Methods to Evaluate and Correct for Bias in Patient-Reported Outcomes in Clinical Trials: A Discussion
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.
History
Publication title
JSM 2020 ProceedingsDepartment/School
Menzies Institute for Medical ResearchEvent title
JSM 2020Event Venue
onlineDate of Event (Start Date)
2020-08-02Date of Event (End Date)
2020-08-06Repository Status
- Restricted