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$125K a dose? Price of game-changing drug for kids frustrates Seattle doctor

Harper and Hendrix Ramos 4-year-old twins from Pasco,. began treatment with Spinraza in February. Since then, they’ve both gained enough strength to roll over, move their wheel chairs and toss a ball. Their mother, Crystal, said the drug treatment has given her hope. (Photo: KOMO News)

SEATTLE -- After 20 years of treating people with spinal muscular atrophy (SMA), Dr. Susan Apkon believes she finally has a treatment that can change her patients’ lives.

“It’s a game-changer,” Apkon said. “For the first time I can actually say to them we have a treatment that may change the course of your child’s disease.”

She’s talking about a new drug called Spinraza, which the FDA approved in December. At Seattle Children’s Hospital, Apkon has already started treatment on around 25 kids with SMA, a genetic disorder that affects the nervous system and robs patients of their muscle control and strength.

Apkon says nearly every SMA patient she is treating with Spinraza has reported some positive results, and her team remains “cautiously optimistic.” But there is one thing complicating the treatment process: the price tag.

One dose of Spinraza costs $125,000.

Apkon was stunned when she saw the price tag. Spinraza, she says, is the most expensive drug she’s seen on the market.

Patients need six doses during their first year (including ‘loading doses’ at the beginning); that’s $750,000 for year one. For every year after that, patients need three doses.

“I think that people around this country should be talking about the cost of medications,” she said, adding “Our system is broken. We need to figure out as a nation how to fix this.”

Because of the massive cost, coverage of Spinraza varies between different state Medicaid programs and private insurers, meaning many families nationwide can’t get treatment.

In February, Apkon spoke in front of the Washington Health Care Authority and asked for Washington’s Medicaid program to cover Spinraza. She succeeded. Medicaid in Washington now covers Spinraza treatment.

In response to the price of Spinraza, the Washington Health Care Authority sent KOMO this statement: “We are very concerned that we are seeing unprecedented prices of drugs to treat these rare conditions. Such pricing appears to be disconnected from any rational economic basis.”

Biogen, the company that makes Spinraza, explained the price in a statement to KOMO News.

“The price of Spinraza was determined through a rigorous and thoughtful process that evaluated a range of information and strived to achieve an appropriate balance among three key principles—clinical value, impact to the healthcare system, and commitment to patients and advancing science through the funding of research and development,” Biogen representative Matt Fearer wrote.

Seattle Children’s Hospital put together a team of doctors, insurance processors, ethicists and more to solve this complex issue. Apkon says they have found coverage for every SMA patient who has sought Spinraza treatment.

The Ramos family is one who has found renewed optimism.

Harper and Hendrix Ramos are 4-year-old twins from Pasco. Both have SMA and began treatment with Spinraza in February. Since then, they’ve both gained enough strength to roll over, move their wheel chairs and toss a ball.

“You don’t know how long you have,” their mother, Crystal Ramos, said through tears. “And now this gives us hope.”

Ramos says private insurance through her teaching job covers most of the treatment, but Medicaid will pick up the last $30,000 of this year’s treatments for the twins. She’s worried though, because she says her school district is switching insurance companies this fall, and the new one may not cover the treatments.

She’s thankful for each day with her sons, watching their strength improve. But she’s also frustrated that other families don’t get the same chance.

“We have a lot of friends who are not covered or who have gone through the denial process time after time, and it’s heartbreaking,” she said.

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