Alternative approaches to outcomes assessment: beyond psychometric tests

Area of Science:

• Clinical Trials
• Neuropsychology
• Behavioral Science

Background:

• Current outcomes assessments in clinical trials for cognition and behavior heavily rely on IQ and neuropsychological tests.
• These standard assessments offer limited insight into real-world, everyday functional abilities.
• Challenges such as low participant functioning or travel limitations can lead to missing data in traditional assessments.

Purpose of the Study:

• To highlight the limitations of traditional cognitive and behavioral assessments in clinical trials.
• To introduce interview-based adaptive behavior assessments as a viable alternative for evaluating everyday functions.
• To demonstrate the utility of these alternative assessments in overcoming challenges faced by specific participant groups.

Main Methods:

• Discusses the limitations of IQ and neuropsychological assessments in capturing functional outcomes.
• Proposes interview-based adaptive behavior assessments as a method to evaluate everyday functions.
• Illustrates the application of these methods using the neurobehavioral component of a myelomeningocele fetal surgery trial.

Main Results:

• Interview-based adaptive behavior assessments reflect actual everyday functions.
• These assessments can be conducted remotely via telephone, overcoming travel barriers.
• The method is suitable regardless of a participant's cognitive functioning level.

Conclusions:

• Interview-based adaptive behavior assessments provide a more comprehensive evaluation of everyday functioning in clinical trials.
• This approach enhances data completeness by accommodating participants with low functioning or travel constraints.
• The myelomeningocele fetal surgery trial serves as a practical example of incorporating these effective alternative assessments.