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]
Official URL: https://ww2.amstat.org/meetings/jsm/2020/onlinepro...
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 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 Group:||Evaluation of health and support services|
|Objective Field:||Evaluation of health outcomes|
|UTAS Author:||Roydhouse, J (Dr Jessica Roydhouse)|
|Deposited By:||Menzies Institute for Medical Research|
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