Recommendations on the Implementation of Performance Outcome Assessments in Clinical Trials: A Good Practices Report of an ISPOR Task Force.

Abstract

Performance outcome (PerfO) assessments are standardized, task-based clinical outcome assessments (COAs) used to evaluate patients' physical, cognitive, sensory, and functional abilities in clinical trials. Building on the first ISPOR PerfO Task Force report, which addressed selection, development, and modification of PerfO instruments, this second task force report focuses on clinical trial implementation considerations that are especially relevant to PerfO assessments, when compared to other clinical outcome assessment types. Examples of PerfO Assessments related to specific aspects of function (sensory, motor, cognitive, daily, ambulatory, and neurodevelopmental) are presented. Practical considerations related to clinical trial design, standardized documentation, administrator training, data collection and use of digital health technologies to enable reliable data collection are provided. Factors that jeopardize validity (including disease progression, learning effects, encouragement effects, and sensory or cognitive limitations) are identified alongside targeted mitigation strategies. As no analytic technique can compensate for inconsistent PerfO assessment implementation, careful planning and consistent monitoring of data collection is foundational to ensuring data are valid and interpretable.