Wednesday , May 18 2022

An expensive new medication is finally available for her rare disease – but she has been fighting for two years to get it


When Annie Wilson was only six months old, she was diagnosed with spinal muscular atrophy, a rare disease that causes progressive muscle weakness and impaired movement – and said she would not live over 3 years of age.

More than 30 years later, the first reading was approved for her permission in Biogen Inc.'s

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and Ionis Pharmaceuticals Inc.

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Spinraza has been noted for being innovative, but it also has its high price tag: up to $ 750,000 in the first year and $ 375,000 a year afterwards.

At the time of approval, Wilson had trouble breathing, talking, and even drove his wheelchair and was excited about the prospect of the new treatment.

But after she had initially told her, she was a good candidate, her doctor, working for the California-based healthcare system Kaiser Permanente, said she was too weak and would come back later, Wilson MarketWatch said.

Wilson has challenged Kaiser's decision through a Medicare appeal process. Nevertheless, the two-year battle has been strenuous, Wilson MarketWatch said in an email. She suspects stress affects her health, and has even considered giving up.

"I did not want to die for a drug that will stop the development of my disease," said Wilson. "I feel that by denying someone, it's just as Kaiser says that your life does not matter to us, but the dollar does."

"If I'd get Spinraza, I do not expect a miracle," she said. "Stopping the development of my disease is what I strive for. Keeping the little movement I have left is the most important for my quality of life."

Annie Wilson

Annie Wilson, a 36-year-old from Alameda, California, was diagnosed first with the rare disease spinal muscular atrophy when she was six months old. She has been fighting for the health system Kaiser Permanente for the past two years to gain access to an expensive therapy that could stop disease progression.

See more: Extremely expensive rare disease drugs can "invite a storm of criticism"

Kaiser has "tremendous empathy" for Wilson and wants her to have support and care, but "we can not recommend a treatment that puts the patient at risk and has no clinical studies showing that the patient is effective," says Dr. Sameer Awsare, Associated Managing Director of The Permanent Medical Group, said in a statement.

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

More and more rare disease medications have been approved in recent years, but Wilson's experience speaks of the confusion of complications that can keep promising but expensive therapies away from the patients.

related: Why insurers do not cover this $ 300,000-a-year rare drug drug

About 10,000 to 25,000 individuals in the United States are expected to have SMA, which is a hereditary disease, according to the SMA Foundation. Biogen estimates that the majority of the total population, or about 60%, are adults.

Read: This precious drug has become Biogen's most noticed product – and can soon be a blockbuster

Spinraza was approved by the end of 2016 for both children and adults, but some healthcare providers – including Kaiser – were balancing on adults, as clinical trials that received Spinraza approved only tested in children.

More adults with SMA are now treated with Spinraza, including Kaiser, Wilson's patient advocate, said joahn ginsberg.

"They have to treat her, and they have to pay for that treatment, because that's what she's paying for Medicare every month," said Ginsberg.

However, the guidelines vary, and major health insurance companies like UnitedHealthcare

UNH, -0.04%

and Cigna Corp.

CI, -1.29%

has a policy that does not cover people who need a prolonged use of fans. Price, in particular, is a key factor affecting restrictions on Spinraza access, perhaps because a rare disease is SMA a relatively common.

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However, adults with SMA are still treated in the United States, including those dependent on fans.

For example, Stanford University Hospital had treated 32 adults from mid 2018, of which 22 were unable to go and three of them were on ventilation support and no serious side effects or medical problems occurred, according to a letter like its neuromuscular law, Wilson's doctor at Kaiser sent this year.

The Stanford team also recommends that "treatment of [Annie] with Spinraza is reasonable and would benefit her to stop disease progression and possibly improve existing function, "according to the letter.

Biogen has attempted to expand its use in adults and estimates at the end of October that it has reached approximately 15% of adult patients, compared with about half of all infants and children.

One spokesman told MarketWatch that Spinraza has "demonstrated significant and clinically meaningful impact … across a wide range of SMA populations."

There is a "large number of untreated adult patients we think may benefit Spinraza," said Jeffrey Capello, Biogen's financial director, at a conference call in late October.

But questions about price tag, patient population and side effects are likely to be delayed, and for Wilson time is all.

"Will it miraculously cure her SMA? No, Ginsberg said." But do we think it can help her? Yes."

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