Precision Medicine- Optimizing Patient Selection Janet Woodcock- “ How can we apply modern tools? ” Mark Rogge- “We need to understand the patients” Participant-Centered.

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

Precision Medicine has up till nowprimarily 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 interventionsEspecially for changes in their quality of life

Phenotypic Data

Asymmetry of Data driving Precision Medicine: The public can help with this

Genotypic Data

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6

Participant –Centered Research Studies with Feedback Loops

Anecdotes into Signals

Partners Partners

iOS based mHealth Research Kit

Framework for Clinical StudiesConsents, Surveys, Tasks, Passive DataOpen Code hosted on GitHub for re-useApple does not see any of the dataSage Bionetworks- as a neutral repository

Tanner Kruger BloemKieburtz

mPower

Trister

Dorsey

Klein

Parkinson mPower Study App

Parkinson mPower Study App

Parkinson mPower Study App

Parkinson mPower Study App

Within first three months of mPower being available

15,439 consented individuals

11,360 enrolled

(largest Parkinson Disease trial ever assembled)

62 y old Man 67 y old Woman

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

Need for better ways to follow Dementia

Federated Approaches for Digital Phenotyping

Open Data

Open Source Code

Direct Comparisons

Diversity of Cohorts

participant centered data from apps

benefits to understanding how to enrollfrom early trials through post-approval

benefits to the individual

( Movement Disorders, Rheumatoid Arthritis, Anemia, Melanoma, Cognition, Mood)

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