A recent study by researchers at Brigham and Women's Hospital and Harvard Medical School, both based in Boston, found only 43% of cancer drugs granted accelerated FDA approval demonstrated clinical benefit in confirmatory trials.
The study, published April 7 in JAMA, analyzed publicly available FDA data from 129 cancer drug-indication pairs granted accelerated approval from 2013 to 2023. It also looked at the demonstrated improvement in quality of life or overall survival in accelerated approvals with more than five years of follow-up, and confirmatory trial end points and time to conversion for drug-indication pairs converted to regular approval.
Among th 46 drugs with more than five years of follow-up, about 63% were converted to regular approval, 22% were withdrawn and 15% remained ongoing after a median of 6.3 years. Only 43% demonstrated a clinical benefit in confirmatory trials.
Here are three other findings:
- The time to withdrawal decreased from 9.9 years to 3.6 years, and time to regular approval increased from 1.6 years to 3.6 years.
- Of the 48 drug pairs converted to regular approval, 40% were converted based on overall survival, 44% on progression-free survival, 10% on response rate plus duration of response, 4% on response rate and 2% despite negative confirmatory trials.
- When comparing accelerated and regular approval indications, 38% of drug trials were unchanged and 63% had different indications.
"Most cancer drugs granted accelerated approval did not demonstrate benefit in overall survival or quality of life within five years of accelerated approval," the study 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."