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Are accelerated approvals having as much impact as the FDA hopes?
The effectiveness of cancer drugs approved quickly is being called into question by some experts.
A 2018 study published in JAMA Oncology study looked at the accelerated approvals of 93 cancer drugs and five had been removed from the market during a 25-year span.
Researchers at Harvard Medical School's Program on Regulation, Therapeutics and Law wanted to take a deeper dive to what had happened with the rest, according to NPR. Follow-up studies conducted by the researchers found that only 19 of the 93 drugs approved via the accelerated FDA approval pathway were deemed to have verified benefits based on improvement in survival reported in confirmatory trials, according to a study published in JAMA Internal Medicine.
“The [study] should mean that some researchers and academics have a different view of appropriate benefit/risk profile for approving drugs than the FDA does,” says Michael Kleinrock, research director, IQVIA Institute. “This suggests that progression-free survival is not an appropriate end point but FDA has clearly indicated that it is. Society has called upon industry and the FDA to bring cancer treatments to people faster and they have done so. These researchers are calling doubt on the value, but that information was not known at the time of approval, and it is not part of their study to see whether providers are still using the drugs for those uses.”
In many cases, according to Kleinrock, IQVIA has found that drugs have a period of uptake and usage that can fade very quickly, particularly when clinical data fails to confirm the previous results. “From our research, we observe the high numbers of approvals based on a single trial and note that the results clearly satisfied the regulator and time will tell if that is supported in the real world,” he says.
“The FDA is not the only limit on the use of the drugs and it would be good to note that having them available may still bring value to some patients, even if the drug fails to demonstrate overalls survival for the median patient,” he says. “These are complex issues and people’s lives are at stake, and the balance of approving more medicines more quickly when promising clinical data is presented is key to FDA’s mission.”