Fast-Tracked Cancer Drugs Still Recommended To Patients Despite Poor Performance In Trials

A new study has found that a third of cancer drugs given accelerated approval by the U.S. Food and Drug Administration (FDA) continue to be recommended in clinical guidelines after they fail to show any benefit in trials.

U.S. and Canadian researchers collaborated on the study, which was published in the British Medical Journal on Sept. 8. The team investigated how the FDA has handled cancer drugs that received accelerated approval but then had negative post-approval trials. They also checked whether these negative trials led to changes in treatment guidelines.


The team considered trials to be negative if the main result being measured showed no improvement under the given treatment. Results could include the number of deaths recorded or the difference in survival rates between the treatment group and control group.

Based on the findings of their study, the researchers called for clinical guidelines to “better align with the results of postapproval trials of cancer drugs that received accelerated approval.”

A cancer patient receives a chemotherapy drip at Cape Fear Valley Medical Center June 17, 2003, in Fayetteville, North Carolina. (Chris Hondros/Getty Images)

Accelerated approval allows drugs to enter the market before their effectiveness has been proven to permit quicker access to them. As part of the process, the drug manufacturer must conduct post-approval trials to confirm a clinical benefit, such as improved mortality among cancer patients. If there is no benefit, approval can be withdrawn.

Post-approval trials can be delayed for years. Until recently, the FDA has been slow to withdraw drugs when trials fail to demonstrate a clinical benefit.

The researchers analyzed an FDA database to identify cancer drugs that had been granted accelerated approval from 1992 until December 2020. They found 18 indications for 10 cancer drugs that failed to show a clinical benefit in post-approval trials. Of these, approvals for 11 (61 percent) were voluntarily withdrawn, one was revoked, and six (33 percent) remained on the drug’s label, over an average of four years.

Next, the team reviewed the latest FDA and National Comprehensive Cancer Network, or NCCN, guidelines. They discovered that most of the drugs they had identified had continued to receive high-level endorsements, even after approval for the given indication had been withdrawn or revoked.

The researchers acknowledged that the study’s limitations, such as relying on publicly available information and pending FDA decisions on certain approvals, may have affected its accuracy. However, they stressed that this was the most comprehensive study published to date, noting that their findings “reflect the lack of fulfillment of the compromise between speed and evidence that underpins the accelerated-approval pathway.”

Edited by Siân Speakman and Kristen Butler



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