Less than half of the cancer drugs recently receiving accelerated approval from the Food and Drug Administration ultimately showed clinical benefit in trials after five years, a new JAMA study finds.
Why it matters: There's been growing scrutiny around the FDA's fast-track process for allowing drugmakers to launch products on preliminary evidence and charge high prices before completing trials.
Previous studies have raised similar concerns, and cancer drugs account for the vast majority of those receiving accelerated approval.
What they found: In the study, presented at the American Association for Cancer Research Annual Meeting, researchers looked at 129 accelerated drug approvals for cancer drugs between 2013 and 2023.
46 of those accelerated approvals were granted between 2013 and 2017, meaning those drugmakers have had at least five years to conduct confirmatory trials.
Of those drugs, the researchers found 63% were converted to regulatory approval, but only 43% demonstrated that they improved overall survival or quality of life.
Between the lines: Researchers also found drugs granted accelerated approval were more quickly withdrawn from market, decreasing from 9.9 years post-approval to 3.6 years between 2013 and 2023.
However, it took longer to get full approval, increasing from 1.6 years to 3.6 years over the study period.
"We believe that conversion decisions should be both timely but — more importantly — supported by high-quality clinical outcomes, and that this is critical to the proper functioning of the accelerated approval pathway," saidstudy co-author Ian T. T. Liu, a researcher at Brigham and Women's Hospital.
The bottom line: Researchers said they hoped the findings would encourage more doctors to discuss with patients the uncertainty surrounding cancer drugs approved on an accelerated basis.
They said they also hoped it would encourage more scrutiny from regulators. The FDA has been considering changes that would require more stringent trials for accelerated approval.