FDA’s Cancer Drug Accelerated Approval Process: How Effective Are We Giving Our Patients?

In a recent study led by Ian Liu, Aaron Kesselheim, and Edward Shaffer Cliff, researchers found that out of 46 cancer treatment drugs approved through an accelerated procedure between 2013-2017, only 63 percent received permanent approval from the FDA. However, only 43 percent of these drugs demonstrated clinical effectiveness, with just 20 out of the 46 showing significant results.

According to Dr. Ido Wolff, director of the oncology department at Ichilov Hospital and head of Tel Aviv University School of Medicine, drugs receiving temporary approval are usually for advanced, terminal diseases with no other treatment options. The FDA sometimes grants conditional approval based on promising initial results, pending further examination of the drug’s effectiveness.

Prof. Wolff notes that while comprehensive clinical studies can take several years, preliminary findings are often available sooner. He emphasizes the need for proper monitoring and control of subsequent studies after temporary approval, as well as transparency in marketing the drug’s status. In Israel, separate registration procedures exist, and drugs must also undergo rigorous evaluation before being included in the medicine basket for use. Despite these measures, Prof. Wolff acknowledges that marketing efforts sometimes influence the use of drugs with unproven effectiveness.

It’s crucial for doctors to communicate clearly with patients when considering costly treatments outside the medicine basket. Patients should be fully informed about the drug’s status and potential benefits before making any decisions regarding their treatment options.

The study highlights the importance of ensuring that drugs receive proper clinical evaluation before being approved for widespread use. While some drugs may receive temporary approval based on promising initial results, it is important to conduct rigorous follow-up studies to ensure their long-term safety and efficacy.

Furthermore, doctors must ensure that they communicate clearly with patients about a drug’s status and potential risks before prescribing it outside of traditional clinical trials or approved channels.

Overall, this study underscores the need for continued vigilance in approving new cancer treatments and ensuring that they are safe and effective before they are widely available to patients.