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April 8, 2024
The U.S. Food and Drug Administration’s (FDA) accelerated approval program, designed to provide early access to promising drugs for patients, is facing scrutiny following a new study that suggests most cancer drugs granted accelerated approval may not demonstrate significant benefits within five years.
Conducted by researchers and published in the Journal of the American Medical Association, the study examined cancer drugs granted accelerated approval between 2013 and 2017. Of the 46 drugs analyzed, only 43% demonstrated a clinical benefit in confirmatory trials within the five-year timeframe. Despite this, 63% of the drugs were converted to regular approval, raising questions about the efficacy and transparency of the program.
Dr. Ezekiel Emanuel, a cancer specialist and bioethicist at the University of Pennsylvania, who was not involved in the research, emphasized the importance of obtaining definitive answers about drug efficacy within a reasonable timeframe. “Thousands of people are getting those drugs. That seems a mistake if we don’t know whether they work or not,” Dr. Emanuel stated.
Initially created in 1992 to expedite access to HIV drugs, the accelerated approval program has since become heavily utilized for cancer drugs, with 85% of accelerated approvals going to cancer treatments. While the program offers patients early access to potentially life-saving medications, it also entails risks, as some drugs may not ultimately demonstrate the expected benefits.
Dr. Edward Cliff of Harvard Medical School, a co-author of the study, raised concerns about whether cancer patients fully understand the uncertainties associated with drugs granted accelerated approval. “We raise the question: Is that uncertainty being conveyed to patients?” Dr. Cliff noted.
Dr. Jennifer Litton of MD Anderson Cancer Center in Houston emphasized the importance of clear communication between doctors and patients regarding the evidence supporting drugs granted accelerated approval. “You can provide the data you have, but you shouldn’t overpromise,” Dr. Litton stated.
In response to these concerns, Congress recently updated the program, granting the FDA more authority and streamlining the process for withdrawing drugs when companies fail to meet their commitments. FDA spokesperson Cherie Duvall-Jones highlighted the agency’s efforts to expedite the verification process for accelerated approval drugs, ensuring timely assessment of their efficacy.
While the accelerated approval program remains a valuable tool for patients seeking access to innovative treatments, the findings of this study underscore the need for enhanced transparency, rigorous testing, and clear communication to ensure that patients receive the most accurate information about the benefits and risks of these medications.
As the FDA continues to refine its approach to drug approval, stakeholders hope to strike a balance between providing timely access to potentially life-saving treatments and ensuring robust evidence of their efficacy and safety.
