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Activity Number: 424
Type: Topic Contributed
Date/Time: Tuesday, August 2, 2016 : 2:00 PM to 3:50 PM
Sponsor: ENAR
Abstract #319268 View Presentation
Title: Developing PRO Instruments in Clinical Trials: Issues, Considerations, and Solutions
Author(s): Cheryl Coon* and Dennis Revicki* and Scott Komo* and Kendra DeBusk* and Lisa Kammerman* and Stacie Hudgens*
Companies: Outcometrix and Evidera and FDA/CDER and Genentech and AstraZeneca and Clinical Outcome Solutions
Keywords: patient-reported outcome (PRO) ; clinical outcome assessment (COA) ; clinical trial endpoints ; psychometrics ; interim analysis ; missing data

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.

Authors who are presenting talks have a * after their name.

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