Patient-Reported Outcomes in Oncology Expected to Make Regulatory Waves
Patient-reported outcomes, or PROs, are going to play a larger role in FDA cancer drug approvals, according to an expert in the field.
Until recently, most collection of PRO data for cancer drug development had not been rigorous or comprehensive, according to Ethan Basch, MD, MSc, professor of medicine at the University of North Carolina (UNC)-Chapel Hill, and director, Cancer Research Outcomes Research Program at the UNC Lineberger Comprehensive Cancer Center, in Chapel Hill, North Carolina. (Dr Basch also serves on the National Cancer Institute (NCI)'s Board of Scientific Advisors and the Patient-Centered Outcomes Research Institute [PCORI] Methodology Committee.)
That has meant that regulatory approvals are frequently made based on incomplete information — and that oncologists are often unable to detail for patients what types of changes in cancer-related pain, sleep disturbance, fatigue, food taste, nausea, or diarrhea, for example, they should expect with a particular treatment, Dr Basch noted.
PRO data is often collected during drug development, but data collection has largely been haphazard and unstandardized, or insufficient for labeling purposes.
“The real problem has been that designing PRO end points in studies has not been sufficiently rigorous or thoughtful,” Dr Basch told Cancer Therapy Advisor. “They are often added at the last minute.”
“By the time an application comes into a regulatory agency or authority, the information is not really what it could be because there wasn't enough interaction with the agency from the beginning,” he explained. “They didn't use a thoughtful implementation, so they have missing data or the tools they used were not appropriate.”