FAST extends its partnership with Duke to support the ORCA measure
FAST extended its partnership with Duke University School of Medicine to gather and document rigorous evidence to support the validity of the Observer-Reported Communication Ability (ORCA) measure for use in clinical trials for individuals living with Angelman syndrome (AS).
What is the ORCA? The ORCA measure is a tool that was developed through a collaboration with FAST and Duke University. This initiative started based on vital feedback from the FDA to FAST after speaking with caregivers and meeting with patients living with AS. Families were asked “what are the most meaningful and important AS symptoms you would like to see improvement in?” Communication ability was at the very top of this list. The FDA listened and encouraged FAST to work with an expert team to develop an AS-specific tool to assess communication ability that is unique and appropriate to the AS population.
The ORCA measure is intended to evaluate an individual's baseline communication ability level and any changes in communication ability that might occur throughout a research study and to capture this in the most sensitive and specific way. This measure was designed to ensure that all individuals living with AS would be captured on this measure and not hit the historical obstacles of traditional neuropsychological tools, where unique and non-vocal forms of communication abilities are missed. This is a serious issue with traditional endpoints in AS and many other neurodevelopmental disorders (NDDs). The ORCA consists of 84 questions with 70 behavioral items within 22 concepts/functions that cover receptive, expressive, and pragmatic areas of communication specific to AS based on nearly 300 parents' input! This is assessed alongside 14 descriptive items that capture essential information about the individual's unique communication styles. The questionnaire is meant to be conducted by the primary caregiver who is most familiar with the individual and how they communicate, rather than expecting individuals living with dyspraxia or apraxia to perform in a clinical or hospital setting.
The ORCA was developed using rigorous quantitative methods and qualitative interviews with parents of individuals with Angelman syndrome and communication specialists who treated individuals with AS and other developmental communication disorders. The team developed questions from the caregiver's perspective, and the questionnaire's target population was any AS individual, including any genotype. The ORCA was designed to include all communication modalities, meaning that an individual's ability is scored on how they communicate, including AAC, signs, gestures, sounds, word approximation, or words.
Now that the ORCA is a tool being used by numerous pharmaceutical companies in their active clinical trials, the tool is being progressed through regulatory agencies. This additional research focuses on analyzing data from longitudinal assessments in relationship with other communication measures so that it can be presented to the U.S. Food and Drug Administration (FDA). This work provides value to our community, the ABOM, and other sponsors using or planning to use the ORCA measure in future clinical trials for AS, which right now include: Ionis, Roche, Ultragenyx, and Neuren. In addition, this communication measure is being developed for 13 other NDDs (Neurodevelopmental Disorders) through a grant funded by the FDA of over $2M.