Janet Woodcock, the top drug evaluator at the FDA, was the driving force behind the FDA’s recent decision to approve eteplirsen (Exondys 51), a drug for patients with Duchenne muscular dystrophy from Cambridge, MA-based Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]).
The decision was controversial: Many FDA scientists didn’t want to approve eteplirsen. They didn’t want to lower the bar for future drug approvals—eteplirsen was approved mainly on data from a flawed study of just a dozen patients—and indicate to drug companies that the agency can be swayed by political pressure and lobbying from patient advocates. But they were overruled by Woodcock, who has been a proponent of increasing patients’ input in the current drug evaluation process.
That decision, and the increasing impact patients are having on drug development, was the topic of a panel held on Tuesday at the Wisconsin BioHealth Summit. The discussion, entitled “How Patient-Centered Care is Driving Change and Propelling Opportunity,” was part of the annual conference put on by BioForward Wisconsin, the state’s flagship life sciences trade group.
Two of the panelists, Tim Cunniff and Erik Eglite, work at Marathon Pharmaceuticals, a Northbrook, IL-based company whose steroid deflazacort could become the next approved Duchenne treatment in the U.S. Unlike Sarepta’s drug, which is approved for a small subgroup of patients whose Duchenne is caused by a specific genetic mutation, deflazacort is meant for all Duchenne patients. The FDA began a review of deflazacort in August. (Deflazacort is already approved in Canada and several European countries for various joint and autoimmune diseases, but not in the U.S.)
Cunniff says that Marathon and Sarepta “are not competitors,” and that the FDA’s approval of eteplirsen “has no bearing” on his company. Still, he calls it “an extraordinary approval,” and says that the influence of patient advocacy groups, Congress, and others was likely a factor.
“They really wanted patients’ voices to be heard,” Cunniff says. “When you go through and look at the level of evidence, with 12 patients, it’s pretty scant.”
This is a complex issue. Patient advocates stumping for approval of drugs for rare diseases with no treatments help drive awareness and funding for research to their causes. Yet many of these groups work with or get funding from drug companies, which critics say make these groups more likely to minimize the risk associated with experimental treatments, and less likely to fight for lower prices (check out this New York Times article for more on the issue).
Cunniff says that in his experiences with patient advocacy groups, he’s found them to be zealous, though not to the point of being intimidating.
“Obviously, they have an agenda,” he says. “Up until recently, there hadn’t been drugs being developed for this condition. They had boys that are dying before their eyes.”
The eteplirsen saga was a test case for the increasing power of patient advocacy in drug development and approvals, which is why advocates for patients with other rare diseases, such as spinal muscular atrophy, were paying close attention. There are currently no approved treatments for spinal muscular atrophy, which was also the case with Duchenne before the FDA gave eteplirsen the green light.
In approving eteplirsen, FDA commissioner Robert Califf wrote that the agency didn’t set a precedent for future drug approvals, and that each future decision will be made on its own merits.
The FDA also ordered Sarepta to perform post-approval studies to prove that eteplirsen can benefit patients. If the results of those studies, which are due to the FDA by 2021, do not
