Regulatory Guidance

Study questions efficacy of cancer drugs in FDA’s accelerated approvals program

May 29, 2019

A new study has revived the debate over whether or not cancer drugs fast-tracked through the FDA’s accelerated approvals program actually help patients. 

The program was designed to help patients with rare or difficult-to-treat forms of cancer gain quicker access to newer drugs. But as part of the process, drug companies are required to perform follow-up studies on the treatment’s efficacy. 

Last year, the FDA boasted that after 25 years, only five of the 93 cancer drugs approved through the program had been removed from the market. But a study recently published in JAMA Internal Medicine points out that only 19 of those 93 drugs were later shown to extend the life of patients.  

The study also criticizes the use of “surrogate endpoints” such as tumor shrinkage (with arbitrary size parameters) as a benchmark for success in confirmatory studies. The authors argued that these targets don’t necessarily reveal whether or not there was a clinical benefit with the use of the treatment, such as an improvement in symptoms or an extended lifespan. 

In response to the study, the FDA told CNN that the agency only approves drugs with a “favorable risk-benefit profile.”  

Since the program was launched in 1992, a number of critics have argued that the agency should take more time in evaluating new drugs. But patients and cancer advocates have long maintained that the program is vital for giving patients access to potentially helpful drugs. 

Read the full NPR report.

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