Researchers have shed light on the efficacy of cancer drugs that have been granted accelerated approval by the U.S. Food and Drug Administration (FDA). This accelerated approval program was initially designed to provide early access to promising drugs for patients in need. However, the study's findings have raised questions about the actual benefits of these drugs in improving or extending patients' lives.
Dr. Ezekiel Emanuel, a prominent cancer specialist and bioethicist at the University of Pennsylvania, expressed the urgency of obtaining definitive answers about the effectiveness of these drugs within five years of their accelerated approval. He emphasized the potential risks associated with administering drugs to thousands of patients without clear evidence of their efficacy, stating, "Thousands of people are getting those drugs. That seems a mistake if we don't know whether they work or not."
Originally established in 1992 with a focus on expediting access to HIV drugs, the accelerated approval program has since evolved, with 85% of accelerated approvals now being granted to cancer drugs. This program allows the FDA to grant early approval to drugs showing promising initial results in treating debilitating or fatal diseases. In return, drug companies are expected to conduct comprehensive testing and produce substantial evidence before obtaining full approval.
However, the tradeoff of early access to medications means that some drugs may not yield the expected benefits. The responsibility falls on the FDA or the drugmakers to withdraw drugs that do not meet the expected standards. In some cases, the FDA has accepted less definitive evidence as sufficient for full approval.
The recent study, published in the Journal of the American Medical Association and presented at the American Association for Cancer Research annual meeting in San Diego, revealed that between 2013 and 2017, 46 cancer drugs were granted accelerated approval. Shockingly, only 43% of these drugs demonstrated a clinical benefit in confirmatory trials, raising concerns about the effectiveness of the accelerated approval process.
Dr. Edward Cliff of Harvard Medical School, a co-author of the study, highlighted the potential lack of understanding among cancer patients regarding drugs granted accelerated approval. "We raise the question: Is that uncertainty being conveyed to patients?" he stated, underscoring the need for transparency and informed decision-making.
Dr. Jennifer Litton of MD Anderson Cancer Center in Houston emphasized the importance of carefully explaining the evidence to patients, particularly in cases where drugs with accelerated approval may be the only option for individuals with rare or advanced cancers. She cautioned against overpromising and stressed the significance of providing clear, accurate information to patients.
In response to these findings, Congress has recently updated the accelerated approval program, granting the FDA more authority and streamlining the process for withdrawing drugs when companies fail to meet their commitments. According to FDA spokesperson Cherie Duvall-Jones, the changes enable the agency to swiftly withdraw approval for drugs approved under accelerated approval when necessary. Moreover, the FDA can now require a confirmatory trial to be underway when granting preliminary approval, expediting the process of verifying the efficacy of a drug.
