1. Precision Medicine- Optimizing Patient Selection Janet Woodcock- “ How can we apply modern tools? ” Mark Rogge- “We need to understand the patients” Participant-Centered Trials using Pervasive Computing Devices to capture real-time data and reveal the actual features of disease Ways to improve pre-enrollment, assessments, and post approval Stephen H Friend MD PhD Sage Bionetworks Center for Health Policy at BROOKINGS Session III July 28, 2015

2. Precision Medicine has up till now primarily focused on defining subgroups It has not focused on realities of fluctuations Hypothesis: Better tracking of patients at baseline and understanding the fluctuations in daily changes will allow more accurate measures of interventions Especially for changes in their quality of life

3. Phenotypic Data Asymmetry of Data driving Precision Medicine: The public can help with this Genotypic Data

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8. Participant –Centered Research Studies with Feedback Loops Anecdotes into Signals Partners

9. iOS based mHealth Research Kit Framework for Clinical Studies Consents, Surveys, Tasks, Passive Data Open Code hosted on GitHub for re-use Apple does not see any of the data Sage Bionetworks- as a neutral repository

10. Tanner Kruger Bloem Kieburtz mPower Trister Dorsey Klein

11. Parkinson mPower Study App

16. Within first three months of mPower being available 15,439 consented individuals 11,360 enrolled (largest Parkinson Disease trial ever assembled)

20. 62 y old Man 67 y old Woman

21. Real loads and reliefs that exist – that modulate severity of symptoms against which the impact of medications should be assessed

24. Need for better ways to follow Dementia

25. Federated Approaches for Digital Phenotyping Open Data Open Source Code Direct Comparisons Diversity of Cohorts

26. participant centered data from apps benefits to understanding how to enroll from early trials through post-approval benefits to the individual ( Movement Disorders, Rheumatoid Arthritis, Anemia, Melanoma, Cognition, Mood)

28. Opportunities for Pervasive Computing to impact Clinical Trials: A.Pre-Enrollment baselines for patients before they enter trials B. New secondary end-points and ways to distinquish from existing therapies C. Post- Approval Surveillance