Examples of Patient-Centric Digital Measures for Rett Syndrome

Examples of Patient-Centric Digital Measures for Rett Syndrome

On June 7th, 2023, Robert Wright, PhD, Senior Research Scientist at VivoSense, and Jana Von Hehn, PhD, CSO at Rett Syndrome Research Trust, presented at the Digital Health Innovation Summit, a forum to learn from the best about top trends in the industry. Their presentation, Developing Patient-Centric Real-World Digital Clinical Measures for Rett Syndrome, summarized years of collaborative work implementing digital health technologies in complex research programs.

Robert and Jana discussed the process from establishing meaningfulness to interpreting the results of studies using digital health technologies to discover valid endpoints.

Here's a quick snapshot of their presentation and a link to download the slides.

Rett Syndrome Overview

Rare neurodevelopmental disorder

  • Occurs in 1:10,000 female births
  • Normal early development followed by developmental delay & regression of acquired skills

Diverse life-long symptoms

  • Autonomic dysfunction, sleep disturbances, GI dysmotility, nutrition issues, scoliosis, contractures, seizures, movement disorders, limited communication, repetitive hand movements, and others

Assessment of Efficacy on Rett Symptoms

The current assessment options are incomplete:

  • Observer and clinician reported questionnaires are subjective and indirect
  • Provides a static snapshot in time or summary based on memory

Biosensors and digital health technologies provide an opportunity to improve measurement and expedite drug development:

  • Biosensors and digital health technologies are objective and direct measures
  • Provides continuous data collection, even at home

Developing Patient-Centric Digital Measures that Matter

The patient’s voice is vital for selecting measures that matter. Meaningfulness was established in two ways:

  1. Qualitative, semi-structured in-depth interviews with caregivers of individuals living with Rett syndrome
  2. The Voice of the Patient Report, Rett Syndrome Externally-Led Patient-Focused Drug Development Meeting on March 11th, 2022, explored meaningful aspects of health as defined by caregivers of individuals with Rett syndrome.

In Rett syndrome, breathing irregularities are an example of patient-centric autonomic dysfunctions amenable to biosensor collection.

  • Meaningful Aspects of Health: Ability to breathe normally
  • Concept of Interest: Hyperventilation, daytime Apnea, hypopnea
  • The outcomes to be measured: Number and duration of events per hour
  • Endpoint: Study-specific, precisely defined, statistically analyzed variable

View the presentation slides for examples of collecting and analyzing patient-centric digital measures using wearable sensors to discover novel digital endpoints and biomarkers.

View Slides

Robert Wright, PhD

Robert Wright, PhD

Skilled researcher with more than 10 years of experience in clinical health care. Adept at designing and conducting rigorous medical and behavioral research studies, advanced quantitative and qualitative methods, building partnerships, and working on innovative cross-functional teams that are focused on improving patient outcomes.

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