With an aim to provide patients with early access to potential life-saving drugs, the U.S. Food and Drug Administration’s accelerated approval program is under scrutiny. However, recent research has raised questions about the effectiveness of these drugs and whether they truly improve or extend patients’ lives.
A Closer Look at the Accelerated Approval Program
The accelerated approval program was initiated in 1992 to expedite access to HIV drugs. Currently, a whopping 85% of accelerated approvals are given to cancer drugs. This program allows for early approval of drugs that show promising preliminary results against debilitating or fatal diseases. In return, pharmaceutical companies are expected to conduct rigorous testing and offer better evidence before acquiring full approval.
The tradeoff here is that patients get access to drugs earlier, but some of these medications might not pan out. The responsibility to withdraw underperforming drugs lies with either the FDA or the drugmaker. Occasionally, the FDA has deemed less definitive evidence sufficient for full approval.
Recent Study Raises Concerns
A new study has discovered that between 2013 and 2017, 46 cancer drugs were granted accelerated approval. Among these, 63% were converted to regular approval, even though only 43% demonstrated a clinical benefit in confirmatory trials.
This research, published in the Journal of the American Medical Association, was also a topic of discussion at the American Association for Cancer Research annual meeting in San Diego on Sunday.
Dr. Ezekiel Emanuel, a cancer specialist and bioethicist at the University of Pennsylvania who was not part of the research, believes that five years post the initial accelerated approval, there should be a definitive answer. He is concerned that thousands of people are receiving these drugs without knowing if they work.
Understanding the Impact on Patients
Dr. Edward Cliff of Harvard Medical School, a study co-author, expresses uncertainty about how much cancer patients understand about drugs with accelerated approval. He questions whether the uncertainty of these drugs is being conveyed to patients.
For patients with rare or advanced cancers, drugs that got accelerated approval may be the only option. Dr. Jennifer Litton of MD Anderson Cancer Center in Houston, who was not involved in the study, emphasizes the importance of doctors carefully explaining the evidence. She cautions against overpromising.
Congressional Updates to the Program
Congress has recently updated the accelerated approval program. The changes have bestowed more authority on the FDA and have streamlined the process for withdrawing drugs when pharmaceutical companies fail to fulfill their commitments. The updated policy will enable the agency to withdraw approval for a drug more quickly when appropriate, according to FDA spokesperson Cherie Duvall-Jones.
With these changes, the FDA can now require a confirmatory trial to be underway when it grants preliminary approval, which accelerates the process of verifying a drug’s efficacy.
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