As the role of biosensors, wearables and mobile health in modern healthcare evolves, the potential of digital biomarkers to continually monitor patient health, rapidly diagnose disease, and accurately predict outcomes becomes increasingly apparent. Physiological data may now be collected via digital devices such as portables, wearables, and implantables. Mobile health, or “mHealth,” promises to transform not only the future of healthcare but also the process of clinical trials. Cambridge Healthtech Institute’s Inaugural Digital Biomarkers: Biosensors, Wearables, and mHealth will focus on the clinical utility of cutting-edge biosensor technology and its impact on drug development.

Final Agenda

Sunday, June 10

4:30-6:30 pm Short Course and Conference Registration

5:00-8:30 Dinner Short Course*

SC1: Fit-for-Purpose Biomarker Assay Development and Validation

*Separate registration required

Monday, June 11

7:00 am Conference Registration and Morning Coffee

8:00 Organizer’s Welcome

OPENING PLENARY SESSION: EMERGING APPROACHES FOR CANCER

8:05 Chairperson’s Opening Remarks

George A. Green, IV, PhD, Head, Pharmacodiagnostics, Bristol-Myers Squibb

8:10 Clinical Genomic Profiling Using the MSK-IMPACT™ Large Panel NGS Assay to Guide Patient Selection for Targeted and Immune Therapies

Marc Ladanyi, MD, William J. Ruane Chair in Molecular Oncology, Molecular Diagnostics Service and Human Oncology & Pathogenesis Program, Memorial Sloan-Kettering Cancer Center

As the centerpiece of an institutional initiative in clinical cancer genomics, we have implemented large scale genomic profiling for targetable cancer drivers and other cancer-relevant alterations in all patients with advanced solid cancers. Since 2014, over 23,000 patients have been profiled using the MSK-IMPACT™ targeted large panel, capture-based DNAseq assay. MSK-IMPACT™, which received FDA clearance in 2017, allows robust detection of somatic mutations in all known cancer genes, copy number changes and select cancer fusion gene rearrangements, as well as assessing overall tumor mutation burden and microsatellite instability. Patients are also screened for oncogenic fusions by targeted RNAseq and for germline cancer predisposition alleles and evidence of clonal hematopoiesis.

8:40 Widgets to Cancer Patient-Specific Digits: The Case for Out-of-Clinic Objective Measures and Their Potential Impact to Remote Patient Monitoring in Precision Oncology and Discovery

Christopher M. Hartshorn, PhD, Program Director, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health

Albeit the case for long-term, out-of-clinic monitoring has been obvious for many chronic diseases, the case for cancer has not been as clear. The National Cancer Institute has begun piloting and funding various aspects to enable an Internet of Cancer Medical Things. This talk will focus on these efforts currently and prospectively as well as the overall vision to coordinate a much broader initiative to improve our understanding of cancer progression and improve the delivery of cancer care.

9:10 Coffee Break in the Exhibit Hall with Poster Viewing

CHOOSING DIGITAL ENDPOINTS IN CLINICAL TRIALS

9:55 Chairperson’s Remarks

Peter Bergethon, MD, Vice President, Quantitative Medicine and Clinical Technologies, Biogen

10:00 The Path toward Meaningful Novel Digital Endpoints in Clinical Trials

Daniel Karlin, MD, MS, Head, Clinical, Informatics & Regulatory Strategy, Digital Medicine and the Pfizer Innovation Research Lab

Dan will walk through how the Digital Medicine group at Pfizer and a number of consortia have been using theory and experience in the digital biomarker space to develop the science of novel digital endpoints and think about clinical meaningfulness of these measures. He will consider the variety of uses of digital endpoints and the ways in which these measures can inform the drug development paradigm from internal decision making through regulatory and market acceptance.

10:30 Digital Biomarkers from Parkinson’s Disease to Melanoma

Dan E. Webster, PhD, Principal Scientist of Digital Health, Sage Bionetworks
Smartphones contain sensors that can monitor disease symptoms, drug response, and potentially predict health outcomes. The mPower Parkinson’s Disease Study uses phone-based measurements to assess tremor, gait, phonation, and more to monitor participant symptoms. To find digital biomarkers predictive of disease from this data, teams competed in the Parkinson’s Disease Digital Biomarker DREAM Challenge. The Mole Mapper app uses a smartphone camera to map and quantitatively measure skin lesions over time to detect markers of progression to melanoma.

11:30 StepWatch™ Accuracy and Reliability Means Greater Probability to Detect Changes in Walking

Teri Chou, PhD, CEO, Modus Health LLC

The accuracy of walking monitors vary widely from consumer products such as Fitbit™ to medical devices such as StepWatch™. This presentation uses independent published studies about the most used walking monitors to emphasize how monitor accuracy and reliability can affect number of study participants needed to detect walking improvements.

12:00 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

BIOSENSORS AND WEARABLES AS BIOMARKERS IN CLINICAL DEVELOPMENT

1:25 Chairperson’s Remarks

Mark Frasier, PhD, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research

1:30 Patient Centered Doesn’t Have to Mean Subjective: The Role of Digital Sensors in Refocusing Clinical Development on the Patient

Daniel Grant, PhD, Director, Early Development Lead, Novartis

Traditionally, the assessment of the impact of treatments on patients outside of the clinic has been dependent on questionnaires. As standards for data quality have become higher, clinical studies are becoming progressively more focused on hard clinically measured endpoints. Alternatively, digital sensors in wearable devices and smart phones present the opportunity for the collection of objective data directly from patients in the real world.

2:00 Wearable Devices in Clinical Trials: Methodological Approaches and a Real-Life Experience

Elena Izmailova, PhD, Senior Director, Novel Data Streams and Devices, Data Science Institute, Takeda Pharmaceuticals International, Inc.

Development of wearable digital technologies in the form of sensors and cell phone apps has opened unprecedented opportunities for health data collection and remote medical care. Multiple applications for wearable technologies have been identified for patient care management and drug development. These applications range from basic physiological data collection to discovery of novel endpoints specific for certain disease areas. This presentation will highlight the methodological approaches and the experience in wearable data collection, analysis and interpretation in clinical trials.

2:30 When You Hear Digital, Think Dynamics: Time and Trajectories in Quantitative Medicine

Peter Bergethon, MD, Vice President, Quantitative Medicine and Clinical Technologies, Biogen

Medicine is systems science in which the patient is a system and their health state is characterized by measurable properties. The change in state over time (dynamics) allows characterization of a sequence of states as a trajectory through growth development, health and disease phases of life. Dynamic transitions define the trajectory from health to illness and with therapeutic intervention to recovery. Capturing time dependence is the power of digital technology.

3:00 Digital Biomarkers in Drug Development – Exploiting the Digital Armamentarium to Fight Heart Failure

Kinjal R. Patel, MHA, MBA, Senior Study Manager, Clinical Sciences, Bayer US

Worldwide, approximately 26 million people suffer from heart failure (HF). The disease has a high impact on patients’ quality of life and life expectancy with an annual mortality of approximately 30%. The use of emerging technologies, which allow remote and continuous patient monitoring will lead to a paradigm shift in the conduct of clinical trials in HF. Opportunities and challenges in this field will be discussed in the presentation.

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing

4:15 A Roadmap to Digital Endpoints for Parkinson’s Disease Using Sensors and Wearable Devices

Mark Frasier, PhD, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research

Parkinson’s disease is a complex heterogeneous neurodegenerative condition characterized by motor and non-motor symptoms affecting millions worldwide. Disease symptoms include tremor, bradykinesia, dyskinesia, sleep disturbance and cognitive impairment. Finding sensitive and objective metrics to track disease progression is difficult. Wearable devices and sensors open the possibility of objectively measuring disease progression. The talk will present a possible roadmap to finding these metrics.

4:45 pm Designing Wearables for Clinical Trials

Tushar Parlikar, PhD, Product Manager, Verily Life Sciences

In this talk, Tushar Parlikar will discuss Verily Life Sciences' investigational device, Study Watch, and its use in clinical trials such as Verily's Project Baseline.

5:15 Human Movement Analytics for Parkinson’s Disease

Erhan Bilal, PhD, Researcher, IBM T.J. Watson Research/Computational Biology Center

Current standards for evaluating the motor symptoms of Parkinson’s patients are based on episodic assessments, such as UPDRS part III, performed by trained physicians in the clinic. Lately, there has been an effort in the field to develop continuous, objective measures of motor symptoms based on wearable sensors and other remote monitoring devices. This talk will focus on current approaches and the progress made towards this goal.

5:45 Welcome Reception in the Exhibit Hall with Poster Viewing

Tuesday, June 12

7:25 am Interactive Breakout Discussion Groups with Continental Breakfast

MOBILE HEALTH IN CLINICAL TRIALS

8:25 Chairperson’s Remarks

Kinjal R. Patel, MHA, MBA, Senior Study Manager, Clinical Sciences, Bayer US

8:30 Regulatory Aspects for Mobile Medical App Development for Commercial and Clinical Trial Use Cases

Michael J. Benecky, PhD, Senior Director, Global Regulatory Affairs, Precision and Digital Medicine, R&D Chief Regulatory Office, GlaxoSmithKline

The presentation will discuss the regulatory aspects for two specific GSK mobile apps: MyAsthma and PARADE. MyAsthma is designed to help people living with asthma manage their condition. The GSK PARADE Study (Patient Rheumatoid Arthritis Data from Real World Study) is the first industry study to utilize the Apple Research Kit. Topics of discussion will include: 1) mobile medical apps are defined as medical devices from its intended use shown through labeling claims, advertising materials, or oral or written statements; 2) mobile medical app regulation is health risk based to balance patient safety and barriers to technological innovation; 3) quality management for mobile medical apps.

9:00 Lessons Learned from Conducting a Non-Interventional Virtual Trial in Migraine Patients

Gabriel Vargas, MD, PhD, Executive Medical Director, Digital Health & Neuroscience Therapeutic Area Head, Early Development, Amgen

The increasing costs of modern drug development have stimulated industry to identify novel approaches in the conduct of clinical trials. One area of great interest is the incorporation of digital health technologies into our clinical trials to enable faster recruitment, provide larger and richer data sets and promote more patient centric trials. This talk will discuss some examples of how Amgen has incorporated digital health into our trials and will describe how taking advantage of the tremendous progress made in consumer electronics over the last several years we have designed a “virtual” trial to understand the relationship between migraines and activity level as measured by an Apple Watch in a migraine patient population using a trial design which has no actual physical sites or study visits.

9:30 Impact of a Smartphone Application on Pain Severity in Patients with Cancer-Related Pain

Amanda J. Centi, PhD, Research Program Manager, Connect Health Innovations, Partners HealthCare

10:00 Internet of Medical Things: Making Intelligent Care Everywhere a Reality

James R. Mault, MD, FACS, Senior Vice President and CMO, Qualcomm Life

The Internet of Medical Things (IoMT) is a transformational era for health care that will shape technology, business culture and the practice of medicine. As this interoperable health care network develops and matures, there will be a number of defining inflection points that will shape the IoMT fabric. Dr. Mault will be sharing benchmarks and early examples covering: 1) parallels and pitfalls from the scaling of the Internet of Things that can inform the IoMT; 2) the investment and return for consumers, manufacturers and health systems; and 3) the stages and advancements, both structured and organic, that will accelerate a viable IoMT fabric.

10:30 Coffee Break in the Exhibit Hall with Poster Viewing

CLINICAL APPLICATIONS OF SMARTPHONES: NEW SOURCE OF DIGITAL BIOMARKERS

11:10 Chairperson’s Remarks

Christian Gossens, PhD, MBA, Global Head, Early Development Workflows, pRED Informatics, Roche Pharmaceutical Research and Early Development

11:15 Digital Biomarkers Collected with Smartphones in Clinical Trials: How Relevant Are They?

Christian Gossens, PhD, MBA, Global Head, Early Development Workflows, pRED Informatics, Roche Pharmaceutical Research and Early Development

Mobile sensors are rapidly becoming part of everybody’s lives, and enhancing clinical trials with real world data is now increasingly possible. This allows for more objective, precise and continuous measurements. Roche has been pioneering a smartphone-based monitoring system since 2014. We share our first real-world digital biomarker results based on active tests and passive monitoring data from several neuroscience clinical trials.

11:45 The Asthma Mobile Health Study

Yu-Feng Yvonne Chan, MD, PhD, Associate Professor, Genetics and Genomics Sciences & Emergency Medicine; Director, Center for Digital Health, Icahn Institute for Genomics and Multiscale Biology, Icahn School of Medicine at Mount Sinai

The Mount Sinai Center for Digital Health team led the pioneering application of Apple’s ResearchKit framework to enable a large-scale clinical research study of asthma, with over 10,000 research participants. The study helped demonstrate the value and utility of consumer-engagement in research, enabled by personal smartphones, with longitudinal, multi-dimensional data collected and analyzed from participants from 3 countries.

12:15 Pixels as Digital Biomarkers: Machine Learning and Beyond with MoleMapper

Tracy Petrie, PhD, MoleMapper Product Manager, Oregon Health & Science University

Smartphones are facilitating the Machine Learning revolution both as data collection devices and inference engines. In recent years, we’ve moved away from telling algorithms what to look for and are now training them to find the relevant and interesting features. Recent successes in classification efforts have fueled the work on Melanoma detection and triage algorithms but the next wave is arriving: supplementing single images with the appropriate context. The primary goal of this effort is to enable early detection of melanomas resulting in decreased mortality rates. With the MoleMapper project, which uses Apple’s ResearchKit platform, we’re building a dataset that includes context while asking, “Is there more we can learn from the data than just the ability to classify lesions?”

12:45 Close of Conference

1:15 Executive ThinkTank*

SC2: Digital Biomarker Implementation Strategies

Discussion Leader:

Daniel Karlin, MD, Head, Experimental Medicine, Informatics & Regulatory Strategy, Pfizer

Panelists:

Michael J. Benecky, PhD, Senior Director, Global Regulatory Affairs, Precision and Digital Medicine, R&D Chief Regulatory Office, GlaxoSmithKline

Peter Bergethon, MD, Vice President, Quantitative Medicine and Clinical Technologies, Biogen

Yu-Feng Yvonne Chan, MD, PhD, Associate Professor, Genetics and Genomics Sciences & Emerging Medicine; Director, Center for Digital Health, Icahn Institute for Genomics and Multiscale Biology, Icahn School of Medicine at Mount Sinai

Christian Gossens, PhD, MBA, Global Head, Early Development Workflows, pRED Informatics, Roche Pharmaceutical Research and Early Development

Daniel Grant, PhD, Director, Early Development Lead, Novartis

Daniel Karlin, MD, Head, Experimental Medicine, Informatics & Regulatory Strategy, Pfizer

Gabriel Vargas, MD, PhD, Executive Medical Director, Digital Health & Neuroscience Therapeutic Area Head, Early Development, Amgen

1:15-1:45 Introductions and Lunch Provided

1:45 Roundtable: Choosing Digital Endpoints in Clinical Trials

• How are digital biomarkers different from traditional biomarkers?
• What clinical evidence is required for using digital biomarkers as clinical outcome measure in clinical trials?
• What is the value proposition for digital endpoints?
• How to progress digital endpoints beyond the exploratory phase
• How to deal with clinical meaningfulness and regulatory acceptability of digital biomarker data
• How to choose and validate a digital biomarker for a new application when there is no current biomarker for reference
• Where to prioritize investment for optimum clinical trial process
• What are the emerging applications for digital biomarkers?

3:00 Networking Refreshment Break

3:45 Roundtable: Technology Advances in Biosensors, Wearables and mHealth for Clinical Applications

• How to validate digital biomarkers
• What would be sufficient qualification for regulatory acceptance?
• What evidence is needed for market access and payer acceptance?
• What are the challenges and opportunities for using wearables, biosensors and smartphones in clinical development?
• Can technology and analytics keep up with endpoint development?
• What data sharing and data standards are needed to enable progression?
• Is there a consortium validation model for digital biomarkers?
• Where will the technology and implementation stand in 5 years?

5:00 Close of ThinkTank

*Separate registration required