Patient-reported outcomes (PROs) are playing a growing role in cancer research and are poised to become an important part of regulatory review in drug development—and even routine clinical cancer care, according to experts at the American Society of Clinical Oncology (ASCO) Annual Meeting 2016, held June 3–7 in Chicago.
During a June 6 session entitled “Integrating Patient-Reported Outcomes into Cancer Clinical Trials and Regulatory Review,” experts discussed PROs, which are measures of patients’ symptoms and physical, cognitive, social and emotional functioning, and health-related quality of life, that are obtained via self-administered questionnaires or structured interview questions.
In research settings, there are indications that PROs might improve early detection of side effects.
“Early data suggest PRO [measures] might also enhance our ability to discriminate adverse events between study arms,” reported Ethan Basch, MD, MSc, of the University of North Carolina at Chapel Hill, a leading researcher in PRO assessments of adverse events among cancer patients.
In one study, the number of adverse events found to be statistically-significantly different between treatments was three times larger when adverse events were measured using PROs instead of traditional clinician-driven adverse-events reporting, Basch noted.
Incorporating PROs in drug development
Such findings have prompted the FDA to take a serious look at how best to incorporate PRO assessments of symptomatic adverse events in drug development, said Paul Gustav Kluetz, MD, a medical oncologist at the FDA’s Office of Hematology and Oncology Products.
“We are now in a different era of drug development; patient voices are more important,” Kluetz said. “Thoughtful incorporation of patients into clinical trials and drug development is becoming a priority.”
Improved assessment of tolerability represents a real “PRO measurement opportunity,” Kluetz said. “Symptomatic adverse events are best assessed by patients themselves.”
Drug developers and regulators have long “looked at drug safety through the clinician’s lens,” Kluetz said. The emphasis is on precisely-measured lab test results and radiographic or histological variables.