JAMA says FDA jumped the gun on Gilenya, Pradaxa

The FDA's balancing act is no easy feat. In fact, it just might be impossible. As soon as the agency gets congressional direction to revamp and speed up approvals, experts in other quarters conclude that approvals are coming too fast. In an article published by the Journal of the American Medical Association, drug-safety advocates point to a few recently approved drugs--Boehringer Ingelheim's Pradaxa, AstraZeneca's ($AZN) Caprelsa, and Novartis' ($NVS) Gilenya--as examples of an FDA rush to judgment.

The JAMA writers--including Thomas J. Moore, whose CenterWatch publication issues drug-safety information based on FDA adverse event reports--said "significant safety questions" were unanswered when the three meds got the agency's blessing. They question Caprelsa's safety and efficacy, citing toxicity issues and a lack of overall survival benefit. AZ told The Wall Street Journal that its trials found a 65% decrease in the risk of progression-free survival.

And then there's Pradaxa. Since its launch in 2010, the Boehringer blood thinner has been linked to thousands of adverse event reports and hundreds of cases of fatal bleeding. The FDA is reviewing case reports; after their own review, European regulators asked for label changes, but said the drug's benefits are still worth the risk for appropriate patients.

Finally, there's Gilenya. As the JAMA article points out, FDA required multiple post-marketing studies when the multiple sclerosis drug was approved. Heart-rate problems flagged early on have surfaced in the "real world," but patient advocates say the drug is still a viable treatment option. The JAMA writers themselves said that Gilenya proved better than an existing drug at preventing MS relapses. "I would hardly say the Gilenya experience constitutes going too fast," the National Multiple Sclerosis Society's Timothy Coetzee told the WSJ.

The FDA's director for drug evaluation and review, Janet Woodcock, points out that for certain diseases, people want treatment options, even if those options could be risky. "I'd like to stress that where there are unmet medical needs, the public has told us they are willing to accept greater risks," Woodcock told the newspaper. "The cancer community in particular says we haven't used accelerated approvals enough."

- see the WSJ story