According to the findings of new research, only about one out of five cancer drugs approved by federal regulators through a fast tracked early approval system end up being effective at helping patients survive.
The FDA offers an accelerated drug approval program to help get critical drugs to patients sooner. However, a new study indicates the program may not be approving effective drugs. Instead, the majority of the drugs approved early through the program don’t actually work.
The findings were published May 28, in the journal JAMA Internal Medicine.
The FDA’s accelerated approval program allows cancer drugs to be approved quickly if a pre-approval study can show some type of benefit in surrogate measures. Then after the drug is approved it must undergo post-approval studies to confirm the benefit in patients.
In this latest review, Harvard researchers conducted an updated review of 93 cancer drugs fast tracked to approval using the FDA’s accelerated approval program from December 1992 through May 2017.
The study indicated only 20% of drugs rapidly approved actually increased patient survival and helped them reach remission. The remaining drugs helped patients improve in other measures, such as tumor reduction, which are not directly correlated to cancer survival.
Surrogate measures are allowed by the FDA to stand-in for actual measures, like overall survival rates. Instead, researchers can use measures like survival without the cancer progressing further or tumor reduction. These measures don’t predict clinical benefit, effectiveness, or survival, but the FDA allows these measures as predictions of a “good” cancer drug.
According to those surrogate measures, 51 of the studies showed clinical benefit. In one case, the FDA approved a drug even though it did not even meet any surrogate measures. However, the new review of the studies indicated only 19 studies showed clinical benefit or improvement in overall survival when those surrogate measures were removed.
Some of the drugs shown to be ineffective, but that still received FDA approval, included Avastin for gliobastoma; Keytruda for urothelial, and for head and neck cancer; and Opdivo for melanoma. However, the FDA says the accelerated approval method is successful because only 5% of drugs approved this way are withdrawn after going to market. It does not account for the drugs that made it to the market, but aren’t effectively treating cancer.
A similar study published in 2017 concluded new medications that are approved through the accelerated program are more likely to need a warning label update for serious life-threatening side effects after patients begin using the drugs. In fact, one-third of all drugs approved this way require safety updates.
Researchers indicate a reassessment of the accelerated program requirements should be conducted to obtain more clinically meaningful information on drugs that patient’s will use.
They also emphasized the study wasn’t meant to criticize the FDA and the accelerated process. Instead, it’s meant to better serve cancer patients and help create drugs that will help patients live longer and better lives.