When it comes to balancing the risks with the benefits of a drug, it’s vital to have the full picture of a patient’s treatment experience. And who better to provide that personal account than the patient herself?
Yet, in cancer clinical trials, the side effects patients often experience are typically reported by physicians — and not directly by the patients. “These range from more technical side effects, such as low blood counts, to symptoms like nausea,” Ethan Basch, MD, MSc, director of the University of North Carolina Lineberger Cancer Outcomes Research Program and associate professor at UNC-Chapel Hill, told Drug Discovery & Development.
“This approach might have made more sense in a time when cancer treatments were not very effective,” Basch told Drug Discovery & Development. “But these days, people live for long periods of time with cancer, and symptoms have a substantial impact on their lives.”
And previous research indicates that doctors underreport side effects of investigational cancer drugs by as much as half, according to a study published in March in the Journal of Clinical Oncology.
In an effort to give patients a stronger voice, researchers have created and implemented a system to enable patients to directly report their symptoms during cancer drug development, according to a study published today in JAMA Oncology. The study was led by Basch, who conducted the research when at Memorial Sloan Kettering Cancer Center and is now with UNC, and supported by the National Cancer Institute (NCI).
The researchers tested 124 measures, called the Patient Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), which had been previously developed by Basch and colleagues for the NCI to survey patients about 78 potential adverse events. The study surveyed nearly 1,000 patients who were undergoing outpatient chemotherapy, radiation therapy, or both (i.e. In the past seven days, how often did you have arm or leg swelling?). To establish the validity of the survey questions, “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.
“Most patients had multiple symptoms, and these were often severe and impacted daily activities,” Basch told Drug Discovery & Development. “Many new drugs in oncology are taken in pill form, and when people experience severe side effects, they often elect to stop taking their medicine.
“We have seen this with multiple cancer drugs. We often find out about these problems after a drug is in the marketplace because we don’t do a good job assessing these side effect symptoms during drug development. If we could have this information earlier in clinical trials, we might pick more tolerable doses,” Basch told Drug Discovery & Development.
The study found that the PRO-CTCAE measures demonstrated validity and reliability, and “establishes that this tool can be trusted in widespread use,” said Basch.
Efforts to highlight patient-reported outcomes (PROs), defined by the FDA as ‘measurements of any aspect of a patient’s health status that come directly from the patient without the interpretation of the patient responses by a physician or anyone else’ include CONSORT (Consolidated Standards of Reporting Trials), the National Quality Forum’s PRO Project, and Patient Reported Outcomes Measurement Information System (PROMIS), funded by the National Institutes of Health.
Filed Under: Drug Discovery