University Of Tasmania

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Methods to Evaluate and Correct for Bias in Patient-Reported Outcomes in Clinical Trials: A Discussion

conference contribution
posted on 2023-05-23, 19:11 authored by Jessica RoydhouseJessica Roydhouse, Cappelleri, JC, Colantuoni, E, Alemayehu, D

Improving the underlying disease or condition is a central goal of drug development. However, understanding patient experience while on therapy is increasingly of interest. The goal is accurate and interpretable patient-centric information that can inform providers and patients when making treatment decisions. Understanding the patient experience requires collecting data from patients. Patient-reported outcomes (PROs) such as symptoms and function are frequently collected on trials on a quantitative scale. These outcomes can provide valuable insight into the patient perspective.

However, like all trial data, PRO results may be biased. PRO data can present additional analytic challenges, and a better understanding of methods to analyze and interpret this data, while taking into account the potential for bias is needed. In this discussion paper, we consider two situations: 1) bias in responder analyses and 2) estimands for analyzing patient function in trials with severely ill patients.


Publication title

JSM 2020 Proceedings


Menzies Institute for Medical Research

Event title

JSM 2020

Event Venue


Date of Event (Start Date)


Date of Event (End Date)


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  • Restricted

Socio-economic Objectives

Evaluation of health outcomes