By Vishal Bhatnagar, Oncology Center of Excellence, US FDA, Lori Minasian, Division of Cancer Prevention, US National Cancer Institute, and Paul G. Kluetz, Oncology Center of Excellence, US FDA
Over the past decade, a series of government-lead events set in motion a sea change in how patient-reported outcomes (PRO) in cancer clinical trials are assessed and reported. PRO measures have long provided an opportunity for the patient’s voice to be heard, and when rigorously implemented, PRO assessments can add quantitative symptom and functional data to help characterize risks and benefits of cancer treatments. An important example is the use of PRO to provide a direct report of symptoms and side effects from the person experiencing them.
The US National Cancer Institute (NCI) and the US Food and Drug Administration (FDA) have created publicly available tools to advance the collection, measurement, analysis, and reporting of patient-reported symptomatic adverse events in cancer trials. These initiatives have had ripple effects, expanding the definition of tolerability, developing guidelines to manage symptoms, providing new tools for industry to enhance dose finding in early-phase trials, and providing PRO data to enhance the patient-clinician interaction at the point of care.
In this article, we discuss:
- NCI’s development of the PRO version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), the FDA’s work to identify analytic and visualization methods to describe PRO-CTCAE data culminating in Project Patient Voice;
- future uses of PRO-CTCAE and other PRO data to inform early- and late-stage drug development; and
- the history of sustained scientific collaboration necessary to advance the rigorous use of PRO data to help characterize tolerability of cancer drugs.