A Family-Centered Approach to Measuring Communication Ability within Clinical Trials
Dr. Christie Zigler from Duke University School of Medicine presented on the Observer Reported Communication Ability Measure (ORCA) at the 2022 FAST Science Summit. The ORCA measure is a survey developed in collaboration with the Angelman Syndrome Research Community to measure communication ability in a meaningful and sensitive way. The measure has undergone qualitative interviews, pilot testing, and psychometric evaluation, resulting in a measure that is well-aligned with the experiences of parents and has documented support for use in clinical trials. The ORCA measure has shown to have a wide impact, with over 300 families providing input, availability in 16 languages, and licenses for use by 38 organizations. It is being used with over 20 different neurodevelopmental disorders and diagnoses. The ongoing work includes exploring how the ORCA complements information from clinicians, measuring responsiveness to changes in communication, and interpreting score changes within clinical trials. The support of FAST and the Angelman Syndrome Research Community has been instrumental in the development and success of the ORCA measure.
In this talk, Dr. Christie Zigler from Duke University School of Medicine discusses the development and impact of the Observer Reported Communication Ability Measure (ORCA). The ORCA measure is a survey that was developed in collaboration with the Angelman Syndrome Research Community to measure communication ability in a meaningful and sensitive way. Dr. Zigler highlights the role of FAST (Foundation for Angelman Syndrome Therapeutics) and ABOM (Angelman Biomarkers and Outcome Measures) in the development of the ORCA measure and discusses the current and future work being done to further validate and utilize the measure.
The Road to the ORCA Measure
The development of the ORCA measure began with the recognition by FAST and the Angelman Syndrome Research Community of the need for a measure that could effectively evaluate communication ability in clinical trials. The Center for Health Measurement at Duke University was approached to collaborate on the development of the measure. The first step was to assess existing measures and identify their strengths and limitations. It was determined that a new measure specific to Angelman syndrome, centered around the experiences of parents, was needed.
Qualitative interviews were conducted with 22 families and six clinical experts to gather rich and detailed information about communication experiences and strengths. This information was used to develop survey questions for the ORCA measure. The survey was then pilot tested with 12 families, and modifications were made based on their feedback. A psychometric evaluation was conducted using an electronic platform, with 249 families providing complete data for analysis. The result of this process was the development of the ORCA measure and documented support for its use in clinical trials.
Addressing Limitations: Avoiding Floor Effects
One of the limitations the development team aimed to address was the presence of floor effects, which can make it difficult to compare between children and detect changes in communication ability. Dr. Zigler presents data from a study showing a floor effect in scores on the Vineland Adaptive Behavior Scale. In contrast, the ORCA measure demonstrated a bell-shaped distribution of scores, indicating a lack of floor effects. This suggests that the ORCA measure is well-aligned with the behaviors observed in children with Angelman syndrome and allows for the detection of change across the score range.
Current and Future Work
The development team is continuously working to further validate and utilize the ORCA measure. Collaborations with FAST and ABOM are ongoing to answer important questions about the measure’s complementarity to clinician assessments, its responsiveness to meaningful changes in communication, and how score changes should be interpreted within a clinical trial. Preliminary data for question one has been presented, and plans are in place to access data for questions two and three in the coming months and year.
Impact of Support
Dr. Zigler expresses gratitude for the support of FAST, ABOM, and the Angelman Syndrome Research Community, highlighting the impact their support has had on the ORCA project and the larger ORCA initiative. Through the support of FAST, collaborations with Boston Children’s Hospital and the Angelman Syndrome Natural History Study have been established to further research and answer important questions related to the ORCA measure. Additionally, the work done in the Angelman Syndrome community has led to similar projects in other rare and ultra-rare neurodevelopmental disorders. The ultimate goal is for the ORCA measure to be deemed fit for purpose as an outcome measure in clinical trials.
ORCA by the Numbers
Dr. Zigler shares some statistics that demonstrate the impact of the ORCA measure. Approximately 300 families from the Angelman Syndrome community provided input in the development of the measure. The ORCA measure is currently available in 16 languages, and qualitative interviews have been conducted with international families. There have been 38 executed licenses for the ORCA measure, indicating its use by various organizations, academic institutions, industry partners, nonprofits, and clinicians. Over 32,000 administrations of the ORCA measure have been conducted or planned, with almost 4,000 of those being with children with Angelman syndrome. The ORCA measure is also being used with over 20 different neurodevelopmental disorders and diagnoses, with efforts to support its use in other populations as well.
Dr. Zigler concludes by expressing gratitude to her team, collaborators, funders, partners, and the Angelman Syndrome Research Community for their support and involvement in the ORCA project. She emphasizes the significant impact that their support has had on the development and utilization of the ORCA measure, and the potential it holds for improving communication assessment in clinical trials.
- Title: A Family-Centered Approach to Measuring Communication Ability within Clinical Trials
- Author(s): Christy Zigler
- Author(s)’ affiliation: Duke University School of Medicine
- Publication date: 2022-12-03
- Collection: 2022 FAST Science Summit