“Our Son’s Fate”: Parents Fighting for Kids’ Spine Drug Eye New Data

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a minority of their patients have begun treatment.

In New Jersey, Dastgir says Goryeb Children’s Hospital has treated four SMA patients with nusinersen; another dozen or so are waiting because of insurance issues.

John Brandsema, the director of the neuromuscular clinic at the Children’s Hospital of Philadelphia, says CHOP has only treated between 10 and 20 SMA patients—many others are trying to get cleared by insurers. The hospital has identified nearly 70 patients interested in getting therapy.

At Stanford, Cho says fewer than 10 of the roughly 100 SMA patients are on nusinersen. Another two dozen are awaiting payer approvals. Cho says he is encouraged by that number, arguing the uptake of drugs like nusinersen for rare disorders is a slow build. “We’ve assessed it, overall, as going pretty well,” Cho says.

Beyond insurers balking at high prices, patients and clinicians interviewed by Xconomy cite a raft of other holdups, too.

“This is the most logistically challenging medicine I’ve ever encountered,” according to Brandsema, who says he has heard the same from many of his colleagues.

Nusinersen requires an injection into a patient’s spinal canal once every few months, and the patient could require anesthesia and an overnight stay, depending on his or her condition. Brandsema says before CHOP began with nusinersen treatments, it had to figure out which patients to treat first and coordinate different hospital groups—pulmonologists, neurologists, and radiologists—to make sure the procedure was safe and efficient.

CHOP also has had to ensure it could supply the drug without risking the loss of hundreds of thousands of dollars if insurers refused to reimburse for it.

These problems all came amidst a crush of patients clamoring for access immediately.

Amy Concilio was eventually successful getting her two-year-old daughter Claire, a type 2 SMA patient, treated at CHOP. “But the fight for it was ridiculous,” she says.

Unaware of the logistical challenges, Concilio called daily, wasn’t hearing back, and eventually fired off an angry e-mail to the hospital. She eventually connected with a clinician but couldn’t get an estimate of when Claire would receive nusinersen.

She began reaching out to other centers, including Children’s National Medical Center in Washington, DC, which she called “every 30 minutes for three days.” She barged in without an appointment, bringing cookies to compensate for the hassle, and got Claire on the nusinersen list. CHOP called soon thereafter, however, and Claire received her first injection there in February.

Concilio says she now devotes hours each day answering questions from other SMA families worried about making too much of a fuss. “There’s a lot of people who are like, ‘I don’t want them to hate me,’” Concilio says. “And I’m like, ‘That’s really sweet. Your kid might not get treated any time soon.’”

Not everyone has a story of frustration to tell. When Beth Kingkiner’s 18-year-old son Griffen, a Type 3 SMA patient, was 11 years old, he had an opportunity to participate in the first human trials of nusinersen. He decided not to, wary of the risk of a new, untested drug. Kingkiner says they eventually learned that patients in that trial weren’t just tolerating nusinersen but in some cases regaining physical abilities. Griffen is now in a wheelchair. “But he doesn’t regret it,” Kingkiner says. “He even wrote his college essay about it.”

The experience gave Kingkiner a crucial network of contacts. Once nusinersen was approved, she called all the SMA doctors she knew, including the neurologist who had suggested Griffen join the trial years ago. The neurologist recommended Dastgir, who had given multiple nusinersen injections as a trial investigator at Columbia.

Dastgir says she hasn’t faced the same logistical problems as some other clinics. Her familiarity with the drug, the procedure, and who to call helped her get things up and running quickly at Goryeb, in Morristown, NJ. Kingkiner called Dastgir in January, and Griffen was cleared by insurance and treated in February. “We were so lucky,” Kingkiner says.

Lucky indeed: Dastgir says since then insurers have been slower to reimburse, setting more limitations, and changing their policies. Yet she’s hesitant to point fingers. “I think the problem is that this is brand new for everybody, even the insurance companies,” she says.

Meanwhile, Stanford’s Cho blames the payer pushback on the drug’s high price. “For obvious reasons, higher priced drugs face more resistance,” he says. That’s important to note, because patient advocacy groups for rare diseases—which have gained power and sophistication—often take drug industry funding. Critics say that makes them less likely to push back on drug prices and, at minimum, creates the appearance of conflict of interest: The groups desperately want the therapies. Will they speak out against the industry developing them? (Xconomy delved into that issue, and its impact on the SMA community, here.)

Better data next week won’t necessarily change nusinersen’s high list price. But they could convince some insurers the high price is worth paying for more patients.

“A lot of the carriers when they were first setting their policies were going by what was publicly available,” Brandsema says. “So I think that that [data] is going to impact things.”

Even with good data from CHERISH, however, Dastgir still foresees “growing pains” for a new, unfamiliar therapy. Access will only open up because patients fight for it. “This is a very active patient population,” she says. “I’m sure whatever kid is out there, their insurance company is already hearing a lot about it.”

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