The FDA recently cleared the way to make it easier for new drugs to get approval with a designation of breakthrough therapy. But speeding up the regulatory process has led to some uncertainty about the effectiveness of new drugs, according to a new study published in the Journal of American Medical Association (JAMA).
Regulators started allowing certain drugs to be given breakthrough status in 2012, if preliminary evidence showed they were better than available treatment options. The term 'breakthrough' had the effect of garnering attention to new drug options, even if they were unproven.
“Although physicians and patients often perceive that breakthrough approvals are based on rigorous clinical evidence, no systematic evaluation of the evidence supporting breakthrough approvals has occurred,” the study reads.
Of the FDA-approved drugs with the breakthrough therapy designation from 2012 to 2017, 40 percent did not go through a randomized trial that's typical when researchers conduct clinical trials.
Overall, speeding up the drug approval process has been successful, though the trials for breakthrough therapy drugs tend to be shorter and smaller, resulting in more uncertainty.
“If we are going to be making this trade-off to allow novel drugs to come to market on the basis of evidence that is generally accompanied by greater uncertainty, we must be committed as a clinical and scientific community to ensuring that high-quality, rigorous post-market trials are conducted within a reasonable period,” Joseph Ross, associate professor of medicine at Yale, told the Washington Post.
Amy joined TriMed Media as a Senior Writer for HealthExec after covering home care for three years. When not writing about all things healthcare, she fulfills her lifelong dream of becoming a pirate by sailing in regattas and enjoying rum. Fun fact: she sailed 333 miles across Lake Michigan in the Chicago Yacht Club "Race to Mackinac."