Abstract:
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This session will address when and how to evaluate the psychometric properties (including meaningful change and responder definitions) of a patient-reported outcome (PRO) instrument or other clinical outcome assessment (COA) used to construct an endpoint in a clinical trial. The focus will be identifying the risks and ramifications of evaluating for the first time the psychometric properties of a PRO in the same pivotal clinical trial in which the instrument is used to construct a primary, co-primary, or key secondary endpoint. In particular, the session will consider issues that can arise when these psychometric properties are assessed at an interim analysis within a clinical trial (i.e., within a "psychometric substudy") and the interim findings are then applied to the interpretation of the final results. While many potential options exist to evaluate psychometric properties during a clinical trial, these options may result in loss of statistical power, over-fitting, blinding/masking issues, unique missing data concerns, and bias. Attendees can expect to gain insights that will help them plan psychometric evaluations within their own drug development timelines.
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