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