Expensive new drug finally available for her rare disease – but she’s been fighting for two years to get it

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When Annie Wilson was just six months old, she was diagnosed with spinal muscular atrophy, a rare condition that causes progressive muscle weakness and impaired movement – and was told she wouldn’t live past 3 years.

Over 30 years later, the first treatment for his disease was approved in Biogen Inc.’s BIIB,
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and IONS of Ionis Pharmaceuticals Inc.,
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Spinraza.

Spinraza grabbed attention for its innovation, but so did its hefty price tag: up to $ 750,000 the first year and $ 375,000 per year thereafter.

At the time of approval, Wilson was struggling to breathe, speak, and even drive her wheelchair, and was excited about the prospect of the new treatment.

But after initially telling her she was a good candidate, her doctor, who works for California’s Kaiser Permanente healthcare system, said she was too weak and should come back later, Wilson told MarketWatch. .

Wilson challenged Kaiser’s decision through a Medicare appeal process. Even so, the two-year battle has been grueling, Wilson told MarketWatch in an email. She suspects that the stress is affecting her health and has even considered giving up.

“I didn’t want to die fighting for a drug that would stop my disease from progressing,” Wilson said. “I feel like denying someone is like Kaiser saying your life doesn’t matter to us, but the dollar does.”

“If I were to get Spinraza, I wouldn’t expect a miracle,” she said. “Stopping the progression of my disease is my goal. Preserving the little movement I have left is the most important for my quality of life.

Annie Wilson, a 36-year-old woman from Alameda, Calif., Was first diagnosed with spinal muscular atrophy, a rare condition, at the age of six months. She has been fighting the Kaiser Permanente health system for two years for access to expensive therapy that could stop the progression of the disease.

Annie wilson

See more : Heavily Expensive Rare Disease Drug May “Invite Criticism Storm”

Kaiser has “enormous empathy” for Wilson and wants her to receive support and care, but “we cannot recommend a treatment that puts a patient at risk and has no clinical studies demonstrating its effectiveness for him. patient, ”Dr. Sameer Awsare, executive managing partner of The Permanente Medical Group, said in a statement.

“At this time, there are no clinical studies indicating that Spinraza is effective in ventilator-dependent adults with spinal muscular atrophy, and there are significant risks, even fatal in some cases. “

More rare disease drugs have been approved in recent years, but Wilson’s experience speaks to the tangle of complications that can keep patients away from promising but expensive therapies.

Related: Why Medicare Won’t Cover This Rare Disease Drug By $ 300,000 Per Year

In the United States, approximately 10,000 to 25,000 people are believed to have SMA, an inherited disease, according to the SMA Foundation. Biogen estimates that most of the total population, about 60%, are adults.

Read: This expensive drug has become Biogen’s most watched product – and may soon become a blockbuster

Spinraza was approved in late 2016 for children and adults, but some health insurers, including Kaiser, were reluctant to cover adults because clinical trials that got Spinraza approved only tested it in children. .

More adults with ADS are now being treated with Spinraza, including by Kaiser, Wilson’s patient advocate, said Joahn Ginsberg.

“They have to treat her, and they have to pay for this treatment, because that’s what she pays in Medicare each month,” Ginsberg said.

However, guidelines vary and major health insurers like UnitedHealthcare UNH,
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and Cigna Corp. THIS,
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have policies that will not cover people who require sustained use of ventilators. Price, in particular, is a key factor influencing restrictions on access to Spinraza, perhaps because for a rare disease, ADS is relatively common.

See: A group of ALS patients unhappy with how the $ 115 million raised by the Ice Bucket Challenge is spent

But adults with ADS are nonetheless treated in the United States, including those who depend on ventilators.

Stanford University Hospital, for example, had treated 32 adults by mid-2018, 22 of whom were unable to walk and three of them were on life support, and there were no serious side effects or medical issues. ‘followed, according to a letter his Neuromuscular Team sent Wilson’s doctor to Kaiser earlier this year.

The Stanford team also recommends that “the treatment of [Annie] with Spinraza is reasonable and would benefit it in stopping the progression of the disease and possibly improving existing function, ”according to the letter.

Biogen has sought to expand use in adults, estimating in late October that it has reached about 15% of adult patients, compared to about half of all infants and pediatric patients.

A spokesperson told MarketWatch that Spinraza has “demonstrated significant and clinically significant efficacy … on a wide range of populations of AMS.”

There are “a large number of untreated adult patients who we believe could benefit from Spinraza,” Jeffrey Capello, Biogen’s chief financial officer, said on a conference call in late October.

But questions about price, patient population and side effects are likely to linger, and for Wilson, time matters.

“Is that going to miraculously cure his SMA?” No, ”Ginsberg said. “But do we think it can help him?” Yes.”

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