UNC Lineberger-led report details strategies for including patient-reported outcomes in cancer drug labeling

A UNC Lineberger-led report published online in JAMA Oncology describes barriers to getting patient-reported outcomes information into cancer trials and drug labels in the United States as well as recommendations for overcoming those obstacles.

UNC Lineberger-led report details strategies for including patient-reported outcomes in cancer drug labeling click to enlarge Ethan Basch, MD, MSc

A report led by a UNC Lineberger Comprehensive Cancer Center researcher has laid out recommendations for how to incorporate data on how cancer drugs make patients feel and function into oncology drug labels.

The article was published online today in the journal JAMA Oncology. It details perspectives and strategies from a panel of pharmaceutical industry representatives, U.S. Food and Drug Administration officers, and patients who met at a FDA-sponsored workshop last year to discuss barriers to getting patient-reported outcomes in oncology drug labels.

“Patients with cancer have many symptoms due to their disease and drug side effects, but we seldom collect this information well in clinical trials,” said Ethan Basch, MD, MSc, director of the UNC Lineberger Cancer Outcomes Research Program, and associate professor of medicine and public health at the UNC School of Medicine, and the report’s lead author. “Without this information, drug developers and the FDA have an incomplete picture of the efficacy and safety of drugs because they don’t fully understand the patient experience. Moreover, after a drug is approved, when patients sit down with their doctors to consider taking that drug, accurate information about how they should expect to feel is not available.” 

The report describes barriers to getting patient-reported information into cancer trials and drug labels in the United States. For example, there is often an accelerated approval process for oncology drugs, but developing measures for patient-reported outcomes for specific diseases can take years. And the trials often include small numbers of patients who are often very sick.

The report notes that from the pharmaceutical industry perspective, there are barriers related to overly rigorous FDA standards for patient-reported outcomes measurement, while from the FDA view, pharmaceutical industry approaches are often flawed. 

“Companies and regulators largely focus on other outcomes besides how people feel in clinical trials,” Basch said. “Patient-reported outcomes are often an afterthought in research design, and as a result are not well designed.” 

And from the patient perspective, the report states that without the inclusion of patient-reported outcomes data in publications and drug labels, patients are frequently turning to the internet to try to understand symptom effects. 

Nonetheless, the report notes recent progress, including formation of a public-private partnership called the PRO Consortium, and a new Patient-Focused Drug Development initiative to move this area forward. 

The report also includes specific recommendations for addressing the issue. The report suggests that the FDA should encourage trial sponsors to include patient-reported outcomes in drug development programs. It also encourages discussions about patient-reported outcomes between pharmaceutical industry representatives and the FDA in any given drug development program. These suggestions are already being implemented by the FDA, Basch said.

“As a result of this and other discussions, there are ongoing, serious efforts at the FDA and in industry in this area,” he said. “It’s still early, but there are real efforts here.”

Other article co-authors include Cindy Geoghegan, BA, of Patient an Partners LLC; Stephen Joel Coons, PhD, of the Patient-Reported Outcomes Consortium and the Critical Path Institute; Ari Gnanasakthy, MSc, MBA, of Novartis Pharmaceuticals; Ashley F. Slagle, PhD, of the U.S. FDA Center for Drug Evaluation and Research Office of New Drugs Study Endpoints Team; Elektra J. Papadopoulos, MD, MPH, of the U.S. FDA Center for Drug Evaluation and Research Office of New Drugs Study Endpoints Team; and Paul G. Kluetz, MD, U.S. FDA Center for Drug Evaluation and Research Office of new Drugs and of the Office of Hematology and Oncology Products.