Most Cancer Drugs Granted Accelerated Approval Later Show Few Real-World Benefits: Study

Researchers urge doctors to tell their patients when a recommended drug went through an accelerated approval process, and to disclose whether the drug has actually been shown to be safe and effective

Nearly half of all cancer drugs that are allowed onto the market through an accelerated approval process are later found to provide few, if any, actual benefits for patients, according to a new study.

The U.S. Food and Drug Administration (FDA) uses the accelerated approval program to allow potentially life-saving cancer treatments to reach the market quickly, without requiring drug makers to complete lengthy and thorough testing required by the full regulatory process. However, these are drugs are often widely adopted and used before real-world data is available.

In findings published this week in the The Journal of the American Medical Association (JAMA), researchers indicate that 41% of cancer drugs that went through the abbreviated FDA approval process failed to improve patient survival or their quality of life by the five-year mark.

FDA Accelerated Drug Approval Program

The FDA’s accelerated drug approval program was created in 1992 to help improve access to HIV drugs. Now, more than 85% of drugs going through the accelerated approval program are designated for cancer treatment.

The program allows the agency to fast-track drugs that may be promising treatments for cancer and other fatal diseases. It is only after the accelerated approval is granted and the medications are on the market that drug companies must perform clinical trials showing the drug’s effectiveness and safety before full approval is granted.

However, the findings of this latest study suggest those post-marketing clinical trials often never happen, and real-world data often shows that the drugs are not that beneficial, potentially depriving patients from using older, proven treatments.

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Researchers from Brigham and Women’s Hospital and Harvard Medical School in Boston, Massachusetts, conducted research on cancer drugs granted fast-track approval by the FDA from 2013 to 2023, focusing on those from 2013 to 2017, along with a five-year follow-up on survival data.

During that time period, 129 cancer drugs were granted accelerated approval, according to the findings, including drugs like Perjeta, Mekinist, Lartruvo, Rubraca, and chemotherapy drugs Pomalyst and Imnovid.

Of those, 63% were later converted to regular FDA approvals, 22% were withdrawn, and 15% continued on the market, even though they had not completed post-marketing clinical trials, an average of six years after their approval was granted.

However, the data indicates 41% of cancer drugs that went through accelerated approval failed to improve patient survival or quality of life after five years on the market.  In some cases, even though the drug trials didn’t show a benefit to patient survival, the FDA still converted the drugs from accelerated approval to full approval, researchers discovered. Data was not yet available for 15% of cancer drugs in the accelerated approval program.

The researchers note that even among those drugs granted full approval, 60% relied on surrogate measures or outcomes, instead of other measures that would have better shown a relationship between how the drug works and whether it was granting patients actual improvements. They noted that 2% of the drugs were approved despite findings disputing their safety and effectiveness in those follow-up clinical trials.

Among the 63% granted accelerated approval, 44% were approved based on patient survival without progressing stages of cancer, and 40% were approved based on overall survival.

Researchers also noted about 63% of cancer drugs were originally intended for another purpose when they were first granted accelerated approval.

Accelerated Approval Program Concerns

The findings are the latest concern over the FDA Accelerated Approval Program, which many have criticized for allowing ineffective, and potentially dangerous, drugs on the market without proper research.

A study published in 2019 indicated only about one-fifth of cancer drugs approved through accelerated approval actually work or increase patients’ odds of survival. Another study indicated drugs approved through the accelerated program are more likely to need safety warning label updates for serious and potentially life-threatening side effects.

More than 94% of drugs that make it through the fast-track program need safety-related label changes every year, including black box warnings, the strictest form of FDA label warning.

Researchers in this latest study warned that patients should be clearly informed if a doctor recommends a drug that received accelerated approval. They also recommended that if the drug does not show benefits in clinical trials, doctors should inform their patients of the deficiencies.

“Most cancer drugs granted accelerated approval did not demonstrate benefit in overall survival or quality of life within 5 years of accelerated approval,” the researchers concluded. “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.”


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