However, others remain skeptical.
Under the law, Tavneos “should not actually remain on the market,” said Joshua Skydel, a clinical fellow in rheumatology at Yale New Haven Hospital and a researcher at the Yale Collaboration for Regulatory Rigor, Integrity, and Transparency. The FDA has standards for drug approval, including substantial evidence of safety and effectiveness. In this case, the crucial study has been called into question, and several of the studies pointed to by Amgen have yet to be published: “I would at least like to peer-review them,” Skydel said, “before I hang my hat on them.”
Høeg, meanwhile, is skeptical that the clinical evidence accumulated since Tavneos’ approval will do much to support his case. “I suspect that real-world data, much of which was already available when we requested the withdrawal in April, will not meet FDA’s evidentiary requirements for approval,” she wrote. “We know that the data was not sufficient for ongoing market approval [in] the EU and the UK.”
Skydel also noted that at the time of Tavneos’ approval, the FDA had requested that additional post-marketing studies be conducted. But Amgen has fallen behind on deadlines for these studies. “This episode really demonstrated to me how drugs can come to market with unanswered questions,” Skydel said. “Our ability to answer these questions is really only as strong as the collaboration between the FDA and industry,” he continued, as well as the FDA’s willingness to use its authority after a drug comes to market.
In its April letter, the FDA argued that rare disease patients aren’t the only ones at stake in this debate, saying that withdrawing the drug “is critical to protecting public health and the integrity of the drug approval process.” Without such a consequence, the agency said, other companies would have an incentive to act fraudulently.
Walter Kraft, who voted for the approval of the drug based on his findings at the time, also fears possible consequences. The clinical trial system “is based on a level of trust,” he said. “And when that trust is compromised for limited church gain, it puts the entire system at risk for other subsequent actors in the space.”
This article was originally published on Undark. Read the original article.
