The Changing Face of Clinical Trials - Patient-Centered Research in the Internet Age

1. The Changing Face of Clinical Trials - Patient-Centered Research in the Internet Age Cal Collins Co-founder & CEO OpenClinica, LLC June 21, 2013

2. What is “Patient-Centered Research”? Part of a “learning healthcare system”? A trendy buzzword? Where the money is? Cheaper and less rigorous than RCTs? A major cultural shift in approaches to research? 2

3. What is “Patient-Centered Research”? It is generally defined as research leading to evidence-based, patient-centered health interventions. Umm, ok. 3

4. Why is Patient-Centered Research Important? It helps people make more informed healthcare decisions by directing research toward questions that are important to patients, measuring outcomes that are noticeable and meaningful to them, and producing results that help them weigh the value of healthcare. Due to a focus on 'real world' factors, it tends to be prominent in certain research designs: • Observational research • Registries • Pragmatic trials But it has applications to the design and methods of explanatory randomized clinical trials as well. 4

5. Why is Patient-Centered Research Important? Less than one-third of people who come in for a screening end up completing a clinical trial.[1] Employing new ways to engage patients and physicians while increasing their level of knowledge and trust can improve the sponsor’s ability to meet recruitment goals. [1] Getz, Ken, The Gift of Participation: A Guide to Making Informed Decisions About Volunteering for a Clinical Trial, 2007, p40. 5

6. Why do people participate in trials? “Big picture” factors: • Access to needed treatment • Altruism (advancing science, helping those with similar conditions)

7. Why do people participate in trials? “On the ground” factors that influence decisions: • Prior participation • Relationships with researchers • Trusted leaders • Trust in organization • Incentives 7

8. Why DON’T people participate in trials? • Recruitment/Matchmaking insufficient • Not convenient • Privacy concerns • Cultural norms • Confusing consent forms • Lack of knowledge about potential to participate • Control over data sharing/secondary use Source: http://dx.doi.org/10.1016/j.atg.2012.07.001 8

9. What roles can technology play? • Increase convenience (ePRO, web-based access to trial materials, email reminders). • Strengthen connections between participants and researchers. Create infrastructure for coordinated team-based care, home-based continuous monitoring, including wireless data reporting from medical devices. • Enhance communication structures - provide trial-specific information at the points in clinician and participant workflow when they can best make use of it. 9

10. What roles can technology play? • Improve recruitment – social networks, cohort identification through patient record mining • Provide rewards/incentives to participation – gamification, access to health information, risk factors, etc on mobile devices • Better secondary data use/feedback loops • The (still elusive) promise of using the Electronic Health Record system in research to identify participants and capture clean, accurate trial data with less duplication 10

11. What is Open Source? Why does it matter? Open Source Software is software that allows programmers to freely run, read, redistribute, and modify the software’s source code. We need new, innovative approaches to improving health outcomes. We need to be integrated and patient-centric, in care and research. Open Source Software (OSS) can deliver improved integration, quality, and efficiency in patient-centered care and research environments. 11

12. Why Open Source? • When people can rapidly download and try things for free, share ideas with a community, and see each others’ source code, a few surprising things happen: • It’s easier to evaluate… all the way down to the code • Technologies get ‘mashed up’ in highly innovative ways • Best practices emerge and are shared quickly • Interoperability is easier • Business can focus on where they contribute the most value, rather than on the ‘plumbing’ 12

13. Prominent Patient Centered OSS Technologies • OpenDataKit (ODK) and OpenXData • Mobile data capture by field workers and patients. • http://opendatakit.org/ • http://www.openxdata.org/ • SMART • The “App Store” for health care • http://smartplatforms.org/ • tranSMART • Translational research platform (based on i2b2 • http://www.transmartproject.org/ 13

14. Key Goals for Patient Centered Capabilities in OpenClinica Patients require a simple interface that can be used without any training. As such, Participant Portal and mobile device solutions must meet several goals:

15. Approach • Develop stand alone modules that leverage core OpenClinica • Study design is done in one place (OpenClinica) and deployed to all modules, including event/CRF design, skip logic, and study calendar => no separate study build process • Provide a simple, intuitive data entry interface and submit data back to the OpenClinica instance. • Provide participants the ability to complete the right forms at the right time plus provide the management tools needed to manage the patient responses.

16. Participant Portal Overview • Web-based survey and outcomes reporting by clinical study participants. • A standalone application that interfaces with existing OpenClinica capabilities. • Leverages the existing investment in OpenClinica’s study design, data management, and extract capabilities.

17. Participant Portal Overview

18. Participant Portal Overview Portal forms support: • Skip patterns • Real-time data checks • Multiple input types • Repeating Groups* • Simple UI with customizable layout • Design in OpenClinica * - not implemented for proof of concept

19. Tablet-based Data Capture Needs: Tablet-based surveys/instruments for completion by patient Provide immediate feedback on health risks and conditions for clinicians to use in the patient visit 19

20. Tablet-based Data Capture • Uses existing open source components (OpenClinica, ODK), same technology foundation as Participant Portal • Uses OpenClinica CRF definitions and rules, no additional programming/design necessary. • Touch screen tablet, with support for other modes (SMS, IVR) in the future. • Standards-based form definition (CDISC ODM) provided by EDC System, automatically converted to XForm for mobile client device. • Open source technologies ‘mashed up’ in 9 month development period • ODK • Dimagi TouchForms • OpenClinica EDC

21. Vision Nations are creating a “health data infrastructure in which patient data… can be accessed in de-identified form by researchers and public health agencies, and in which a market for applications that enhance EHR usability and patient involvement can flourish” “Sound medicine needs to be based on empirical evidence… Aggregated de­identified information could enable a wide range of studies on such topics as prevention strategies, frequency complications in particular settings, and the individuals response to specific drugs based on genotype” True transformation of healthcare must deliver on potential of research via integration into practice (translational research and precision medicine) PCAST report on Health IT, Dec. 2010:

22. Thank You!Questions? Cal Collins OpenClinica, LLC ccollins@openclinica.com 22