest of data for learning!

26. Personal Health Records Stay Tuned!

27. Biosamples • Saliva, blood, stool, etc. • Combined with patient-reported data and medical records can facilitate studies of: • Prognosis and risk stratification • Effects of environment/lifestyle factors • Response to treatment (med, surgery, diet) • Side effects of treatment • How will we obtain biosamples: • New sample collection: recruiting from our engaged patient network • Using existing samples: linking with other studies

28. New Patient-inspired functionality Data as a two way street Moving beyond the conventional research study:

29. New Patient-inspired functionality • Use data to motivate, engage, and empower our network participants • Design a dashboard where patients can view/understand current health status • Compare with others • View trends over time and observe correlations • Communicate better with their HCPs • Predict future outcomes based on today’s behaviors

30. Enhancing Patient Governance and Engagement • Stay tuned

31. High level project goals • Integrate communities • Expand capacity • Grow membership • 6 Key strategies • Become part the U.S. National Patient Centered Research Infrastructure

32. Membership growth • Make membership irresistible! • Engage physicians and other HCPs • CCFA Clinical Research Alliance as a place to start • Partnering with other IBD cohorts • Collaboration with PCORnet • Stay tuned • Search Engine Optimization • Qualitative research to learn more about potential barriers/solutions

33. High level project goals • Integrate communities • Expand capacity • Grow membership • Become part the U.S. National Patient Centered Research Infrastructure • PCORnet • 17 other PPRNs • 11 CDRNs

34. PCORnet

35. The most disruptive change of all • Has nothing to do with technology!

36. The most disruptive change of all • Has nothing to do with technology! • And everything to do with patients!

37. Its not clinical research anymore! • In clinical research, patients are subjects in studies planned by clinicians/scientists, intended to answer questions of paramount importance to the clinical or research community • How the CCFA, NIH, have traditionally operated • How myself, and others in this room have previously conceptualized research • Involves patients, may even involve patient reported outcomes. . . • “Patient scented” versus “Patient Centered”

38. Patient-Centered Outcomes Research • Helps people and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options. This research answers patient-centered questions such as: • “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?”

39. Patient Powered Research Networks “Comprised of patients and/or caregivers who are motivated to build an ideal network and play an active role in patient-centered comparative effectiveness research” “Control of the research process is held by the patients. The active involvement of patients . . . ensures a central role for patient participation in governing the network and its uses, identifying and selecting research questions to be studied, identifying and recruiting patients to participate in the research, and in conducting the research itself”

40. 3 Levels of Patient Governance • Scientific leadership • Patient leadership • Community responsiveness

41. Scientific Leadership • Dr. Balfour Sartor (Network PI) • Adult gastroenterologist and microbiome expert • Chief Medical Advisor to the CCFA • Diagnosed with Crohn’s disease at age 21 (~40 years ago) • Sean Ahrens • Founder of Crohnology • Computer programmer • Diagnosed with Crohn’s disease during childhood • Both will serve on the network executive committee • Dual perspectives critical to informing policies related to data sharing and return of research findings • Should patients be informed of genetic results? • Might there be unintended consequences?

42. Patient Governance Committee (PGC) • Key responsibilities: • 1) Define network’s mission, • 2) Assist in prioritizing patient-driven research agenda • 3) Assist in the review of proposed network studies/partners • 4) Develop policies regarding use and sharing of data • 5) Assist in developing plans to facilitate communications among network participants • 6) Receive and act upon input from broader network community • PGC will report directly to the Executive committee • 2 members of the PGC will hold seats on the Executive Committee (total of 4 patients) • PGC must independently approve all substantial changes in the direction of the network

43. PGC Member Selection and Training • 5 lay patients • 2 with prior experience serving on CCFA grant review committees • Former Chair of the CCFA National Council of College Leaders program • 2 selected by popular election by Crohnology community • Information about the PPRN posted on Crohnology • Self-nominations were invited. • Candidates posted description of interests and qualifications • On-line election • All will complete CCFA stakeholder training course

47. Crowdsourcing to broaden responsiveness • Draft policies to be posted online • All network members will be able to comment, suggest changes, or contribute more substantially through the use of “wiki-style” editing. • The PGC will incorporate member feedback into the final version of all policies.

48. Communication and dissemination of research findings • Network updates will be posted on discussion pages (also distributed by emails) • Member comments and “hearts” will be periodically reviewed by the PGC and the network’s scientific leadership • Research findings will be communicated using lay summaries and infographics

49. Infographics

50. Infographics • In 4 weeks: • 528 shares/views • 116 likes • 67 comments • 92% indicated that the infographic “effectively summarized the research findings”