Almost a third of drugs approved by the Food and Drug Administration (FDA) over a 10-year period had problems related to safety that were not evident before approval, according to a study by Nicholas Downing, a health policy researcher at Brigham and Women's Hospital in Boston, and colleagues.
“The fact that so many new safety risks are being identified after FDA approval indicates that the FDA is taking its responsibility of ensuring the safety of new drugs throughout their lifetime seriously,” Downing said in a news release, adding that the postapproval risks emerge, on average, four years after approval. “This means that many patients are exposed to these medications before the risks become clear.” He called on everyone involved in the drug development process to “commit to the generation of clinically useful information both before and after regulatory approval.”
Although the appropriate role of the FDA has been debated for years, its key mission is ensuring safety. This was famously shown in the 1960s with the drug thalidomide. Although thalidomide had been quickly approved outside the United States for morning sickness, the FDA opted for continued study, thus saving many American mothers and their children from the worldwide epidemic of severe birth deformities associated with thalidomide's use during pregnancy.
Concerns have been raised about the 21st Century Cures Act, signed into law by President Obama last December, which aims to streamline the development and FDA approval of new drugs, among other goals. Some experts believe its provisions favor the pharmaceutical industry and weaken the FDA's standards of evidence for approval. Another concern is that the agency's new commissioner, Scott Gottlieb, has had long-standing relationships with several drug companies through his venture capital firm and also as a board member or adviser. Gottlieb has promised to recuse himself for a year from FDA deliberations related to any of those 20-plus companies.
A relevant finding of the Downing and colleagues study was that biologics, psychiatric medications, and drugs approved on an accelerated basis or near the regulatory deadline were most likely to have new “post-market” adverse events.—Serena Stockwell
Downing NS, et al JAMA 2017 317 18 1854–63