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Science update: Learn more about outcome measures

An outcome measure is a test used to determine if a treatment or therapeutic had some sort of effect on an individual. Typically, this measure is taken before a treatment/therapeutic for baseline results then readministered after the intervention to measure changes due to intervention.

An example of this could be weight measurements. Let’s say an individual was planning to enroll in a clinical trial testing the effectiveness of a diet drug. First, they would have to take their initial (baseline) weight. Then they would take the diet drug for a specified amount of time according to that trial and, upon completion of the trial, they would record a final weight. Here, the outcome measure is weight before and after participation in a diet drug trial. You can see here that outcome measures can show positive change (weight loss), negative change (weight gain), and no change (no weight change), thus useful in understanding the effect of a treatment or therapeutic. You can also see that it is imperative to have strong, reliable, and sensitive outcome measures because if the test is not accurate it cannot inform on the effect of that therapeutic/treatment.

Outcome measures are particularly important in the Angelman syndrome community as we need to be able to determine if a therapeutic is effectively treating AS symptoms in a clinical trial. In an effort to best capture potential AS outcome measures for communication, a research team at Duke University, led by Drs Christina Zigler and Bryce Reeve and funded by FAST, have developed an assessment tool called the Observer-Reported Communication Ability (ORCA) to evaluate the communication abilities (including receptive, expressive, and pragmatic) of individuals with Angelman syndrome. In two new publications out this week, this team presented findings from two rounds of cognitive interviews and a quantitative study based on caregiver reports. The results support the overall content validity, construct validity, and dependability of the ORCA measure for individuals with Angelman syndrome. With clinical trials already underway, this tool is proving to be a valuable means of sensitively monitoring improvements in communication abilities for those living with Angelman syndrome.

Learn more about the ORCA here.