Fast-tracked cancer drugs often fail to prove benefit
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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Between the lines: Researchers also found drugs granted accelerated approval were more quickly withdrawn from market, decreasing from 9.9 years post-approval to 3.6 years between 2013 and 2023.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," said study 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.
