Landmark study validates method for patient reporting of adverse events related to cancer drugs
August 13, 2015
Side effects experienced by patients in cancer clinical trials typically are reported by physicians, not by the patients themselves. Previous research has shown that doctors under-report these side effects.
Dr. Ethan Basch
Dr. Bryce Reeve
Ethan Basch, MD, associate professor of health policy and management at the UNC Gillings School of Global Public Health and director of the UNC Lineberger Cancer Outcomes Research Program, has led studies and national initiatives to aid patients in directly reporting their own symptoms during cancer drug development.
In an article published online Aug. 13 in the Journal of the American Medical Association (JAMA) Oncology, a study led by Basch and colleagues for the National Cancer Institute shows that a system they developed accurately and reliably captures the patient experience with cancer drug side effects.
Bryce B. Reeve, PhD, professor of health policy and management at the Gillings School, member of UNC Lineberger Comprehensive Cancer Center and research fellow at UNC’s Cecil G. Sheps Center for Health Services Research, is a co-author.
“This is a landmark study because patient-reported information has not been used for measuring side effects in cancer research,” said Basch, who also is associate professor of medicine in UNC’s School of Medicine and the paper’s senior author. “This study shows that these patient-reported measures perform well, and are ready for implementation in cancer research.”
The study tested the reliability and validity of a group of measures previously developed by Basch and colleagues for the National Cancer Institute (NCI) to test patient-reported adverse events. Basch and colleagues were contracted to develop the measures, which are called the Patient Reported Outcomes version of the Common Terminology Criteria for Adverse Events, or PRO-CTCAE.
The researchers tested 124 of the measures that had been developed for the NCI to question patients about 78 potential adverse events. The study involved nearly 1,000 patients who were undergoing outpatient chemotherapy, radiation therapy or both at one of nine U.S. cancer centers or oncology practices.
“In order to establish the validity of the 124 questions in the survey we developed using the NCI measures, we conducted statistical analyses to compare the new measurement tools to other metrics, such as to clinician reports of patient function and to data on the underlying characteristics of the patients’ cancer and their treatments,” Basch said.
The study found that the PRO-CTCAE measures performed extremely well, demonstrating validity and reliability across items.
Basch said the study is important as it validates measures developed to obtain reports of adverse events directly from patients. He noted a study by researchers in Europe published earlier this year in the Journal of Clinical Oncology found that doctors under-report side effects by as much as half in cancer trials.
“Patient reporting improves the quality of information that we have about how patients feel and function, and it provides a fuller picture of the impact of treatments on people’s lives,” Basch said. “This is important because in risk-benefit balancing for drugs, we need to have comprehensive and valid information about risk.”
Numerous co-authors of the study include first and corresponding author Amylou C. Dueck, PhD, in the Department of Health Sciences at the Mayo Clinic in Scottsdale, Arizona; and researchers from the National Cancer Institute, University of Texas M.D. Anderson Cancer Center, Memorial Sloan Kettering Cancer Center, Dana-Farber Cancer Institute, Duke University Medical Center, Emory University, and others.
The study was supported by contracts from the National Cancer Institute.
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Gillings School of Global Public Health contact: David Pesci, director of communications, (919) 962-2600 or dpesci@unc.edu
