Agendas

Session 1: Existing Digital Endpoint Validation Efforts for Measuring Motor Functions

Moderators: Indu Navar, MS and Emily Carifi, PhD

Opening comments: Indu Navar (5 min)

Speakers (15-20 min talks):

• Paul Strijbos, PhD – Stride Velocity at the 95th Centile (SV95C): the first regulatory-qualified real-world digital outcome measure for use as primary endpoint in pivotal studies of Duchenne Muscular Dystrophy
  
• Jamie Adams, MD – Digital Measures in Early Parkinson’s Disease: the WATCH-PD Study
• Gul Erdemli, PhD – MOBILISE-D Towards Qualification of Digital Mobility Outcomes

Panel discussion and Q&A with speakers (30-40 min): lessons and best practices from digital endpoint validation efforts.

Session 2: Established and Emerging Technologies for Measuring Motor Functions

Moderators: Mindy Leffler, MEd and Glen Nuckolls, PhD

Opening comments: Mindy Leffler (5 min)

Speakers (15 min talks):

• William Marras, PhD – The Spine Phenome Project: Enabling Technology for Personalized Medicine
• Fay Horak, PhD, PT – Development of Wearable Technology for Neurological Clinical Trials
• Ashkan Vaziri, PhD – Wearable Sensors and Digital Health Technologies for Tracking Disease Progression in Neurological and Neuromuscular Disorders

Panel discussion & Q&A with speakers (30 min): lessons and best practices in advancing digital wearable technologies.

Emerging and established technologies use cases: lightning talks (5-7 min) from junior investigators/trainees and small businesses.

Moderator: Glen Nuckolls, PhD

• Melissa McIntyre, PT, DPT – Beyond the Clinic: Multiday Analysis of Leg Movement Quantity and Kinematic Characteristics in Infants with SMA
• Tina Duong, PT, PhD – Video Kinematic Analysis of Movement: Complimentary Clinical Measures
• Margaux Poleur, MD – Digital Outcome Measure for Longitudinal Follow-up of Patients with Amyotrophic Lateral Sclerosis
• Damiano Zanotto, PhD + Jacqueline Montes, PT, EdD, NCS – AI-Enabled Insoles: A New Tool for Assessing NMD Walking Function in Controlled & Natural Environments
• Henry Kaminski, MD – Quantitative, Objective Neuromuscular Examination Obtained by Telemedicine
• Dustin Heldman, PhD – Clinical Applications of Wearable Sensors in Movement Disorders

Session 3: Panel discussion:  Gaps and opportunities for assessing motor function in neurological and neuromuscular disorders.

Moderators: Toyin Ajisafe, PhD and Fay Horak, PhD, PT

Panelists:

• Linda Pax Lowes, PT, PhD
• Steven Cramer, MD
• R. James Cotton, MD, PhD
• Adeline Vanderver, MD

Session 4: Regulatory overviews and reflection panel

Moderators: Ted Zheng, MD, PhD and Carol Taylor-Burds, PhD

Speakers (15 min talks):

• Matthew Diamond, MD, PhD – Overview of digital health technologies: understanding requirements and guidelines for marketing authorization of a device vs. use in a clinical trial.
• Michelle Campbell, PhD – Engaging with CDER for drug development approvals.

4:45 – Panel discussion and Q&A (40 min)

• Susan Miller, MD, Centers for Medicare & Medicaid Services
• Matthew Diamond, MD, PhD, FDA/DHCE
• Michelle Campbell, PhD, FDA/CDER
• Michael McKnight, PhD, FDA/CDRH

Session 1:  Existing Digital Endpoint Validation Efforts for Measuring Sleep and Pain  

Moderator:  Brooks Gross, PhD

Speaker:

• Walter Maetzler, MD – IDEA-FAST: Lessons learned from a large EU project on the validation of digital endpoints for fatigue and quality of sleep

Q&A (30 min):  lessons and best practices in advancing digital endpoints.

Session 2: Established and Emerging Technologies for Measuring Sleep, Pain, and Autonomic Function

Moderators: Ted Zheng, MD, PhD and Adissa Silue, PhD

Speakers (20 min talks):

• Anton Bowden, PhD – Segmental spine biomechanics-based phenotypes of chronic low back pain based on inexpensive wearable strain sensors
• Justin Brooks, MD, PhD – Leg Movements and Cortical Arousals in Sleep: Biomarkers for Diagnosis, Treatment, and Research
• Jeffrey Durmer, MD, PhD – Quantifying Autonomic Health During Sleep

Panel discussion & Q&A with speakers (30 min): lessons and best practices in advancing digital wearable technologies.

Session 3: Panel Discussion: Gaps and opportunities for assessing autonomic function, sleep and pain in neurological and neuromuscular disorders.

Moderators: Carol Taylor-Burds, PhD and Janet He, PhD

Panelists:

• Ammar Al-Chalabi, MB, ChB, PhD
• Christina L. Master, MD, FAAP, CAQSM, FACSM, FAMSSM
• Azizi Seixas, PhD
• Yo-El Ju, MD, MSCI

Session 4:  Breakout Session:  Open Discussion with Audience

Breakouts (1 hour)

• Breakout 1: Measuring and Monitoring Pain
  • Moderators:  Ram Arudchandran, PhD and Adissa Silue, PhD
• Breakout 2: Measuring and Monitoring Sleep
  • Moderators:  Marishka Brown, PhD and Brooks Gross, PhD

Session 1: Existing Digital Endpoint Validation Efforts for Measuring Cognitive Function

Moderators: Chris Weber, PhD and Yuan Luo, PhD

Speakers:

• Jason Hassenstab, PhD – What can be learned from high-frequency cognitive assessments?
• Ioannis Tarnanas, PhD and Anna-Katharine Brem, PD, PhD, MASNP – Digitally-enhanced precision medicine: accurate selection of Preclinical and Prodromal AD individuals and measuring progression of MCI.
• Supriya Bhavnani, PhD – Harnessing digital technology to assess cognition in early childhood in India – evaluating the DEEP tool.

Panel discussion & Q&A with speakers (30 min): lessons and best practices in advancing digital endpoints.

Session 2:  Established and emerging technologies for assessing cognitive function.

Moderators: Andrew Potter, PhD, Rebecca Hommer, MD

Speakers (15 min talks):

• Olu Ajilore, MD, PhD – Passive assessment of cognitive constructs via smartphone interactions
• Adam Vogel, PhD – Speech and language biometrics for measuring communication, cognition and motor performance
• Francesca Cormack, PhD

Panel discussion & Q&A with speakers (30 min): lessons and best practices in advancing digital wearable technologies.

Emerging and established technologies use cases: lightning talks (5-7 min) from junior investigators/trainees and small businesses.

Moderator: Becky Berman, PhD

• Alena Stasenko, PhD – Multidimensional Assessment of Cognition via Smartphones & Wearables
• Kelly Donegan – Cannon Blast: a Smartphone Measure of Model-Based Planning
• Andrew Byun – Understanding Human Behavior Through The Lens of Motor Learning, Digital Phenotyping, and More
• Sunghye Cho, PhD – Natural Speech as a Screening and Monitoring Tool for Alzheimer’s Disease and Related Dementias (ADRD)
• Emily Paolillo, PhD – Smartphone Digital Endpoints in Frontotemporal Lobar Degeneration (FTLD)

Session 3: Panel Discussion: Gaps and opportunities for assessing cognitive function in neurological and neuromuscular disorders.

Moderator: Kristi Hardy, PhD and Carol Taylor-Burds, PhD

Panelists:

• Rhoda Au, PhD
• Richard Gershon, PhD
• Rosie Curiel Cid, PsyD

Session 4: Perspectives on Advancing Digital Endpoint Development Through Public Private Partnerships

Moderator: Amir Tamiz, PhD – Director, Division of Translational Research, NINDS

• Diane Stephenson, PhD – Critical Path Institute
• Abhishek Pratap, MS, PhD – Boehringer Ingelheim
• Penny Dacks, PhD – The Association for Frontotemporal Degeneration
• Steve Hoffmann, MS – Foundation for the National Institutes of Health (FNIH)