A decade ago, the US Food and Drug Administration (FDA) issued broad guidance for PROs. But that guidance was widely seen as overly technical and restrictive, prompting complaints from drug developers and caused them to think, ‘forget this – it isn’t worth it,’ Dr Basch said. “They said, ‘we’ll just focus on radiographic and biomarker end points, or overall survival’.”
But that is now changing.1-3
Regulators and professional organizations are providing more practical guidance for the use of PRO measures in clinical trials and regulatory filings that will help close the information gap, he said.
“Some of this is because of the US FDA,” he noted. “But it is also because of the European regulatory authorities, which ask for certain types of PRO information to understand the value of products.”
Dr Basch further noted that the FDA’s 2017 creation of the Oncology Center of Excellence to streamline development of precision cancer treatment and the 21st Century Cures Act, passed into law in 2016, have helped to “substantially” refocus drug development on patient-focused outcomes.3
“I think it is widely recognized that PROs are a big part of this effort,” he said. “There are multiple leaders within oncology at the FDA who have become very knowledgeable and committed to PROs and they’re hiring more people who are methodologists within oncology and who are focused on PROs — and they’re encouraging industry to think earlier about including PROs.”
The FDA has focused on 3 key areas for PRO measures: disease symptoms, physical function, and symptomatic adverse events. The FDA is developing a rubric for inclusion of all of these elements via public meetings, Dr Basch noted.
“Those are their big 3,” Dr Basch said. “Notice that global quality of life [QoL] is not one of them. They consider that to be a little too broad or multidimensional for regulatory purposes. They’re really focusing on more discrete, clinically actionable domains. And the idea is that there should be more standardized ways to integrate PROs into clinical trials from the beginning. QoL is still important, but in different contexts — and the Europeans are still interested in QoL.”