Advances towards Remote Assessment of Disease and Relapse in Multiple Sclerosis

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Advances towards Remote Assessment of Disease and Relapse in Multiple Sclerosis Jane Rhodes March 31 st 2016 CAMD Digital Biomarkers Conference Baltimore, MD

Emerging technology for the enhancement of patient monitoring and assessment ipad -based assessment tool Integrated, standardized, quantitative MRI measures for efficient evaluation of MS disease activity and progression Wearable sensors to monitor patient mobility MS Care Today Future MS Care IT platform for integration and visualization of clinical, radiologic, and biomarker data to quantify individual ongoing risk of MS progression Genetic and molecular profiles for characterizing patients, predicting MS prognosis, and monitoring treatment responses Leveraging clinical, radiologic, and molecular measures to inform treatment choices and provide decision support Note: All components are under development and/or being explored.

Challenges in using Digital Biomarkers for Assessing Function in Multiple Sclerosis Functional measures are challenging to collect objectively in MS : Currently accepted primary endpoints in MS clinical trials focus on inflammatory events in relapsing forms of disease and are not effective functional measures of chronic progression. Validated functional outcome measures, most suited to a digital format, are not routinely used to assess MS patients hence there is a lack of evidence to support actionable outcomes from digital data Construct validity and clinical meaningfulness are frequently overlooked in App and wearable development 3

Partnering across Industry and Academia to Develop and Assess Digital Biomarkers in MS 4

Developing tools to help improve outcomes through passive and active data collection Consumer Wearable Devices & Sensor Technology for Passive Data Collection Medical Devices & Apps for Active Data Collection Chronic Disease Management Leverage emerging patient monitoring tools to generate novel data or extend window of observation with the goal of better disease management at a lower time burden and financial cost

Deploying Wearable Sensors to MS patients: The purpose of the study was to understand the deployment and use of consumer activity tracking devices in the MS population Objectives: Assess likelihood of use of devices Assess feasibility of collecting data from patients via devices Additional questions What features do patients want in a patient-centric device/app? Does a wearable device impact patient behavior, patient-provider relationship, disease understanding? How does social and peer-to-peer connectivity affect utilization of wearables by patients? 6

Study engagement rates observed were very high, likely the result of high patient interest and value Note: Invites sent to 1,600 385 137 30 65 Patient demand for study was high, shown by full enrollment time frame of approx. 24 hours 27 248 35 57 Opened invitation Did not reply 14% 213 23% 191 Opted out Screened out* Did not complete 248 (100%) 203 (82%) 191 (77%) Invited Did not participate Participated Did not authorize Authorized Did not complete survey Completed Survey Note: Authorization refers to connecting Fitbit profiles with PLM profiles. Sync refers to uploading data from device to Fitbit.com (so that data can be imported into PLM). *the reasons for the screen out of 10 members could not be identified 7

Clinical Correlation: Participants with lower MSRS-R walking scores also had lower activity Higher walking ability Lower walking ability Note: Data presented is last reported Walking Domain of MSRS; 15 members did not report MSRS Source: PatientsLikeMe profile data for study participants, Fitbit data as recorded on PatientsLikeMe; PatientsLikeMe analysis 8

Clinically meaningful or actionable? This is going to be a big help for me to prove that if I change my ways and take better care of my health my MS will be manageable. Quote from actual patient that participated in the study; stock photo used to protect patient identity

Systems Biology Study of Multiple Sclerosis 10

Molecular and Digital Biomarker studies underway 11

Device signals Aim: Develop a sensitive objective measurement of features of mobility, dexterity, balance and gait to allow sub-stratification of disease phenotypes Method: Digital signals being compared against traditional measures (EDSS) and validated functional outcome measures (MSFC). 12

Developing tools to help improve outcomes through passive and active data collection Consumer Wearable Devices & sensor technology for Passive Data Collection Medical Devices & Apps for Active Data Collection Chronic Disease Management Leverage emerging patient monitoring tools to generate novel data or extend window of observation with the goal of better disease management at a lower time burden and financial cost

to collect, analyze and visualize clinical data Patients can use this ipad-based medical device to Conduct selfadministered neurological tests that quantify walking ability, dexterity, vision, and cognition Developed in Partnership with 14

Clinical data collection device designed to be used in routine practice for comprehensive patient assessment 15

MS Performance Test domains 16

Processing Speed Test based on the symbol digit modalities test Patient will be instructed to match the numbers and symbols according to the key provided. The trial lasts 120 seconds using audio and visual instructions 17

Instructional videos are embedded in the app to facilitate the user experience 18

Data Dashboard for Physician use 19

Applications to help patients living with chronic disease

Mood Energy Activity Sensation

Barriers that impede advancement of digital biomarkers in MS Clinical validity: Correlation with conventional measures Clinical meaningfulness: Actionable or useful output Standardization of Minimal Viable Product attributes for clinical use Sensitivity to change over time Sensitivity/utility in the patient s own environment Differentiation between non-clinical and clinically relevant digital tools Physician acceptance 22

Back up slides

Partnering to assess digital biomarkers in MS Biogen gave out 250 Fitbit bands to MS patients in the U.S. last spring to track their level of activity and sleep patterns. Biogen says collecting data on a daily basis about how much and how fast MS patients walk could yield data about the progression of the disease and lead to better treatments. Bloomberg, Dec 2014 Biogen partners with Altas 5D to evaluate the use of in-home sensor technology to monitor MS patient symptoms. Precise measurement of neurological and neuropsychological impairment and disability in multiple sclerosis is challenging. We report methodologic details on a new test, the Multiple Sclerosis Performance Test (MSPT), a computer-based platform for precise, valid measurement of MS severity. Rudick et al, MSPT: An ipad-based Disability Assessment Tool, June 2014 Google will join Biogen Idec to study environmental and biological contributors to the debilitating disease s progression. Using sensors, software and data analysis tools, the companies will collect and sift through data from people with the disease. The goal is to explain why multiple sclerosis progresses differently from patient to patient. Bloomberg, Jan 2015 IMI2 initiative to develop and test a transformative platform of remote monitoring of disease in CNS disorders including MS 24