WASHINGTON (AP) — The U.S. Food and Drug Administration's accelerated approval program aims to provide patients with quicker 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 that receive accelerated approval do not demonstrate such benefits within five years.
"Five years after the initial accelerated approval, you should have a definitive answer," said Dr. Ezekiel Emanuel, a cancer expert and bioethicist at the University of Pennsylvania, who was not involved in the research. “Thousands of people are getting these medicines. "It seems like a mistake if we don't know whether they work or not."
The program was created in 1992 to provide rapid access to HIV medications.Today, 85% of accelerated approvals go to cancer drugs.
This allows the FDA to quickly approve drugs that show promising early results for treating debilitating or fatal diseases. In return, drug companies are expected to conduct rigorous trials and produce better evidence before receiving full approval.
Patients get medications earlier, but tradeoffs mean some medications don't finish. Withdrawal of disappointing drugs is up to the FDA or the drug manufacturer, and sometimes the FDA has decided that less certain evidence is sufficient for full approval.
The new study found that between 2013 and 2017, 46 cancer drugs were granted accelerated approval.Of those, 63% were converted to routine approval, even though 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 not clear how much cancer patients understand about drugs with accelerated approval, said Dr. Edward Cliff of Harvard Medical School, a co-author of the study.
"We raise the question: Are patients being made aware of that uncertainty?" Cliff said.
Dr. Jennifer Leighton of MD Anderson Cancer Center in Houston, who was not involved in the study, said the drugs that received accelerated approval may be the only option for patients with rare or advanced cancers.
It's important for doctors to interpret the evidence carefully, Litten said.“This could be a shrinkage of the tumor. This may depend on how long the tumor remains stable, Litten said. “You can provide the data you have, but you should not overpromise.”
Congress recently updated the program, giving the FDA more authority to streamline the process for recalling drugs if companies don't meet their commitments.
FDA spokeswoman Cheri Duvall-Jones wrote in an email that the changes allow the agency to "withdraw approval for a drug approved without accelerated approval, when appropriate, more quickly." The FDA may now require that a confirmatory trial be ongoing when it grants initial approval, he said, which speeds up the process of verifying whether a drug works.
In a new study, researchers found that most cancer drugs that receive accelerated approval do not demonstrate such benefits within five years.
"Five years after the initial accelerated approval, you should have a definitive answer," said Dr. Ezekiel Emanuel, a cancer expert and bioethicist at the University of Pennsylvania, who was not involved in the research. “Thousands of people are getting these medicines. "It seems like a mistake if we don't know whether they work or not."
The program was created in 1992 to provide rapid access to HIV medications.Today, 85% of accelerated approvals go to cancer drugs.
This allows the FDA to quickly approve drugs that show promising early results for treating debilitating or fatal diseases. In return, drug companies are expected to conduct rigorous trials and produce better evidence before receiving full approval.
Patients get medications earlier, but tradeoffs mean some medications don't finish. Withdrawal of disappointing drugs is up to the FDA or the drug manufacturer, and sometimes the FDA has decided that less certain evidence is sufficient for full approval.
The new study found that between 2013 and 2017, 46 cancer drugs were granted accelerated approval.Of those, 63% were converted to routine approval, even though 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 not clear how much cancer patients understand about drugs with accelerated approval, said Dr. Edward Cliff of Harvard Medical School, a co-author of the study.
"We raise the question: Are patients being made aware of that uncertainty?" Cliff said.
Dr. Jennifer Leighton of MD Anderson Cancer Center in Houston, who was not involved in the study, said the drugs that received accelerated approval may be the only option for patients with rare or advanced cancers.
It's important for doctors to interpret the evidence carefully, Litten said.“This could be a shrinkage of the tumor. This may depend on how long the tumor remains stable, Litten said. “You can provide the data you have, but you should not overpromise.”
Congress recently updated the program, giving the FDA more authority to streamline the process for recalling drugs if companies don't meet their commitments.
FDA spokeswoman Cheri Duvall-Jones wrote in an email that the changes allow the agency to "withdraw approval for a drug approved without accelerated approval, when appropriate, more quickly." The FDA may now require that a confirmatory trial be ongoing when it grants initial approval, he said, which speeds up the process of verifying whether a drug works.