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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.

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This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.