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


Roydhouse, J and Cappelleri, JC and Colantuoni, E and Alemayehu, D, Methods to Evaluate and Correct for Bias in Patient-Reported Outcomes in Clinical Trials: A Discussion, JSM 2020 Proceedings, 2-6 August, online (2020) [Non Refereed Conference Paper]

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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.

Item Details

Item Type:Non Refereed Conference Paper
Keywords:clinical trial, patient-reported outcomes, biostatistics
Research Division:Biomedical and Clinical Sciences
Research Group:Other biomedical and clinical sciences
Research Field:Other biomedical and clinical sciences not elsewhere classified
Objective Division:Health
Objective Group:Evaluation of health and support services
Objective Field:Evaluation of health outcomes
UTAS Author:Roydhouse, J (Dr Jessica Roydhouse)
ID Code:142750
Year Published:2020
Deposited By:Menzies Institute for Medical Research
Deposited On:2021-02-10
Last Modified:2021-02-19

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