2024-04-07 22:09:36
In a new study, researchers found that most cancer drugs granted accelerated approval do not demonstrate such benefits within five years.
“Five years following the initial accelerated approval, you should have a definitive answer,” said Dr. Ezekiel Emanuel, a cancer specialist and bioethicist at the University of Pennsylvania, who was not involved in the research. “Thousands of people are receiving these medications. It seems like a mistake if we don’t know whether they work or not.”
The program was created in 1992 to accelerate access to HIV medications. Today, 85% of accelerated approvals are for drugs to fight cancer.
It allows the FDA to grant early approval to drugs that show promising initial results for treating debilitating or life-threatening diseases. In return, pharmaceutical companies must conduct rigorous testing and produce better evidence before gaining full approval.
Patients get access to medicines sooner, but the trade-off means some medicines don’t work. It’s up to the FDA or the drug manufacturer to remove disappointing drugs, and sometimes the FDA decides that less definitive evidence is enough for full approval.
The new study found that between 2013 and 2017, 46 cancer drugs received accelerated approval. Of these, 63% were converted to regular 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 well cancer patients understand drugs with accelerated approval, said study co-author Dr. Edward Cliff of Harvard University School of Medicine.
“We ask the question: is this uncertainty transmitted to patients?” emphasized Dr. Cliff.
Drugs granted 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 for doctors to carefully explain the evidence, Dr. Litton said.
“It might be a shrinkage of the tumor. That might depend on how long the tumor remains stable, she said. You can provide the data you have, but you should not overpromise.”
The U.S. Congress recently updated the program, giving more authority to the FDA and simplifying the process of withdrawing drugs when companies fail to meet their commitments.
The changes allow the agency “to more quickly withdraw approval of a drug approved under accelerated approval, where appropriate, more quickly,” wrote FDA spokeswoman Cherie Duvall- Jones, in an email. The FDA can now require that a confirmatory trial be underway when it grants preliminary approval, speeding up the process of verifying a drug’s effectiveness, she said.
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