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Understanding Clinical Outcome Assessments (COAs) Used in Clinical Trials

The Observer Reported Communication Ability Measure (ORCA), which is a caregiver reported assessment of expressive, receptive and pragmatic communication is just one type of clinical outcome assessment used in clinical trials for Angelman syndrome.

The ORCA measure was specifically created by FAST with Dr. Bryce Reeve’s team at Duke University for assessment of communication in individuals living with AS. The ORCA focuses on all communication methods that are meaningful to those living with AS, including AAC. The ORCA is translated into many languages. This measure has been so successful in AS that the FDA gave a grant of more than $2.5M to modify it for use in assessing communication for 12 other neurodevelopmental disorders. 

Click here to watch the latest presentation on the ORCA.

What other types of Clinical Outcome Assessments (COAs) are used? At the 2023 Global Science Summit, impressive work was presented by Dr. Anjali Sadhwani from Boston Children’s Hospital focused on clinical outcome assessments used in clinical trials.  Key highlights to her talk included:

  • Dr. Sadhwani first reviewed types of outcomes measures used in clinical trials including:

    • performance based outcomes where a participant is asked to perform a task

    • patient-reported outcomes measures

    • observer-reported outcome measures

    • clinician-reported outcome measures

  • Outcome measures must be valid, reliable, and sensitive to change.

  • Dr. Sadhwani described the Bayley Scale of Infant and Toddler Development (BSID) as one of the common measures used to assess development  in AS. This is an in-person, performance-based assessment looking at development across multiple domains (cognitive, language [receptive/expressive], and motor [fine/gross]). The BSID  has been used across many studies for over 17 years, and has tasks that many individuals with AS can complete at first assessment, while still allowing them to show  progress in development over time.

  • She described the Vineland Adaptive Behavior Scales (VABS) used to assess adaptive functioning in AS. The VABS assesses development across 5 domains 

    • Communication (receptive/expressive)

    • Motor (gross/fine)

    • Socialization (interpersonal, play and coping skills)

    • Daily living skills (personal, domestic, and community)

    • Behavior

New funding from ABOM is supporting work to improve the consistency of administration of the Bayley and Vineland by involving advocacy groups, parent/caretakers, and evaluators to address challenges in executing the measure consistently. They plan to share a standardization manual so that administration of the Bayley becomes more consistent across all clinical trials in AS.

Watch the full presentation: