Duchenne Lessons: Advocates For Rare Spinal Disease Have Eyes on FDA

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more thorough, “patient-led” meeting will take place within the next year. This meeting, he says, would be to convey information that Cure SMA believes “should be taken into account” both for nusinersen’s coming application and “for all potential future drug applications for SMA.”

People with SMA can share the same malfunctioning SMN1 gene but exhibit a broad range of different outcomes, classified as types 1 through 4. Type 1, for example, is diagnosed in infants and the impact—many die within two years without help from a ventilator—is much more significant than the other types.

Hobby says a drug focused on that genetic problem, like the Biogen-Ionis drug and others in development, should be applicable to more than just a fraction of patients. Cure SMA doesn’t know how the FDA will stand on that issue, however, so through the meeting, Hobby hopes to “ensure this perspective is taken into account.”

“Rather than just presenting stories and anecdotes, we’ll provide data and statistics to the FDA that they can use,” he says.

But industry watchdogs say pressure from advocacy groups can lead to approval of ineffective or unsafe therapies. And funding from drug makers creates a potential conflict of interest when the two campaign for a drug’s approval.

Sharon Batt, a bioethics professor at Dalhousie University in Nova Scotia, points to the case of bevacizumab (Avastin). Already approved for several other cancers, bevacizumab got the FDA’s conditional nod for metastatic breast cancer in 2008 based on a thinner body of evidence than usual. At the time, as this New York Times article points out, “oncologists and patients advocates were divided, in part because of the drug’s sometimes severe side effects.” The FDA’s compromise was to approve the drug on a conditional basis, subject to further testing that could pave the way for a wider approval.

The data from additional studies didn’t support that claim, however. The agency revoked the approval three years later after more conclusive results showed bevacizumab wasn’t helping patients live longer, and worse, was leading to serious health risks like hemorrhages.

Batt says some patient advocates supported the FDA’s decision, but the majority “pleaded and protested” for the drug to keep its breast cancer approval; as the Washington Post wrote in 2011, some patients “likened the FDA’s rejection to a death sentence.” The situation, the Post wrote, “sparked one of the more emotional and acrimonious debates in years over a medical treatment among patients, oncologists, women’s health advocates, health-care policymakers, politicians and the pharmaceutical industry.”

The calculus isn’t all that different with rare devastating diseases. SMA is the leading genetic cause of infant deaths. Duchenne muscular dystrophy puts most patients in wheelchairs by their teens and kills them—typically from heart or lung complications—by their mid-20s. Rare disease advocates acknowledge the danger of pressing too hard, but they say the stakes are higher.

“If we end up with a dozen really bad [approval] decisions, that’s going to throw everything backwards,” says Durhane Wong-Rieger, the president and CEO of the Canadian Organization for Rare Disorders (CORD), which advises several patient advocacy organizations. “But a patient group might say, ‘We’re willing to take that risk because we don’t have any other options.’ Can I ask those parents to wait, knowing that by the time [a safe, effective drug] gets there it may be too late?”

This scenario is currently playing out publicly with Duchenne muscular dystrophy. The patient community has rallied around a group of drugs that, one by one, have come up for FDA review over the past year with significantly flawed data. The FDA has rejected two of them, from BioMarin Pharmaceutical (NASDAQ: BMRN) and PTC Therapeutics (NASDAQ: PTCT). A decision on Sarepta’s eteplirsen could come at any moment. As with the other two, the FDA can either approve a drug it normally wouldn’t because the need is so significant, or deny a drug for sick and dying children.

“Whenever the pressure succeeds, it creates a model that patient groups and drug companies see as worth repeating,” says bioethicist Batt. “I know it’s really hard for the scientists or the people on the FDA committee to take a strong stance and to sort of be held up as heartless, but they really have to do it. That’s their responsibility.”

Batt says it’s important to hear from patient groups about their needs. But it’s also important to know where their funding comes from. Patient groups that take money from drug makers, she says, are less likely to challenge the high cost of drugs or talk about serious problems like side effects. The nonprofit group Public Citizen recently published a report showing that the majority of patient groups publicly opposing a Medicare B proposal to reduce drug costs—110 out of 147 total—received Big Pharma funding.

“You don’t have very many critical voices within the patient community,” Batt says.  “They’re now kind of in a box, because they realize that if they do speak critically they risk losing funding that they’re now dependent on.”

Funding levels vary. Cure SMA, for instance, drew $228,675, or just 4 percent, of its roughly $5.4 million in funding in fiscal 2015 from “national corporate supporters,” some but not all of whom are drug makers, says the nonprofit.

CORD receives about half its funding from corporate entities, according to Wong-Rieger.

Cure SMA CEO Hobby says his organization manages potential conflicts that could arise from industry funding in a few ways. It controls the content for all of its events. It assists with … Next Page »

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