PURPOSE: This workshop builds on ISPOR task force recommendations for selection, modification, and/or development of performance outcome (PerfO) assessments by reviewing unique aspects of PerfO assessments relative to other clinical outcome assessment (COA) types and addressing how these differences impact the implementation of PerfO assessments in clinical trials.

DESCRIPTION: When evaluating clinical benefit of therapeutic interventions, it is critical that the outcomes assessed reflect aspects of health that are clinically meaningful to patients. Ideally, PerfO assessments are used when the optimal means of capturing clinical benefit is through the completion of defined tasks involving physical, cognitive, and/or sensory function that reflect activities that are meaningful in daily life. PerfO assessments are based on standardized tasks actively undertaken by a patient according to a set of instructions and administered by a trained individual or completed by the patient independently.

Although PerfO assessments share many of the same scientific and methodological considerations with the other COA types, they require special attention due to the nature of the relevant meaningful aspects of health, their relationship to concepts of interest, and the often indirect method of assessing them. PerfO assessments are frequently used in pediatric populations and patient groups with cognitive challenges, requiring additional considerations.

Ms. Eremenco will chair the session and provide a brief overview of areas where PerfO assessments differ from other types of COAs with regard to their selection, modification, and/or development; Ms. Bush and Dr. Ballinger will address the implementation of PerfO assessments in multinational clinical trials, with a focus on the unique considerations related to task-based assessments and the special populations often involved; and Dr. Campbell will provide her perspective on regulatory expectations regarding fit-for-purpose PerfO assessments in the evaluation of clinical benefit. A moderated discussion with polling will follow to engage the audience in discussing implementation challenges and good practices.