a reformulated chemotherapy drug, it was approved by the FDA in the summer of 2012. And the agency was quick to take credit for its role in bringing forward a new medicine for leukemia patients. “Marqibo’s approval demonstrates the FDA’s commitment to the development and approval of drugs that address serious, unmet medical needs,” said Richard Pazdur, the head of the FDA’s cancer office, in a statement. For me, an equally big eye-opener came in June when FDA staff said in a briefing document about Onyx Pharmaceuticals’ carfilzomib (Kyprolis) that the “FDA is very concerned with the severe toxicities, including deaths that are associated with the use of this agent. The pathogenesis of these toxicities is not understood. Considering these factors, the risks of carfilzomib may not outweigh its benefits.” A couple days later, an FDA advisory committee voted 11-0 to recommend the drug be approved, and the FDA cleared it a month later.
In addition to those surprising approvals, Miller adds that he’s seen no recent examples of “forehead slapping” decisions, which he defines as good drug applications being stalled or rejected because of some unreasonable FDA demand.
Given that we are in an election year, and the FDA is a government body, it would be naïve to think that politics has no role whatsoever in the overall FDA attitude. For the past couple years, venture capitalists and industry trade groups have mounted a dedicated lobbying campaign to get the FDA to become more industry friendly. The VCs and trade groups have grabbed the attention of Congress and the White House, arguing that the FDA’s past intransigence and impossible demands have made it almost impossible for them to invest in new companies with innovative new medicines in development. These arguments gained a lot of currency when the VCs started that contending the FDA was essentially undercutting one of the few industries in which the U.S. is still No. 1. Many good-paying jobs are being sent overseas, and many more are bound to go there, right at a time of worrisome high unemployment in the U.S., they argued.
While those points were falling on sympathetic ears in Congress and at the White House, the industry and the FDA were also working together behind the scenes on the Prescription Drug User Fee Act, the critical governing document on industry/FDA relations. Not everybody got everything they wanted in the renewal of the PDUFA legislation, but just about everybody claimed victory in the end, as the FDA got more resources to do its job, and the industry got some accelerated approval language for reviews of innovative new medicines.
FDA commissioner Margaret Hamburg, speaking on a panel with industry leaders in June at the Biotechnology Industry Organization convention, was positively beaming about how her agency fared in those behind-the-scenes talks. She went so far as to suggest that the agency is being empowered to take a more balanced view of drug risks and benefits, because the public is becoming more sophisticated. To paraphrase, she said the public no longer expects new FDA approved drugs to be absolutely safe in all cases, and that people now understand that an FDA approval means the agency thinks a drug’s benefits outweigh the risks. Presumably, if the public believes that, then it ought to be more understanding when the next Vioxx comes along and causes dangerous side effects, because prescription drugs aren’t Cheerios. If a drug harms you and helps 1 million other people, you can’t really break out the pitchforks and march on FDA headquarters, because the agency warned you that drugs aren’t absolutely safe—they just offer more benefit than risk.
I’ve been saying for a long time to anyone who will listen that the FDA has an incredibly tough job, making decisions with incomplete information, and with millions of dollars and many lives on the line. It has often done a poor job explaining its work, and not surprisingly, it has been frequently misunderstood.
Even though the agency appears to be leaning in favor of industry this year, it would be wrong to conclude that the FDA has turned into an industry lacky that shouldn’t be trusted. I don’t see any decisions here that look like drugs were recklessly approved on half-baked datasets. Many of its cautious decisions of the past must be considered in the broader context of an agency that made a few mistakes and swung too far in the direction of excessive risk-aversion, partly to satisfy an ill-informed public and Congress. Now the pendulum is swinging the other way, in favor of industry, and in favor of patient advocacy groups that argue the FDA should allow patients in need to get access to groundbreaking medicines. That’s just this writer’s view. If you know of examples that either buttress this argument or poke holes in it, please leave a comment in the space below. I could be wrong about change at the FDA, but at least from reviewing the agency’s actions in 2012, it looks like a pretty good time to be in the business of developing new medicines for patients.
