Online Program Home
My Program

Abstract Details

Activity Number: 476 - SPEED: Clinical Trial Design, Longitudinal Analysis, and Other Topics in Biopharmaceutical Statistics
Type: Contributed
Date/Time: Wednesday, August 1, 2018 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #330574
Title: Single Item Analysis of Patient-Reported Outcome Measures in a Phase III Randomized Controlled Trial
Author(s): Stacie Hudgens* and Lysbeth Floden
Companies: Clinical Outcome Solutions and Clinical Outcome Solutions
Keywords: Patient Reported Outcomes; randomized controlled trials; clinical interpretation

Background: Patient-reported outcome (PRO) measures are increasingly used to derive endpoints in randomized controlled trials (RCTs). PRO instruments are often composed of items which, when scored, form multidimensional scales but are treated as unidimensional in the interpretation. In the regulatory setting there is growing interest in identifying and interpreting the single items that best inform the patient experience. Methods: We demonstrate through case-studies the use of a single-item analysis of categorical change to assess the likelihood of improvement during treatment in a phase III RCT setting. Specifically, we use mixed-effect logistic regression to estimate the odds of experiencing improvement on items measuring symptoms and compare to domain results. Results: We found that individual symptom items were better able to detect improvement in patients leading to relevant clinical interpretation compared to symptom domain scores. This method was also more efficient for detecting treatment differences. Single item analysis of PRO measures in RCTs lends to the interpretation of meaningful change of the domain.

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

Back to the full JSM 2018 program