The U.S. Food and Drug Administration’s accelerated approval program is intended to give patients early access to promising drugs. But how often do these drugs actually improve or extend patients’ lives?
In a new study, researchers found that most cancer drugs granted accelerated approval do not demonstrate such benefits within five years.
“Five years after the first accelerated approval, you should have a definitive answer,” says Dr. Ezekiel Emanuel, a cancer specialist and bioethicist at the University of Pennsylvania, who was not involved in the study. “Thousands of people receive those medications. That seems like a mistake if we don’t know if they work or not.”
The program was created in 1992 to accelerate access to HIV medications. Today, 85% of accelerated approvals go to cancer drugs.
This allows the FDA to grant early approval to drugs that show promising early results for treating debilitating or fatal diseases. In return, pharmaceutical companies are expected to conduct rigorous testing and produce better evidence before receiving full approval.
Patients get access to medications earlier, but the trade-off means some medications don’t work. It’s up to the FDA or the drug manufacturer to withdraw disappointing drugs, and sometimes the FDA has decided that less definitive evidence is good enough for full approval.
The new study found that 46 cancer drugs received accelerated approval between 2013 and 2017. Of those, 63% converted to mainstream approval, although only 43% demonstrated clinical benefit in confirmatory trials.
The research was published in the Journal of the American Medical Association and discussed Sunday at the annual meeting of the American Association for Cancer Research in San Diego.
It’s unclear how much cancer patients understand about drugs with accelerated approval, said study co-author Dr. Edward Cliff of Harvard Medical School.
“We ask the question: is that uncertainty conveyed to patients?” Cliff said.
Drugs that received accelerated approval may be the only option for patients with rare or advanced cancers, said Dr. Jennifer Litton of MD Anderson Cancer Center in Houston, who was not involved in the study.
It’s important that doctors explain the evidence carefully, Litton said.
“Maybe it’s a shrinking tumor. It could be how long the tumor remains stable,” Litton said. “You can provide the data you have, but you shouldn’t overpromise.”
Congress recently updated the program, giving the FDA more authority and streamlining the process for withdrawing drugs when companies fail to meet their obligations.
The changes allow the agency to “rescind approval for a drug approved under accelerated approval, more quickly, if necessary,” FDA spokesperson Cherie Duvall-Jones wrote in an email. The FDA can now require a confirmatory study to be initiated when it grants preliminary approval, speeding up the process of verifying whether a drug works, she said.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely responsible for all content.