A recent study has raised concerns about the effectiveness of numerous cancer drugs that received fast-track approval through the U.S. Food and Drug Administration’s accelerated program. This program, initially established in 1992 primarily for HIV medication, aims to provide quick access to promising treatments, especially for severe or life-threatening conditions. Currently, cancer drugs make up 85% of these expedited approvals.
The study discovered that a significant portion of these cancer treatments, despite being fast-tracked, failed to show substantial benefits within five years. Dr. Ezekiel Emanuel, a cancer expert and bioethicist at the University of Pennsylvania not involved in the study, emphasized the need for conclusive evidence of efficacy, considering the large number of patients receiving these medications.
The FDA’s approach allows early access to drugs based on preliminary positive results. Drug manufacturers, in return, are expected to conduct thorough subsequent testing to obtain full approval. However, this method has resulted in the release of some drugs that ultimately don’t meet expectations. Withdrawal of such drugs falls on either the FDA or the drugmaker, and in some cases, the FDA has accepted less conclusive evidence for full approval.
Among the 46 cancer drugs granted accelerated approval between 2013 and 2017, the study found that only 43% showed clinical benefit in follow-up trials, although 63% were converted to regular approval. This research was published in the Journal of the American Medical Association and was a topic of discussion at the American Association for Cancer Research’s annual meeting in San Diego.
The understanding of accelerated approval among cancer patients remains uncertain, according to Dr. Edward Cliff of Harvard Medical School, a co-author of the study. He questions whether patients are fully aware of the potential uncertainties associated with these drugs.
For patients with rare or advanced cancers, these drugs might be the only treatment option, points out Dr. Jennifer Litton from the MD Anderson Cancer Center in Houston, who was not involved in the research. She stresses the importance of doctors being transparent about the evidence and not overpromising outcomes.
Recent legislative updates have strengthened the FDA’s authority, enabling swifter withdrawal of drugs under accelerated approval if companies fail to fulfill their obligations. Cherie Duvall-Jones, an FDA spokesperson, highlights these changes, noting that the FDA can now demand that confirmatory trials begin at the time of preliminary approval, thereby hastening the validation process for these drugs’ efficacy.