Just 43 Percent of Cancer Drugs Greenlit Under FDA’s Accelerated Approval Process Had Confirmed Benefit


‘Our results suggest that the FDA may be approving many costly, toxic drugs that do not improve overall survival.’

A new study into the U.S. Food and Drug Administration’s (FDA) accelerated medicine approval process found that just 43 percent of the cancer drugs launched under the program between 2013–2017 demonstrated a confirmed clinical benefit, even though 63 percent of them were converted to regular approval.

The FDA established its accelerated approval pathway over 30 years ago to allow for earlier approval of drugs that treat serious conditions such as HIV, with roughly 85 percent of accelerated approvals nowadays going to cancer drugs.

Any pharmaceuticals that are approved under the accelerated program must undergo follow-up trials to confirm that they deliver the promised clinical benefit.

A previous study carried out in 2015 showed, of the cancer drugs that received accelerated FDA approvals between 2008 and 2012, just 14 percent went on to show an overall survival benefit.

“Our results suggest that the FDA may be approving many costly, toxic drugs that do not improve overall survival. Enforcement of postmarketing studies is therefore of critical importance,” the authors of the earlier study wrote.

Since then, researchers have wanted to determine how the use of accelerated approvals has evolved. This led to the latest study, which was published in the Journal of the American Medical Association (JAMA) on April 7.

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The new study focused on 129 cancer drugs that were given accelerated approval from 2013 to 2023.

Among the drugs evaluated were 46 cancer drugs approved between 2013–2017, meaning they had more than 5 years of follow-up research to determine clinical benefit in confirmatory trials.

The study found that, of the 46 cancer drugs granted accelerated FDA approval and evaluated over the five-year period in question, 63 percent were converted to regular approval even though just 43 percent of them demonstrated a benefit in confirmatory trials.

“Most cancer drugs granted accelerated approval did not demonstrate benefit in overall survival or quality of life within 5 years of accelerated approval,” the study’s authors wrote.

In the past, some researchers and government watchdogs raised concerns about FDA oversight of the accelerated approvals program, including delays in quickly removing drugs with failed or missing confirmatory studies. The latest study adds to those concerns.

“Patients should be clearly informed about the cancer drugs that use the accelerated approval pathway and do not end up showing benefits in patient-centered clinical outcomes,” the researchers wrote in the study.

The FDA did not immediately respond to a request for comment on the study nor on broader concerns about the agency’s accelerated approvals pathway.

Accelerated Approvals in Focus

The FDA’s accelerated approval track allows for expedited approval of pharmaceuticals that cure critical diseases while the drugs are still undergoing larger and longer studies under the condition that the medicines fill an unmet medical need.

If the drugs are shown, through clinical studies, not to show benefit, it’s up to the drugmaker or the FDA to remove the medicines from the market. In some cases, drugmakers have resisted calls from the FDA to voluntarily withdraw drugs that have failed to show benefits in follow-up trials.

However, if studies show therapeutic benefits, the agency may grant conventional approval. At times, the FDA has decided that less definitive evidence is good enough for a full approval.

“The FDA relinquishes significant leverage once it converts accelerated approvals to regular approval—it is harder to ensure timely completion of further trials, and harder to withdraw drugs—so the evidence used to justify these decisions is important,” said Edward Cliff, a postoctoral fellow and senior author of the study, according to a press release from the American Association for Cancer Research (AACR).

The JAMA research was discussed at the annual meeting of the AACR in San Diego over the weekend.

Besides seeking to evaluate how the cancer drugs that were granted accelerated approval between 2013–2017 performed after getting a green light, the study also considered whether the drugs were found to improve the patients’ quality of life, a factor that previous studies did not evaluate.

“We found seven drugs that were converted to regular approval based on response rate, i.e., tumor shrinkage, but this leaves a great deal of uncertainty regarding whether the drugs ultimately benefit patients,” Mr. Cliff said, per the AACR press release.

Among the 46 cancer drugs that were approved between 2013 and 2017, 10 were withdrawn.

Congress recently updated the FDA’s accelerated approvals program, giving the agency more authority and streamlining the process for withdrawing drugs when companies don’t meet their commitments.

The JAMA study also found that, between 2013 and 2023, the time to withdraw drugs that didn’t show confirmed benefits decreased from 9.9 years to 3.6 years, while the time to regular approval increased from 1.6 years to 3.6 years.


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