Are Patient-Reported Outcomes in Open-Label Cancer Trials Biased?

Mar 4, 2019 | Bias, Cancer Clinical Trials, Oncology, Patient Reported Outcomes

patient engagement

Members of the U.S. Food and Drug Administration published a report in JAMA Oncology recently to address a concern that patient-reported outcomes (PROs) in open-label cancer trials could be biased.  Given the expected growth of such open-label trials, the authors’ sought to articulate thoughts on ways bias might occur.  The common concern is a patient’s knowledge of a treatment could influence their view and reporting of symptoms. The anticipated growth of PRO arises from recent legislative efforts such as the 21st Century Cures Act which emphasize capture and communication of patient experience.  PRO assessments represent a common method to measure symptoms, function, and health-related quality of life.  Back in 2009, the FDA released its first guidance on PROs which communicated that PRO labeling claims in open-label trials could become problematic.  Regardless, PRO measures have been incorporated in cancer trials for many years. These trials are increasingly single-arm or open-label comparative studies not supportive of blinded features due to deferring administration routes or differential overt toxic effects.  One specific concern with PROs in open-label trials is the patient’s perception of their symptoms or function may be influenced by knowledge of their assigned treatment.  Consequently, the authors produced the report articulating the ways in which open-label bias may arise. They address ways to address these concerns—follow the link and secure the report to learn more.


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