Measuring Communication Ability in Clinical Trials: A Caregiver-Centered Approach for Angelman Syndrome

Quick Overview

Dr. Bryce Reeve from Duke University presented on the Observer-Reported Communication Ability (ORCA) measure, which was developed to assess communication ability in individuals with Angelman Syndrome. The ORCA measure is designed specifically for use in clinical trials and can be completed by caregivers independently. It captures various forms of communication, including gestures, vocalizations, and the use of low-tech and high-tech devices. The measure has been translated into multiple languages and is available for use in research. Ongoing evaluation and feedback are being sought to further enhance the measure. The ORCA measure has been well-received and is being used in various research studies and clinical trials.

Introduction

In this talk, we will discuss the work done by Dr. Bryce Reeve and his research team at Duke University in developing and evaluating a measure of communication ability for individuals living with Angelman Syndrome. The goal of this measure is to be used in upcoming clinical trials.

Background

The need for an effective measure of communication ability for individuals with Angelman Syndrome was identified through a survey conducted by the FAST organization in 2018. Speech and communication were identified as the highest selected outcome that parents wanted to see improvement in if there is an effective treatment for their child.

Existing measures for assessing communication ability in individuals with Angelman Syndrome had several limitations. Many of these measures required a speech and language pathologist or a trained administrator to assess the child’s communication ability, which may not accurately reflect their abilities in a natural setting. Additionally, these measures were not specifically designed for use in clinical trials and did not capture the unique ways in which children with Angelman Syndrome communicate, such as through gestures, signs, or the use of devices.

The Observer-Reported Communication Ability (ORCA) Measure

To address these limitations, Dr. Reeve and his team developed the Observer-Reported Communication Ability (ORCA) measure. The ORCA measure was designed specifically for use in clinical trials and can be completed by parents or caregivers independently, without the need for an administrator. It captures different ways in which children with Angelman Syndrome communicate, including gestures, vocalizations, and the use of low-tech and high-tech devices.

The ORCA measure includes 23 different communication concepts, categorized into expressive, receptive, and pragmatic communication. These concepts were identified through discussions with parents and caregivers of children with Angelman Syndrome. The measure was constructed using a hierarchy of ability levels, with questions tailored to each level of ability.

The ORCA Metric

The ORCA measure uses an interval-based scale to assess communication ability. High scores represent higher levels of communication, while low scores represent lower levels of communication. The metric is scaled on a sample of children with Angelman Syndrome, with a mean score of 50 representing the average communication ability in this population. The ORCA measure has been translated into 16 other languages to facilitate its use in international trials.

Use and Evaluation of the ORCA Measure

The ORCA measure is available for use in research studies through the Duke University School of Medicine and Pattern Health. It has been used in various research studies, including the BOSS National History Study and clinical trials. Ongoing efforts are being made to collect more evidence about the validity and reliability of the ORCA measure, and feedback from parents and caregivers is welcomed to enhance its relevance for the Angelman Syndrome population.

The success of the ORCA measure has led to its expansion to other neurodevelopmental diseases. A grant from the FDA is supporting the adaptation of the measure for use in 12 other neurodevelopmental diseases that also lack quality measures.

Conclusion

The development of the Observer-Reported Communication Ability (ORCA) measure has provided a caregiver-centered approach to assessing communication ability in individuals with Angelman Syndrome. The measure addresses the limitations of existing measures and has been widely adopted in research studies. Ongoing evaluation and feedback from parents and caregivers will continue to enhance the measure’s relevance and effectiveness in clinical trials.